The Pfizer/BioNTech and Moderna Covid-19 vaccines are not designed to alter your genes. (Photo by: ... [+] Daniel Garzon/VW Pics/Universal Images Group via Getty Images)

The Pfizer/BioNTech and Moderna Covid-19 vaccines are not genes in a bottle.

Yet, some on social media are now suggesting that such mRNA vaccines are actually gene therapy. For example, Alex Berenson, who has described himself as a former reporter for The New York Times and a current thriller novel author, mentioned the words gene therapy in a recent tweet. And presumably he wasnt referring to Gene Simmons therapy:

As you can see, Berensons tweet prompted a response and a correction from Seth Trueger, MD, MPH, an Assistant Professor of Emergency Medicine at Northwestern University.

Then theres the Its Gene Therapy, Not a Vaccine with Dr. David Martin podcast on the Weston A. Price Foundation website. This is the Foundation that once issued a press release entitled FDA and CDC Bias Against Raw Milk. Yes, the CDC has warned about the possibility of getting dangerous infections from raw milk, otherwise known as unpasteurized milk, as I covered previously from Forbes.

The Foundation website doesnt really describe Martins scientific background, simply saying that he received his Undergrad degree from Goshen College, Masters of Science from Ball State University and Doctorate from the University of Virginia, without specifying the actual subject matters of his degrees. So it isnt clear why they chose him to talk about Covid-19 versus someone else who happened to go to college and some kind of graduate school.

On the podcast, Martin said the following: The problem is that in the case of Moderna and Pfizer, this is not a vaccine. This is gene therapy. Its a chemotherapy agent that is gene therapy. It is not a vaccine. Chemotherapy? How did that word get in there? The National Cancer Institute (NCI) defines chemotherapy as treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Isnt mentioning chemotherapy when you are talking about a vaccine against a virus like mentioning spaghetti and hot dogs when talking about building a bear in a Build A Bear Workshop? Its mixing different things that dont seem to be related.

Martin then tried to explain, What is this doing? Its sending a strand of synthetic RNA into the human being and is invoking within the human being, the creation of the S1 spike protein, which is a pathogen. Its a toxin inside of human beings. This is not only not keeping you from getting sick, its making your body produce the thing that makes you sick.

No, no, no. Spike proteins line the surface of the severe acute respiratory syndrome coronavirus 2 (SARS-CoV2), which looks like a spiky massage ball but shouldnt be used as one. The spike protein helps the virus enter your cells. But the spike protein itself is not supposed to make you sick. Claiming that the spike protein alone is responsible for illness would be like claiming that a bleached blonde wig with frosted tips alone can cook like celebrity chef Guy Fieri.

The rest of the podcast didnt exactly scream science. It included the statement by Martin that Covid 19 is not a disease. It is a series of clinical symptoms. It is a giant umbrella of things associated with what used to be associated with influenza and with other febrile diseases. No. Covid-19 is not a giant umbrella. Where the ella, ella, eh, eh did that come from? Covid-19 is not the flu either. Repeat, having Covid-19 is not the same as having influenza.

Oh, and holy conspiracy theories, Batman, Martin asserted that Anthony Fauci, MD, Director of the National Institute of Allergy and Infectious Disease (NIAID) is manipulating this situation to force a vaccine on a population.

How much real evidence did Martin offer to support his claims? Well, the answer rhymes with not much. Its easy to say anything when the interviewer doesnt ask you to provide real concrete evidence behind what you are saying.

The Covid-19 mRNA vaccines are not gene therapy because they are not designed to alter or change your genes in any way. The U.S. National Library of Medicine (NLM) describes gene therapy as a technique that may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. For example, doctors may be able to either inactivate or replace a mutated gene that isnt functioning properly or place a new gene in your body that will do something to combat a disease.

