Category Archives: Gene therapy

A gene therapy approach using a so-called antibody-drug conjugate (ADC) conditioning regimen led to safe and sustained production of factor VIII (FVIII) in platelets, and prevented joint bleeding in a mouse model of hemophilia A, according to new research.

The study, Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice, appeared in the journal Blood Advances.

Prior work in mice showed that stem cell-based gene therapy specifically targeting platelets leads to the production of FVIII the clotting protein missing or defective in people with hemophilia A and induces immune tolerance (no development of exacerbated immune response).

However, the procedure required a conditioning regimen with chemotherapy or total body irradiation (TBI) that may be toxic to genetic material (or genotoxic), so patients may not be agreeable to using the protocol.

Safer approaches may come from ADCs, which use antibodies against cell surface proteins for more specific targeting of cell populations.

A team of researchers from the Blood Research Institute, in Milwaukee, tested ADC-based conditioning with stem cell-based F8 gene therapy that targets platelets in a mouse model of hemophilia A. This ADC consists of saporin a plant-derived toxin that halts protein production bound to antibodies specific for the CD45.2 and CD117 cell surface proteins.

The scientists found that, three weeks after hematopoietic stem cell transplant (HSCT), peripheral blood counts were higher with ADC conditioning than with TBI. This suggested a lower risk of cytopenia (reduced number of mature blood cells), the investigators said.

Then they observed that the new conditioning regimen led to effective engraftment, the process through which transplanted stem cells establish themselves in the bone marrow and start producing new blood cells. At 20 weeks after transplant, all mice receiving the ADC regimen had more than 15% of donor-derived white blood cells.

Sixteen weeks after HSCT, the number of copies of therapeutic genes was similar in the mice receiving ADC and the controls on TBI. Two-thirds of the mice receiving the viral-delivered gene therapy under ADC conditioning showed sustained increases in FVIII levels in platelets.

The ADC regimen also led to increasing reconstitution of platelets and white blood cells, meaning a higher percentage of cells derived from donors.

Subsequent experiments showed that ADC also targeting the CD8 protein led to significantly higher white blood cell reconstitution, frequency of regulatory T-cells (which dampen excessive immune responses), and FVIII levels in platelets than the regimen specific for CD117 and CD45.2 only.

These effects were sustained long-term, as found when transplanting bone marrow cells from animals that had undergone HSCT to new mice under the same conditioning regimen.

Blood clotting was normalized with this gene therapy approach, and hemoglobin levels were higher than in mice with no FVIII. Joint bleeding and limping were effectively prevented in a knee joint injury model. Also, no animal with sustained FVIII expression in platelets developed anti-FVIII inhibitors.

The central finding of this report is that platelet-directed HSC-based FVIII gene therapy is safe and effective for eliminating [hemophilia A] using nongenotoxic hematopoietic-targeted ADC conditioning, the researchers wrote.

This safe and effective treatment strategy could be especially meaningful for [hemophilia A] patients who are especially wary of standard preconditioning, they added.

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

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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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Hemophilia A Study Finds Benefits With New Gene Therapy Approach - Hemophilia News Today

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, today announced senior appointments to its leadership team. Sadik Kassim, PhD, a cell and gene therapy bioprocessing and translational research expert, joins Vor from Kite Pharma as Chief Technology Officer. Tirtha Chakraborty, PhD, a hematological and gene engineering research specialist with experience at Sana Biotechnology and CRISPR Therapeutics, joins as Vice President of Research. These new positions follow Vors recent move into an integrated headquarters in Cambridge, Mass., the appointment of Robert Ang, MBBS, MBA, as President and Chief Executive Officer and a $42 million Series A financing directed at developing Vors platform technology and advancing its pipeline of eHSC-based candidates.

Vor is bringing a fundamentally novel approach to hematopoietic stem cells to empower targeted cancer therapies, and we are rapidly building an industry-leading team to realize the value in this scientific foundation, said Dr. Ang. Dr. Kassim brings his substantial experience with the complex methods and processes that are required for manufacturing genetically-manipulated cell therapies, and Dr. Chakraborty provides deep expertise in hematology and genetic engineering. Their complementary knowledge will aid Vors expansion, platform development and the move towards our first Investigational New Drug filing for VOR33.

I am impressed that compelling in vivo data already supports the potential of Vors cellular engineering platform to protect healthy cells from antigen-directed therapies via antigen removal, said Dr. Kassim. This is especially noteworthy when therapeutic effectiveness is so often highly limited by co-location of target antigens on healthy immune cells, creating a huge opportunity for Vor to significantly broaden the applicability of these and future therapies.

