StemCell Therapy

Promising preliminary data from one of the first human trials testing the safety and efficacy of a CRISPR gene therapy has just been revealed. Although it is too early to evaluate long-term effects, the initial reports are impressively successful for two patients with severe genetic blood diseases.

Until February of this year, when pharmaceutical companies CRISPR Therapeutics and Vertex began a large global trial into a treatment called CTX001, no human outside of China had been officially treated with a CRISPR-based gene editing therapy.

CTX001 was developed to treat two types of inherited blood disease, beta-thalassemia and sickle cell disease. Both conditions are caused by a mutation in a single gene and the treatment involves engineering a patient's stem cells with a single genetic change designed to raise levels of fetal hemoglobin in red blood cells.

The newly announced data from the first two patients treated with CTX001 is nothing short of extraordinary. The first patient was treated with the CRISPR therapy at the beginning of 2019 for transfusion-dependent beta-thalassemia. The patients illness was so severe they required around 16 blood transfusions every year. Nine months after the single CTX001 treatment the patient was completely independent of the need for blood transfusions and their total hemoglobin levels were near normal.

The second patient, treated for sickle cell disease, demonstrated similar remarkable responses to the one-off gene therapy treatment. Four months after the CTX001 infusion the patients total hemoglobin levels had returned to normal and many of the disease symptoms had disappeared.

We are very encouraged by these preliminary data, the first such data to be reported for patients with beta thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate, CTX001, says Samarth Kulkarni, CEO of CRISPR Therapeutics. These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention.

Both patients did suffer from a small number of serious adverse events following the CTX001 treatment, however, the researchers conducting the trial are confident none of these side effects were related to the gene therapy. Instead, these side effects seem primarily related to a pre-CTX001 treatment involving the elimination of pre-existing mutated bone marrow cells to enable the healthy CRISPR-edited cells to reproduce.

It is certainly very early days for the research, and a number of patients are yet to be enrolled and treated in this current trial. Both the beta-thalassemia and sickle cell disease trials plan to enroll up to 45 patients, with a two-year follow-up planned to evaluate safety and efficacy. The Phase 1/2 open-label trials precede larger Phase 3 trials required for ultimate market approval, so these new therapies are still at least a decade away from clinical implementation.

Still, these initial results are as positive as one could hope for at this stage, establishing CRISPR gene editing as having exciting curative potential in human subjects. Whether the treatment holds for extended periods of time, and demonstrates longer safety profiles is yet to be determined. But, these early results are leaving researchers cautiously optimistic.

The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta thalassemia, says CEO of Vertex, Jeffrey Leiden. While the data are exciting, we are still in the early phase of this clinical program.

Source: CRISPR Therapeutics

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Encouraging early results from first human CRISPR gene therapy trials - New Atlas

Thanks to a $62 billion acquisition of Shire, Takeda is one of the world's largest developers of rare disease drugs.

Despite that, the 238-year-old Japanese pharmaceutical company lacks any mid- or late-stage cell or gene therapies, two technologies that figure to play a large role in how many rare cancers and inherited diseases will eventually be treated.

It's a mismatch Takedais putting substantial effort into addressing. Last week, executives made cell and gene therapy a notable focus of the company's first R&D day since closing its Shire deal.

"We have a world-class gene therapy platform," Dan Curran, head of Takeda's rare disease therapeutic area unit, told investors and Wall Street analysts gathered in New York city.

"We intend to build on that over the next five years. Because as we look to lead in the second half of [next]decade, we believe patients will demand and we can deliver transformative and curative therapies to patients globally."

But right now that's just an ambition. While Takedahas begun to explore how it can improve on current gene therapies, its candidates are early stage and lag their would-be competitors.

"Our heme A program we're behind. Our heme B program we're behind," admitted Curran in an interview. "But we're behind the first generation and when has there only been one generation of anything?"

Takeda's hemophilia A program is currently in Phase 1, with the hemophilia B candidate about to join it in human testing well back from leaders BioMarin Pharmaceutical, Spark Therapeutics and SangamoTherapeutics in hemophilia A and UniQure in hemophilia B.

Curran laid out three priorities for Takeda'spush: exploring whether gene therapy, typically pitched as a one-time treatment, can be re-dosed; lowering the doses currently used for first-generation therapies; and developing alternative gene delivery vehicles than the adeno-associatedand lentiviralvectors that are predominant today.

"We need to figure out how to re-dose AAVvectors if we want to provide functional cures for patients for the rest of their lives."

How long a gene therapy's benefit lasts is a critical question. In theory, it could last decades or potentially for life, depending on the treatment's target.

But clinical evidence presented to date suggests that benefit for some therapies could wane over time. BioMarin, for example, presented data this year that it argued is proof its gene therapy could raise Factor VIII expression levels in patients with hemophilia A above the threshold for mild disease for at least eight years a long time, to be sure, but not life-long.

Still, it's an unusual objective. Much of gene therapy's promise lies in the potential for it to be given just once and still deliver lasting benefits. And the therapies that have reached market most notably Spark Therapeutics' Luxturna, Novartis' Zolgensma and Bluebird bio's Zynteglo are among the most expensive drugs to ever reach market. Were a gene therapy to be re-dosed, the current value proposition those drugmakers describe would need to be re-evaluated.

Curran recognizes that bringing down costs substantially will be essential to any attempt to advance a multi-use gene therapy. But Takeda might have an advantage. In buying Shire, the pharma inherited a viral vector manufacturing plant, originally built by Baxalta, that Curran calls the company's "best kept secret."

"It's an enormous competitive advantage," he said, adding that Takeda believes it's among the industry's top three facilities by production capacity. "Roche trying to acquire Spark, Novartis and AveXis a significant component of value of those transactions was that these companies had actually invested in manufacturing capabilities."

Curran emphasized that Takeda's ambitions in gene therapy will require it to partner with academic leaders in the field, a playbook that it's followed over the past three years as it's worked to expand into cell therapy.

"In the cell space, there's more innovation you can bring up into proof of principle milestones in academia," said Andy Plump,Takeda'shead of R&D, in an interview.

"An academic can manipulate a cell, but it's very hard in an academic setting to optimize a small molecule," he added. "This is a space where Novartis, and now we, have been quite successful in creating those relationships."

Takeda has put partnerships in place with Japan's Center for iPS Cell Research and Application, GammaDelta, Noile-Immune Biotech, Memorial Sloan Kettering Cancer Center and, just this month, The University of Texas MD Anderson Cancer Center.

That last collaboration gives Takeda access to a chimeric antigen receptor-directed natural killer, or NK, cell therapy.The drugmaker believes NK cells could offer advantages over the T cells modified to create the currently available cell therapies Kymriah and Yescarta.

Most notably, MD Anderson's approach uses NK cells isolated from umbilical cord blood, rather than extracting T cells from each individual patient a time-consuming and expensive process that has complicated the market launch of Kymriah and Yescarta. Cord blood-derived NK cells are designed to be allogeneic, or administered "off the shelf."

Additionally, CAR NK cells haven't been associated (yet) with cytokine release syndrome or neurotoxicity, two significant side effects often associated with CAR-T cell therapies. That could help Takeda position its cell therapies as an outpatient option.

"Even if we were a company that entered a little bit later into the immuno-oncology space, we've very much tried to turn this into an advantage," said Chris Arendt, head of Takeda's oncology drug discovery unit, at the company's event.

"We believe we have a chance to establish a leadership position rather than jumping on the bandwagon and being a follower."

While Takeda's choice to pursue NK cell therapy stands out, its choice of target does not. TAK-007, a drug candidate from MD Anderson that is now Takeda's lead cell therapy program, is aimed at a cell surface protein called CD19 that's found in leukemias and lymphomas.

Both Yescarta and Kymriah target CD19, and a recent count by the Cancer Research Institute tracked 181 cell therapy projects aimed at the antigen.

Takeda is planning to advance TAK-007 into pivotal studies in two types of lymphoma and chronic lymphocytic leukemia by 2021, with a potential filing for approval in 2023.

