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To Reinvent Healthcare, Stop Treating Patients and Start Building Communities – Singularity Hub

Posted: November 20, 2019 at 1:45 pm

Its not news that healthcare costs in the US are sky-high. What might be news to some, though, is that year over year, were spending more money to treat illness and disease, but getting less out of it.

According to Asif Dhar MD, Chief Informatics Officer at Deloitte, spending on diabetes alone went up by $75 billion in the five years from 2012-2017. Addictionfrom alcoholism to prescription drugs to illegal drugscosts Americans $740 a year in healthcare, crime, and lost productivity. The suicide rate in the US has gone up 33 percent in less than 2 decades; were 3 times more likely to kill ourselves than we are to be killed.

Of the top ten reasons people die globally, five are inherently related to behaviors: what we eat, how we sleep, the medications we take, Dhar said in a talk at Singularity Universitys Exponential Medicine summit last week. He emphasized that spending more money isnt the way to better health; rather, we should shift some of our focus away from treating sick individuals and towards creating healthy communities.

80 percent of the problem has to do with the way we live, how we engage in healthy behaviors, and what our communities are like, Dhar said. We can move from healthcare to health by expanding the aperture of what were reinventing.

Average life expectancy in the US is declining for the first time since 1918, and if you look at the top causesdrug overdoses, suicide, and liver diseaseit can be pretty easily argued that were dying younger not because we cant cure disease, but because were not living well.

The emperor of all maladies (an apt moniker bestowed on cancer by physician and author Siddharta Mukherjee), is a good example. 42 percent of all cancers are preventable, stemming from factors like an unhealthy diet, smoking, and a lack of exercise. That effectively means that 659,000 cancer cases never needed to happen, Dhar said. Is that a question of care, or is it a question of how we live?

To be sure, our healthcare system is flawed. Medical error rates are high, care is absurdly expensive, and there are curable diseases that havent been cured, Dhar pointed out. But by the time people get to a doctor or hospital, theyre already sick; the damage has already been done. So why arent we trying harder to dig down to the roots where that damage starts? How do we figure out where those roots are? And how big of a difference could it make if we focused on nourishing roots rather than bandaging or reviving dying trees?

Dhar and his colleagues think communities are at the root of our health, and that if we focus on building healthier communities, the payoffs in quality of life, years of life, and dollars saved will all be significant. A team at Deloitte came up with five consistent themes for how communities can reduce costs while improving the health of their people.

1. Empower proactive health and well-being management

2. Foster a sense of community and well-being

3. Engage digital technologies with behavioral science

4. Meaningfully use data to improve outcomes

5. Enable new innovative models for the way these societies work

For a concrete example of what it looks like to apply these steps, Dhar pointed to Oklahoma City. After it was designated Americas fattest city in 2007, the citys mayor decided to fight back by implementing a program called This City Is Going on a Diet. A website was created to track progress, with around 47,000 people signing up to report their weight loss. The government poured money into building sidewalks, bike trails, parks, and wellness centers, encouraging people to ditch their cars and use more active modes of transportation. Within five years, the citys residents collectively lost a million pounds.

The one thing we know that gets people to walk is to create places that are enticing so they want to walk there, said Dick Jackson, a UCLA medical professor and former director of the Centers for Disease Controls National Center for Environmental Health. Telling people to exercise, to go to the gym, doesnt seem to work. You have to build it into daily life.

Geisinger Health System (serving Pennsylvania and New Jersey) used a community focus in a slightly different way than Oklahoma City. After realizing they were spending more on treating type 2 diabetes patients but outcomes werent improving, the health system decided to take another route and zeroed in on patients diets. They started thinking about food as medicine, Dhar said, asking if they could start prescribing food. They did, and the results were astounding.

Geisinger created a Fresh Food Farmacy, sourcing food from local food banks and providing patients with two meals worth of food per day, five days per week, for as long as they need it. Among pilot participants, there was a reduction of 80 percent in treatment cost and an average 2.1 percent reduction in patients HbA1C levels.

When it comes to the social determinants of health, we know there are many more causes impacting the health of a population than access to quality medical care, said David Feinberg, Geisingers former CEO. We want to transform healthcare at its core by focusing on preventative care, behavioral health, and economic growth.

Creating health-focused communities and empowering people to take their wellness into their own hands will have huge implications for the American healthcare system. Dhar emphasized that while people dont always make optimal health choices for themselves, theyre far more likely to engage in healthy behaviors when theyre in an environment that enables, encourages, and models healthy choices.

Examining the root causes of poor health in a community then transforming those factors from the ground up will require larger up-front investments, but will save cities, health systems, and individuals millions of dollars over timeand give everyone a higher quality of life in the process.

What was thought of as completely impossible is completely doable today, Dhar concluded.

Image Credit: Image by S. Hermann & F. Richter from Pixabay

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To Reinvent Healthcare, Stop Treating Patients and Start Building Communities - Singularity Hub

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Breaking Down The Invitae Short (Podcast Transcript) – Seeking Alpha

Posted: November 20, 2019 at 1:45 pm

Editors' Note: This is the transcript of the podcast we published last week on Invitae (NYSE:NVTA). We hope you enjoy.

Daniel Shvartsman: Welcome to the Razor's Edge. I'm Daniel Shvartsman. I'm joined by Seeking Alpha author Akram's Razor on this show. Each episode we take an investing idea or theme that Akram has been looking at for his personal investing as well as the Seeking Alpha marketplace service he runs, also called The Razor's Edge. We look at the ideas themselves, stress test them, try to figure out where they might go right or wrong, talk about what's been going on, and talk about the research and analysis that led to this take.

The idea to share some current investing ideas here into consideration, but also get the ins and outs of deep fundamental market research today. This week's topic has a lot behind it. The ticker symbol is NVTA. The stock is Invitae. Akram released a short case on the company on Seeking Alpha on October 11th, that went long in terms of breaking down why the genetic testing company was more like a WeWork than an Amazon. In other words, the company has had prodigious revenue growth, but it's come at a cost of increasingly negative cash flows and limited competitive advantage, in Akram's view. Was a thoroughly researched short case and as is often the case it's attracted a lot of attention, both positive and negative.

On today's Razor's Edge, we're going to talk about what brought Akram to this case, what investors are missing, and some of the reactions since he went public on Invitae. We're also joined by a colleague of Akram's, James who can add some insight on this topic based on research he's done on the stock as well.

Before we begin a quick disclaimer and disclosure; The Razor's Edge is a podcast on Seeking Alpha's The Investing Edge channel. The views discussed belong to either Akram, James in this case, or me respectively, and nothing on this podcast should be taken as investment advice. We'll disclose any positions in any stocks discussed at the end of the podcast, though upfront I can say I have no positions in any of the stocks we plan to discuss, Akram is short Invitae and long Myriad Genetics, and James is short, Invitae. We're recording this on the morning of November 4th.

Listen to or subscribe to The Investing Edge on these podcast platforms:

All right, guys. Good morning. Welcome.

Akram Razor: Good morning.

James: Good morning. Thanks.

DS: So let's just go really basic, why Invitae? What brought -- why does your radar get on this stock? Where did they come up?

AR: That's definitely an interesting part of the story. I guess the starting point was, there was people shorting Myriad. So I follow the Southern Investigative Reporter. And they've done a couple short pieces on that. And I kind of have taken a look at it briefly, which caused me to take a brief look at Invitae, and that was probably like, May or June. And then Ilumina missed big time on earnings, and I've traded Ilumina several times of the year. I've never traded any of these other stocks in terms of that. But it kind of got me interested in the space. And I guess what kind of, I mean, like, I think I had pinged James on it a couple times being like this is like this is worth looking into, but like neither of us had really gotten that excited about it.

And then there was a report that came out by a short seller, recommending the stock is an amazing long idea, which I read, because I know the short seller and his work. Once I read that, I was like, I need to look at this company a little closer. And then just the typical process, like pulling up their filings, seeing exactly what's going on financially, and I was just like, wow, this is just an incinerator. What are they doing? Why is -- like, what's the business model?

And that brought me to, there's an author on Seeking Alpha who had published a lot on this over the years, capital markets, laboratories and read their work, and they made these Amazon compares where, obviously, that's where you get a little bit of a heightened radar, when a laboratory diagnostic company is being compared to Amazon, which I mean, I don't know also if you know -- and I had like a brief experience with Theranos in 2000 it was say like, early 2014, like back when it was like -- I was picking on the Decacorns then, my favorite, like when I used to write my market commentary and I'd have a little bit of fun with it.

At the time, the two I was having fun with were Zenefits and Theranos. But I mean in Theranos' case, like I was just curious as far as what like -- I mean, it was a private company, $10 billion, obviously a lot of hype. I was like, wow, do I short Quest and LabCorp, right? I mean, I can't make -- I can't invest in Theranos. But if this company is so revolutionary like it, there's this pretty simple thesis out there in the public markets where I mean, if they're going to stick a lab in a box, I should go short Quest Diagnostics and LabCorp.

