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One-time gene therapy injection could provide HIV treatment that may last a lifetime – OHSU News

Posted: July 11, 2022 at 2:38 am

Jonah Sacha, Ph.D., at OHSU's Oregon National Primate Research Center and Vaccine & Gene Therapy Institute. (OHSU/Kristyna Wentz-Graff)

A new pre-clinical study in nonhuman primates will evaluate an experimental drugs potential use as a gene therapy that could prevent people who have HIV from having to take daily antiviral drugs for the rest of their lives.

The research will be led by Oregon Health & Science University researcher Jonah Sacha, Ph.D., who also serves as a scientific adviser to CytoDyn, the biotechnology company developing the drug, called leronlimab. The study is funded by a five-year grantof up to $5 million that was recently awarded to OHSU by the National Institute of Allergy and Infectious Disease, which is part of the National Institutes of Health.

This grant will fund the development and early study of leronlimab as a potential single-injection gene therapy, said Sacha, professor at OHSUs Vaccine and Gene Therapy Institute and Oregon National Primate Research Center. If this approach works as hoped, it could provide a functional cure for HIV, meaning it could suppress HIV enough that patients would no longer need to take daily antiviral pills for the rest of their lives."

In an earlier study, Sacha and colleagues found leronlimab completely preventednonhuman primates from being infected with the monkey form of HIV. That result indicated leronlimab held promise as a potential pre-exposure drug to prevent human infection from the virus that causes AIDS.

Now, this study aims to design a way to offer leronlimab as gene therapy. Sacha and colleagues will explore how to contain the coding sequence of the experimental drug inside a lab-made form of the adeno-associated virus, an approach that gene therapy researchers call an AAV vector. The resulting therapy would then be injected inside the body where muscle cells would make leronlimab long term.

Leronlimab is a monoclonal antibody that blocks HIV from entering immune cells through a surface protein called CCR5. The drug has demonstrated it can mimic a CCR5-deficient donor by occupying all available CCR5 molecules, but this would require a new method for delivery as a gene therapy. Viral vectors have been used to deliver antigens from specific pathogens for decades.

In this project, researchers will design a synthetic AAV vector to enable the long-term production leronlimab inside the body. The goal is to develop a safe and effective single injection that suppresses HIV replication and eliminates the need for life-long antiretroviral therapy.

Currently, patients often take multiple antiretroviral pills daily, said Sacha. Removing this pill burden could not only improve patients quality of life, but remove problems with adherence.

Rhesus macaques at OHSUs Oregon National Primate Research Center that have been exposed to a monkey version of HIV will be given a single AAV injection that contains leronlimab. Researchers will monitor the nonhuman primates for years to assess the safety and efficacy of this approach.

This research is supported by the National Institute of Allergy And Infectious Diseases of the National Institutes of Health under Award Number R01AI166969. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

In our interest of ensuring the integrity of our research and as part of our commitment to public transparency, OHSU actively regulates, tracks and manages relationships that our researchers may hold with entities outside of OHSU. In regard to this research, Dr. Sacha has a significant financial interest in CytoDyn, a company that may have a commercial interest in the results of this research and technology. Reviewdetails of OHSU's conflict of interest programto find out more about how we manage these business relationships.

All research involving animal subjects at OHSU must be reviewed and approved by the universitys Institutional Animal Care and Use Committee (IACUC). The IACUCs priority is to ensure the health and safety of animal research subjects. The IACUC also reviews procedures to ensure the health and safety of the people who work with the animals. No live animal work may be conducted at OHSU without IACUC approval.

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One-time gene therapy injection could provide HIV treatment that may last a lifetime - OHSU News

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Can Philly’s Developers Shepherd Cell And Gene Therapy Startups Through The ‘Valley Of Death’? – Bisnow

Posted: July 11, 2022 at 2:38 am

The life sciences real estate market in Philadelphia is coming into its own just as its cell and gene therapy industries stare down their first economic cycle with high inflation and spiking federal interest rates.

Bisnow/Virginia Baker

Mosaic Development Partners founder and co-owner Greg Reaves and Brandywine Realty Trust Senior Managing Director Jeff DeVuono at Bisnow's Philly Life Sciences Summit event at Budd Bioworks on June 28, 2022

Despite the success of Spark Therapeutics, the vast majority of cell and gene therapy candidates remain in early stages of testing, with relatively few in clinical trials and only a handful commercially available. So much of the public and private sector in the area is focused on sustaining the growth of the past few years, but both are being called on to shepherd startups through the valley of death, or the period between early stage trials and commercialization when funding is hardest to come by and most desperately needed.

One of the costs that balloon during that period is real estate, as the space a biotech startup requires for advancing a therapy through testing phases is often what takes it from a couple of stations at an incubator to its first proper lease. But that graduation space, as the industry calls it, is in extremely short supply across the Philadelphia region, Longfellow Real Estate Partners Managing Director of Research Lauren Gilchrist said at Bisnows Philadelphia Life Sciences Summit on June 28.

Our incubator spaces and our [life sciences] coworking spaces are filling up with companies that are probably ready for their graduation spaces, and in other markets would be in graduation space, Gilchrist said. But we have not yet provided that space and the right continuum of services in Philadelphia.

While several ground-up construction projects are underway, the two most significant adaptive reuse projects in and around Center City 401 North Broad St. and The Curtis are filling up space virtually as soon as they can convert it.

To meet the timeline specific to life sciences tenants, Philadelphia developers have had to get comfortable with starting buildings on speculation, with multiple such projects underway inUniversity City and the Philadelphia Navy Yard. One uCity Square, set to deliver this fall, is up to 80% leased, said Wexford Science + Technology Director of Development Pete Cramer, whose company is developing the building in partnership with the University City Science Center and Ventas. But the upfront cost and risk associated with speculative development keep rising.

Bisnow/Virginia Baker

Longfellow Real Estate Partners Managing Director of Research Lauren Gilchrist and Delaware Innovation Space founder and CEO Bill Provine

With financing costs rising precipitously amid higherinterest rates, capital markets activity is cooling in commercial real estate, all while inflation and supply chain snags persist to make projects more expensive and more unpredictable in terms of cost. But if a company has the money, spending it now seems to be the order of the day, panelists at the event agreed. Even if specialized equipment is more expensive than it has been for years, future price hikes and surprise inventory disruptions are being treated as a virtual certainty across the industry.