By then, Kymriah and Yescarta will have been on the market for six years and current bottlenecks in cell therapy treatment could be solved, helping both Takeda's potential entry as well as the host of competitors it will likely face.

Next year will be a test of how productive Takeda'scell therapy unit can be. In addition to TAK-007, the pharmaexpects to have four other CAR-T and gamma delta cell therapies in the clinic, two of which will target solid tumors.

Cell and gene therapy are part of what Takeda calls its "second wave" of R&D projects, a group of early-stage drugs and programs that it sees as progressing to regulatory stages by 2025 or later.

In the nearer term, the drugmakeris advancing a "first wave" of clinical candidates that it told investors will deliver 14 new molecular entities by 2024. Five of those will come in rare disease, with the others spread across oncology, neuroscience, gastro-enterology and vaccines.

"We think the cascade of news coming forward on these programs will transform how people view Takeda," Curran said.

More importantly to the investors gathered in New York, Takeda expects these experimental drugs will eventually earn $10 billion in peak annual sales, which would represent a sizable addition to a business that generated $30 billion in sales last year.

Excerpt from:
Takeda sees cell, gene therapy in its future. Is it too late? - BioPharma Dive

CAMBRIDGE, Mass. Gene therapy pioneer Dr. James Wilson is disappointed by the progress in his field and expects current therapies and technologies to be soon surpassed by new approaches.

In five years, when we look back on the way were executing on gene therapy now, were going to realize that things are going to be very different, Wilson said at the STAT Summit on Thursday. The way in which were going to treat Duchenne muscular dystrophy, potentially cure it, is not the way in which its being evaluated in the clinic now.

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!

STAT Plus is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis.Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

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Gene therapy pioneer: Field is behind, and delivery tech is 'embarrassing' - STAT

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When its not working and we keep getting the same outcomes, something will change and it will be an urgent matter. The Alachua County School Board is starting to put together bold plans for closing the achievement gap between the districts black and white students. (WUFT News)

Global company Thermo Fisher Scientific has concluded its $6 million expansion at Progress Park in Alachua. One of its vice presidents celebrated that progress Friday, saying, Ive been in this industry for over 20 years in gene therapy, and over the last three years, its completely transformed. (WUFT News)

Marketplace visited Gainesville to see a University of Florida professors efforts to improve the flavor of tomatoes.

State Sen. Keith Perry is taking an interest in the problem of police arresting people with epilepsy during their seizures. (WTSP)

The first two Democrats vying to replace state Rep. Clovis Watson, who is term-limited from running again next November, have filed paperwork during the past month or so. Watson now intends to unseat Alachua County Sheriff Sadie Darnell. (WUFT News, News Service of Florida)

UFs fall semester is wrapping up in the next few weeks, but as it does so, theres a racial and political mess simmering that on Friday afternoon prompted a campuswide email from a vice president. (The Alligator)

One of Micanopys longtime religious leaders is stepping down after 37 years. Les Singletons final service as vicar took place yesterday at Church of the Mediator. (WCJB)

Three years ago, it seemed Amtrak was on a path to returning to serving parts of north Florida. The process of getting passenger trains going again between Pensacola and Jacksonville has moved extremely slowly. (WUFT News, Pensacola News Journal)

A slice of old Florida has been preserved at the Orange Lake Overlook at a cost of $1.3 million. Another piece of local Florida natural history was celebrated this weekend at Little Orange Creek. (Ocala Star-Banner, WUFT News)

Correction from Fridays edition, which had the incorrect title of Ibram X. Kendis latest book. It is called How to Be an Antiracist.

Theres no denying the importance of a good education.

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WFSU put together a helpful guide on how state Supreme Court justices come to hold that position. Its particularly timely with the governor set to make two more selections.

USA Today tracked a man who in the 1990s was to face trial on rape charges in Vermont but who has been living in Florida without consequence for more than two decades.

CBS spotlights the role Florida played in the 2016 election hacking by Russian operatives, including what happened within Annette Taddeos failed run for the U.S. House.

Not everyone is on board with recreational marijuana in Florida, and that group now has a political action committee. (BayNews9)

Being a prisoner (of high water) in Key Largo is not that bad, but it is reality, a resident there tells the New York Times. Its report on King Tide flooding is worth your time.

Whats west of Key West? The Dry Tortugas, and they have a fascinating 500-year history. (Florida Memory Blog)

Heres a helpful guide on some of the states worst invasive plant species. (Florida Today)

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The Point, Nov. 25, 2019: Gene Therapy Work Gets $6 Million Home In Alachua - WUFT

MANHATTAN, Kan., Nov. 21, 2019 /PRNewswire/ -- Libella Gene Therapeutics, LLC("Libella") announces an institutional review board (IRB)-approved pay-to-play clinical trial in Colombia (South America) using gene therapy that aims to treat and ultimately cure aging. This could lead to Libella offering the world's only treatment to cure and reverse aging by 20 years.

Under Libella's pay-to-play model, trial participants will be enrolled in their country of origin after paying$1 million. Participants will travel to Colombia to sign their informed consent and to receive the Libella gene therapy under a strictly controlled hospital environment.

Traditionally, aging has been viewed as a natural process. This view has shifted, and now scientists believe that aging should be seen as a disease. The research in this field has led to the belief that the kingpin of aging in humans is the shortening of our telomeres.

Telomeres are the body's biological clock. Every time a cell divides, telomeres shorten, and our cells become less efficient at dividing again. This is why we age. A significant number of scientific peer-reviewed studies have confirmed this. Some of these studies have shown actual age reversal in every way imaginable simply by lengthening telomeres.

Bill Andrews, Ph.D., Libella's Chief Scientific Officer, has developed a gene therapy that aims to lengthen telomeres. Dr. Andrew's gene therapy delivery system has been demonstrated as safe with minimal adverse reactions in about 200 clinical trials. Dr. Andrews led the research at Geron Corporation over 20 years ago that initially discovered human telomerase and was part of the team that led the initial experiments related to telomerase induction and cancer.

Telomerase gene therapy in mice delays aging and increases longevity. Libella's clinical trial involves a new gene-therapy using a proprietary AAV Reverse (hTERT) Transcriptase enzyme and aims to lengthen telomeres. Libella believes that lengthening telomeres is the key to treating and possibly curing aging.

Libella's clinical trial has been posted at the United States National Library of Medicine (NLM)'s clinicaltrials.gov database. Libella is the world's first and only gene therapy company with a clinical trial posted at clinicaltrials.gov that aims to reverse the condition of aging.

On why they decided to conduct its project outside the United States, Libella's President, Dr. Jeff Mathis, said, "Traditional clinical trials in the U.S. can take years and millions, or even billions,of dollars. The research and techniques that have been proven to work are ready now. We believe we have the scientist, the technology, the physicians, and the lab partners that are necessary to get this trial done faster and at a lower cost in Colombia."

Media Contact:Osvaldo R. Martinez-ClarkPhone: +1 (786) 471-7814Email: ozclark@libellagt.com

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william-bill-andrews-ph-d.jpg William (Bill) Andrews, Ph.D. Dr. Bill Andrews is a scientist who has spent his entire life trying to defeat the processes that cause us to age. He has been featured in Popular Science, The Today Show, and numerous documentaries on the topic of life extension including The Immortalists documentary.

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Dr. Bill Andrews speech at RAADfest 2018 (Sept 21, San Diego, CA)

bioaccess: Libella's CRO partner in Colombia.

SOURCE Libella Gene Therapeutics, LLC

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Breakthrough Gene Therapy Clinical Trial is the World's First That Aims to Reverse 20 Years of Aging in Humans - PRNewswire

NEW YORK--(BUSINESS WIRE)--IVERIC bio, Inc. (NASDAQ: ISEE) announced today the appointment of Guangping Gao, PhD, as Chief Strategist, Gene Therapy. Dr. Gao brings over 30 years of scientific research experience in gene-based treatments. As one of the worlds leading gene therapy experts, Dr. Gaos highly distinguished career includes major contributions to the development of adeno-associated virus (AAV) gene delivery technology. Dr. Gao is the current President of the American Society of Gene and Cell Therapy (ASGCT). In his advisory role, Dr. Gao will help shape IVERIC bios gene therapy strategy going forward.