And I have, on the medical side, an extensive network of friends and family. So I ended up doing just one call with someone who'd been at Quest and in the laboratory diagnostic space, pretty senior for about 20 years. And like, I mean, it was like a 30 minute conversation. I felt stupid by the time it was over. It was just like, he's like, are you an idiot, you can't stick a Lab-in-a-Box. It was like -- it was literally like that, after like being very polite for a little while. So that type of stuff kind of intrigued me. And Theranos ended up being Theranos. I never really did much more with it after that. And then Wall Street Journal came after it, and it became this cautionary tale. I don't know if James -- did you follow Theranos very closely, I don't even know if we really discussed that?

J: No, I had heard from some VC friends who were very skeptical of it all the way through. But I wasn't that close to the name myself.

AR: Yeah, I mean, neither of us I would say, I would characterize myself as close to it. But I mean, I did take the time to literally be like, hey, do I short these stocks because of Theranos, right? It was like an investment idea at the time.

So yeah, I mean, I guess that's what started it, right. I mean, like Theranos had this -- and then the message was just kind of just like affordable blood testing for all, a social cause being compared more to, like technology differentiating companies and I mean, Elizabeth Holmes like to associate herself when she talked about things, when she compared herself to the Google Founders and Facebook and like, I mean, if you saw the documentaries like here we were at, in Brazil, and this is where we -- who was sitting there, and the Google guys, and the Amazon and Facebook, and we are the stars of the show, right?

And if you look at Invitae, I mean, like on the surface, it's like there's a social enterprise element to it, right? Where it's just like, we have a mission, and this mission is to make genetic testing affordable for all. I mean, like, they don't care, they don't describe themselves as like a laboratory diagnostics company, right?

DS: Well, they --

AR: That was a genetic information company. Go ahead.

DS: Yeah. I mean, if you look, I have got their 10-K open, their last one and it's -- our goal is to aggregate a majority of the world's genetic information into a comprehensive network that enables sharing of data among network participants to improve healthcare and clinical outcomes. So it's this.

AR: Yeah, so what does that mean?

DS: Right. You know, it is this very -- it is very big mission, right. It is very big.

AR: Yeah, I mean, James, obviously has some views on this. Like, I mean, what do you think of that part?

J: Well, I think that really comes down to the crux of the investment case here, which is, just because something sounds good as a sound bite doesn't mean that it actually makes sense in business. And so the idea of accumulating the world's genetic information, you would argue, seems like if you could do that would allow you to capture rents on that database. But there are very real issues with that. Namely the company has said that they won't do that. And then when they're pressed about how -- whether or not they will do that, they kind of seem to give non answers what I can tell.

So that's one of the questions right that that I think would be very helpful for the company to lay out there, very specifically, which is, if we gather all this data up, and specifically what data is being gathered? Are they -- is that data, they've also released to a common database, like they say they have been doing, is there additional data that they're not releasing?

AR: Well, I mean, the data that they're sharing with the common database is the variant of unknown significance data, right? So I mean, that's what they're contributing to Clinvar. We don't really know. But like, I mean, I'll be honest, like if I was to go in and have hereditary cancer test, I would have never thought once about, like, who the lab doing the test is or ask the oncologist anything, but like literally now knowing this business, I mean like, make sure you're not using Invitae, because I have no clue what they're going to do with that data.

Like my DNA is a product for them to figure out somewhere down the road just to do something with it, I mean, it's so -- like James just said, it's very bizarrely unclear, which is decidedly convenient when you're running a business model like this.

DS: So step back for a second. So the thesis -- the company's thesis is that they're providing genetic tests. And then they're aggregating that, they provide it at -- arguments are they providing it for below the cost that they actually have to pay to their providers to actually deliver the test but low cost testing that they can then -- if we're going to use the Silicon Valley jargon, that they get the flywheel of more genetic data and improve, they get a lot of volume, and eventually, that's both going to give them scale to lower the costs, but then also they're aggregating this huge genetic database, which as you kind of point out, Akram, they're going to have to do something with to make it reasonable, which raises concerns in of itself. But the idea is that eventually they'll get scale from offering at cost that will allow them to become a profitable business, I think. That's how I understood the company's take and the company's argument.

AR: I mean, like James just said the company hasn't explained that. So if you look at this company's history, this isn't like a startup, number one, right. So this company was founded in 2009. They've been at this for a decade, okay. Initially, they were kind of rare disease focused, right?

Like where they're deriving the revenue, if they were to actually describe themselves accurately, they would be like, we do hereditary cancer testing far cheaper than Myriad, right? And I mean, like, I think that's an important thing to look at this. I mean, there are sources of revenue, which is generally speaking what has attracted people to the stock, it's not science, right. It's not like you've developed a test or you're like Foundation Medicine and you've got this companion diagnostic clinical trials and you are about to do something where you're going to earn a high margin, because you've developed something nobody else has. That's typically, what people get excited about in science and biotechnology, right?

You get rewarded for R&D, okay. These guys have approached the market that Myriad discovered the BRCA mutation in 1994, okay. That's like what the indication of hereditary cancer right? I understand cancer, 90% of it is not hereditary. So you're looking -- when you talk hereditary, there's a less than 10% chance that it's something hereditary. That's helpful from a clinical standpoint, as far as your treatment for cancer diagnosis, whether it's early or late stage or whatnot.

So this company came into this space in the sense that Myriad had a monopoly on this gene, right? They started doing their first BRCA testing lab kit was like 1996. They had the patent on that gene till 2013 when the Supreme Court struck it down, right. So these guys entered this space, essentially, from that standpoint as a competitor against Myriad. And once that space kind of opened up in hereditary cancer testing, I mean like, well, you can't patent these genes, right? Which is great for competition, but what's the flip side of it?

If I make a major discovery on gene X correlates to disease Y, right? And I can't patent it and I'm running like a test that kind of identifies that historically, kind of tough to build a very profitable business around it. So when we go back to like what you were saying about like this whole genetic information and whatnot, the most interesting thing about this business is look at the rest of the landscape. Nobody's selling the story, like Myriad's still the revenue leader in hereditary cancer testing.

The other space the company is in deriving revenue from is reproductive health, carrier screening, non-invasive prenatal screens. And that's the most crowded market ever. It's got Ilumina and Sequenom, which is owned by LabCorp, like pretty much, they control the IP there. And then there's like a half a dozen other competitors owned by large companies.

So like, in hereditary cancer, you've got Myriad. And then you kind of have this like at a huge, huge, huge discount to Myriad prices, Invitae, right? I mean, like now they're running what, $99 tests, okay? So you look at it and you can sit here we can just, like figuring out what they want to do data wise and the fact that it's Vegas kind of important. It's also important in the context of -- well, there's a whole industry here, right? I mean, this is at least what -- we got some heat when we cautioned this like, some of this has attracted obviously a lot of retail investors by using the Amazon compare. And, in the initial thesis it was like a cautionary mention at the end, like that the laboratory diagnostic space doesn't need another Theranos.

And at least in Theranos' case, what they were selling to investors was we're building a better mousetrap essentially, right? Like we've got -- we've engineered something that's going to change the way we are able to do testing. And that's not the story here. The story here, I mean, if you go back to the founder in 2016, there's a couple of interviews with him. And he literally says like, we're providing the same test that everyone else is providing, we're just making them more affordable. That was that's their initial story, right.

So I mean, you do kind of run into something like that, which is where, like some people may think it like, you know, oh, it's very, self-serving or opportunistic to compare this to WeWork. It is, exactly like WeWork from a business standpoint. Like you can't get around that fact. And I think the Amazon like -- and this is not like some guy writing a really bullish Seeking Alpha article and using hyperbole, the management sticks to compares. I mean, they -- literally they have a slide in their investor deck, what we can learn from Amazon, and they make statements like our competitors -- what did they say, James, is like the competitors margin is our opportunity or

J: Yeah, something that effect. Yeah, you've heard a lot of ground there, Akram. I think the important aspect of what you've said and the commonality among all those points is that the management team has made some very high level statements about the promise of this industry and the promise of their company. And they could really answer a lot of these questions if they wanted to, right? They could say, okay, here is our revenue breakdown from cancer or nips [ph], kind of other panels, which don't have necessarily the clinical efficacy.

Or they could say, here's our -- here's what we're getting from the various cost cutting or network effects or economies of scale that we promised. And here's how this is going to develop, but they don't they just say, trust us, it's all going to work out. And the way that it's not [ph] going to work out is that it worked out for Amazon. So it's there's not much transparency, there's a lot of just kind of big picture verbiage I would say.