Its exciting to join the Vor team during this period of accelerated expansion, said Dr. Chakraborty. As a geneticist and cell biologist, I look forward to developing this new approach to treat a range of devastating cancers, beginning with VOR33 in acute myeloid leukemia.

Dr. Kassim is a former Executive Director at Kite Pharma where he led the development of manufacturing processes for autologous CAR- and TCR-based gene-modified cell therapies. Prior to Kite, he served as Chief Scientific Officer at Mustang Bio, where he was the first employee and oversaw the foundational build-out of the companys preclinical and manufacturing activities. Prior to Mustang, Dr. Kassim was Head of Early Analytical Development for Novartis Cell and Gene Therapies Unit, where he contributed to the BLA and MAA filings for Kymriah. Earlier in his career, Dr. Kassim was a research biologist at the National Cancer Institute, where he was involved in early research and CMC work that led to the development of several first-in-human TCR and CAR-T products, including Kites Yescarta. Dr. Kassim has also conducted preclinical immunology research at Janssen and was a research fellow in the University of Pennsylvania Gene Therapy Program, where he led the initial discovery and preclinical studies for an AAV8 gene therapy for familial hypercholesterolemia, a program that is now in the clinic. Dr. Kassim earned his BS in Cell and Molecular Biology from Tulane University and received his PhD in Microbiology and Immunology from Louisiana State University.

Dr. Chakraborty joins Vor from Sana Biotechnology, where he served as the Vice President of Cell Therapy Research. Prior to Sana, Dr. Chakraborty was the Head of Hematology at CRISPR Therapeutics, where his teams work on hemoglobin disorders paved the way for the first clinical trial for the CRISPR industry. Before that, at Moderna Therapeutics, Dr. Chakraborty led synthetic mRNA platform technology research. He was trained as an RNA biologist and an immunologist during his postdoctoral research at Harvard Medical School. Dr. Chakraborty received his PhD from the Tata Institute of Fundamental Research in Mumbai, India.

About VOR33Vors lead engineered hematopoietic stem cell (eHSC) product candidate, VOR33, is in development for acute myeloid leukemia (AML). VOR33 is designed to produce healthy cells that lack the receptor CD33, thus enabling the targeting of AML cells through the CD33 antigen, while avoiding toxicity to the bone marrow. Currently, targeted therapies for AML and other liquid tumors can be limited by on-target toxicity. By rendering healthy cells invisible to CD33-targeted therapies, VOR33 aims to significantly improve the therapeutic window, utility and effectiveness of these AML therapies, with the potential to broaden clinical benefit to different patient populations.

About Vor BiopharmaVor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. Vors eHSCs are designed to generate healthy, fully functional cells with specific advantageous modifications, protecting healthy cells from the toxic effects of antigen-targeted therapies, while leaving tumor cells vulnerable.

Vors platform could potentially be used to change the treatment paradigm of both hematopoietic stem cell transplants and antigen-targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. A proof-of-concept study for Vors lead program has been published in Proceedings of the National Academy of Sciences.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil. Vor was founded by Dr. Mukherjee and PureTech Health and is supported by leading investors including 5AM Ventures and RA Capital Management, Johnson & Johnson Innovation JJDC, Inc. (JJDC), Novartis Institutes for BioMedical Research and Osage University Partners.

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Vor Biopharma Hires Senior Cell and Gene Therapy Leaders as Chief Technology Officer and Vice President of Research - Business Wire

This week, were looking at the enormous medical potential now being realised by gene therapy. Well be hearing how these approaches can battle blindness, halt Duchenne Muscular Dystrophy, and even cure HIV... but first, what do we actually mean by gene therapy? Phil Sansom is here with the quick-fire science...

Phil - Most diseases that you can get have their roots in your DNA, but ever since medicine began, treating those diseases hasn't actually involve tackling those root problems. That is until 40 years ago when doctors successfully inserted new DNA into five people to help treat their melanoma. Welcome to a new type of medicine; gene therapy. So how does it work? Well a gene is a bit of DNA, and there are different ways a gene can malfunction to give you a disease. So different types of gene therapy might do different things.

One treatment might replace a harmful mutated gene with the non-mutated healthy version, or if the mutated gene isn't vital the treatment might want to stop it from working altogether. That's called knocking it out. In other cases your body might be totally missing a gene that you need. Gene therapy in this case could just add it back in. Here's the problem though; to efficiently add bits of DNA into your cells, you usually need some kind of vector.