The joint venture of Ensemble Real Estate Investments and Mosaic Development Partners tasked with leading the Navy Yards next wave of development, which has one spec lab building under construction, plans on front-loading more of its capital expenditures for its next building, which is planned for Current Good Manufacturing Practices-compliant biomanufacturing, Mosaic founder and co-owner Greg Reaves said at the event.

The GMP that you knew before Covid is not the same GMP [as today], Reaves said. Purchasing materials upfront is a requirement today, and I dont think you can avoid that. If you expect to be open anytime in 2023 or 2024, you have to be thinking seriously about moving forward on risk.

To build on speculation is to rely on the Field of Dreams line oft-quoted in real estate, If you build it, they will come. But in a sector where the majority of tenants are pre-revenue, selecting a tenant from a pool of interested parties is an exercise in risk assessment. Multiple landlords and brokers at the full-day event stressed the importance of believing in a tenants research or, more importantly, its leadership.

A lot of these early stage companies can't scale because they don't have the expertise, they don't have the network and they don't know what they don't know, HDR Managing Principal Elizabeth Mahon said. In the cell and gene therapy world, there's not enough leadership talent to go around. So when you're dealing with a lot of technical founders, scientific founders, first-time CEOs and [chief science officers], you're really limited because they're learning on the fly.

Bisnow/Virginia Baker

University Place Associates President Anthony Maher, Center for Breakthrough Medicines co-founder Audrey Greenberg and Tactix Real Estate Advisors Senior Broker Gary Zoloff

Having a leader with deep experience or a wide network is crucial for a life sciences startups appeal to landlords, not least because it gives those startups an edge in recruiting venture capital investors. In situations when a landlord may believe in a tenant even if the latter lacks its own access to capital, some landlords are tapping their own capabilities or partnerships.

University Place Associates has connected potential tenants with its capital partners, the joint venture of Cantor Fitzgerald and Silverstein Properties that has financed 3.0 University Place, set to deliver next year, and with which UPA is negotiating financing terms for the following 4.0 University Place next door, UPA President Anthony Maher said.

My guess is that [a startups] investors are not looking at this as a gulp moment of hyper-leverage, Maher said. They're looking at it as a serious way to get these companies into occupancy and production by paying rent and other fees and moving along in the life cycles of their businesses.

The Center for Breakthrough Medicines, which operates both an accelerator and a contract development and manufacturing organization at the Discovery Labs complex in King of Prussia, uses a milestone-based lease structure with some promising companies, CBM co-founder Audrey Greenberg said. Under that system, CBM initially leases space at cost to startups, andleases grow in cost commensurate with the funding and/or revenue those startups wind up bringing in.

CBM announced in late June that it plans to expand its facility by 1M SF, including 90K SF of cGMP space, with a focus on cell therapy. That expansion, as well as CBMs leasing model, is made possible by a $350M equity investment the facility received at the start of the year from SK Inc., the investment arm of South Korean conglomerate SK Group. That investment was announced only a few weeks before the Russian invasion of Ukraine, which the Federal Reserve cited as an inflationary factor in raising its interest rate, which in turn has made capital more expensive.

We have to acknowledge that pricing has changed, Greenberg said. I think that more can be done in terms of taking risk on the tenant base and underwriting credit in a creative way. But it's a challenging time to do that. So you know if it was last year or the year before, it might be different.

Bisnow/Virginia Baker

Hargrove's Justin Pagliaro, Breakthrough Properties' Joe Traynor, Colliers' Joseph Fetterman and Plymouth Group's Michael Davis at Bisnow's Philly Life Sciences Summit event, hosted at Plymouth's Budd Bioworks development in June 2022.

Panelists throughout the day pleaded for more assistance from state government. The commonwealth of Pennsylvanias Redevelopment Assistance Capital Program served as a small-yet-key financing piece for tenant space at 3.0 University Place and 401 North Broad, Maher said. A RACP-type program specific to life sciences, or private capital deployed in a similar way, would go a long way toward stabilizing many companies slogging through the biotech valley of death.

A backstop for a non-credit tenant that you believe in, or [funding] an incubator, could go a long way, Maher said. I can show you [potential tenants] that could stabilize on revenue. But you have a blip in the first six months, then you have a blip again at month 18, someone needs to fill that void. And the ask would not be $50M, it would be $5M.

Massachusetts and California created programs that filled crucial financing gaps in the past couple of decades, and their life sciences hubs are better for it, Mahon said. But part of the difficulty facing the Philadelphia market is that its suburbs are split across multiple states, introducing a competitive factor that ultimately works against the region on the whole, Delaware Innovation Space founder and CEO Bill Provine said. But New Jersey Gov. Phil Murphy has not let the presence of New York in the north and Philly in the south get in the way of offering economic incentives to companies through the New Jersey Economic Development Authority.

It's not just the state, every level has to be engaged in this, Philadelphia Department of Commerce Senior Director of Business Development Sam Woods-Thomas said. And that's why you see places like Massachusetts, New York and California eating our lunches, frankly. We hope that won't continue. We're going to work hard to make sure that doesn't continue.

Though it only represents a part of the regions ecosystem and has a much smaller budget, the city of Philadelphia is working on a way to address the problem of institutional support, Woods-Thomas said.

In the next year or so you're going to see policies and incentives coming from us that begin to make a lot more sense than what we have right now, he said.

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Can Philly's Developers Shepherd Cell And Gene Therapy Startups Through The 'Valley Of Death'? - Bisnow

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AAVIATE: Gene therapy via suprachoroidal drug delivery may lower treatment burden for patients with AMD – Modern Retina

Posted: July 11, 2022 at 2:38 am

Emily Kaiser [EK]: Hello, welcome. I'm Emily Kaiser, editor of Modern Retina. I'm sitting down with Dr. Rahul Khurana to discuss the Aaviate study. Dr. Khurana, can you tell us a little bit about the Aaviate data presented at the Angiogenesis meeting?