On behalf of the Board of Directors and management team at IVERIC bio, we are extremely excited to welcome Dr. Gao to IVERIC bio, stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. Our goal at IVERIC bio is to become a leading retina company with a diversified portfolio addressing significant unmet medical needs in large market age-related retinal indications and inherited retinal diseases by utilizing both therapeutics and gene therapies. Dr. Gaos extensive background in gene therapy will be instrumental in shaping the direction of our gene therapy portfolio. It is a true privilege to work with him closely and benefit from his extensive expertise during these exciting times at our company.

I am delighted to have the opportunity to be a part of the IVERIC bio team, stated Dr. Gao. I am impressed with the progress that IVERIC bio had made with their diverse pipeline of novel gene therapy solutions to treat orphan inherited retinal diseases (IRDs). I look forward to working closely with the team to help build and maximize the potential of the Companys gene therapy programs.

Dr. Gao is a world-renowned scientist and researcher who is a pioneer in AAV gene delivery technology, stated Kourous A. Rezaei, MD, Chief Medical Officer of IVERIC bio. We believe that the combination of Dr. Gaos expertise in gene therapy and our knowledge and experience in drug development for retinal diseases will help set the stage for the proficient execution of our gene therapy strategy, advancing it to the next stage of evolution. We will also continue to move forward our Zimura, complement C5 inhibitor, programs as expeditiously as possible.

About Guangping Gao, PhD

Dr. Gao is the Co-Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems and Penelope Booth Rockwell Professor in Biomedical Research, at the University of Massachusetts Medical School; an elected fellow of both the US National Academy of Inventors (NAI) and the American Academy of Microbiology; and the current President of the American Society of Gene and Cell Therapy.

Dr. Gao is an internationally recognized gene therapy researcher who has played a key role in the discovery and characterization of a new family of adeno-associated virus (AAV) serotypes, which was instrumental in reviving the gene therapy field, providing technology to enable potential treatments for many currently untreatable human diseases. For nearly 30 years of his scientific research career, Dr. Gao has primarily focused on molecular genetics and viral vector gene therapy for rare genetic diseases, with research encompassing disease gene cloning, causative mutation identification, pathomechanism investigation, animal modeling, novel viral vector discovery and engineering for in vivo gene delivery, vector biology, preclinical and clinical gene therapy product development, and viral vector manufacturing for preclinical and clinical gene therapy applications, as well as development of technology platforms for novel approaches for human gene therapy.

Dr. Gao has published 267 research papers, 6 book chapters, and 5 edited books and serves as Editor of Human Gene Therapy, Senior Editor of the Gene and Cell Therapy book series, Associate Editor of Signal Transduction and Targeted Therapy, and on the editorial boards of several other gene therapy and virology journals. Dr. Gao holds 135 patents with 239 additional patent applications pending. Dr. Gaos inventions have been licensed to and are currently in development by over ten pharmaceutical companies. Recently, Dr. Gao was ranked as #4 on Nature Biotechnologys list of the Worlds Top 20 Translational Researchers for 2017.

About IVERIC bio

IVERIC bio is a biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. Vision is Our Mission. For more information on the Company please visit http://www.ivericbio.com.

IVERIC bio Forward-looking Statements

Any statements in this press release about the Companys future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include any statements about the Companys strategy, future operations and future expectations and plans and prospects for the Company, and any other statements containing the words anticipate, believe, estimate, expect, intend, goal, may, might, plan, predict, project, target, potential, will, would, could, should, continue, and similar expressions. In this press release, the Companys forward looking statements include statements about the implementation of its strategic plan to seek to address significant unmet medical needs in large market age-related retinal indications and inherited retinal diseases by utilizing both therapeutics and gene therapies, the timing, progress and results of clinical trials and other research and development activities, including manufacturing activities, the potential utility of its product candidates and the potential for its business development strategy. Such forward-looking statements involve substantial risks and uncertainties that could cause the Companys development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, those related to the initiation and the conduct and design of research programs and clinical trials, establishment of manufacturing capabilities, availability of data from these programs, reliance on university collaborators and other third parties, expectations for regulatory matters, need for additional financing and negotiation and consummation of business development transactions and other factors discussed in the Risk Factors section contained in the quarterly and annual reports that the Company files with the Securities and Exchange Commission. Any forward-looking statements represent the Companys views only as of the date of this press release. The Company anticipates that subsequent events and developments will cause its views to change. While the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so except as required by law.

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IVERIC bio Appoints Guangping Gao, PhD, Internationally Recognized Gene Therapy Pioneer as Chief Strategist, Gene Therapy - Business Wire

The gene therapy field made significant strides this year, and 2020 looks set to bring further advances, particularly as a three-way race to develop a hemophilia A gene therapy progresses.

BioMarin Pharmaceutical, Spark Therapeutics and Sangamo Therapeutics are all developing gene therapies for hemophilia A, the inherited disorder characterized by frequent bleeding episodes due to the lack of an essential blood-clotting protein.

While drug candidates from all three biotechs are currently in clinical testing, each is in a distinctly different spot as the companies head into 2020. Here's an overview of how the hemophilia gene therapy space is shaping up and what to watch for over the next 12 months.

BioMarin is well-positioned to be first to market with a gene therapy known as valrox, short for valoctocogene roxaparvovec.

The San Rafael, California-based biotech reported interim Phase 3 results from 16 patients in May, and stated it would file for accelerated approval with the Food and Drug Administration by year end. Since then, BioMarin executives have reiterated that regulatory timeline.

Yet even as BioMarin nears an FDA submission, clinical questions still surround valrox.

Interim Phase 3 data fell short of an earlier trial, leading company executives to blame a difference in timing of steroid use. In the prior study, steroids were typically given to study participants three weeks after infusion, while the Phase 3 trial prescribed steroid treatment after 10 weeks.

And three-year follow-up data from that Phase 1/2 trial showed patient levels of the Factor VIII clotting protein declined over time, raising questions on the product's long-term durability.

That sets up 2020 as the potential year for valrox's approval and launch, which could make it the third or fourth gene therapy for an inherited disease to reach market.

If that occurs, BioMarin will face an important decision on setting valrox's price.Unlike Novartis' Zolgensma, a gene therapy for spinal muscular atrophy that launched this year with a $2.1 million price, hemophilia patients have a range of existing treatments that are used to manage and control their disease.

Particularly if the therapy's price falls in the seven figures, payers could very well push back and negotiate. Concepts such as installment payment plans or outcome-based arrangements have floated as options around by gene therapy companies but remain incremental changes to a system still geared toward chronic therapy.

BioMarin also plans to present four-year follow-up data from the Phase 1/2 study in mid-2020 and Phase 3 data on reducing annual bleed rates in the fourth quarter of 2020 or first quarter of 2021.

At least publicly, Spark has gone quiet on how it's hemophilia candidate is progressing. The Philadelphia-based biotech stopped providing updates on its clinical testing since announcing a deal in February to be acquired by Roche. But that $4.8 billion acquisition has been held up for months by the Federal Trade Commission.

A portion of that scrutiny is presumably over Roche acquiring a gene therapy candidate while also marketing Hemlibra, a prophylaxis factor treatment first approved in 2017. Hemlibra has quickly become a key drug for the Swiss pharma, recording $930 million in sales through the first nine months of 2019.

As FTC mulls the deal, Spark has pushed off sharing interim updates on its gene therapy SPK-8001. While company slides presented at the beginning of 2019 showed plans for a midyear update, that never materialized.

"The guidance we provided about a hemophilia A update in mid-2019 was made prior to the announcement of the pending Roche acquisition," a Roche spokesperson wrote to BioPharma Dive in July. "We have no further comment at this time while we focus on completing the transaction."

In the months since, the biotech has stayed quiet. The latest clinical update is more than a year old, with a data cut-off of Nov. 2, 2018 for its Phase 1/2 trial. Those results included 12 patients, with seven tested at the high dose.

While most patients stopped suffering from bleeds and needing infusions, one patient treated with SPK-8001 was hospitalized and needed adrenocortical steroids. Spark stated it planned to include prophylactic steroids going forward in testing.