A gene consists of DNA and serves as the basic physical and functional unit of heredity, according to the NLM. Messenger RNA, known as mRNA for short, is different from DNA. RNA stands for ribonucleic acid. DNA stands for deoxyribonucleic acid. DNA serves as the library for instructions to produce different proteins. When a cell wants to produce a protein, it uses the DNA to produce a copy of mRNA. That mRNA then serves as a blueprint for the protein that is built by the ribosomes in your cells. The DNA is in the nucleus of the cell. The ribosomes are not. Thus, the mRNA from a Covid-19 vaccine will not go into the nucleus but instead will simply go to the ribosomes, which in turn will manufacture the spike protein.

This PBS News Hour video shows this process and emphasizes that mRNA should not alter your DNA:

The Pfizer/BioNTech and Moderna Covid-19 vaccines are not gene therapy. Repeat, these vaccines are not gene therapy. In fact, they dont really involve your genes, unless Gene Simmons or Gene Hackman happen to be getting the vaccines. Or you accidentally drops the vaccines on your jeans and then misspell the word jeans.

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As calls for mandatory COVID-19 vaccination grow around the world, its becoming ever more crucial to understand what these injections actually are. The mRNA vaccines created by Moderna and Pfizer are in fact gene therapies.

Interestingly enough, mainstream media, fact checkers and various industry front groups insist the gene therapy claim is bogus, even though every single detail about the vaccines shouts otherwise. Why are they spreading this disinformation? Why do they not want you to know what these injections actually are?

To start, lets take a look at some basic definitions of words. According to the U.S. Centers for Disease Control and Prevention, a vaccine is:1

Immunity, in turn, is defined as:

Thats the medical definition. The legal definition, in the few cases where it has been detailed, is equally unequivocal:

These definitions, both medical and legal, present problems for mRNA vaccines, since:

We should not be fooled by attempts to condition the public to accept redefined terms. As of February 2019, Merriam-Webster defined5 vaccine as a preparation of killed microorganisms, living attenuated organisms, or living fully virulent organisms that is administered to produce or artificially increase immunity to a particular disease. By February 26, 2021, they had updated the definition of vaccine to:6

A preparation that is administered (as by injection) to stimulate the bodys immune response against a specific infectious disease:

a: an antigenic preparation of a typically inactivated or attenuated pathogenic agent (such as a bacterium or virus) or one of its components or products (such as a protein or toxin)

b: a preparation of genetic material (such as a strand of synthesized messenger RNA) that is used by the cells of the body to produce an antigenic substance (such as a fragment of virus spike protein)

Lets be clear. Merriam-Webster does not dictate medical terminology. It can be used, however, to confuse people. For now, all medical dictionaries still show the traditional definition of vaccine,7 as Merriam-Webster did up until this year. That said, I would not be surprised if changes are made there as well, eventually, if the misrepresentation of COVID-19 mRNA vaccines is allowed to stand.

Theres also the issue of whether a gene therapy can be mandated, and this may hinge on it being accepted as a vaccine. The 1905 Supreme Court ruling in Jacobson v. Massachusetts8 essentially established that collective benefit supersedes individual benefit.

Since mRNA therapies do not render the immunized person immune, and do not inhibit transmission of the virus, they cannot qualify as a public health measure capable of providing collective benefit that supersedes individual risk, and therefore cannot be mandated.

Put another way, the ruling argues (although legal experts diverge on some of the finer details of its interpretation) that its acceptable for some individuals to be harmed by a public health directive as long as it benefits the collective. However, if vaccination is a public health measure meant to protect and benefit the collective, then it would need to accomplish two things:

Were now back to the original problem that mRNA therapies for COVID-19 do not accomplish either of these things. Since these gene therapies do not render the person immune, and do not inhibit transmission of the virus, they cannot qualify as a public health measure capable of providing collective benefit that supersedes individual risk.

On the contrary, the only one benefiting from an mRNA vaccine is the individual receiving the gene therapy, since all they are designed to do is lessen clinical symptoms associated with the S-1 spike protein.