Rahul Khurana, MD [RK]: For sure, Emily.

Aaviate is a really exciting clinical trial that I've been involved with and at Angiogenesis this year, we presented the updated data on the first two cohorts. And so to give everyone kind of a background, obviously, gene therapy is a very exciting area of interest right now.

We know there's a lot of unmet needs in our treatments of macular degeneration. You know, these treatments often are very effective, but they require a lot of treatments. And there's a high burden with our current set of treatments out there, and gene therapy offers the potential for a one-time treatment to give us long-term, anti-VEGF suppression and really a long-term answer to this kind of chronic disease.

And so Aaviate is along with many gene therapy programs, or studies, that are looking to kind of tackle this. And the thing which is interesting about Aaviate is that most of gene therapy has really looked at it the traditional approaches. Either theyve gone intravitreal, which is something we're very used to because we do these injections, or done a subretinal gene therapy delivery, which requires surgery, which is much more invasive. And Aaviate utilizes a suprachoroidal approach.

And the benefit of this is that we get good drug delivery of inhibiting anti-VEGF gene therapy, but by delivering it in the office. And one of the benefits of suprachoroidal delivery over intravitreal delivery is that in intravitreal, there is a potential for a lot of exposure of the medicine to not just the back of the eye, but also the front of the eye. And we've seen in some early gene therapy programs, a lot of complications involving inflammation and hypotony.

In a suprachoroidal approach, you can get a very high concentration to the retina with very low concentration to the anterior segment.

EK: That's really interesting. Has anything developed since the presentation?

RK: the study has been ongoing. So what I presented at Angiogenesis was the first two cohorts in the sense that those patients had been fully enrolled, and we had up to date up to six months. So that was really exciting. And we'll kind of delve into some of the details there. But there's still cohorts three, four, and five, which are now fully enrolled. So since that time, we've now fully enrolled those patients. And we're basically waiting to hear back on updates, or we were waiting to hear back those results. And we need to once they're fully enrolled, we need to have the subsequent time to see how these patients do.

EK: And what are the next steps?

RK: Part of the next steps for gene therapy is really to finish up the clinical study. The patients are all enrolled, which is wonderful. Now we want to see how they did in these higher enrolling cohorts. So one thing that we haven't talked about is what were the results that we actually found from the first two cohorts? And so as I mentioned before, Aaviate takes patients who have been previously treated so these patients who were in the study were basically patients who needed to get multiple injections.

On average, they average nearly nine injections in the previous year, which is about an injection every five weeks. And we took those patients who basically needed regular anti-VEGF therapy, and we basically offered them a super coronal injection of RGX-314, which involves a novel Aaviate vector, which encodes for an anti VEGF monoclonal antibody fragment, which is transduced, or basically transvexed, the patient's own retinal cells to produce anti-VEGF protein to effectively give you long term suppression.

And the data showed that in the first two cohorts where this was done, not only was the treatment quite safe, there was a very low rate of inflammation and no serious adverse events. But more impressively, that the number of treatments had gone down dramatically. The patients in the study were able to maintain their visual acuity, which was wonderful to see. But more importantly, the number of injections went down significantly.

As I told you, before, most of these patients needed about an injection every five weeks, and in the study, the number of injections went down nearly 70 to 79% than they had before receiving the gene therapy, they were able to maintain the visual acuity, maintain the retinal anatomy, and the number of patients who didn't even need injections was nearly 30% in the first cohort, and nearly 40% in the second cohort. And that was quite exciting because this truly was kind of delivering on the promise of a once-and-done therapy. But as I said before, we really need the long-term data to kind of see how this translates and also we need to see how higher doses if we can get better efficacy and also maintain a very good safety profile.

EK: Wow. So what does this mean for clinicians and for patients?

RK: I think it offers a really exciting hope for both our patients and physicians. As we mentioned before, we have a lot of treatment options for anti-VEGF therapy and they do work very well. The problem is that they require a lot of treatments and there's a high treatment burden, and this is challenging for patients because not all patients can come back in there's a high rate of lost to followup, non-compliance, and non-adherence to the treatment regimens. And we've seen in Phase 3 clinical studies, especially in follow up in real world practice that when patients are not getting regular treatments, they lose vision. And that's why we've it's been hard to replicate the excellent results we've seen in the Phase 3 studies in real world practice. And the hope is that if one of these gene therapy treatments can work, we can offer a really one-and-done or a much more sustainable treatment therapy for our patients, which ultimately lead to better compliance and better visual outcomes.

EK: Fantastic. Well, thank you so much for the update.

RK: My pleasure. Thanks for having me.

Note: This transcript has been lightly edited for clarity.

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Myrtelle Announces Positive Data for Its investigational Proprietary rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the National Tay Sachs &…

Posted: July 11, 2022 at 2:38 am

WAKEFIELD, Mass.--(BUSINESS WIRE)--Myrtelle Inc. (Myrtelle or the Company), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced in a presentation by Armen Asatryan, MD, Chief Medical Officer, at the National Tay Sachs and Allied Diseases Annual Family Conference held in Denver, Colorado, that the Companys recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy for Canavan disease (CD) has shown encouraging early efficacy data and a favorable safety profile in the first three patients treated through their six month visit post-treatment in the Companys open-label Phase 1/2 First-in-Human (FIH) clinical trial at Dayton Childrens Hospital (Dayton, Ohio). Myrtelles FIH trial utilizes the Companys proprietary rAAV vector to directly target oligodendrocytes, the affected brain cells in CD responsible for producing myelin the insulating material that enables proper neuronal function. In CD, the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) encoding the enzyme Aspartoacylase (ASPA). The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function, thus enabling metabolism of N-Acetylaspartate (NAA), a neurochemical that is abundant in the brain, and thereby supporting myelination. Five patients have been treated in the ongoing Phase 1/2 study with favorable safety and tolerability results to date and no study drug-related adverse events.