AR: Yeah. I mean, 100% and like, I mean, you get the Amazon story, Daniel, right. I mean, if you look at it, people like, if you look, well, come on, Amazon was losing money. No, Amazon was improving operating cash flow from literally from day one. I mean, people forget, like, you make money selling DVDs and books online, particularly if you don't pay your suppliers for 100 days, right? I mean, like, where was Amazon after a decade? It was already a behemoth, right?

So when you look at it, Amazon had someone else, i.e., their suppliers funding their growth. It's free, cheap capital. This company went and IPOed in 2015, at $16 a share, had like 25 million shares outstanding. They're at 100 now, four years later, right? I mean, it's like, they keep going back to the well. So if you're an investor and you're looking at it from a return on investment on money, you're giving them. I mean, you have a serious problem. And if you look at it, and you say, hey, I'm going to compare myself to Amazon. Well, Amazon had a cost structure that attacked this cost structure of brick and mortar, okay?

They benefited from so many things. We didn't have sales taxes, if you were paying on Amazon as a consumer. They benefited from the fact that they went into markets with huge existing volume already, books and DVDs. They didn't have to convince people to buy X, Y and Z. They were already huge volume markets. I mean, I'm sorry, but like hereditary cancer testing. It's not something people get excited about to go online or buy as a gift for a friend.

I mean, like, we get the DTC space, and even that has already slowed down drastically, and that's Ancestry. And if Ancestry slowed down at like 30 million tests, right, like, you really think people are going to be super excited as individual consumers to be like, I really need to figure out whether I have a history of cancer right now. I mean

J: Well, and more importantly, more importantly, I think there's a healthy amount of skepticism in the medical community whether or not these tests are useful, right? Like the issue with any test is, if you find something, an indicator of a disease. Does that help you catch the disease? Does it help you catch it earlier than existing testing or physical exams, or other ways of seeing the diseases there? And then can you do anything about it?

So, I think one of the issues here is even if you were to send your saliva [ph] sample out, and you came back with saying indication that you might have liver cancer potentially. A, you wouldn't know, if that were real, because it's really just a correlation at this point. There's not enough data. B, even if you knew, it was real, there's really not much to do except worry about it. And so you have a lot of additional burdens on the patient and the healthcare system in terms of emotional and financial burdens, without any clear benefit.

And so I think within -- as I'm saying, within breast cancer, that there is a clear benefit in terms of efficacy and outcomes. But for the rest of the space, it's really not clear. And that's why, if you look at the treatment protocols for most of the commercial payers, they don't pay for all these tests, because the research doesn't demonstrate that they should.

DS: So one of the things I'm -- as we're throwing around these comparisons. I'm thinking about the -- there's a notion in Silicon Valley and sort of abroad, the market, the idea of tech, tech as a category is becoming less and less meaningful, because companies are adopting online models or tech models to different verticals. And what I think about with Theranos and Invitae, specifically, healthcare is a very complicated sector, both in terms of the way payers work and we can get into Medicare in a little bit, but the way the payment system works.

And I'm not talking even about what might or might not happen in the future with changes to insurance. But just -- that's, I think, always been fairly -- you have to really work through it. And the articles for example, there's looking at the different reimbursement codes and that sort of thing. Like, it's more than you have to do to figure out, well, are they going to buy this software tool or not?

And then also, Theranos is a problem, because they were actually -- there were issues of fraud around people, things that were supposed to help people's health. And so I guess I'm just kind of, I guess, I wanted to hear a little bit more about like, because -- a lot of the response.

AR: Well, I mean, look -- let's, I mean, if you think about Theranos, where really was the fraud. The fraud like, I mean, if you read the indictment, what she's really on the hook for the biggest time is misleading investors, okay. I mean, that's the biggest part.

Yes, correct, like at the end, there was issues with the lab testing and they were getting inaccurate results because by the time she did that deal with Walgreens, and clearly they were at a point where they were desperate for showing meaningful revenue, because they need to raise more cash, right? Because they still haven't made the Edison work, right? They're trying to engineer a problem. They're working on it.

She didn't set out to commit fraud, right? She set out to build -- to stick a Lab-in-a-Box, right? But she had bigger aspirations. She wanted to take -- that she wanted to aggregate your data. She wanted to stick it in this cloud called Yoda, right? I mean, if you look today, you still have people who defend -- I mean, what's his name?

DS: Yeah.

AR: Tim Draper has really defended her. And what does he defend her on the point, and his point is that look, she had this vision to give you a movie of your health, i.e. like look, I get my blood work once a month, and that goes into a cloud, right? And my physician can track it. And I'm building a historical picture of a trend, by having more real time information on my blood work, cholesterol, everything right? So that creates preventative medicine in their view, right? Like your ability to have an earlier and more accurate versus a snapshot, right?

So he's like, look, she had that and was great and I genuinely have discussed this with James. I believe if she had IPOed this company and the company had started out as like, hey, she's got this Edison and this finger stick. It would have been just like binary tech play, right? She either makes it or she doesn't. And people would have debated that and thought that out there would been the believers. And then there would have been the skeptics, but she would have had plenty of time, as she gauged how that was working, okay?

To find something that generates revenue, which investors are willing to pay for, like with her inflated market cap on the Edison optimism, where she could just do regular lab testing like Quest and like LabCorp, but she would obviously do it at a lower price, right? But she would sell you the story that I'm going to stick this in the cloud, and you're going to pay a subscription fee, right? And that subscription fee to that cloud, Yoda, where all your information is and your general practitioner can access it and whatnot, that's the business. That's where I make money.

Of course, what's the problem with that business? And that business is -- well, I mean, that you're going to be like, well, you're losing a lot of money per test, doing your tests at a lower price than Quest and LabCorp or whatever to provide this back end service, why can't they do that, right? I mean, like, that becomes the same thing because you're going to need the same infrastructure. That's where she ran into issues. She's collecting data and she doesn't have the lab infrastructure to do it at the scale that these guys are doing.

If she'd been like these DTC companies, 23AndMe ancestry, she could have actually struck a deal, which is like, I'll be the cloud and I'll outsource the testing to them. And I will take a loss on the tests, because my investors are going to subsidize it, right? I mean, there was -- there would have been many Ways, but of course, she was also trying to kill their businesses. So it didn't work, right, with her engineering. but like, I mean, the bottom-line is, is if you look at it, like, it's something where you had a potential business model in that sense, where we would be asking the same questions that you'd ask about in detail, right? Like what are you doing that's different?

If you look at them today, Quest and LabCorp, they've entered into direct-to-consumer testing. I can go online and order my own tests and schedule the appointment and go pick them up, right? I'd like it's really, like, it's something where I don't even need to go through my medical practitioner if I want to get tested. And I don't know, to the degree I mean, I've discussed this with other people in the medical community on the testing side and I'm just, why isn't there a VIP service like, if I'm an extremely wealthy individual, where they come to my house, do the work, store in a cloud. And there's access to my blood work, on let's say -- but we don't have to do a monthly but let's say every three months, right?

These -- I mean like these are obviously options. So when you look at something like that and you see what went wrong with this company, her biggest mistake was being private. It's like -- because with her turtlenecks and Steve Jobs and Stanford dropout, I mean, the benefit of doubt, she would have gotten, if you look at the benefit of doubt, for example, that this company, Invitae has gotten. I mean, their CEO says one thing, and then he does the other three months later and nobody has cared like, at all. No one's asking questions. I mean, I don't know if you -- have you watched the CNBC, the Invitae interview with him on CNBC, Daniel?

DS: No. No, I haven't pulled that up.

AR: If you watch that, you would not understand what the company does. It's like we are the company the key opinion leaders turn to and this -- like, he does not say I'm a laboratory diagnostics genetic testing company, who derives primarily its revenue from doing these types of tests. And we're doing them at a significant discount to a competitor, who has had a monopoly in the space for ages. And we're using that to generate volume and we're hoping to parlay that into other sectors. And this is like -- this is our business model. Because to be -- to tell you the truth, if you look at this closely, I don't think they figured it out. They are trying to figure it out as they go along and that's part of the problem here.

DS: Isn't -- you said, if Theranos that they could have arguably sold tests below costs, but then put the -- like, isn't that essentially what the Invitae has? They haven't maybe laid out that vision but they're essentially selling below cost to get into -- like they could build that into the cloud sort of approach like?

AR: Okay. No, let's not make that mistake, okay? I'm saying that Theranos, if they wanted to, okay, and wanted to pivot for a story to sell, that sells well, when you're dressed like Steve Jobs, and you dropped out of Stanford, and you're a unique character and you're -- you've got a Board that has these people on it. And you've convinced Tim Draper and Larry Ellison to invest in you and whatnot, right? When you've dressed something like that, and you've ticked all those boxes, okay, you could just be like, hey, I'm going to do the same blood work everyone else is doing, I'm going to do it cheaper. So come to me. And how I'm going to make money off of it down the road, is I'm going to store that data, and it's going to give you a real time picture.