In other words, they wont keep you from getting sick with SARS-CoV-2; they are only supposed to lessen your infection symptoms if or when you do get infected. So, getting vaccinated protects no one but yourself. Since youre the only one who will reap a benefit (less severe COVID-19 symptoms upon infection), the justification to accept the risks of the therapy for the greater good of your community is blatantly irrational.

Since mRNA vaccines do not meet the medical and/or legal definition of a vaccine, referring to them as vaccines, and marketing them as such, is a deceptive practice that violates9 15 U.S. Code Section 41 of the Federal Trade Commission Act,10 the law that governs advertising of medical practices.

The lack of completed human trials also puts these mRNA products at odds with 15 U.S. Code Section 41. Per this law,11,12 it is unlawful to advertise that a product or service can prevent, treat, or cure human disease unless you possess competent and reliable scientific evidence, including, when appropriate, well-controlled human clinical studies, substantiating that the claims are true at the time they are made.

Heres the problem: The primary end point in the COVID-19 vaccine trials is not an actual vaccine trial end point because, again, vaccine trial end points have to do with immunity and transmission reduction. Neither of those was measured.

Whats more, key secondary end points in Modernas trial include prevention of severe COVID-19 disease (defined as need for hospitalization) and prevention of infection by SARS-CoV-2, regardless of symptoms.13,14 However, Moderna did not actually measure rate of infection, stating that it was too impractical to do so.

That means theres no evidence of this gene therapy having an impact on infection, for better or worse. And, if you have no evidence, you cannot fulfill the U.S. Code requirement that states you must have competent and reliable scientific evidence substantiating that the claims are true.

Making matters worse, both Pfizer and Moderna are now eliminating their control groups by offering the real vaccine to any and all placebo recipients who want it.15 The studies are supposed to go on for a full two years, but by eliminating the control group, determining effectiveness and risks is going to be near impossible.

Alright. Lets move on to the definition of gene therapy. As detailed on MedlinePlus.govs What Is Gene Therapy page:16

Gene therapy is an experimental technique that uses genes to treat or prevent disease Researchers are testing several approaches to gene therapy, including: Introducing a new gene into the body to help fight a disease

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures.

Here, its worth noting that there are many different treatments that have been shown to be very effective against COVID-19, so it certainly does not qualify as a disease that has no cure. It makes sense that gene therapy should be restricted to incurable diseases, as this is the only time that taking drastic risks might be warranted. That said, heres how the U.S. Food and Drug Administration defines gene therapy:17

Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use. Gene therapy is a technique that modifies a persons genes to treat or cure disease. Gene therapies can work by several mechanisms:

Replacing a disease-causing gene with a healthy copy of the gene

Inactivating a disease-causing gene that is not functioning properly

Introducing a new or modified gene into the body to help treat a disease

November 17, 2020, the American Society of Gene + Cell Therapy (ASGCT) announced COVID-19 Vaccine Candidates Show Gene Therapy Is a Viable Strategy, noting that:18

Two COVID-19 vaccine trials, both of which use messenger RNA (or mRNA) technology to teach the body to fight the virus, have reported efficacy over 90 percent.

These findings, announced by Moderna on Nov. 16 and by Pfizer and its partner BioNTech on Nov. 9 demonstrate that gene therapy is a viable strategy for developing vaccines to combat COVID-19.

Both vaccine candidates use mRNA to program a persons cells to produce many copies of a fragment of the virus. The fragment then stimulates the immune system to attack if the real virus tries to invade the body.

As explained in the ASGCTs video above, mRNA are molecules that contain genetic instructions for making various proteins. mRNA vaccines deliver a synthetic version of mRNA into your cells that carry the instruction to produce the SARS-CoV-2 spike protein, the antigen, that then activates your immune system to produce antibodies. Then theres Modernas trial website,19 where they describe their technology thus:

Typical vaccines for viruses are made from a weakened or inactive virus, but mRNA-1273 is not made from the SARS-CoV-2 virus. It is made from messenger ribonucleic acid (mRNA), a genetic code that tells cells how to make protein, which help the bodys immune system make antibodies to fight the virus.