Analyses of the first three patients at 6-months post-treatment by Magnetic Resonance Imaging (MRI) demonstrated increases in white matter and myelin in the brain. Improvements were also exhibited in scores on the Gross Motor Function Measure (GMFM) and Mullen Scales of Early Learning (MSEL), validated functional scales. Observed improvements contrast the continuous clinical deterioration expected with the natural progression of the disease.

The 6-month functional and anatomic data observed in the first three patients elicit hope when we compare it with the natural course of this devastating disease. Equally encouraging are positive changes in the patients reflexes, eye tracking, vocalizations to communicate, and overall awareness said Robert Lober, MD, PhD, FAANS, Co-Principal Investigator and Associate Professor of Pediatrics at Wright State University Boonshoft School of Medicine and Attending Neurosurgeon at Dayton Childrens Hospital in Dayton, OH. These improvements suggest the gene therapy and the route of administration are directly targeting the oligodendrocytes, the key cells affected in Canavan disease.

Per the FIH trial protocol, the gene therapy is administered as a single total dose of 3.7 x 1013 vg delivered by intracerebroventricular (ICV) injection to target the oligodendrocytes of the brain where ASPA activity is deficient in CD patients.

Myrtelles current investigational gene therapy targeting oligodendrocytes with a unique rAAV vector builds upon over two decades of science and effort with the potential, if successful, to usher in new treatment options for children with Canavan disease, commented Armen Asatryan, MD, MPH, Chief Medical Officer of Myrtelle. We intend to build on these encouraging initial findings and complete our current Phase 1/2 clinical study, following which we plan to engage with regulatory authorities to advance the program to the later stages of clinical development.

ABOUT MYRTELLE

Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. For more information, please visit the Companys website at: http://www.myrtellegtx.com.

ABOUT CANAVAN DISEASE

Canavan disease (CD) is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent the normal expression of Aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD, and only palliative treatments are available.

More information on Myrtelles clinical study in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com.

Forward-Looking Statements

This press release contains forward-looking statements. Words such as may, believe, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding encouraging early data support further development of rAAV-Olig001-ASPA as a potential therapeutic approach for children diagnosed with Canavan disease, the improvements suggesting that the gene therapy and the route of administration are directly targeting the oligodendrocytes, the key cells affected in Canavan disease, the potential of Myrtelles current investigational gene therapy targeting oligodendrocytes, to usher in new treatment options for children with Canavan disease. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based in information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelles program demonstrating safety and efficacy, as well as results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelles expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events or otherwise.

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Positive Initial Clinical Data from the B-LIEVE Dose-Confirmation Trial for FLT180a in … – Tyler Morning Telegraph

Posted: July 11, 2022 at 2:38 am

FLT180a generated protective FIX levels with no bleeding or need forFIX replacement

The treatmentwaswelltolerated witha good safety profile

LONDON, July 10, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced the presentation of safety and initial efficacy data from the first cohort of the Phase 1/2 dose-confirmation B-LIEVEtrial for FLT180a, the companys AAVS3-based gene therapy candidate for people with hemophiliaB, at the International Society on Thrombosis and Haemostasis (ISTH) Congress being held in London, July 9-13, 2022.

As of the data cut-off of May 23, 2022, a one-time FLT180a dose of 7.7e11 vg/kg generated a rapid increase of coagulation factor IX (FIX), reaching levels in the normal range (93, 92 and 80 IU/dL) for the three patients in cohort 1 through days 77, 56 and 36, respectively. Patients stopped FIX prophylaxis and did not require FIX replacement or experience bleeding following treatment with FLT180a.

The treatment and the prophylactic immune management regimen were well tolerated. No serious adverse events or infusion reactions were observed, and there has been no evidence of FIX inhibitors. All adverse events (AEs) were mild, and most were transient. AEs related to immune management were consistent with the known profiles of corticosteroids and tacrolimus.

Dosing of cohort two was completed in June, with early results showing a similar initial response to FLT180a. Based on the data from cohort one and consistent with the advice of the independent Data Monitoring Committee of the B-LIEVE trial, patients in cohort two received the same dose of FLT180a and prophylactic immune management regimen that were used in the first cohort.

As the data continue to evolve since the data cut-off for cohort one, two patients have experienced a decrease in FIX expression together with a mild and transient increase in liver enzymes. All patients continue to have expression levels above baseline, and no patient has experienced a bleed or required FIX supplementation.

The initial data show that FLT180a provides rapid and consistent elevations in FIX to normal levels, which can prevent bleeding and the need for regular FIX replacement in people with hemophilia B, said Pamela Foulds, MD, Chief Medical Officer of Freeline. Emerging data suggest that while FIX expression was maintained at protective levels, a further refined immune management regimen may be required to avoid mild and transient transaminitis and to sustain FIX levels in the normal range. Potential adjustments in cohort two and forthcoming results from that cohort will help us interpret this further.

Our strategy is to advance gene therapy programs that have the potential to deliver best-in-class or first-in-class treatments, said Michael Parini, Chief Executive Officer of Freeline. While we continue to believe FLT180a has the potential to deliver a best-in-class gene therapy for people with hemophilia B, the availability of other treatment options and the need to prioritize our valuable resources dictate that we evaluate strategic options for FLT180a. These include, but are not limited to, seeking a partner that would enable the continuation of FLT180a through Phase 3 development.

With two other promising programs in Fabry disease and Gaucher disease in the clinic, each with the potential to be first-in-class gene therapy treatments for these debilitating diseases, we will continue to focus our attention and resources on those programs that offer the highest value for patients and Freelines shareholders, said Parini.

The poster #PB0213 entitled Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients with Hemophilia B by Guy Young et al. will be presented from 6:30pm-7:30pm BST today and will be made available on the Investors section of Freelines website.

About the B-LIEVE Dose-Confirmation Trial

B-LIEVE is a Phase 1/2 dose confirmation trial of FLT180a using a short course of prophylactic immune management with the goal of normalizing FIX levels in patients with severe and moderately severe hemophilia B. The starting dose of 7.7e11 vg/kg was selected based on the results of the Phase 1/2 B-AMAZE dose-finding trial and modeling. The goal of the B-LIEVE trial is to finalize a dose for a pivotal Phase 3 trial that enables predictable and sustained factor IX (FIX) expression at protective levels.