Now if you were to compare this on genetic information, my DNA isn't constantly changing, right? So if I'm doing -- if my focus is hereditary screening, i.e., what's been passed on to me, and what does that indicate?

What is the usefulness of that sitting there, right? Number one. And number two, in her case, it would be like, well, you still need to build the lab infrastructure to do the tests. You're going to have to do huge volume. So any business that -- like I mean, if you listen to the CEO, he literally sits on conference calls. And he's like, we hope to do half a million tests this year and reach a million people next year, and on our way to billions across the world. Well, what kind of infrastructure, you need Amazon infrastructure for that, right? I mean, how many geneticists do you need, genetic counselors? The industry doesn't even have the employees. I mean, we were discussing this, like, how big is that industry James.

J: It's well smaller than then I thought it was. I think it's in the thousands of genetic counselors. I forget it, 10,000 or 15,000.

AR: So you're going to need lab technicians, genetic counselors, you're going to need the physical footprint. You're going to need logistics. I mean have you looked -- like part of the thing that like -- we found kind of interesting is just look at Quest Diagnostics. I mean, there was a $100 price target slapped on this thing. That's the market cap of Quest, okay? They have 3,500 trucks like 26 planes, 6,000 patient access points, right? They have infrastructure to test everybody.

The internet bull on this stock, he's been close to management. He's done a lot of write ups on it. One of his write ups was just recently, and I read it and he's like, I visited the company and the CEO told me that they're actually paying for the trucks to go to FedEx, to pick up the samples and bring them back instead of waiting for FedEx to bring them to the lab. And he's like, I've never seen a company who cares about the customer so much. And I am like -- I mean, sorry, logistics are part of his business. Collecting the samples and the turnaround time and what the infrastructure you need to do it, right?

Like that's not something of like, hey, I really care about my customer. It's something you have to do and unfortunately, Quest and LabCorp are sitting there with huge economies of scale and scope and infrastructure and the same machines available to them, and the lab technicians and the geneticists and everything to flip this switch on, and they're not flipping it on. Why?

DS: Right.

AR: It doesn't make money, because the volume isn't significant enough and the cost isn't at that point. So when this company talks about driving down costs, no, they're not driving down costs. Everyone else has a lower cost per test already established, because they have higher volumes in the space, right? If you look at it Myriad's cost per sample is in the $140, $150 range. If you look across all these other labs, who are doing the stuff and the testing on the reproductive health they're all far lower, right?

So this is a last person in the space coming and trying to get to the volume, trying to get to the economies of scale and trying to drive it down, right? But they still are subject to the same cost infrastructure limits. It's not amazon.com. They haven't eliminated the blockbuster employees sitting, that when they're competing against in DVDs like a Netflix or an Amazon or whatnot, they haven't eliminated the huge physical retail footprint that a Barnes & Noble needed, right? Like, they still have the same limitations, from a cost standpoint. They're relying on Ilumina machines, consumables, Agilent, Read [ph] everybody -- like it's the vials from, what's the company that sells the vials?

J: OraSure

AR: OraSure, right, like you're buying the same stuff from the same people. So it's when you look at it from that standpoint, like if they were to sell you a story about data or whatever, it's like, well, everybody else can sell us a story about data. Why are they selling it to us?

J: It's an interesting dynamic here, because when there was the rebuttal by this bullish commentator/endorsed analyst of the company, there was a comment that, hey, it's not just the raw data, it's not just the genetic information that is useful, because genetic information in and of itself doesn't tell you enough about the disease. And I think that's a partial indictment of the whole process. But more importantly, what the analyst said is, what the company does is they take that information and they combine it with a patient's medical record. That includes all of their scans, their CTs, their MRIs, all their historical blood tests, all their physical exams, and then it takes that data. And if you get enough of that data, then you can start running effectively very large statistical relationships and figure out, okay, which genetic mutations might be associated with which diseases.

The problem with that is, as far as we know, that's not what's happening. And yet the company, again, they've kind of endorsed this, this guy is an analyst of record, the company hasn't come out and said that. But that's not what's happening. Because if it is what is happening, I think they're serious privacy concerns. So I think it's very different, if you as patients and [indiscernible] into 23AndMe, you might sign a paper, some paperwork somewhere, but if they're actually signing away the rights to all their medical records. Again, I don't believe they are, but again, this is what would need to happen in order for this data to be proprietary and useful. Then I would imagine the consumers are not aware of that.

And so you kind of have this catch 22, if you're getting the data, whatever, this guy refers to it as the golden data, whatever it is, if you're getting that data, such that it's useful, you're probably in violation of some privacy laws, whether or not you are in terms of you're covered legally, I think just patients don't understand that's what's happening. And if you're not getting that data, then there's a very real question as to what exactly is the use of just this genetic information without putting it into -- in context without kind of correlating it to these other disease markers?

And again, this is a question that could -- that's very answerable by the company as far as we know, has decided not to answer.

J: I mean, look, there's also two elements of that, right. If you remember also, in his rebuttal, he pointed out to a subscription model, right ,where he was even saying that this should be looked at from a dollar-based retention standpoint like a SaaS company. So I mean, again, if he's saying this, it's something that well was spoon fed to him, okay? And that's part of the element here, when you're dealing with something like this, and you look at that, it's like, all right, so like, I go in, I do a test for cancer, but you know, a BRCA screen. And you're saying -- are you essentially saying that, in year one, you generated revenue off of the actual testing, but then in year two, and year three, supposedly, my DNA is something that just sits there, and they can find ways to make money off of, by farming it for some sort of data that they can sell to pharma.

And it's tenuous at best to even understand how that model would work, because if you look at the rest of the industry, you just have to assume, they're all idiots. I mean, how many tests has Myriad done? I mean, look, when you go back to this thesis, Daniel, one of the most important things here is, when I put this on, this company was bigger than Myriad, literally in enterprise value, it was bigger than a company with $850 million in revenue, 20 years of testing. They've done 6 million tests through some -- to that effect I think it is at this point.

They have a database that they've made a trade secret since 2004, as far as variant data. They have four times the employees of Invitae. I mean, if you were to look at this company from there -- one notable institutional bull in the space, like this bull doesn't own any Myriad, okay. And they have a genetic spawn, and they did like kind of throw like a little bit of a shade at this thesis when it came out. And I mean, I can imagine they got a lot of questions because they own a lot of the stock and they were like, these are the companies leading in AI.

And then they like -- they listed Invitae like two other names. And then they put in their tweet, which was almost essentially directed, not my gen [ph], which I mean, for someone like me, I just -- I didn't even really spend much time getting into the AI nonsense. But if you look at Myriad how many people with machine learning backgrounds and data scientists do they employ, plenty.

You could just go on LinkedIn, look at it, and draw your conclusion. But they're not out running around saying, hey, we got AI, we're doing stuff. We're literally running machine learning models on your DNA. We're figuring out better ways, a secret sauce. Like who would advertise that? If you actually have made the data a trade secret, and you refuse to share it and they've gotten a lot of heat for it, literally the whole industry had to band together to contribute data freely to Clinvar, because Myriad won't share, because they're like, hey, fine, you took away our patent, but who cares about that. Our ability to interpret this is better than everybody else at this juncture.

So again, you look at it, at something like that, and you're just like, well, there's companies who've been doing this for decades. And you're just supposed to assume that like data science is completely irrelevant to them. But the company that acquired an AI startup in July, by September is a leader in this space. I mean what, you know.

DS: Solet me -- so there are a few directions to go here. But let's quickly touch on Myriad. It's -- you're using it as a payer here, as I think you mentioned, it's -- short sellers have kind of had their eyes on it. Somebody like Southern Investigative Reporting Foundation has reported about it. Why are you comfortable with that as the other side of this trade given the fact that they've also come under fire?

AR: I mean, I don't know James, you want to tackle this? I'm obviously a lot more bullish on Myriad then most people. I would say, like, I can't really get my head around the short thesis, and I can't get my head around the short thesis in a relative context. I mean, if you've looked at the space there are some companies with some pretty crazy valuations. Myriad is not a hard business to understand. They have a cash cow in hereditary cancer. They've used that to diversify into companion diagnostics, into carrier screening, now into these pharmaco-genetic tests, psychotropic like for depression, which is a very controversial area.