Spark has started a run-in study for the Phase 3 program, the company disclosed in its first quarter financial filing. While the company has not clarified where that program now stands, a federal database of clinical trials shows the lead-in study actively recruiting for 55 patients with a completion date of July 2020.

Sangamo has been the wild card in the race with impressive, albeit early, efficacy and safety data.

Early results from a Phase 1/2 trial turned heads in April by showing a small handful of patients treated with SB-525 had normal Factor VIII activity levels six weeks after treatment. And a further update in July backed that result up, showing more patients sustaining normal levels of the essential blood-clotting protein, so far at least.

Pfizer will take over late-stage testing of SB-525 through a 2017 licensing deal with Sangamo. CEO Albert Bourla said it has completed transferring the manufacturing process and enrolled the first patient in October for a six-month lead-in trial for the Phase 3 program.

Phase 3 dosing is expected to begin in the first half of 2020, Bourla said on Pfizer's third quarter earnings call.

For its part, Sangamo will also present updated data from its Phase 1/2 trial on Dec. 7 at the American Society of Hematology's annual meeting in Orlando, Florida.

The hemophilia gene therapy race is poised to evolve in 2020. BioMarin could submit valrox for potential approval, while industry leaders in Roche and Pfizer will both be working to advance Spark's and Sangamo's products, respectively.

And while BioMarin has a sizable lead, the biotech will still need to capitalize on its pole position to gain FDA approval and establish the market.

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The hemophilia gene therapy race faces a critical year in 2020 - BioPharma Dive

BioMarin Pharmaceuticals is seeking marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A.

The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the experimental gene therapy, formerly known as BMN 270. Administered as a single infusion, the therapy uses adeno-associated virus (AAV) vectors to deliver a functional copy of clotting factor VIII, the protein that is missing in people with hemophilia A.

An ongoing Phase 3 trial, GENEr8-1 (NCT03370913), is investigating the treatments safety and efficacy, and is still recruiting adult patients. Go here for more information on trials locations and here for eligibility criteria.

The EMA had previously given valoctocogene roxaparvovec the designation of priority medicines, or PRIME, in 2017. Now, the potential therapy has been granted accelerated assessment, which may potentially shorten its MAA review process from 210 to 150 days.

Accelerated assessment is given by the EMAs Committee for Medicinal Products for Human Use and Committee for Advanced Therapies to innovative medications that are of major interest to public health. This endorsement is meant to speed up the review process of eligible medications, but does not impact the committees decision to recommend their approval.

BioMarins MAA submission was based on updated three-year data from a Phase 1/2 study (NCT02576795)and on an interim analysis of the ongoing Phase 3 GENEr8-1 trial (NCT03370913), which is still recruiting an anticipated 130 patients from 73 sites around the world to test the dose of 6e13 vg/kg (vector genomes per kilogram). Another Phase 3 trial, the GENEr8-2 (NCT03392974) study, is also ongoing and testing a lower dose (4e13 vg/kg).

Three-year data from the Phase 1/2 trial showed that a single administration of valoctocogene roxaparvovec at the higher dose markedly reduced bleeding episodes and the need for factor VIII infusions in a small group of adults with severe hemophilia A. Specifically, there was a 96% reduction in both the mean ABR (annualized bleed rate) and the mean factor VIII usage over the three years.

The levels of clotting factor VIII remained stable over the course of three years following treatment.

Valoctocogene roxaparvovec was generally well-tolerated by patients. None of the participants developed inhibitors to factor VIII, and none withdrew from the study due to adverse events.

We are grateful to the study participants, who have made this progress possible in the span of approximately four years since the first participant was enrolled in the clinical program, Hank Fuchs, MD, president of BioMarins global research and development, said in a press release.

We are very pleased with the level of engagement we have had with global health authorities, as it aligns with our belief that gene therapy represents the next wave of innovation and potentially could be a meaningful advancement for treating people with severe hemophilia A, Fuchs said.

Valoctocogene roxaparvovec will be the first gene therapy for hemophilia whose MAA will be reviewed by health authorities for potential approval in the E.U. BioMarin is expecting the EMA to start reviewing its application in January 2020 and said it will provide an update at that time.

In the meantime, the company is planning to submit a biologics license application for valoctocogene roxaparvovec to the U.S. Food and Drug Administration (FDA) by the end of the year. The investigational treatment has been given a breakthrough therapy designation by the FDA, as well asorphan drug status from both the FDA and EMA.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

Total Posts: 121

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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For Hemophilia A, BioMarin Seeks Approval of Its Gene Therapy in Europe - Hemophilia News Today

Dive Brief:

Fujifilm is looking to take advantage of the gene therapy market which, for CDMOs, is expected to reach $1.7 billion by 2025, according to Fujifilm Diosynth Biotechnologies' market research.

"We are very much aware of the incredible growth in such an important therapeutic space," Martin Meeson, president and chief operating officer of FDB U.S., said in a Nov. 14 statement. "We know that we need to invest now, in technology, assets and people in order to achieve a market leadership position."

The new Gene Therapy Innovation Center on the existing FDB campus in Texas will be about 60,000 square feet and operational starting in fall 2021. FDB expects the center to triple its gene therapy development capabilities and add about 100 jobs.

Meanwhile, the first stage of the expansion for the existing manufacturing facility should be complete by the spring of 2021, Fujifilm said.

The Japan-based company's fresh investments in gene therapy come amid a wave of expansions, mergers and partnerships in the growing field.

This month, Swiss manufacturer Lonza announced a new partnership with cold chain specialist Cryoport as part of its goal to provide a seamless "vein-to-vein" network in cell and gene therapy. Lonza also opened a 300,000-square-foot plant last year in Texas dedicated to manufacturing the therapies.

Catalent earlier this year bought Paragon Bioservices for $1.2 billion to strengthen its position as a CDMO of gene therapies. And Thermo Fisher acquired viral vector manufacturer Brammer Bio for $1.7 billion.

Fujifilm forecasts sales of 100 billion yen for its CDMO business in the fiscal year ending March 2022.

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Fujifilm to invest $120M in gene therapy, build center in Texas - BioPharma Dive

The future treatment of heart attacks could see doctors deploying gene therapy treatments that deactivate key genes, thanks to research underway at the University of Sheffield.

Researchers at the university, which has been awarded 800,000 from the British Heart Foundation to conduct the project, are working on developing a drug to deactivate the gene that is key to the development of cardiovascular disease (CVD).

We will be identifying a drug to target a gene which contributes to the build-up of fatty material in the arteries, explained Professor Paul Evans, from the University of Sheffields Department of Infection, Immunity and Cardiovascular Disease (IICD), who is leading the project.

Switching this gene off will make the arteries in the heart less leaky, preventing cholesterol from building up and causing blockages which present as heart attacks and stroke.

Atherosclerosis, where theres a build-up of fatty substances in the blood vessels, is an underlying cause of life-threatening conditions such as heart attacks and strokes, added Professor Jeremy Pearson, associate medical director at the British Heart Foundation.

We know that variations in our genes play a part in the development of atherosclerosis, so if these genes can be switched off, many lives could be saved from heart and circulatory diseases.

Gene therapy is an emerging field within medicine that is often characterised as one of the most high-potential areas for future healthcare.

The first gene therapy drug to enter the market only gained approval in 2017, making it a very young field, and one where there is still considerable potential for development.

Treating heart attacks and other CVDs with gene therapy remains a very novel concept, requiring considerable cross-disciplinary skills to explore.

As a result, Evans team is collaborating with IICD and gene specialists in the Department of Neuroscience, as well as colleagues at other institutions, both nationally and internationally.

We are partnering with many organisations known for their research excellence, including scientists at the Georgia Institute of Technology in the USA who will contribute their expertise and technology to process and develop the resulting gene therapy, said Evans.