About FLT180a for People with Hemophilia B

Freelines FLT180a candidate uses a potent AAVS3 capsid rationally designed for effective targeting and transduction of liver cells and containing an expression cassette encoding a gain of function Padua variant of human factor IX (FIX). FLT180a has been studied in B-AMAZE, a Phase 1/2 dose-finding trial with the goal of normalizing FIX activity in patients with moderately severe and severe hemophilia B. Patients treated in B-AMAZE are being followed in a long-term follow-up study. A Phase 1/2 dose-confirmation trial of FLT180a called B-LIEVE to finalize a dose for a Phase 3 pivotal trial is in progress.

About Hemophilia B

Hemophilia B is a rare, debilitating, hereditary bleeding disorder caused by a defect in the gene encoding coagulation factor IX (FIX). Hemophilia B is linked to the X chromosome and mainly affects boys and men; however, women who carry an affected copy of the coagulation factor gene may also experience symptoms. Hemophilia B is classified as mild, moderate or severe, depending on the level of FIX in the blood, and is diagnosed through blood tests. The 2020 Annual Global Survey by the World Federation of Hemophilia estimates that there are approximately 15,000 patients with hemophilia B in the United States, Europe and Japan. A meta-analysis using national registries in Australia, Canada, France, Italy, New Zealand and the UK estimated a prevalence in males of 3.8 in 100,000 or approximately 1 in 30,000.1

About Freeline Therapeutics

Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies. The company is dedicated to improving patient lives through innovative, one-time treatments that may provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally designed AAV vector, along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into the patients bloodstream. The companys integrated gene therapy platform includes in-house capabilities in research, clinical development and commercialization. The company has clinical programs in hemophilia B, Fabry disease, and Gaucher disease Type 1. Freeline is headquartered in the UK and has operations in Germany and the U.S.

Forward-Looking Statements

This press release contains statements that constitute forward looking statements as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the opinions, expectations, beliefs, plans, objectives, assumptions or projections of Freeline Therapeutics Holdings plc (the Company) regarding future events or future results, in contrast with statements that reflect historical facts. Examples include, among other topics, statements regarding the timing, progress and results of the Companys Phase 1/2 B-LIEVE dose confirmation clinical trial of FLT180a and data readouts from that trial, whether a further refined immune management regimen may be required, the potential of FLT180a to deliver a best-in-class gene therapy for people with hemophilia B, whether the Companys evaluation of strategic options for FLT180a will result in any particular course of action and the potential of the Companys programs in Fabry disease and Gaucher disease to be first-in-class gene therapy treatments for such diseases and offer the highest value for patients and the Companys shareholders. In some cases, you can identify such forward-looking statements by terminology such as anticipate, intend, believe, estimate, plan, seek, project or expect, may, will, would, could or should, the negative of these terms or similar expressions. Forward-looking statements are based on managements current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties, including the Companys recurring losses from operations; the uncertainties inherent in research and development of the Companys product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work and regulatory review, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preclinical and clinical data, including the possibility of unfavorable new preclinical, clinical or safety data and further analyses of existing preclinical, clinical or safety data; the Companys ability to design and implement successful clinical trials for its product candidates; whether the Companys cash resources will be sufficient to fund the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements for the Companys expected timeline; the potential for a pandemic, epidemic or outbreak of infectious diseases in the United States, United Kingdom or European Union, including the COVID-19 pandemic, to disrupt and delay the Companys clinical trial pipeline; the Companys failure to demonstrate the safety and efficacy of its product candidates; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing experience, which could result in delays in the development, regulatory approval or commercialization of its product candidates; and the Companys ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. We cannot guarantee that any forward-looking statement will be realized. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in the Companys Annual Report on Form 20-F for the fiscal year ended December 31, 2021 and in subsequent reports on Form6-K, in each case including in the sections thereof captioned Cautionary Statement Regarding Forward-Looking Statements and Item 3.D. Risk factors. Many of these risks are outside of the Companys control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law. For further information, please reference the Companys reports and documents filed with theU.S. Securities and Exchange Commission (the SEC). You may review these documents by visiting EDGAR on theSECwebsite at http://www.sec.gov.

Media Contact:

Arne Naeveke, PhD Vice President, Head of Corporate Communications arne.naeveke@freeline.life +1 617 312 2521

IR Contact:

investor@freeline.life

References 1 Iorio A et al. Annals of Internal Medicine 2019;171(8):540-7

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Positive Initial Clinical Data from the B-LIEVE Dose-Confirmation Trial for FLT180a in ... - Tyler Morning Telegraph

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TC BioPharm CEO, Bryan Kobel to Speak at the "Innovating Cell and Gene Therapy Quality Control Conference 2022" – PR Newswire

Posted: July 11, 2022 at 2:38 am

During the fireside chat, moderated by Caroline Peachey, Editor of the European Pharmaceutical Review, Kobel will focus on TC BioPharm's journey as a CGT startup, the challenges the Company has faced, and the impact of quality control on its development. Kobel will also offer advice to other cell and gene therapy manufacturers and discuss the industry landscape.

The "Innovating Cell and Gene Therapy Quality Control Conference 2022" will take place virtually on July 13, 2022. For more information, visit the conference's website.

TC BioPharm manufactures young, active gamma-delta T cells exogenously using donor blood, expanding the gamma delta t-cell population into the billions and infusing these healthy donor cells into cancer patients. The Company's allogeneic unmodified gamma-delta T cell product, OmnImmune has shown positive results from its Phase 1a/2b human study evaluating its safety and tolerability. OmnImmune targets the potential treatment of relapse/refractory Acute Myeloid Leukemia ("AML"). Additionally, the FDA granted orphan drug status for OmnImmune after reviewing the trial results. TC BioPharm also received MHRA and Research Ethics Committee approvals to initiate Phase 2b/3 gamma-delta T cell therapy clinical trials of OmnImmune.