November 18, 2020, Wired magazine made a big deal about COVID-19 vaccines being genetic vaccines, noting:20

The active ingredient inside their shot is mRNA mobile strings of genetic code that contain the blueprints for proteins. Cells use mRNA to get those specs out of hard DNA storage and into their protein-making factories. The mRNA inside Pfizer and BioNTechs vaccine directs any cells it reaches to run a coronavirus spike-building program.

Importantly, as reported by David Martin, Ph.D.,21,22 Moderna describes its product not as a vaccine, but as gene therapy technology in SEC filings. This is because neither Moderna nor Pfizer make any claims about their products creating immunity or preventing transmission. Additionally, Modernas SEC filings specifically state that Currently, mRNA is considered a gene therapy product by the FDA, as well.23

In a February 2021 article, MIT Technology Review reviewed the history of mRNA technology in general, and Modernas in particular, stating:24

Vaccines were not their focus. At the companys founding in 2010, its leaders imagined they might be able to use RNA to replace the injected proteins that make up most of the biotech pharmacopoeia, essentially producing drugs inside the patients own cells from an RNA blueprint. We were asking, could we turn a human into a bioreactor? says Noubar Afeyan, the companys cofounder

Bloomberg, in August 2020, reported25 that the Moderna vaccine would seek to transform your body into a vaccine-making machine. The New York Times was more to the point. In May 2020, they reported26 that Researchers at two Harvard-affiliated hospitals are adapting a proven form of gene therapy to develop a coronavirus vaccine. Read it again A proven form of gene therapy.

So, to summarize: The definition of genetic is something relating to genes, and the definition of therapy is the medical treatment of a disease. The definition of gene therapy is the process of modifying or manipulating the expression of a gene, or altering the biological properties of living cells.

mRNA are snippets of genetic code that instructs cells to produce proteins. mRNA COVID-19 therapies deliver genetic instructions into your cells, thereby triggering your body to produce a fragment of the virus (the spike protein). So, mRNA vaccines ARE gene therapy. Theres simply no way around this. They fulfill all the definitions of gene therapy and none of the definitions for a vaccine.

Theres yet one more potential problem with the COVID-19 vaccine narrative as a whole, which Martin unpacked in a January 25, 2021, interview on the Wise Traditions podcast (above).27 In it, he explains:

COVID-19 is not a disease. It is a series of clinical symptoms. It is a giant umbrella of things associated with what used to be associated with influenza and with other febrile diseases.

The problem that we have is that in February [2020], the World Health Organization was clear in stating that there should not be a conflation between [SARS-CoV-2 and COVID-19]. One is a virus, in their definition, and one is a set of clinical symptoms. The illusion in February was that SARS-CoV-2 caused COVID-19.

The problem with that definition, and with the expectation, is that the majority of people who test positive using the RT-PCR method for testing, for fragments of what is associated with SARS-CoV-2, are not ill at all. The illusion that the virus causes a disease fell apart. Thats the reason why they invented the term asymptomatic carrier.

In short, SARS-CoV-2 has yet to be definitively proven to be the actual cause of COVID-19. So, a gene therapy that instructs your body to produce a SARS-CoV-2 antigen the viral spike protein cannot even be touted as a preventative against COVID-19, as the two have not been shown to be causally linked.

They have been willfully lying since the inception of this, Martin says in the interview. There is not a causal link between these things It has never even been close to established.

We have a situation where the illusion of the problem is that people say, I dont want to get COVID-19. What they mean is they dont want to get infected with a virus. The problem is those two things are not related to each other. A viral infection hasnt been documented in the majority of what is called cases.