Read more: A groundbreaking gene therapy is offering hope for a haemophilia cure

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Scientists target gene therapy to 'switch off' heart attack-causing genes - Verdict

SOUTH SAN FRANCISCO, Calif., Nov. 21, 2019 /PRNewswire/ --CODA Biotherapeutics, Inc., a preclinical-stage biopharmaceutical company developing a chemogenetic gene therapy platform to treat neurological diseases, today announced the appointment of Annahita Keravala, Ph.D., as Senior Vice President, Gene Therapy. In this role, Annahita will lead the gene therapy aspects of CODA's chemogenetic platform. The company also announced the promotions of Orion Keifer, M.D., Ph.D., to Vice President, Discovery and Translational Research, and Steve Dodson, Ph.D., to Vice President, Pharmacology and Early Development, respectively.

Michael Narachi, President and Chief Executive Officer, said, "We are excited to have Annahita, Orion, and Steve in these vital roles as we advance our lead candidates toward the clinic, as well as build our pipeline based on CODA's chemogenetic gene therapy platform. Their combined gene therapy, neuroscience and small molecule expertise, and experience in early stage research and development, will prove invaluable. With this leadership team, we will fulfill our mission of discovering and developing transformative therapies for patients with intractable neurological diseases for whom limited or no treatment options exist."

Annahita brings more than two decades of experience in gene therapy using viral and non-viral vectors. In particular, she has extensive expertise in discovering novel vector technologies and gene therapy drug development for ophthalmic, systemic and inflammatory diseases. Annahita joins CODA from Rocket Pharmaceuticals, where she was Associate Vice President, AAV Platform. At Rocket, she provided strategic, scientific and operational leadership, oversaw all aspects of discovery research, preclinical and assay development, and provided technical insight to the Chemistry, Manufacturing and Controls (CMC) team. This culminated in a successful Investigational New Drug (IND) application filing.

"I am thrilled to be joining CODA at such an exciting time in the Company's development and growth. What attracted me was the opportunity to partner with Mike, Orion, Steve and our talented team to help bring cutting-edge therapeutic options to patients living with some of the most challenging and debilitating neurological conditions and disorders like chronic neuropathic pain and focal epilepsy for which there remains great unmet medical need," said Dr. Keravala.

Prior to her tenure at Rocket Pharma, Annahita held several positions of increasing responsibility at Adverum Biotechnologies (formerly Avalanche Biotechnologies). As Director of Adverum's Novel Vector Technology group, she designed the overall research strategy, led her team to discover and optimize next-generation adeno-associated virus (AAV) vectors, and oversaw process development and preclinical testing to support the company's pipeline. Earlier, Annahita was a Research Scientist at Stanford University School of Medicine. An author of multiple patents, she is also widely published in prestigious scientific journals. Annahita earned a Ph.D. in molecular genetics and biochemistry from the University of Pittsburgh, a M.Sc. in life sciences and biotechnology from the University of Bombay, Bombay, India, and a B.Sc., with Honors in life sciences and biochemistry from St. Xavier's College, Bombay, India. She completed a post-doctoral fellowship in the Department of Genetics at Stanford University School of Medicine.

CODA's Vice President, Discovery and Translational Research, Orion Keifer, M.D., Ph.D., is a neuroscientist with neurosurgical training and hands-on expertise in small and large animal models, focused small molecule, and cell and gene therapies for neurological diseases. Before joining CODA, he worked as consultant translational scientist and surgeon for Above and Beyond focusing on precision medicines for neurodegenerative disorders. Orion earned an M.D. and a Ph.D. in neuroscience from Emory University, and a M.S. in brain and cognitive neurosciences and B.S. degrees in biomedical engineering, applied psychology and applied biology with Highest Honors from Georgia Tech. He completed his post-doctoral training in the Department of Neurosurgery at Emory University.

Steve Dodson, Ph.D., is CODA's Vice President, Pharmacology and Early Development.Prior to joining CODA, Steve served as Senior Director, Drug Discovery and Development at Second Genome, Inc. Previously he held positions of increasing responsibility at NeuroTherapeutics Pharma, Inc., and Renovis, Inc., where his work focused on the discovery and development of small molecule therapeutics to treat pain, central nervous system disorders and inflammation. Steve received his Ph.D. in biological sciences from Stanford University and a B.S. in genetics from University of California, Berkeley.

About CODA BiotherapeuticsCODA Biotherapeutics, Inc., is a preclinical-stage biopharmaceutical company developing an innovative gene therapy platform to treat neurological disorders and diseases. The company is creating the ability to control neurons with its revolutionary chemogenetics-based technology. CODA is located in South San Francisco, CA. For more information, please visit http://www.codabiotherapeutics.com.

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SOURCE CODA Biotherapeutics, Inc.

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CODA Biotherapeutics Deepens Gene Therapy Expertise with Industry Veteran, Annahita Keravala, Ph.D., and Key Promotions - P&T Community

Gene therapy is an emerging field and industry is yet to work out how best to manufacture these life-saving products says expert. Interest in gene therapy is growing, says Andrew Powaleny, public affairs director at the Pharmaceutical Research and Manufacturers of America (PhRMA).

As of December 2018, the last time we did our analysis, there were 289 cell and gene therapies in development either in clinical trials or awaiting FDA approval, he adds.

And the interest is understandable. Gene therapies let doctors treat the root causes of disease, potentially curing patients. Also judging by products like Zynteglo and Zolgensma, which cost $1.7m and $2.1m, respectively, gene therapies will generate significant revenue for firms able to commercialize them successfully.

But manufacturing a gene therapy remains a complex technical challenge. Just ask Zynteglo developer, Bluebird Bio.

Zynteglo is designed to treat patients with transfusion-dependent -thalassemia (TDT). Development has not always been smooth. For example, in 2017 Bluebird announced it had made manufacturing process improvements for Zynteglo after the product disappointed in an earlier trial. However, in June this year it appeared Bluebird had turned things around when the EMA granted Zynteglo conditional clearance. But, in the approval presentation Bluebird announced it would not be able to launch the product until 2020.

Bluebird explained the delay was to allow it to work with the EMA to finalize commercial drug product specifications and manufacturing parameters. The situation changed again last month. The EMA accepted refined commercial drug product manufacturing specifications for Zynteglo. And Bluebird now expects the therapy to launch this year.

Similarly, Novartis, owner of the spinal muscular atrophy therapy Zolgensma, has encountered manufacturing issues. In October the firm said the EMA and Japans PMDA had extended their assessments of Zolgensma and asked for more CMC information.

Bluebird and Novartis travails are the high-profile examples, but the wider gene therapy sector faces manufacturing challenges according to Ashleigh Wake, laboratory director, Intertek Pharma Services Manchester.

Given the newness of medicines of this type there is limited historic information available on which to build strategy and as such adds extra uncertainty to any regulatory submission, says Wake. When considering how to build an effective CMC for a gene therapy IND, selection of critical assays will depend on the specific product but may include assays for concentration, purity such as determination of residual cellular DNA or empty viral particles, identity, activity, potency and stability.

Understanding which tests are critical to determining product quality is a key part of winning approval, continues Wake. From a regulatory perspective, an understanding of the critical quality attributes (CQAs) which impact product safety, purity, and potency are required. For gene therapy products this means developing and validating analytical assays to assess, vector productivity, vector purity, biological activity and safety.

With this in mind Intertek recently announced plans to expand its service center in Manchester in the UK.

Our expansion in laboratory footprint and recruitment of specialists in gene therapy analytics coupled with our integrated approach to analytical method lifecycle will mean we can develop methods which are best suited for the intended use at the various clinical stages on the way to commercialization, he points out.

The challenges will keep coming, according to Wake. She says therapies that use viral vectors will increase demand for quality control expertise and analytical technology.The inherent complexity of viral vector-based products makes physical and biological characterization highly challenging, she explains, citing the ability to differentiate between full capsids and empty ones as an example. In order to provide a complete quality control package, a range of analytical methods and technology are required, which incorporate instrumentation such as cryoelectron microscopy, qPCR or DDPCR which are not commonly associated with pharmaceutical quality control, in addition to techniques such as HPLC or analytical ultracentrifugation.

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Gene Therapy Production and Quality Control - Genetic Engineering & Biotechnology News

NEW YORK, Nov. 18, 2019 /PRNewswire/ --

The global cell and gene therapy market is growing at a CAGR of over 24% during the forecast period 20182024.