About TC BioPharm (Holdings) PLC

TC BioPharm is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of gamma-delta T cell therapies for the treatment of cancer and viral infections with human efficacy data in acute myeloid leukemia. Gamma-delta T cells are naturally occurring immune cells that embody properties of both the innate and adaptive immune systems and can intrinsically differentiate between healthy and diseased tissue. TC BioPharm uses an allogeneic approach in both unmodified and CAR modified gamma delta t-cells to effectively identify, target and eradicate both liquid and solid tumors in cancer.

TC BioPharm is the leader in developing gamma-delta T cell therapies, and the first company to conduct phase II/pivotal clinical studies in oncology. The Company is conducting two investigator-initiated clinical trials for its unmodified gamma-delta T cell product line - Phase 2b/3 pivotal trial for OmnImmune in treatment of acute myeloid leukemia and Phase I trial for ImmuniStim in treatment of Covid patients using the Company's proprietary allogenic CryoTC technology to provide frozen product to clinics worldwide. TC BioPharm also maintains a robust pipeline for future indications in solid tumors and other aggressive viral infections as well as a significant IP/patent portfolio in the use of CARs with gamma delta t-cells and owns our manufacturing facility to maintain cost and product quality controls.

Forward Looking Statements

This press release may contain statements of a forward-looking nature relating to future events. These forward-looking statements are subject to the inherent uncertainties in predicting future results and conditions. These statements reflect our current beliefs, and a number of important factors could cause actual results to differ materially from those expressed in this press release. We undertake no obligation to revise or update any forward-looking statements, whether as a result of new information, future events or otherwise. The reference to the website of TC BioPharm has been provided as a convenience, and the information contained on such website is not incorporated by reference into this press release.

SOURCE TC BioPharm

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Hyperhidrosis Treatment Market: Rise in Demand for Minimally-invasive and Non-invasive Therapeutic Procedures to Boost Market – BioSpace

Posted: July 11, 2022 at 2:38 am

Wilmington, Delaware, United States, Transparency Market Research Inc.: Patients suffering from idiopathic hyperhidrosis experience extreme sweating of the axilla, soles, palms, or face. Elevated environmental temperatures or emotional stress worsens sweat secretion; however, this extra sweating can be caused without any clear triggers. Typically, these symptoms develop in adolescence, get worse after puberty, and reduce during old age.

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Individuals may express inconvenience associated with humid footwear or trouble handling paper, tools, musical instruments, or sports equipment. More importantly, patients are embarrassed while shaking or holding hands, meeting new people, and intimacy, consequently leading to social withdrawal.

The global market for hyperhidrosis treatment reached a valuation of ~US$ 1.2 Bn in 2018, and is projected to grow at a CAGR of ~4% between the years 2019 to 2027.

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Rise in Demand for Minimally-invasive and Non-invasive Therapeutic Procedures to Boost Market

Globally, the demand for non-surgical cosmetic and aesthetic procedures such as the botulinum toxin procedure for hyperhidrosis treatment, which is minimally-invasive or non-invasive in nature, has increased in the last two decades.

According to the American Society of Plastic Surgeons, the U.S. recorded 819% increase in botulinum toxin procedures from 2000 to 2017

The demand for non-surgical topical treatments, non-surgical microwave energy destruction of sweat glands, and botulinum toxin procedures is rising, owing to benefits such as minimum or no downtime, no wear and tear of skin surface, and minimal side effects.

Geographically, North America dominated the global hyperhidrosis treatment market, accounting for the largest share in 2018. The regions significant market share is attributed to high prevalence of hyperhidrosis among the population in the U.S.

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Asia Pacific Hyperhidrosis Treatment Market to Grow at a Rapid Pace

Asia Pacific was the third-largest market for hyperhidrosis treatment in 2018. The hyperhidrosis treatment market in the region is expected to expand at a high CAGR during the forecast period, due to new product launches in countries such as India and China, and rise in awareness about the available treatment options for hyperhidrosis treatment.

Key players operating in the global hyperhidrosis treatment market include GlaxoSmithKline plc, Ulthera, Inc., TheraVida, Dermira, Inc., Revance Therapeutics, Inc., Allergan plc, and Miramar Labs, Inc.

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Global Hyperhidrosis Treatment Market: Segment Analysis

In terms of treatment type, the global hyperhidrosis treatment market has been classified into botulinumtoxin A, iontophoresis, surgical treatments, topical treatments, non-surgical microwave energy destruction of sweat glands, and others. The botulinum toxin A segment is projected to dominate the global hyperhidrosis treatment market during the forecast period.

Based on hyperhidrosis type, the global hyperhidrosis treatment market has been categorized into axillary hyperhidrosis, palmar hyperhidrosis, plantar hyperhidrosis, and others (craniofacial, etc.). The axillary hyperhidrosis segment is anticipated to dominate the global hyperhidrosis treatment market from 2019 to 2027.

In terms of end user, the global hyperhidrosis treatment market has been divided into hospitals, ambulatory surgical centers, and others (dermatology clinics & cosmetic centers). In terms of market share, the hospitals segment is expected to dominate the global hyperhidrosis treatment market during the forecast period.

The market size and forecast for each of the segments have been provided in terms of US$ Mn for the period from 2017 to 2027, along with their respective CAGRs for the forecast period from 2019 to 2027, considering 2018 as the base year.

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Global Hyperhidrosis Treatment Market: Regional Outlook

Geographically, the global hyperhidrosis treatment market has been segmented into five major regions: North America (the U.S. and Canada), Europe (the U.K., Germany, France, Spain, Italy, and Rest of Europe), Asia Pacific (India, China, Japan, Australia & New Zealand, and Rest of Asia Pacific), Latin America (Brazil, Mexico, and Rest of Latin America), and Middle East & Africa (South Africa, GCC Countries, and Rest of Middle East & Africa).

The market size and forecast for each of these regions and the mentioned countries/sub-regions have been provided for the period from 2017 to 2027, along with their respective CAGRs for the forecast period from 2019 to 2027, considering 2018 as the base year. The research study also covers the competitive scenario in these regions.