There is no basis for that conflation other than the manipulation of the public. Thats the first half of the problem. The second half of the problem is that what is being touted as a vaccination is not a vaccine. This is gene therapy

What is this doing? Its sending a strand of synthetic RNA into the human being and is invoking within the human being, the creation of the S1 spike protein, which is a pathogen A vaccine is supposed to trigger immunity. Its not supposed to trigger you to make a toxin

Its not somewhat different. Its not the same at all Its not a prohibiting infection. Its not a prohibiting transmission device. Its a means by which your body is conscripted to make the toxin that then, allegedly, your body somehow gets used to dealing with, but unlike a vaccine which is to trigger the immune response this is to trigger the creation of the toxin.

As for why drug companies are misrepresenting this technology, Martin suspects its done exclusively so that they can get themselves under the umbrella of public health laws that exploit vaccination.

Experimental gene therapies do not have financial liability shielding from the government, but pandemic vaccines do, even in the experimental stage, as long as the emergency use authorization is in effect. This is indeed a major incentive to make sure this technology is perceived as a vaccine and nothing else.

So, by maintaining the illusion that COVID-19 is a state of emergency, when in reality it is not, government leaders are providing cover for these gene therapy companies so that they are insulated from any liability.

Ive written many articles detailing the potential and expected side effects of these gene therapy vaccines. If all of this is new to you, consider reviewing How COVID-19 Vaccine Can Destroy Your Immune System, Seniors Dying After COVID Vaccine Labeled as Natural Causes and Side Effects and Data Gaps Raise Questions on COVID Vaccine.

The take-home message here is that these injections are not vaccines. They do not prevent infection, they do not render you immune and they do not prevent transmission of the disease. Instead, they alter your genetic coding, turning you into a viral protein factory that has no off-switch. Whats happening here is a medical fraud of unprecedented magnitude, and it really needs to be stopped before its too late for a majority of people.

If you already got the vaccine and now regret it, you may be able to address your symptoms using the same strategies youd use to treat actual SARS-CoV-2 infection. I review these strategies at the end of Why COVID Vaccine Testing Is a Farce.

Last but not least, if you got the vaccine and are having side effects, please help raise public awareness by reporting it. The Childrens Health Defense is calling on all who have suffered a side effect from a COVID-19 vaccine to do these three things:28

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COVID-19 'Vaccines' Are Gene Therapy - Atlanta Business ...

One year after receiving an experimental gene therapy developed by the Dutch drugmaker UniQure, patients with hemophilia B aren't having nearly as many bleeding issues as they used to have.

In hemophilia, genetic mutations prevent the body from making proteins needed to clot blood. People with the less common, "B" form of the disorder are missing a protein known as Factor IX. UniQure's medicine is meant to provide a working version of the gene so patients can generate their own clotting protein, and rely less or ideally, not at all on so-called replacement factor treatments.

UniQure said Tuesday that, a year after receiving the company's therapy, patients enrolled in the company's HOPE-B clinical trial were experiencing 80% fewer bleeding episodes that require treatment.

At the trial's start, these patients were having an average of three to four bleeding episodes annually. Now, according to UniQure, they're having less than one. The use of replacement factor therapy declined 96% during the 52-week period, as all but two of the 54 participants successfully discontinued their preventive infusions.

What's more, patients had almost 42% the amount of clotting protein typically observed in people who don't have hemophilia B. That's a slight improvement from six-month data presented in December, which showed protein levels at 39% of normal.

Yet, in spite of the positive data, UniQure shares dipped more than 5% in early afternoon trading Tuesday. CSL Behring, which spent $450 million to acquire the rights to UniQure's therapy, also saw its stock trade down.

Some of that stock market reaction may be related to an update Uniqure disclosed alongside the fresh data about the approval process for its therapy. According to UniQure, the Food and Drug Administration primarily wants to see evidence the therapy has a lasting effect, so the agency's focus will be on 52-week data. However, that time frame must start from when patients' protein levels stabilize, rather than from when they first received UniQure's therapy.

The company said all participants in its study achieved steady-state protein levels by week 26. As a result, UniQure is now measuring the study's success by annualized bleeding rates at week 78, with the expectation that patients to complete 78-week follow-up visits by the end of September. That should allow UniQure and CSL to file for approval in first quarter of 2022, months later than when the companies had estimated last year.