A lot of a lot of volatility around Myriad lately, let's say the last six months, has been tied to this gene site division, and the way the FDA wants to treat these tests, where I get a DNA test that like tells me, I'm more tolerant for Zoloft over Prozac. And no science has shown any clinical efficacy yet, and it's a controversial area, because there's obviously some doctors, and they've been doing -- they've been running clinical studies to try to get this approved. And they missed the primary endpoints on it. But there's also an argument that in depression, which is like opioids, a national crisis, essentially speaking, and it's not going to get better, that there's nothing, and something is better than nothing. I mean, two-thirds of antidepressants are prescribed by general practitioners. Not -- you're not talking about the psychiatry side here. These are not experts on these drugs or on mental health.

And the argument is that maybe you give them something that starts this out with a little bit more direction, however little incremental it is. Now when the stock got hammered in the summer, on its last earnings, was because they said the FDA is pushing back on them on the labeling. And then recently, there was another genetic psychotropic-related test company where the FDA allowed them to resume sending the test information, but to the doctors. So the patient just gets this, like here's what your genetics say, but nothing about the drugs. But the doctor actually gets the drug indications. And then that doctor can see that and that doctor can use that as part -- like as a helpful part of his treatment.

But again, you go back to -- they haven't been able to show scientifically and it's hotly debated. I mean, there was just recently something in -- two Harvard doctors had published something in one of the big journals on mental health, same thing with oncology, like outside of BRCA, like there was a recent paper basically like these other genes are like no better than a placebo.

So this is part of the problem in the space but I mean, I haven't -- I don't have the numbers in front of me. I mean, James do you remember off the top of your head, but I mean, I think it's like $850 million in revenue and like $150 million in EBITDA, something like that against a company with -- that did what, like $144 million in revenue last year and lost what $100 million.

J: Yeah, but larger now.

DS: Yeah, $850 million, trailing 12 months revenue, looks like EBITDA of over $100 million at least.

AR: Yeah. So like, you can look at that, I mean, and this is something when you look at stocks, I mean I was long, some Pinterest against the Snapchat short. And I thought about closing it before earnings. And the reason I thought about closing it is that you know, Snap is $16 billion and Pinterest is 15 billion and Twitter after its 35% decline is 18.2 billion AV. Yeah, Twitter's growing slower than the other two. But it's like three to four times the revenue base. And you got to kind of adjust for that, when you get to that point.

And you're like, this is a company that is in the advertising space, and it's doing -- it's going to do whatever issues its got, it's going to do 3.5 times what this is going to do and their enterprise values are a hair apart. So when you -- like this wasn't -- this is a case where if you would look at an Invitae, you'd be like, this has got to be like a quarter a fifth of the size of like, even if you are a believer and you're willing to buy into the speculation of a Myriad.

And then the other problem you have with it is that they're interrelated. All the revenue growth that is coming for Invitae is coming because Myriad hasn't come down in pricing. They've been fighting this price decline, because they've had the luxury to fight it as the leader in the space and they're extracting a premium for their testing, because they -- like there's a compelling argument, at least from their end, that based on the data we have in the history, our tests can more accurately predict what you have, as far as a likelihood of a hereditary cancer and indication reliably.

And it's ironic, and we were discussing this when we were working on this, like this company throw shade at who -- I mean, they throw shade at the DTC companies. Like they literally just gave a scientific presentation at this Houston Cancer Conference with Genetics or whatever, just like two weeks ago, where they were like, here's 23andMe's tests, okay, and this is what's wrong with it. Like 23AndMe gives you a BRCA test, that only is designed to detect three variants. Basically, if you're not an Ashkenazi Jew, it's useless.

But it's literally a report that is bundled in with the ancestry, with the health with the 50, 60, 70 reports, you get for $100 okay. It's not like you're going into buy this or you're going to your doctor and you're like, okay, I had breast cancer, I'm worried about a potential recurrence. Let's see family history. Do I need to get a mastectomy early because I have this mutation and that's a good preventative measure, et cetera, et cetera. They're not looking at 23AndMe. Like, it's -- you're competing in the clinical grade medical diagnostics market. But here's this company attacking the DTC companies who are not really their competitors.

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Genome editing: a broad perspective on a precision technology – PHG Foundation

Posted: November 20, 2019 at 1:45 pm

Genome editing goes prime-time

Genome editing was in the news again recently, following the publication of a paper that describes prime-editing, a new method of altering genetic information. Whilst more information is needed to accurately assess the true potential of this technique, prime-editing does represent another addition to the ever-growing suite of genome editing tools many of which will have an impact on treatment approaches to genetic diseases. In our latest set of policy briefings, we outline the current state of somatic genome editing in health, discussing areas of greatest progress, limitations and the evolving regulatory landscape and ethical considerations surrounding its application.

Genome editing describes a suite of techniques that can be used to alter genetic sequences. These all use a cutting enzyme and a targeting mechanism to make cuts and induce repair in DNA, altering the genetic sequence in the process. CRISPR-Cas has received the greatest attention in recent years, owing to its precision and relative ease of use.

The new technique prime-editing differs from standard CRISPR-Cas by making a cut in only one of the two DNA strands and using different methods to induce repair and add new information to the DNA. The technique could potentially be used to make alterations in genomic regions which preceding techniques cannot. However, it also has its limitations.

The hype surrounding both somatic and germline genome editing has reflected the general excitement and rapid progress in a field that stole the spotlight in 2012, thanks to the arrival of CRISPR-Cas9 for DNA editing. While germline editing is still being hotly debated, and dominates the headlines, greater awareness is needed of the current scope and applicability of somatic genome editing for health, and the broader progress that is being made in addressing genetic disease. The focus on human germline editing has somewhat obscured the impacts of somatic gene editing on individuals and populations.

Several therapies based on genome editing techniques in somatic cells have now made their way into clinical trials, including several therapies for rare blood disorders such as sickle cell disease and retinal disorders such as Leber congenital amaurosis. Genome editing is also having an impact on therapies and diagnostics beyond its direct applications for rare disease; for instance, CAR-T therapies a type of immunotherapy for blood cancer and in research into cancer diagnostics that utilise CRISPR for detecting the presence of genetic variants.

Like all disruptive technologies, somatic genome editing poses legal and ethical challenges, which should be considered alongside the potentially life-changing benefits for patients. Although somatic editing physically impacts only the individual whose cells are edited, its use could still have wide-reaching societal implications. For example, genome editing in one individual will inevitablyhave an impact upon other patients may be stigmatised or discriminated against if they are unable or unwilling to recieve the same therapy. The circumstances in which genome editing is used will also have an impact upon public views of the technology, as studies show that whilst there is widespread support for genome editing for therapeutic applications, this does not extend to non-therapeutic use. High-profile cases may further influence this view one way or another.

Although the drive to develop genome editing comes predominantly from therapeutic need, these techniques could theoretically be put to non-therapeutic use i.e. enhancement. Much policy and public discussion assumes a meaningful distinction between the two. But in recent years, as preventative health has taken centre stage, this distinction has become harder to maintain; medicine is not just about treating acute disease, its about identifying and reacting to risk. Is editing out a mutation that carries an increased risk of developing cardiovascular disease a therapeutic intervention? Navigating this blurred line is hugely debated, and societal debate on such issues will become more important as the associated risks and benefits become clearer, influencing how genome editing might be used in the future.

Whilst momentum is building around the use of genome editing technologies, the significant journey from the bench to the bedside is often underestimated, and it can be easy to forget that current developments are built on decades of contributory research in related fields. Patients are beginning to see benefits from genome editing, but use is currently limited to a small number of rare disease and cancer patients taking part in clinical trials. However, looking to the future, new techniques such as prime-editing and base-editing could expand the applications of genome editing. There is much excitement and optimism among researchers about these techniques, but this is only the beginning in terms of exploring how they could practically be used. In time, these techniques might be used to develop new therapies, once their safety and efficacy have been established and technical obstacles such as how to deliver the editing tools to cells have been overcome. A further challenge will be to ensure that the regulatory landscape is sufficiently robust and dynamic to be able to accommodate these novel technologies.

For more information, see our policy briefings:

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BT, UHB demonstrate 5G-connected ambulance in Birmingham – Government Computing Network

Posted: November 20, 2019 at 1:45 pm

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BT and the University Hospitals Birmingham NHS Foundation Trust (UHB) have entered into a partnership to demonstrate a remote diagnostic procedure using a 5G-connected ambulance in Birmingham.

BT said that the technology has the capability to transform healthcare services in the UK and bring about major cost-savings by reducing the number of patients being admitted to hospitals.

The demonstration follows the launch of BTs 5G network in Birmingham earlier in 2019.

It also follows the UKs first demonstration of a remote-controlled ultrasound scan over the 5G network in June.

The presentation was hosted at the Medical Devices Testing and Evaluation Centre (MD-TEC) in UHBs simulation lab located in the Institute of Translational Medicine.

The demonstration involved a 5G-connected ambulance, which was provided by South Central Ambulance NHS Foundation Trust, to allow remote diagnostics by connecting practitioners in the field with consultants or surgeons in real-time.