Companies Mentioned in Report

The report also profiles major players in the global hyperhidrosis treatment market based on various attributes such as company overview, financial overview, treatment types, business strategies, and recent developments. These include

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Hyperhidrosis Treatment Market: Rise in Demand for Minimally-invasive and Non-invasive Therapeutic Procedures to Boost Market - BioSpace

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Why Shares of Bluebird Bio, CRISPR Therapeutics, and Editas Medicine Soared This Week – The Motley Fool

Posted: July 11, 2022 at 2:38 am

What happened

The downtrodden biotech space has kicked off the second half of 2022 with a boom. Hard-hit gene-editing and gene therapy companies in particular have started the back half of the year on the right foot. Underscoring this point, Bluebird Bio (BLUE 2.59%) stock has already risen by 17% over the holiday-shortened week as of Thursday's closing bell, according to data provided by S&P Global Market Intelligence.

What's more, shares of CRISPR Therapeutics (CRSP -1.92%) have gained 22.6% over the same period, and fellow gene editor Editas Medicine (EDIT -1.32%) also saw its equity rise in price by a healthy 20.7% this week. By contrast, Bluebird and Editas shares both fell by over 50% over the first six months of 2022, while CRISPR's stock price stumbled by a noteworthy 20% during the first half of the year.

Image Source: Getty Images.

What's behind this sudden trend reversal? The most likely explanation is simply short-sellers covering their positions (buying back their borrowed shares). In keeping with this theme, Bluebird, Editas, and CRISPR all saw a sharp rise in their short interest during the first six months of 2022. Short-sellers piled into these three names earlier this year due to the fact that they are all cash flow negative, which is a tough spot to be in during a persistent bear market and an era of rising interest rates. Bluebird, in fact, is staring down a serious cash crunch at the moment.

Short-sellers, for their part, are probably backing away at this stage for no other reason than to play it safe in the event that big pharma starts to go bargain shopping.

Why might big pharma target beaten-down gene-editing and gene therapy companies in the second half of the year? The key reason is that these high-value fields are starting to move beyond the research stage of their life cycle and into the realm of commercially available therapies.

Speaking to this point, Bluebird's gene therapies for beta thalassemia and cerebral adrenoleukodystrophy appear to be on their way toward a formal approval from the Food and Drug Administration (FDA) following a pair of positive advisory committee votes last month. What's more, CRISPR is also expected to file for regulatory approval for its Vertex Pharmaceuticalspartnered blood disorder candidate, exa-cel, later this year.

Are any of these three biotech stocks still worth buying? CRISPR is arguably the most attractive bargain among the three. The company's ex-vivo gene-editing platform has posted stellar trial results so far, and Vertex could very well decide to buy its partner as a result.

Bluebird, on the other hand, is a tough call. The company ought to have a compelling buyout case if the FDA does grant it a pair of approvals soon. The bad news is that the biotech's balance sheet may force a sale at a heavily discounted price (relative to the commercial potential of its lead assets).

Finally, Editas might simply get lost in the mix when everything is said and done. There are several gene-editing companies vying for the spot of top dog, and Editas' clinical pipeline lags in several key areas at the moment. Time will tell.

George Budwell has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends CRISPR Therapeutics, Editas Medicine, and Vertex Pharmaceuticals. The Motley Fool recommends Bluebird Bio. The Motley Fool has a disclosure policy.

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Why Shares of Bluebird Bio, CRISPR Therapeutics, and Editas Medicine Soared This Week - The Motley Fool

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Advanced Therapy Medicinal Products Market [REVENUE SOURCE] 2022 Gross Margins and Forecast Research Report till 2030 – Taiwan News

Posted: July 11, 2022 at 2:38 am

Key Companies Covered in the Advanced Therapy Medicinal Products Market Research are Spark Therapeutics, Inc., Bluebird Bio, Inc., Novartis AG, uniQure N.V., Celgene Corporation, Gilead Lifesciences, Inc., Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., MEDIPOST, Vericel Corporation and other key market players.

Global Advanced Therapy Medicinal Products Market is valued approximately at USD 7.9 Million in 2020 and is anticipated to grow with a healthy growth rate of more than 13.2% over the forecast period 2021-2027.

Advanced Therapy Medicinal Products have enabled the pharmaceutical industry and healthcare industry to open new modes for the treatment of incurable diseases and cancer types. The global Advanced Therapy Medicinal Products market is being driven by growing prevalence of diseases, an expanding healthcare industry and increasing research and development.

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For instance, in 2020, WuXi Advanced Therapies launched new closed process CAR-T Cell Therapy Platform to accelerate the timeframe for cell and gene therapy expansion, which is expected to boost the market growth. In 2020, EdiGene and Immunochina collaborated on Research & Development to develop allogeneic CAR-T therapy for cancer. Furthermore, the strategic development between hospitals and companies along with the government regulations and approvals will provide new opportunities for the global Advanced Therapy Medicinal Products industry. However, high manufacturing costs and low investments by the governments of less developed countries in the healthcare may impede market growth over the forecast period of 2021-2027.

In the regional analysis of the global Advanced Therapy Medicinal Products Market, North America is dominating the market while Asia-Pacific is expected to have the fastest growth rate among the key regions such as Asia Pacific, North America, Europe, Latin America, and Rest of the World. North America is the leading region across the world in terms of market share due to growing demand for therapies in ambulatory centers and hospitals, technological progressions coupled with the well-established healthcare infrastructure which has stimulated product demand in the region. Whereas, high growth rate of Asia-Pacific can be attributed to the increasing number of cancer patients and people getting more aware due to government and private investments in improving healthcare infrastructure in the region.

The objective of the study is to define market sizes of different segments & countries in recent years and to forecast the values to the coming eight years. The report is designed to incorporate both qualitative and quantitative aspects of the industry within each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, the report shall also incorporate available opportunities in micro markets for stakeholders to invest along with the detailed analysis of competitive landscape and product offerings of key players.

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The detailed segments and sub-segment of the market are explained below:

By Type:CAR-T TherapyGene TherapyCell TherapyTissue Engineered Product

By Region:North AmericaU.S.CanadaEuropeUKGermanyFranceSpainItalyROE

Asia PacificChinaIndiaJapanAustraliaSouth KoreaRoAPACLatin AmericaBrazilMexicoRest of the World

Furthermore, years considered for the study are as follows:

Historical year 2018, 2019Base year 2020Forecast period 2021 to 2027.