If approved, UniQure's gene therapy would become the first cleared to treat any kind of hemophilia. Another, for hemophilia A, was up for approval last year, but the FDA rejected it in in a major surprise. In issuing its rejection, the FDA asked for two years of follow-up data from the therapy's developer, BioMarin Pharmaceutical, a task that can't be completed until November 2021.

Estimates hold there are as many as 33,000 people living with hemophilia in the U.S., with the B form being about four times less common.

Though the FDA has approved around half a dozen drugs to treat hemophilia B, patients taking them can still experience bleeds. Additionally, they can develop a type of antibody called an inhibitor, which blocks the effects of replacement factor.

That's made gene therapy, with its potential as a one-time fix for the disease, an attractive option for some patients and physicians.

Still, concerns remain. UniQure's study was stopped late last year, for example, after researchers saw an unexpected case of liver carcinoma in one participant, reigniting long-held fears about gene therapy's potential to drive cancer.

UniQure investigated the case and recently concluded, along with an independent laboratory and outside experts, that its therapy was "highly unlikely" to have caused the cancer.

There are also worries that gene therapy won't work for everyone. Some people have pre-existing "neutralizing antibodies" that attack the viruses used to deliver functioning genetic material.

UniQure has found its therapy appears, for the most part, to bypass this issue of neutralizing antibodies. The company said Tuesday it hasn't seen any "significant correlation" in how patients who have these antibodies at least to a certain range are responding to its therapy.

UniQure said that range is high enough that it should encompass more than 93% of the general population. The company noted in December how one of the study participants who didn't respond its therapy had neutralizing antibody levels that were five times greater than anyone else in the group.

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A year after getting UniQure's gene therapy, hemophilia patients are still doing better - BioPharma Dive

Vedere Bio II is fresh off a $77 million raise, and the follow-up act to the original Vedere Bio now has a big-name chief scientific officer in Gabor Veres, Ph.D.

The Cambridge, Massachusetts-based biotech is at work on next-gen eye disease gene therapies, aiming to restore sight, or help those keep it, with patients suffering from vision loss due to photoreceptor death.

To help in that mission, the company has poached Veres from rival biotech BioMarin, where he was vice president and head of gene therapy research focusing on AAV platform discovery and seeking out new indications. He also served stints at bluebird bio and Applied Genetic Technologies.

At VB II, he will provide strategic leadership to Vedere's research organization and will be responsible for driving and expanding the company's pipeline of ocular gene therapies, the biotech said in a statement.

The biotech has a history that while short, is very dense: Cyrus Mozayeni, M.D.s first go at vision-restoring gene therapies got snapped up as it was just getting started. Lots of suitors came knocking even when that company, Vedere Bio, was working under the radar, and Novartis eventually ponied up $150 million upfront to buy it last fall, with a promise of $130 million more.

Buoyed by that success but clearly still hungry for more, Mozayeni, its CEO and Atlas Venture entrepreneur in residence and his team last month nabbed $77 million to bankroll their second chapter: Vedere Bio II.

Although its technically a new entity, its working on gene therapies for vision loss with the same founders, management team and employees as its predecessor. Vederes focus is vision loss caused by the death of photoreceptors or light-sensing cells in the retina rather than blindness stemming from damage to the brain or optic nerve. With Veres, that team just got a little more diversified.

RELATED: Spark grabs FDA nod for Luxturna, a breakthrough gene therapy likely bearing a pioneering price

"Gabor has over 15 years of experience leading preclinical research and development for international pharmaceutical and biotechnology companies and holds 20 years of experience in the development of cell and gene therapy products, said Mozayeni.

His extensive track record designing and executing gene therapy research programs for a range of diseases, including conditions of the eye, will be an incredible asset to our team as we continue our work to restore vision to patients with both genetic and non-genetic causes of vision loss.