It showed a paramedic functioning from an ambulance in the Edgbaston area of the city being linked over the 5G network to a clinician based over two miles away at MD-TEC.

UHB chief executive Dave Rosser said: We are excited by the huge potential of 5G technology and how it can help transform healthcare in the future. We believe it has the potential to create more efficient use of healthcare resources, particularly with regards to easing the burden on A&E services which are facing unprecedented demand.

UK-based telecommunications company BT has worked with its research partners, including Ericsson and Kings College London, as well as Voysys to showcase how 5G technology can offer digital transformation of critically important services and facilitate real-time collaboration. It also shows the potential of 5G beyond enhanced mobile broadband.

BT Enterprise CTIO and 5G executive lead Fotis Karonis said: Not only is 5G capable of ultrafast speeds it has much lower latency meaning there is little to no delay when transmitting data over the network.

This means things happen in real time so this is of significant interest to the NHS because of its potential for medical applications, such as diagnostics and preventative healthcare. This capability provides efficiency opportunities for both hospital and ambulance trusts by reducing the number of referrals into hospital and patient trips.

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Company claims signs of success with CRISPR-edited stem cell transplants for two genetic diseases – Science Magazine

Posted: November 19, 2019 at 10:52 pm

By Jon CohenNov. 19, 2019 , 5:40 PM

Companies and academic groups have begun to use the genome editor CRISPR to try to correct various diseases, but none has so far published, or even announced, evidence that patients have been helpeduntil this week. In an update for investors today, CRISPR Therapeutics of Zug, Switzerland, reported that one patient with sickle cell anemia and another with beta thalassemia appear to have benefited from the same CRISPR-based intervention for up to 9 months, STAT reports. (The company gave STAT an early look at the data but did not allow outside commenters to see the results.) Before the treatment, both patients required multiple infusions each year of red blood cells. CRISPR Therapeutics, collaborating with Vertex Pharmaceuticals, removed blood stem cells from their bodies and modified them with CRISPR to knock out a gene that shuts down production of fetal hemoglobin. When the edited cells were put back in each patients body through a stem cell transplantwhich required a toxic chemotherapy to kill their own stem cellsboth people produced high levels of fetal hemoglobin and no longer needed transfusions. These early findings are a triumph for patients, CRISPR, and genetics, Bruce Conklin, a clinician who focuses on stem cell treatments of cardiovascular disease at the University of California, San Francisco, told Science after reviewing the limited data the companies provided in the call and a press release. They chose patients with severe versions of the disease and the results are truly remarkable. Other companies are working on competing gene therapy and gene-editing technologies for the same disorders.

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Cellect Biotechnology Reports Third Quarter 2019 Financial and Operating Results – BioSpace

Posted: November 19, 2019 at 10:52 pm

TEL AVIV, Israel, Nov. 19, 2019 /PRNewswire/ --Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of innovative technology which enables the functional selection of stem cells, today reported financial and operating results for the third quarter ended September 30, 2019 and provided a corporate update.

Recent Highlights

"Our clinical and regulatory teams remained focused during the third quarter and the more recent positive developments position us to achieve our goals, both in the U.S. and Israel," commented Dr. Shai Yarkoni, Chief Executive Officer. "In the U.S., the IND approval is a significant achievement and represents our first-ever FDA IND in the U.S., with Washington University School of Medicine. In Israel, our Phase 1/2 clinical study of ApoGraft is progressing slowly and we expect to complete the recruitment around the end of the year."

"With our prudent use of cash during the third quarter and the anticipated cash usage needs over the coming quarters, we continue to believe we have the resources to execute our clinical and regulatory plans for the foreseeable future," said Eyal Leibovitz, Chief Financial Officer.

ThirdQuarter 2019 Financial Results:

*For the convenience of the reader, the amounts above have been translated from NIS into U.S. dollars, at the representative rate of exchange on September 30, 2019 (U.S. $1 = NIS 3.482).

Strategic Review Progress Update

On May 16, 2019, the Company disclosed that it commenced plans to explore strategic alternatives to maximize shareholder value. Potential strategic alternatives that may be evaluated include, but are not limited to, an acquisition, merger, business combination, including in other business fields than the Company's in-licensing, or other strategic transaction involving the Company or its assets. The Company continues to evaluate business development opportunities and will keep investors informed as they mature or warrant investor disclosure.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (APOP) has developed a breakthrough technology, for the selection of stem cells from any given tissue, that aims to improve a variety of stem cell-based therapies.

The Company's technology is expected to provide researchers, clinical community and pharma companies with the tools to rapidly isolate stem cells in quantity and quality allowing stem cell-based treatments and procedures in a wide variety of applications in regenerative medicine. The Company's current clinical trial is aimed at bone marrow transplantations in cancer treatment.

Forward Looking Statements

This press release contains forward-looking statements about the Company's expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as "believe", "expect", "intend", "plan", "may", "should", "could", "might", "seek", "target", "will", "project", "forecast", "continue" or "anticipate" or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. These forward-looking statements and their implications are based on the current expectations of the management of the Company only and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: the Company's history of losses and needs for additional capital to fund its operations and its inability to obtain additional capital on acceptable terms, or at all; the Company's ability to continue as a going concern; or maintain its current operations; uncertainties involving any strategic transaction the Company may decide to enter into as the result of its current efforts to explore new strategic alternatives; uncertainties of cash flows and inability to meet working capital needs; the Company's ability to obtain regulatory approvals; the Company's ability to obtain favorable pre-clinical and clinical trial results; the Company's technology may not be validated and its methods may not be accepted by the scientific community; difficulties enrolling patients in the Company's clinical trials; the ability to timely source adequate supply of FasL; risks resulting from unforeseen side effects; the Company's ability to establish and maintain strategic partnerships and other corporate collaborations; the scope of protection the Company is able to establish and maintain for intellectual property rights and its ability to operate its business without infringing the intellectual property rights of others; competitive companies, technologies and the Company's industry; unforeseen scientific difficulties may develop with the Company's technology; and the Company's ability to retain or attract key employees whose knowledge is essential to the development of its products. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2018 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov, and in the Company's periodic filings with the SEC.

Cellect Biotechnology Ltd

Consolidated Statement of Operation

Convenience

translation

Nine months

ended

Nine months ended

Three months ended

September 30,

September 30,

September 30,

2019

2019

2018

2019

2018

Unaudited

Unaudited

U.S. dollars

NIS

(In thousands, except share and per

share data)

Research and development expenses

2,743

9,551

9,473

2,465

4,125

General and administrative expenses

2,249

7,832

11,001

2,768

3,929

Operating loss

4,992

17,383

20,474

5,233

8,054

Financial expenses (income) due towarrants exercisable into shares

(2,303)

(8,020)

(2,935)

(910)

(1,320)

Other financial expenses (income), net

393

1,369

(1,177)

489

64

Total comprehensive loss

3,082

10,732

16,362

4,812

6,798

Loss per share:

Basic and diluted loss per share

0.015

0.051

0.127

0.021

0.052

Basic and diluted loss per ADS

0.30

1.02

2.54

0.42

1.04

Weighted average number of sharesoutstanding used to compute basic anddiluted loss per share

208,771,303

208,771,303

129,139,278

224,087,799

130,192,799

Cellect Biotechnology Ltd.

Consolidated Balance Sheet Data

ASSETS

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These Scientists May Have Found a Cure for ‘Bubble Boy’ Disease – Smithsonian.com

Posted: November 19, 2019 at 10:52 pm

On the morning of April 25, 2018, in Fort Wayne, Indiana, Omarion Jordan came into the world ten-fingers-and-toes perfect. His mother, Kristin Simpson, brought her dark-haired newborn home to a mostly empty apartment in Kendallville, about 30 miles to the north. Shed just moved in and hadnt had time to decorate. Her son, however, had everything he needed: a nursery full of toys, a crib, a bassinet and a blue octopus blanket.

Still, within his first couple of months, he was plagued by three different infections that required intravenous treatments. Doctors thought he had eczema and cradle cap. They said he was allergic to his mothers milk and told her to stop breastfeeding. Then, not long after he received a round of standard infant vaccinations, his scalp was bleeding and covered with green goop, recalled the first-time mother, who was then in her late teens. She took him to the hospital emergency room, where, again, caregivers seemed puzzled by the babys bizarre symptoms, which didnt make any sense until physicians, finally, ordered the right blood test.

What they learned was that Omarion was born with a rare genetic disorder called X-linked severe combined immunodeficiency (SCID), better known as the bubble boy disease. Caused by a mutated gene on the X chromosome, and almost always limited to males, a baby born with X-linked SCID, or SCID-X1, lacks a working immune system (hence the unusual reaction to vaccination). The bubble boy name is a reference to David Vetter, a Texas child born with SCID-X1 in 1971, who lived in a plastic bubble and ventured out in a NASA-designed suit. He died at 12, but his highly publicized life inspired a 1976 TV movie starring John Travolta.