Target Audience of the Global Advanced Therapy Medicinal Products Market in Market Study:

Key Consulting Companies & AdvisorsLarge, medium-sized, and small enterprisesVenture capitalistsValue-Added Resellers (VARs)Third-party knowledge providersInvestment bankersInvestors

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Table of content

What is the goal of the report?

The market report presents the estimated size of the Market at the end of the forecast period. The report also examines historical and current market sizes. During the forecast period, the report analysis the growth rate, market size, and market valuation. The report presents current trends in the industry and the future potential of the North America, Asia Pacific, Europe, Latin America, and the Middle East and Africa markets. The report offers a comprehensive view of the market based on geographic scope, market segmentation, and key player financial performance.

What is the key information extracted from the report?

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Cell therapy cancer centre takes shape in Bengaluru, trials are on – The Indian Express

Posted: July 11, 2022 at 2:38 am

In 2019, when Immuneel Therapeutics Ltd, founded by Pulitzer winning author and US oncologist Dr Siddhartha Mukherjee and leading biotech entrepreneur Kiran Mazumdar Shaw, planned to set up a breakthrough cell therapy facility for cancer treatment in India, its top team never imagined the world would be hit by Covid-19. It faced the brunt of the pandemic, but the team adapted fast from vacating its one-room office in Bengaluru to locating the construction crew near the hospital and finding Indian vendors instead of outsourcing the project to a global firm through the last two years to get the facility up and running.

No amount of preparation would have helped us plan for something like this. There were several challenges: one, you are building something in a hospital, two, its a cancer facility, three, there were restrictions on the number of people you could bring in at a time, four, the entire supply chain was disrupted, and you couldnt get anything shipped from any of the usual suppliers. And we had to ensure high quality GMP (good manufacturing practices) production of cell therapies in a country like India where this had never been done before, said Dr Arun Anand, Chief Operating Officer, Immuneel Therapeutics.

A similar therapy has also been indigenously developed by Mumbais Tata Memorial hospital and IIT Bombay. It is at an early Phase I clinical trial stage.

Not only did Immuneel have to design the centre with GMP (good manufacturing practices), but the team conducting the trial also had to ensure adequate training for the staff, with many posted on Covid-19 duty. The centre is located on the eighth floor of Mazumdar Shaw Medical Center at Narayana Hrudayalaya Health City in Bengaluru.

The centre would conduct Phase II trial of a Spanish CAR-T therapy for treating patients with leukaemia and lymphoma. It has a strategic collaboration with Spain-based Hospital Clinic de Barcelona (HCB) and it has successfully conducted remote tech transfer with HCB on manufacturing protocols. The therapy modifies the patients T-cells using an inactivated HIV virus to weaponise them to fight the cancer cells. In CAR-T cell therapy, we extract t cells from a cancer patients body and use a virus a modified version of HIV virus that has no risk for HIV to deliver genes to the cells that weaponise the T-cells and can attack the cancer cells, said Dr Mukherjee.

We wanted to be in a hospital ecosystem for the team to feel connected to the mission of serving patients. A feeling of empathy is necessary to do something creative; to come up with solutions. We are on the eighth floor in the hospital, and the bone marrow transplant centre is on the seventh floor. Every day when patients go up and down the elevators, we are also travelling, and we see the patients and physicians. This is unique for a company; most companies prefer to set up in lovely ecosystems, glass-faade buildings, outside of the city, far from where the real work happens, said Dr Anand.

Explaining the process, Dr Mukherjee said, We remove the T-cells from the body through apheresis, they go into an extremely sterile laboratory where everyone is working with hoods and masks, no one is allowed to enter except for the people manipulating them, these T-cells are then weaponised using this gene therapy, and then they are grown in the lab in incubators until they increase to a large number, then they are frozen. This process takes about ten to fourteen days.

Then you have to do very careful quality control, which involves making sure that the product is sterile, that the gene therapy has actually worked, that the product has actually expanded and expanded in the right way. And, then it is released. In the hospital, it is transfused back into patients, he said.

With trials and innovations in India, the team hopes to bring down the cost of the therapy from the current US $350,000 to $400,000. The clinical trial has already started, and we have dosed several patients, said Dr Mukherjee who was in India recently for the project.

We were a small team then just 20 people and we occupied a small room on the seventh floor of the hospital. And one evening I was informed by the hospital administrator that they needed the entire floor because they wanted to convert that into a Covid-19 ward. They did it in the next two days. So, overnight we did not even have a room to sit in. What that meant is we implemented an early work-from-home plan, said Dr Anand.

When our construction started in early 2020, we arranged for the contractor to house the entire crew very close to the hospital. The entire crew was in 3-4 houses taken on long-term lease just a kilometre away from the hospital. The crew did not need to travel by bus or public transport, they could just walk. And all of them were tested on a periodic basis (for Covid-19), he said.We did a session with the workmen and the contractor, telling them what we are trying to do. When they realised their work would save the lives of children, they were motivated. I am a firm believer if the purpose is noble, there will be support from the ecosystem to make it happen, he said.

The construction period was stretched to accommodate for breaks and shorter shifts in view of protective equipment being worn by the workers. Instead of nine months, the project was completed in 12. The crew was also trained on wearing masks, and were regularly tested, but still ended up getting the infection, said Anand.

Dr Sharat Damodar, one of the principal investigators of the clinical trial, said, Training the staff took some time till recently Covid-19 was the priority, so most of our nurses and doctors were posted on Covid-19 duty so to pull them out in the middle of it for training for such a new therapy was obviously difficult. Most of the people were drawn from a pool of nurses and doctors who were not doing Covid-19 duty, who were helping in bone marrow transplant and high-end chemotherapy.

He said it was good the clinical trial started as soon as the Covid-19 cases dropped because patients were happy to come in for the new therapy, which promised a better outcome than existing therapies. Emphasising the need to bring the technology to India, he said prior to the pandemic, many Indians travelled to China for treatment, at a lower cost.

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Cell therapy cancer centre takes shape in Bengaluru, trials are on - The Indian Express

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