Read the full report: https://www.reportlinker.com/p05827567/?utm_source=PRN

The major drivers contributing to the growth of the global cell and gene therapy market are the growing incidence of several chronic and terminal diseases, including cancer, the launch of new products, the increasing availability in clinical evidences of these products in terms of safety and efficacy, the rapid adoption of CAR T-cell therapy, favorable regulatory support in the development of these treatment, and improved manufacturing expertise in these products.

The following factors are likely to contribute to the growth of the cell and gene therapy market during the forecast period: Increased Pool of Patient Population with Several Ailments Favorable Regulatory Support and Increasing Special Designations for Cell and Gene Therapy Products Growing Demand for CAR T-cell Therapy Products Increasing Strategic Acquisition Activities

The study considers the present scenario of the cell and gene therapy market and its market dynamics for the period 2018?2024. It covers a detailed overview of several market growth enablers, restraints, and trends. The study covers both the demand and supply aspects of the market. The report profiles and examines leading companies and other prominent companies operating in the market.

Cell and Gene Therapy Market: Segmentation

This research report includes detailed market segmentation by product, application, end-user, and geography. The global cell therapy market is growing at a steady rate, and this trend is expected to continue during the forecast period due to the increased patient base with a wide range of diseases/ailments. The segment is likely to witness upward growth on account of expanded expertise in the manufacturing of stem cell-based products.

The gene therapy segment is expected to witness faster growth as the penetration of these products is increasing at a significant rate, especially in developed economies. The market is expected to grow during the forecast period due to the increased patient base for the existing gene remedy products, expected the launch of other gene therapy-based products for several indications, and expanded indication approvals for existing commercially available products.

The oncology segment accounts for the highest share of the global market. The growth of the oncology segment is increasing at a fast rate on account of the growing prevalence of several types of cancers. Currently, the available products not only modify the disease but also improve the quality of the patient's life, thereby decreasing the mortality rate. The market in the dermatology segment is increasing at a steady rate. This segment owns its growth to the increasing incidence and prevalence rate of several types of wounds, which are difficult to treat under normal conditions and the launch of innovative products. The dermatology segment is likely to showcase growth due to the high product availability of wound care products in the market.Hospitals are the leading end-user segment. The segment is growing mainly due to the increasing incidence/prevalence of chronic diseases such as cancer, cardiovascular diseases, diabetes, and chronic wound on account of diabetes feet, pressure ulcers, and other injuries.

Market Segmentation by Products Cell Therapy Gene TherapyMarket Segmentation by Distribution Channel Type Oncology Dermatology Musculoskeletal OthersMarket Segmentation by End-users Hospitals Wound Care Centers Cancer Care Centers Ambulatory Surgical Centers Others

Geographical Insights

The US market dominates the cell and gene therapy market in North America due to the high prevalence of chronic diseases and other conditions, which require these treatment methods. There is also comparably high utilization and wide accessibility of these therapies. The oncology segment is likely to witness significant growth in North America.The market in Europe is expected to witness upward growth in the near future on account of the growing prevalence of chronic diseases and rising elderly population. In Europe, cell and gene therapy products are considered to be part of the Advanced Therapy Medicinal Products (ATMPs), which are commonly known as regenerative medicine globally.

Market Segmentation by Geography North Americao USo Canada APACo Japano Chinao South Koreao Australia Europeo Germanyo Franceo UKo Spaino Italy Latin Americao Brazilo Mexico MEAo Turkeyo Saudi Arabiao UAE

Key Vendor AnalysisThe global market is characterized by the presence of a few global, large-scale companies and several small to medium-scale companies offering one or two cell and gene therapy products. Global players are majorly offering innovative products with the potential of disease-modifying characteristics that are generating significant revenues, especially in Europe and US regions. Most innovative and breakthrough products are approved in the European countries and the US. Vendors are targeting mostly developed economies such as the US, Germany, France, the UK, Spain, and Japan as the uptake of these products is higher in these countries than low and middle-income countries. However, the market in these regions is at the nascent stage.

Key Vendors Gilead Sciences Spark Therapeutics Novartis AG Organogenesis Amgen Osiris Therapeutics Dendreon Vericel

Other Prominent Vendors Anterogen Tego Sciences Japan Tissue Engineering JCR Pharmaceuticals Medipost MolMed AVITA Medical CollPlant Corestem Biosolution Stempeutics Research Orchard Therapeutics Takeda Pharmaceutical Company CHIESI Farmaceutici CO.DON AnGes GC Pharma JW CreaGene APAC Biotech Nipro Corp. Terumo Orthocell bluebird bio

Key Market InsightsThe report provides the following insights into the market for the forecast period 20192024. Offers sizing and growth prospects of the market for the forecast period 20192024. Provides comprehensive insights on the latest industry trends, forecast, and growth drivers in the market. Includes a detailed analysis of growth drivers, challenges, and investment opportunities. Delivers a complete overview of segments and the regional outlook of the market. Offers an exhaustive summary of the vendor landscape, competitive analysis, and key strategies to gain a competitive advantage.

Read the full report: https://www.reportlinker.com/p05827567/?utm_source=PRN

About Reportlinker ReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________ Contact Clare: clare@reportlinker.com US: (339)-368-6001 Intl: +1 339-368-6001

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The global cell and gene therapy market is growing at a CAGR of over 24% during the forecast period 2018-2024 - Yahoo Finance

The Food and Drug Administration has halted a clinical trial involving a Duchenne muscular dystrophy gene therapy from Solid Biosciences (SLDB) after a patient suffered serious kidney and blood-related injuries, the company said Tuesday.

This is the third time that the Cambridge, Mass.-based Solid has run into a serious safety problem with its gene therapy, called SGT-001. The FDA placed similar clinical holds on the same clinical trial after each prior incident, but later allowed the company to proceed with patient dosing.

SGT-001 uses an inactivated virus to deliver a miniaturized but functional version of the dystrophin gene to muscle cells. The gene therapy is designed to be a one-time and potentially curative treatment for all Duchenne patients, regardless of the mutation that causes their disease.

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Sarepta Therapeutics (SRPT) and Pfizer (PFE) are also developing their own gene therapies targeted at Duchenne.

Six patients have been dosed with SGT-001, starting with three at a lower dose; interim results in those patients were previously reported and found to be disappointing. Three more patients were then treated at a higher dose of SGT-001.

The sixth patient became ill soon after being treated in October, experiencing an over-activation of the immune system, an acute kidney injury, reductions in platelets and red blood cells, and cardio-pulmonary insufficiency, Solid said.

All of the toxicities were deemed related to SGT-001 by the patients treating doctor. The patient is being treated and is recovering, Solid said.

Solid reported the patients status to the FDA, which then placed the clinical trial on hold. In a statement, the company said it will work with the FDA in an effort to resolve the hold and determine next steps for the clinical trial.

Pfizers Duchenne gene therapy has also been tied to similar immune system over-activation and related kidney toxicity, although its clinical trial remains active.

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Solid's Duchenne gene therapy trial halted after patient suffers toxicity - STAT

18 November 2019

A gene therapytrial for Duchenne muscular dystrophy (DMD) has been halted after a patient experienced serious side effects.

The clinical trial, run by the US life science company, Solid Biosciences, was put on hold by the FDA (Food and Drug Administration)after the patient had an adverse reaction in response to the experimental gene therapy, called SGT-001. The symptoms included a decrease in red blood cell count, acute kidney injury, over-activation of the immune system and reduced heart and lung function.

'We are encouraged that this patient is recovering,' said Ilan Ganot, chief executive officer and co-founder of Solid Biosciences in Cambridge, Massachusetts. 'In the coming weeks, we anticipate that we will have a better understanding of the biological activity and potential benefit of SGT-001.'

DMD is a severemuscle-wasting disease that affects around one in every 3500 newborn boys. The disease is caused by a mutation in the gene responsible for making an essential muscle protein, called dystrophin. The resulting deficiency or absence of dystrophin leads to a progressive decline in muscle strength. There is currently no cure for the disease.