Today, technological advances in hospitals provide a kind of bubble, protecting SCID-X1 patients with controlled circulation of filtered air. Such safeguards are necessary because a patient exposed to even the most innocuous germs can acquire infections that turn deadly. As soon as Omarion tested positive for the disorder, an ambulance carried him to Cincinnati Childrens Hospital in nearby Ohio and placed him in isolation, where he remained for the next few months. I had no idea what would happen to him, his mother recalled.

Approximately one in 40,000 to 100,000 infants is born with SCID, according to the Centers for Disease Control and Prevention. Only about 20 to 50 new cases of the SCID-X1 mutationwhich accounts for about half of all SCID casesappear in the United States each year. For years, the best treatments for SCID-X1 have been bone marrow or blood stem cell transplantations from a matched sibling donor. But fewer than 20 percent of patients have had this option. And Omarion, an only child, was not among them.

As it happened, medical scientists at St. Jude Childrens Research Hospital in Memphis, Tennessee, were then developing a bold new procedure. The strategy: introduce a normal copy of the faulty gene, designated IL2RG, into a patients own stem cells, which then go on to produce the immune system components needed to fight infection. Simpson enrolled Omarion in the clinical study and Cincinnati Childrens Hospital arranged a private jet to transport her and her son to the research hospital, where they stayed for five months.

St. Jude wasnt the first to try gene therapy for SCID-X1. Nearly 20 years ago, researchers in France reported successfully reconditioning immune systems in SCID-X1 patients using a particular virus to deliver the correct gene to cells. But when a quarter of the patients in that study developed leukemia, because the modified virus also disrupted the functioning of normal genes, the study was halted and scientists interested in gene therapy for the disorder hit the brakes.

At St. Jude, experts led by the late Brian Sorrentino, a hematologist and gene therapy researcher, set out to engineer a virus delivery vehicle that wouldnt have side effects. They started with a modified HIV vector emptied of the virus and its original contents, and filled it with a normal copy of the IL2RG gene. They engineered this vector to include insulators to prevent the vector from disturbing other genes once it integrated into the human genome. The goal was to insert the gene into stem cells that had come from the patients own bone marrow, and those cells would then go on to produce working immune system cells. It was crucial for the viral vector to not deliver the gene to other kinds of cellsand thats what the researchers observed. After gene therapy, for example, brain cells do not have a correct copy of the gene, explained Stephen Gottschalk, who chairs St. Judes Department of Bone Marrow Transplantation and Cellular Therapy.

In the experimental treatment, infants received their re-engineered stem cells just 12 days after some of their bone marrow was obtained. They went through a two-day, low-dose course of chemotherapy, which made room for the engineered cells to grow. Within four months, some of the babies were able to fight infections on their own. All eight of the initial research subjects left the hospital with a healthy immune system. The remarkably positive results made news headlines after being published this past April in the New England Journal of Medicine. Experimental gene therapy frees bubble boy babies from life of isolation, the journal Nature trumpeted.

So far, the children who participated in that study are thriving, and so are several other babies who received the treatmentincluding Omarion. As a physician and a mom, I couldnt ask for anything better, said Ewelina Mamcarz, lead author of the journal article and first-time mother to a toddler nearly the same age as Omarion. The children in the study are now playing outside and attending day care, reaching milestones just like my daughter, Mamcarz says. Theyre no different. Mamcarz, who is from Poland, came to the United States to train as a pediatric hematologist-oncologist and joined St. Jude six years ago.

Other medical centers are pursuing the treatment. The University of California, San Francisco Benioff Childrens Hospital is currently treating infant patients, and Seattle Childrens Hospital is poised to do the same. Moreover, the National Institutes of Health has seen success in applying the gene therapy to older patients, ages 3 to 37. Those participants had previously received bone marrow transplants from partially matched donors, but theyd been living with complications.

In the highly technical world of medicine today, it takes teamwork to achieve a breakthrough, and as many as 150 peoplephysicians, nurses, regulators, researchers, transplant coordinators and othersplayed a role in this one.

Sorrentino died in November 2018, but hed lived long enough to celebrate the trial results. In the early 90s, we thought gene therapy would revolutionize medicine, but it was kind of too early, said Gottschalk, who began his career in Germany. Now, nearly 30 years later, we understand the technology better, and its really starting to have a great impact. We can now develop very precise medicine, with very limited side effects. Gottschalk, who arrived at St. Jude a month before Sorrentinos diagnosis, now oversees the hospitals SCID-X1 research. Its very, very gratifying to be involved, he said.

For now the SCID-X1 gene therapy remains experimental. But with additional trials and continued monitoring of patients, St. Jude hopes that the therapy will earn Food and Drug Administration approval as a treatment within five years.

Simpson, for her part, is already convinced that the therapy can work wonders: Her son doesnt live in a bubble or, for that matter, in a hospital. He can play barefoot in the dirt with other kids, whatever he wants, because his immune system is normal like any other kid, she said. I wish there were better words than thank you.

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These Scientists May Have Found a Cure for 'Bubble Boy' Disease - Smithsonian.com

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Vertex and Molecular Templates Establish Collaboration to Discover and Develop Novel Targeted Conditioning Regimens to Enhance Hematopoietic Stem Cell…

Posted: November 19, 2019 at 10:52 pm

DetailsCategory: More NewsPublished on Tuesday, 19 November 2019 12:39Hits: 258

-Molecular Templates to receive $38 million upfront payment, including equity investment, with potential for additional milestone and royalty payments on future sales-

BOSTON, MA and AUSTIN, TX, USA I November 18, 2019 I Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Molecular Templates, Inc. (Nasdaq: MTEM; Molecular Templates or MTEM) today announced that the two companies have entered into a strategic research collaboration to discover and develop novel targeted conditioning regimens that may enhance the hematopoietic stem cell transplant process, including transplants conducted as part of treatment with ex vivo CRISPR/Cas9 gene editing therapies such as CTX001. CTX001 is currently being evaluated in two ongoing Phase 1/2 studies in patients with transfusion-dependent beta thalassemia and severe sickle cell disease, where a hematopoietic stem cell transplant is required as part of treatment with CTX001. The collaboration will seek to discover a new conditioning regimen utilizing MTEMs engineered toxin body (ETB) platform, which is designed to specifically target and remove specific cells to enable successful engraftment of new cells.

We believe that gene editing holds significant promise in the treatment of severe hemoglobinopathies such as sickle cell disease and beta thalassemia, and Molecular Templates unique technology platform could play an important role in creating a targeted conditioning regimen that could replace chemotherapy currently required in conditioning regimens and thus enhance the overall future treatment experience for patients, said David Altshuler M.D., Ph.D., Executive Vice President and Chief Scientific Officer at Vertex.

Vertex has a proven history of employing novel technologies to develop innovative medicines for diseases with high unmet medical needs, making them an ideal partner to expand the use of MTEMs ETB platform for therapeutics outside of oncology, said Eric Poma, Ph.D., Molecular Templates Chief Executive and Scientific Officer. MTEM is excited to be working with Vertex to focus on discovering and developing the next generation of targeted conditioning agents. This collaboration further validates the potential of our ETB platform and provides meaningful capital to support continued advancement of our own product pipeline.

Under the collaboration, MTEM will conduct research activities for the use of ETBs for up to two targets selected by Vertex. The initial research will be focused on discovering a novel conditioning regimen using MTEMs ETB technology platform. In addition, Vertex has an option to select a second target as part of the collaboration. Upon designation of a clinical development candidate that emerges from the research efforts, Vertex has the option to exclusively license molecules against the designated target. Vertex will have exclusive rights to develop molecules that emerge from the research program for any indication.

Vertex will make an up-front payment of $38 million to MTEM, including an equity investment. MTEM is also eligible to receive future development, regulatory and sales milestones and option payments of up to $522 million (across two targets) and tiered royalty payments on future sales.

About Molecular TemplatesMolecular Templates is a clinical-stage oncology company focused on the discovery and development of differentiated, targeted, biologic therapeutics for cancer. We believe our proprietary biologic drug platform technology, referred to as engineered toxin bodies, or ETBs, provides a differentiated mechanism of action that may address some of the limitations associated with currently available cancer therapeutics. ETBs utilize a genetically engineered form of Shiga-like Toxin A subunit, or SLTA, a ribosome inactivating bacterial protein, that can be targeted to specifically destroy cancer cells. Additional information about Molecular Templates can be obtained at http://www.mtem.com.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

Founded in 1989 inCambridge, Mass., Vertex's global headquarters is now located inBoston's Innovation Districtand its international headquarters is inLondon, UK. Additionally, the company has research and development sites and commercial offices inNorth America,Europe,AustraliaandLatin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years onSciencemagazine's Top Employers list and top five on the 2019 Best Employers for Diversity list byForbes. For company updates and to learn more about Vertex's history of innovation, visitwww.vrtx.comor follow us onFacebook, Twitter, LinkedIn,YouTubeand Instagram.