Gene therapies have long held much promise, but safety issues and manufacturing complexities have always dampened companies capacity to deliver. Gene therapies for eye disorders have been among the few to see R&D success translate into the market, most recently with Roche/Sparks Luxturna, though sales forecasts are fairly modest, and it requires specialist centers for administration.

Vedere's approach to vision restoration holds great potential to treat vision loss regardless of underlying genetic cause or disease stage, and I'm looking forward to helping build out and progress the company's unique platform and product pipeline, added Veres.

Continue reading here:
BioMarin's gene therapy lead jumps ship to rival biotech Vedere Bio II - FierceBiotech

When you have retinitis pigmentosa, the world slowly goes dark. Most patients with RP lose most of their sight by young adulthood and are often legally blind by age 40. The genetic disorder affects around one in 4,000 people, causing the retinas photoreceptive cells to degrade over time. But a new gene therapy is giving hope and producing life-changing resultsand it could lead to treatments for far more common retinal diseases.

Bedford-based Nanoscope Therapeutics is trying to turn the light back on for RP patientsby developing gene therapies using light-sensitive molecules that could re-sensitize the retina to detect low light levels. That could restore vision in millions of visually impaired people who suffer from RP and other retinal degenerative diseases, like Stargardt disease and dry age-related macular degeneration (AMD). The startups gene therapies, called optogenetics, aim to bring sight to the blind. Nanoscope is one of a number of teams and companies exploring optogenetics as a treatment for RP.

There is no treatment for the diseases that were working on, Nanoscopesco-founder, president, and chief scientific officer, SamarendraMohanty, told Dallas Innovates. So theres a real unmet need that we are trying to fill.

Samarendra Mohanty [Photo: Nanoscope Therapeutics]

Nanoscope, a clinical-stage biotech company, announced earlier this month that its Phase 1/2a clinical study using Multi-Characteristic Opsin (MCO) on RP patients had restored clinically meaningful vision. Significant dose-dependent improvement of visual acuity was demonstrated at 16 weeks, and continued through one year in patients suffering from severe RP, the company said.

The study included 11 patients with advanced RP who had either no light perception or just perception of light in the study eye and no better ability than counting fingers in their other eye. The studys initial positive results were reported at the American Academy of Ophthalmologys 2020 annual meeting last November.

After MCO treatment, the patients reported long-lasting improvements in outdoor light sensitivity and daily activities, saidthe principal investigator, Dr. Santosh Mahapatra, an ophthalmologist and eye surgeon, in a statement. We were pleasantly surprised that after eight weeks of treatment, some subjects could attend their follow-up visits during the study without the assistance of a chaperone. Some of the patients even gained the ability to read letters on a wall or even the large text in a newspaper, use a cell phone, watch television, and could even thread a needle.

Another benefit of the treatment: Vision restoration was produced without the need of stimulating retinal implants or explants (goggles).

Nanoscope co-founder and CEO Sulagna Bhattacharya said the trial studys impact on patients lives has been powerful.

Their quality of life improves significantly, she told us. This is a relief to patients family members, healthcare systems, and society as a whole.

Sulagna Bhattacharya [Photo: Nanoscope Therapeutics]

We expect to begin the first randomized, placebo-controlled, double-masked Phase 2b multi-center optogenetic trial in the U.S. this summer to further validate our gene therapys ability to improve clinically meaningful vision in RP patients, Bhattacharya said in the statement. If successful, it will be the first-ever restorative drug for millions of RP patients worldwide.

The 2b trial will involve eight to 10 centers in the U.S., from Beverly Hills to Florida, to other locations on the east coast.

[Image: Ivan-balvan/istockphoto]

Nanoscopes RP gene therapy has received orphan drug designation from the FDA. It uses a proprietary AAV2 vector to deliver MCO genes into the retina. This mutation-independent gene therapy is delivered via a single injection through the eye administered in a doctors office.

All 11 subjects participating in the trial had objective and subjective improvement in functional vision, Nanoscope reports. Shape discrimination accuracy improved more than 90% in all the subjects compared to baseline. Further, the performance in two different mobility tests improved by 50%, including the reduction in time to touch a lighted panel. Nanoscope says the test outcomes were highly correlated with improved patient-reported outcomes.