To date, six patients in the IGNITE DMD clinical trial, taking place at the University of Florida, have received a one-off intravenous infusion of SGT-001. The treatment is an adeno-associated virus (AAV)-based gene therapy, in which an inactivated virus is designed to deliver a synthetic, functional form of dystrophin, called microdystrophin, to muscles.

The first three patients, aged between fourand 17 years, were given the lowest dose outlined in the study protocol. A second cohort of three patients subsequently received a higher dose, which was believed to have led to the adverse reaction reported to the FDA.

The trial waspreviously halted in March 2018, after a patient receiving the low dose of the therapy experienced a similar reaction, from which he later recovered. The trial was resumed in June of the same year after the study design was amended.

Solid Biosciences have reported that all five other patients dosed in the trial are doing well and continue to be examined. They added that they will work with the FDA to determine the next steps for the trial, including how best to manage administration of the therapy.

'We remain committed to bringing meaningful new therapies to the Duchenne community and continue to believe in the differentiated construct of SGT-001 and the potential benefits it may offer to patients,' Ganot said.

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Duchenne gene therapy trial halted after serious reaction - BioNews

Dive Brief:

The companies identified Rett, Angelman and Dravet syndromes and Niemann-Pick disorder as the first four conditions that the new collaboration will try to treat. Four more could emerge, which would trigger an additional $42.5 million in additional payments.

Sarepta is paying the initial $48 million upfront fee in the form of combined cash and shares. StrideBio will be eligible for additional undisclosed development, regulatory and sales milestones, as well as royalties. The privately-held partner also will have an option to co-commercialization rights to one of the gene therapies, if successful.

The four StrideBio agents will join a Sarepta pipeline that already has 23 identified projects in clinical or pre-clinical development.

Cambridge, Massaschusetts-based Sarepta stated the collaboration will utilize StrideBio's "unique approach" to engineering capsids, the shells surrounding the adeno-associated virus (AAV) used by many researchers to deliver genes to cells.

StrideBio's technology tries to better target which cells their AAV-based therapies reach, as well as avoid triggering neutralizing antibodies, which can reduce the effectiveness of a gene therapy.

Immune responses to some AAV-based therapies have raised safety concerns. On Tuesday, Solid Biosciences announced the Food and Drug Administration had put a hold on its AAV9-based gene therapy for Duchenne muscular dystrophy (DMD) because of immune responses, although Sarepta's own DMD gene therapy has not seen anything similar.

As part of the agreement, Sarepta and StrideBio "plan to focus on strategies intended to address re-dosing challenges in patients who have received AAV-delivered gene therapy."

Re-treatment of patients who don't respond or have unlimited response to gene therapies is an unanswered question in this quickly evolving field, and drug developers and payers alike will closely watch any developments that emerge from the Sarepta-StrideBio re-dosing work.

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Sarepta brings in more gene therapies with StrideBio deal - BioPharma Dive

Tokyo-based FUJIFILM will invest 13 billion yen (about $120 million) to expand the companys gene therapy business and establish anew Gene Therapy Innovation Center adjacent to its current facility in College Station, Texas and add about 100 jobs.

The investment will include the addition of dedicated gene therapy laboratories and will be part of the existing FUJIFILM Diosynth Biotechnologies (FDB) in Texas, which opened last year. That site has been the companys center for excellence in gene therapy since 2014. The company is expanding its contract and development services for gene therapies as the market for CDMOs in gene therapy is expected to increase to $1.7 billion by 2025, the company said in its announcement.

The Gene Therapy Innovation Center, supported by a $55 million investment, will be approximately 60,000 square feet and will house state-of-the-art upstream, downstream and analytical development technologies. The facility is expected to be operational in the fall of 2021. Gene Therapy remains a strategic investment area for FUJIFILM.

Gerry Farrell, COO at FUJIFILM Diosynth Biotechnologies in Texas, said they anticipate breaking ground on the new facility in the first quarter of 2020. The new Texas sit will triple the companys gene therapy development capabilities and will add approximately 100 jobs to its Texas campus, Farrell said.

Martin Meeson, president and chief operating officer of the U.S. division of FUJIFILM Diosynth Biotechnologies, said the investment will allow FUJIFILM to support the incredible growth that the gene therapy sector has experienced over the past few years.

We know that we need to invest now, in technology, assets and people in order to achieve a market leadership position. The expansion through the construction of the Gene Therapy Innovation Center demonstrates our ongoing commitment for growth, Meeson said in a statement.

FUJIFILMs main goal behind its new strategy is to position itself as a key provider of leading, future-proofed end-to-end gene therapy solutions, from pre-clinical to commercial launch. For the company, this investment builds on earlier plans to introduce its gene therapy fill finish services.

For FUJIFILM, this investment in Texas comes several months after it snapped up Biogens biologics manufacturing operations in Denmark. FUJIFILM paid the Cambridge, Mass.-based company $890 million for the site that had about 800 employees. The acquisition is part of the companys expanded manufacturing strategy. Last year, the company acquired two biotechnology units from JXTG Holdings Inc. for about $800 million.

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FUJIFILM Expands Texas Holdings with New Gene Therapy Center - BioSpace

GERMANTOWN, Md., Nov. 18, 2019 /PRNewswire/ --Triple-Gene LLC, a clinical stage cardiovascular gene therapy company and majority owned subsidiary of Intrexon Corporation (NASDAQ: XON), yesterday presented preliminary data from the Phase I trial (clinical trial identifier: NCT03409627) of INXN-4001, a multigenic investigational therapeutic candidate under evaluation for the treatment of heart failure, in a poster at the American Heart Association (AHA) Annual Meeting.1 On November 7, 2019, Triple-Gene announcedthat enrollment in this Phase I study has been completed.

"We are pleased to have had the opportunity to share our early clinical data from this first-in-human study with the cardiology community at this prestigious meeting," stated Thomas D. Reed, PhD, Co-founder and Managing Director of Triple-Gene. "The data presented yesterday suggest that the combination of our transiently expressed, non-integrating naked plasmid DNA with the focused cardiac delivery enabled by Retrograde Coronary Sinus Infusion (RCSI) has the potential to open a new biologics treatment paradigm for treating cardiovascular diseases."

Triple-Gene's investigational therapy uses non-viral delivery of a constitutively controlled multigenic plasmid designed to express human S100A1, SDF-1, and VEGF165 gene products, which affect progenitor cell recruitment, angiogenesis, and calcium handling, respectively, and target the underlying molecular mechanisms of pathological myocardial remodeling. The plasmid therapy is delivered via RCSI, which allows for cardiac-specific delivery to the ventricle.

Dr. David Bull, who was the first investigator to dose a patient with INXN-4001, stated, "Having carefully reviewed the science behind Triple-Gene's product candidate, I was very excited to initiate the INXN4001 clinical trial at the University of Arizona. Heart failure is a devastating disease, and my patients on Left Ventricular Assist Devices (LVAD) have very few therapeutic treatment options. Triple-Gene's novel triple-effector plasmid, as delivered by the minimally invasive RCSI procedure, represents a potential game-changing approach for addressing disease pathology in this high-risk patient population."

Amit N. Patel, MD, MS, Co-founder and Clinical Director of Triple-Gene added, "We are pleased to have completed dosing of the twelve patients in this clinical trial between our two clinical sites at the University of Arizona, Tucson, AZ and The Christ Hospital, Cincinnati, OH, and look forward to sharing additional data for this investigational therapy once the trial is complete. Based upon the promising early results to date, we are now exploring clinical trial designs that contemplate repeat dosing as well as additional orphan-like heart failure subtypes."

1Jaruga-Killeen E, Bull DA, Lotun K, Henry T, Egnaczyk G, Reed TD and Patel AN. Safety of first-in-human triple gene therapy for heart failure patients. Presented at the American Heart Association Annual Meeting, November 17, 2019.

About Triple-GeneTriple-Gene LLC is a clinical stage gene therapy company focused on advancing targeted, controllable, and multigenic gene therapies for the treatment of complex cardiovascular diseases. The Company's lead product is a non-viral investigational gene therapy candidate that drives expression of three candidate effector genes involved in heart failure. Triple-Gene is a majority owned subsidiary of Intrexon Corporation(NASDAQ: XON) co-founded by Amit Patel, MD, MS, and Thomas D. Reed, PhD, Founder and Chief Science Officer of Intrexon. Learn more about Triple-Gene at http://www.3GTx.com.