SOURCE: Vertex Pharmaceuticals

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Vertex and Molecular Templates Establish Collaboration to Discover and Develop Novel Targeted Conditioning Regimens to Enhance Hematopoietic Stem Cell...

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Bone marrow transplant: What it is, uses, risks, and recovery – Medical News Today

Posted: November 19, 2019 at 10:52 pm

Bone marrow is soft, spongy tissue within some bones, including those in the hips and thighs. People with certain blood-related conditions benefit from a transplant that replaces damaged cells with healthy cells, possibly from a donor.

Bone marrow transplants can be lifesaving for people with conditions such as lymphoma or leukemia, or when intensive cancer treatment has damaged blood cells.

This type of transplant can be an intensive procedure, and recovery can take a long time.

Here, we provide an overview of bone marrow transplants, including their uses, risks, and recovery.

Bone marrow contains stem cells. In healthy people, stem cells in bone marrow help create:

If a medical condition such as one that damages the blood or immune system prevents the body from creating healthy blood cells, a person may need a bone marrow transplant.

A person with any of the following conditions may be a candidate for a bone marrow transplant:

There are three types of bone marrow transplant, based on where the healthy bone marrow cells come from.

In many cases, the donor is a close family member, such as a sibling or parent. The medical name for this is an allogenic transplant.

Transplants are more likely to be effective if the donated stem cells have a similar genetic makeup to the person's own stem cells.

If a close family member is not available, the doctor will search a registry of donors to find the closest match. While an exact match is best, advances in transplant procedures are making it possible to use donors who are not an exact match.

In a procedure called an autologous transplant, the doctor will take healthy blood stem cells from the person being treated and replace these cells later, after removing any damaged cells in the sample.

In an umbilical cord transplant, also called a cord transplant, doctors use immature stem cells from the umbilical cord following a baby's birth. Unlike cells from an adult donor, the cells from an umbilical cord do not need to be as close a genetic match.

Before a bone marrow transplant, the doctor will run tests to determine the best type of procedure. They will then locate an appropriate donor, if necessary.

If they can use the person's own cells, they will collect the cells in advance and store them safely in a freezer until the transplant.

The person will then undergo other treatment, which may involve chemotherapy, radiation, or a combination of the two.

These procedures typically destroy bone marrow cells as well as cancer cells. Chemotherapy and radiation also suppress the immune system, helping to prevent it from rejecting a bone marrow transplant.

While preparing for the transplant, the person may need to stay in the hospital for 12 weeks. During this time, a healthcare professional will insert a small tube into one of the person's larger veins.

Through the tube, the person will receive medication that destroys any abnormal stem cells and weakens the immune system to prevent it from rejecting the healthy transplanted cells.

Before entering the hospital, it is a good idea to arrange:

A bone marrow transplant is not surgery. It is similar to a blood transfusion.

If a donor is involved, they will provide the stem cells well in advance of the procedure. If the transplant involves the person's own cells, the healthcare facility will keep the cells in storage.

The transplant typically takes place in several sessions over several days. Staggering the introduction of cells in this way gives them the best chance of integrating with the body.

The healthcare team may also use the tube to introduce liquids such as blood, nutrients, and medications to help fight infection or encourage the growth of bone marrow. The combination depends on the body's response to treatment.

The procedure will temporarily compromise the person's immune system, making them very susceptible to infection. Most hospitals have a dedicated, isolated space for people undergoing bone marrow transplants to help reduce their risk of infection.

After the last session, the doctor will continue to check the blood each day to determine how well the transplant has worked. They will test whether new cells are beginning to grow in bone marrow.

If a person's white blood cell count starts to rise, it indicates that the body is starting to create its own blood, indicating that the transplant has been successful.

The amount of time that it takes for the body to recover depends on:

Many other factors can affect recovery, including:

Some people are able to leave the hospital soon after the transplant, while others need to stay for several weeks or months.

The medical team will continue to monitor the person's recovery for up to 1 year. Some people find that effects of the transplant remain for life.

A bone marrow transplant is a major medical procedure. There is a high risk of complications during and after it.

The likelihood of developing complications depends on various factors, including:

Below are some of the more common complications that people who receive bone marrow transplants experience:

Some people die as a result of complications from bone marrow transplants.

A person who receives a bone marrow transplant may also experience reactions that can follow any medical procedure, including:

The body's response to a bone marrow transplant varies greatly from person to person. Factors such as age, overall health, and the reason for the transplant can all affect a person's long term outlook.

If a person receives a bone marrow transplant to treat cancer, their outlook depends, in part, on how far the cancer has spread. Cancer that has spread far from its origin, for example, responds less well to treatment.

According to the National Marrow Donor Program, the 1-year survival rate among people who have received transplants from unrelated donors increased from 42% to 60% over about the past 5 years.

A bone marrow transplant is a major medical procedure that requires preparation. This involves determining the best type of transplant, finding a donor, if necessary, and preparing for a lengthy hospital stay.

The time that it takes for the body to recover from a transplant varies, depending on factors such as a person's age and overall health and the reason for the transplant.

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Bone marrow transplant: What it is, uses, risks, and recovery - Medical News Today

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Nanotechnology for disease diagnosis and treatment earns Florida Poly professor international award – Yahoo Finance

Posted: November 19, 2019 at 10:50 pm

Florida Poly assistant professor Dr. Ajeet Kaushik has received the 2019 USERN Prize in biological sciences, an international award recognizing his work in the field of nanomaterials for the detection and treatment of diseases.

LAKELAND, Fla., Nov. 18, 2019 /PRNewswire-PRWeb/ -- Dr. Ajeet Kaushik is determined to make detecting and treating diseases easy, accessible, and precise through the use of nanomaterials for biosensing and medicine.

His extensive work and resolute desire to improve the delivery of healthcare has earned Kaushik the prestigious Universal Scientific Education Research Network (USERN) Prize. He was named a laureate in the field of biological sciences during the group's fourth annual congress on Nov. 8 in Budapest, Hungary.

USERN, a non-governmental, non-profit organization and network dedicated to non-military scientific advances, is committed to exploring science beyond international borders.

"I was speechless for a while," said Kaushik, who is an assistant professor of chemistry at Florida Polytechnic University.

Kaushik did not attend the awards ceremony in person but did submit a video to be played at the event. He was among hundreds vying for the prize and one of five people who were recognized in different areas of study.

His submitted project, Nano-Bio-Technology for Personalized Health Care, focuses on using nanomaterials to create biosensors that will detect the markers of a disease at very low levels.

"Biosensing is not a new concept, but now we are making devices that are smarter and more capable," Kaushik said.

He cited the recent zika virus epidemic that affected pregnant women and their fetuses, leading to significant health complications upon birth. "There was a demand to have a system that could detect the virus protein at a very low level, but there was no device. There was no diagnostic system," he said.

Kaushik worked on the development of a smart zika sensor that could detect the disease at these low levels. "The kind of systems I'm focusing on can be customized in a way that we carry like a cell phone and do the tests wherever we need to do them," he said.

In addition to using nanotechnology for the detection of diseases like zika, his research on nanoparticles is advancing efforts to precisely deliver medicine to a specific part of the body without affecting surrounding tissue or other parts of the body.

"The drugs we use now do not go only where they need to go, or sometimes they have side effects. We are treating one disease but creating other symptoms," Kaushik said. "I'm exploring nanotechnology that can carry a drug, selectively go to a place, and release the drug so we avoid using excessive drugs."

This nanomedicine could be used to precisely target brain tumors or other difficult-to-treat conditions. He has published papers in scientific journals about this work and also holds multiple patents.

"My whole approach is using smart material science for better health for everybody, which is accessible to everybody everywhere," Kaushik said.

In addition to his USERN prize, Kaushik was named a USERN junior ambassador for 2020 and will work to advance the organization's mission in the United States.

For the most recent university news, visit Florida Poly News.

About Florida Polytechnic University:

Florida Polytechnic University is accredited by the Southern Association of Colleges and Schools Commission on Colleges and is a member of the State University System of Florida. It is the only state university dedicated exclusively to STEM and offers ABET accredited degrees. Florida Poly is a powerful economic engine within the state of Florida, blending applied research with industry partnerships to give students an academically rigorous education with real-world relevance. Connect with Florida Poly online at http://www.floridapoly.edu.

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Nanotechnology for disease diagnosis and treatment earns Florida Poly professor international award - Yahoo Finance

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