Nanoscopes co-founder, Mohanty, is the inventor of the technology used in the trial.

Optogenetics is a powerful research tool, he said in a statement, but had limited scope of clinical benefit because the opsins had a narrow band of activation, unlike natural light environment. MCO is sensitive to broadband light and activatable by ambient light, thus eliminating the risk of photo-toxicity from long-term continuous use of external intense light stimulation devices.

Nanoscope Therapeutics got a $2 million grant from the National Eye Institute in June 2020 and closed anoversubscribed Series A funding roundin July 2020 to help fund its clinical trial.The startup, a TechFW client, is a spinout from Nanoscope Technologies, serving as a commercialization partner for the R&D enginesMCO vision restoration work. It has 12 employees and a group of consultants and advisors.

Nanoscope Technologies, meanwhile, has received around$10 million in grants from theNational Institutes of Health to help fund its R&D,Bhattacharya said.

The CEO added that the RP study could lead to pivotal results.

Were very excited about our Phase 1/2a results, Bhattacharya said. This trial has the potential to become pivotal, which will allow our product to be available in the clinics to treat millions of blind individuals for whom there is no treatment so far.

Beyond the RP trials, Mohanty says his company placing a big focus on dry age-related macular degeneration (AMD),

Thats a big indication that we are targeting, Mohanty said of AMD, since unlike RP, AMD is super prevalent. According to the National Eye Institute, 11 million Americans have AMD.

Mohanty said that AMD is very rapidly progressing as we are aging and worldwide there are major concerns.

Nanoscope plans to initiatemultiple trials to treat both dry AMD and Stargardt disease, another inherited retina disorder.

Nanoscope Technologies was founded in 2009 by Mohanty. Bhattacharya joined the company as co-founder in 2013. Mohanty and CFO Anthony Togba told Dallas Innovates they are open-minded to the option of going public with their company, and doing due diligence to prepare for that potentiality.

CFO Anthony Togba [Photo: Nanoscope Therapeutics]

Togba says the company is performing readiness activities, undertaking internal processes, and putting in place the structures to be ready to go public.

The timeline? By late Q4 or before, well have a better idea about where were headed, Togba said. The upcoming clinical trial is the preoccupation now to make sure that we have a flawless execution to obtain those results that we expect.

Quincy Preston contributed to this report.

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The investment was led by Advantech Capital, a PE fund based in China that focuses on TMT, pharmaceuticals, and healthcare. This combined with the support from the Cancer Prevention and Research Institute of Texas (CPRIT), which granted OncoNano $9.97 million last year, will support Phase 3 clinical trials for the biotech's technology that can diagnose and treat cancer with high specificity.

Taysha Gene Therapies, which rocketed from a UTSW spinout to a $157 million IPO in under six months last year, has gone global with rights to TSHA-120, apromising AAV9 clinical-stage genetherapy. It's a historic announcement. There are no current treatments for giant axonal neuropathy, or GAN,a severe, progressive disease that affects the central and peripheral nervous systems.TSHA-120 is the first-ever successful in-human intrathecal (spinal) gene transfer.

In this weekly roundup of executive moves in North Texas, you'll also find news from Liberty Capital Bank, Krista Software, Tuesday Morning, Trive Capital, Cantey Hanger, UNT, JUNO, NuZee, Jaunt Air Mobility, Korbyt, and ID90 Travel.

The Series C funding brings Allied BioSciences total to more than $80 million, the team told us. It will be used to grow the biotech's flagship product, SurfaceWise2, which is an active surface coating that can continuously destroy 99.9 percent of viruses on surfaces. Last year, SurfaceWise2 was the first and only surface coating that the EPA approved for continuous protection against COVID-19 with a single application.

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Bedford Biotech Restores "Meaningful Vision" in Blind Patients With Gene Therapyand May Soon Go Public Dallas Innovates -...