About Intrexon CorporationIntrexon Corporation (NASDAQ: XON) is Powering the Bioindustrial Revolution with Better DNAto create biologically-based products that improve the quality of life and the health of the planet through two operating units Intrexon Health and Intrexon Bioengineering. Intrexon Health is focused on addressing unmet medical needs through a diverse spectrum of therapeutic modalities, including gene and cell therapies, microbial bioproduction, and regenerative medicine. Intrexon Bioengineering seeks to address global challenges across food, agriculture, environmental, energy, and industrial fields by advancing biologically engineered solutions to improve sustainability and efficiency. Our integrated technology suite provides industrial-scale design and development of complex biological systems delivering unprecedented control, quality, function, and performance of living cells. We call our synthetic biology approach Better DNA, and we invite you to discover more at http://www.dna.comor follow us on Twitter at @Intrexon, on Facebook, and LinkedIn.

TrademarksIntrexon, Powering the Bioindustrial Revolution with Better DNA,and Better DNA are trademarks of Intrexon and/or its affiliates. Other names may be trademarks of their respective owners.

Safe Harbor Statement Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.

For more information contact:

Investor Contact:

Steven Harasym

Vice President, Investor Relations

Intrexon Corporation

Tel: +1 (301) 556-9850

investors@dna.com

Corporate Contact:

Marie Rossi, PhD

Vice President, Communications

Intrexon Corporation

Tel: +1 (301) 556-9850

publicrelations@dna.com

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Triple-Gene Presented Preliminary Phase 1 Trial Data of Investigational Multigenic Therapeutic Candidate INXN-4001 for Treatment of Heart Failure at...

NEW YORK and BASEL, Switzerland, Nov. 18, 2019 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (NASDAQ: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the Companys investigational gene therapy, AXO-AAV-GM1, for the treatment of GM1 gangliosidosis. GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene leading to impaired production of the beta-galactosidase enzyme. The Company is planning to complete enrollment of patients in Part A of the AXO-AAV-GM1 registrational program evaluating safety and efficacy and expects to announce 6-month data from Part A in mid-2020.

We are pleased to receive orphan drug designation for our GM1 gangliosidosis program as we advance the development of this potentially transformative, one-time gene therapy, said Dr. Gavin Corcoran, chief R&D officer at Axovant. There are currently no FDA approved therapies for patients with GM1 gangliosidosis, and the orphan drug designation underscores the significant unmet medical need. We look forward to increased interaction with the FDA to facilitate the development of AXO-AAV-GM1 and address this unmet need.

FDA orphan drug designation is granted to investigational therapies which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation provides several benefits to drug developers, which in addition to others, include increased FDA interaction, eligibility for 7-year market exclusivity, potential for tax credits towards the cost of clinical trials, and prescription drug user fee waivers at the time of filing a biologics license application (BLA). For more information about orphan drug designation, please visit the FDA website at http://www.fda.gov.

About AXO-AAV-GM1

AXO-AAV-GM1 is an investigational gene therapy that delivers a functional copy of theGLB1gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

About Axovant Gene Therapies

Axovant Gene Therapies, part of the Roivant family of companies, is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visitwww.axovant.com.

In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

About Roivant

Roivantaims to improve health by rapidly delivering innovative medicines and technologies to patients.Roivantdoes this by buildingVants nimble, entrepreneurial biotech and healthcare companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. Roivant today is comprised of a central technology-enabled platform and 20 Vants with over 45 investigational medicines in clinical and preclinical development and multiple healthcare technologies. For more information, please visitwww.roivant.com.

Forward Looking Statements and Information

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, might, will, would, should, expect, believe, estimate, and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding the initiation, timing, progress, and reporting of results of its preclinical programs, clinical trials, and research and development programs; cash to be used in operating activities; its ability to advance its gene therapy product candidates into and successfully initiate, enroll, and complete clinical trials; the potential clinical utility of its product candidates; its ability to continue to develop its gene therapy platforms; its ability to develop and manufacture its products and successfully transition manufacturing processes; its ability to perform under existing collaborations with, among others, Oxford BioMedica and theUniversity of Massachusetts Medical School, and to add new programs to its pipeline; its ability to enter into new partnerships or collaborations; its ability to retain and successfully integrate its leadership and personnel; and the timing or likelihood of its regulatory filings and approvals are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovants management that, although Axovant believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Axovant expected.Such risks and uncertainties include, among others, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the expectations for regulatory submissions and approvals; the continued development of its small molecule and gene therapy product candidates and platforms; Axovants scientific approach and general development progress; and the availability or commercial potential of Axovants product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovants most recent Quarterly Report on Form 10-Q filed with theSecurities and Exchange CommissiononNovember 8, 2019, as updated by its subsequent filings with theSecurities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made.Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media and Investors

Parag MeswaniAxovant Gene Therapies(212) 547-2523investors@axovant.commedia@axovant.com

Originally posted here:
Axovant Gene Therapies Receives Orphan Drug Designation from FDA for AXO-AAV-GM1 for the Treatment of GM1 Gangliosidosis - BioSpace

CHICAGO, Nov. 13, 2019 /PRNewswire/ -- According to Arizton's recent research report, Cell and Gene Therapy Market - Global Outlook and Forecast 2019-2024 is expected to grow at a CAGR of more than 24% during the forecast period.

Key Highlights Offered in the Report:

Key Offerings:

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Cell and Gene Therapy Market Segmentation

Market Segmentation by Products

Market Segmentation by Distribution Channel Type

Market Segmentation by End-users

Cell and Gene Therapy Market Dynamics

CAR T-cell therapy has gained significant traction in recent years. It is the single most rapidly growing type of product in the market that generates revenue at a phenomenal rate. At present, it is the fastest advancing technology in cancer treatment and has the capability to replace many existing therapies. CAR T-cell therapy addresses current challenges in cancer care through superior efficacy, safety, and delivery mechanisms. CAR T-cell therapy has brought itself into focus due to the personalized nature of this therapy and the utilization of advanced genetic engineering technology. The wide acceptance and use of CAR T-cell therapy is fueling the growth of the global cell and gene therapy market.

Key Drivers and Trends fueling Market Growth:

Cell and Gene Therapy MarketGeography

The US dominates the cell and gene therapy market in North America due to the high prevalence of chronic diseases and other conditions. There is also comparably high utilization and wide accessibility of these therapies. In Europe, cell and gene therapy products are considered to be part of the Advanced Therapy Medicinal Products (ATMPs), which are commonly known as regenerative medicine globally. The major factors leading to the growth in APAC region are the growing prevalence of cancers, osteoarthritis, burns, and other chronic wounds, the introduction of advanced products in Japan, advanced R&D activities in countries such as South Korea, India.

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Market Segmentation by Geography

Major Vendors

Other vendors include - Anterogen, Tego Sciences, Japan Tissue Engineering, JCR Pharmaceuticals, Medipost, MolMed, AVITA Medical, CollPlant, Corestem, Biosolution, Stempeutics Research, Orchard Therapeutics, Takeda Pharmaceutical Company, CHIESI Farmaceutici, CO.DON, AnGes, GC Pharma, JW CreaGene, APAC Biotech, Nipro Corp., Terumo, Orthocell, and bluebird bio.

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About Arizton:

Arizton Advisory and Intelligence is an innovation and quality-driven firm, which offers cutting-edge research solutions to clients across the world. We excel in providing comprehensive market intelligence reports and advisory and consulting services.

We offer comprehensive market research reports on industries such as consumer goods & retail technology, automotive and mobility, smart tech, healthcare, and life sciences, industrial machinery, chemicals and materials, IT and media, logistics and packaging. These reports contain detailed industry analysis, market size, share, growth drivers, and trend forecasts.

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The Cell and Gene Therapy Market to Reach Revenues of Over $6.6 billion by 2024 - Market Research by Arizton - PRNewswire