Certain viruses are really good vectors because that's how they infect you anyway, by inserting their genes into your cells to hijack them and make more of themselves. Gene therapy can exploit that by modifying the virus, stripping out the viral genes, and replacing them with useful gene therapy ones instead. But putting foreign material like viruses into your body can be risky. It can trigger your immune system to attack, potentially leading to lethal inflammation. Plus there are other risks too. With some therapies, there's a chance that messing with your body's genetic code could cause cancer. That's why right now, doctors are moving cautiously and they usually prioritise diseases with a bad prognosis and no other cures. But for those diseases it can be a lifeline.

And as it gets safer gene therapy looks to become a pretty powerful tool.

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What is gene therapy? | Interviews - The Naked Scientists

Tenaya CEO Faraz Ali(Tenaya Therapeutics)

Although heart disease remains the leading cause of death worldwide, its an area that hasnt seen as much interest or investment as other areas, and its treatments still focus on dealing with symptoms. With a multipronged approach and a fresh infusion of $92 million, Tenaya Therapeutics is trying to change that.

The heart is a complicated organ, and it can go wrong in different ways. Part of what weve learned the hard way is that prior approaches are not working, Tenaya CEO Faraz Ali told FierceBiotech.

Founded in 2016 by scientists from the Gladstone Institute in California and the University of Texas Southwestern Medical Center, Tenaya is going after the underlying causes of heart disease to head off heart failure. It raised $50 million in its series A round from The Column Group, which also pitched into its $92 million B round alongside the likes of Casdin Capital and GV.

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RELATED: GV leads $58.5M round for Verve, a startup looking to pit gene editing against heart attacks

In an age where companies are forming themselves around one platform thats disease-agnostic and can be applied to many different areas, South San Francisco-based Tenaya is doing the opposite. It's working on three different platforms for heart disease:regenerative treatments, gene therapies and precision medicines.

The company was founded with a big, bold mission to follow the science and use the right tool for the job, Ali said. If the problem is loss of cardiomyocytes (the muscle cells that make the heart beat), such as after a heart attack, we can look for a way to generate new myocytes, create new tissue and improve the ability of the heart to contract that way.

Thats where regenerative treatments come in. Tenaya's approach delivers transcription factors that can nudge heart fibroblasts, cells that play a role in scar formation after a heart attack, to become heart muscle.

If the problem stems from geneticsif a faulty gene doesnt cause heart muscle to die, but leads to an arrhythmia or scarring, stopping it from working properlythe solution is not to create new muscle, but to get the muscle thats there to work, Ali said. And thats where gene therapy, adding a healthy copy of a defective gene, comes in.

Finally, Tenayas taking a leaf out of the book of cancer drug developers: Its become quite the norm to look for therapies that work in a particular genetic background, he said. The companys precision medicine platform uses stem cell-derived heart muscle cells as disease models to identify new targets for heart failure and screen new drugs. Its first focus is on small molecules for the treatment of dilated cardiomyopathiesa group of conditions in which an enlarged heart chamber makes it less efficient at pumpingin genetically defined patient groups.

What the funding allows us to do is advance multiple promising projects from each of the platforms out of the pure research stage and into the clinic, Ali said. Tenaya hopes to move to human studies over the next few years.

RELATED: Renovacor bags $11M to push precision medicine for rare heart disease

Though the company isnt divulging just yet which targets its going after, Ali did say some of its programs are chasing more prevalent conditions such as heart attacks while others are looking at smaller, more defined populations.

Once we get a signal of efficacy and safety and advance into later-stage clinical development, we could potentially expand into larger, more prevalent indications, he said.

Tenaya has about 45 staffers who are mostly focused on research, early development and manufacturing. If all goes to plan, the company plans to double its size by the end of 2021, adding more employees in development and manufacturing.

Two-thirds of our platforms that were working on [gene therapy and regenerative treatments] are heavily dependent on viral vectors, adeno-associated viruses Everyone learned in the last decade or so that manufacturing is the Achilles heel of the gene therapy spaceit's difficult to do and highly technical, its nothing like small molecule manufacturing, Ali said. We made the decision to invest early, and well ahead of being in the clinic, to invest in manufacturing.

Tenaya isnt just doing it because other gene therapy players have proved it necessary. If it wants to go after larger heart disease populations rather than the smaller groups affected by rare disease, its going to need a lot of viral vector to deliver its treatments.

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Tenaya Therapeutics bags $92M to develop triple threat for heart disease - FierceBiotech

By Erin Harris, Editor-In-Chief, Cell & Gene Follow Me On Twitter @ErinHarris_1

Move over, religion and politics. Drug prices are one of the new polarizing topics on the minds of anyone paying attention.

Headlines about high-priced cell and gene therapies specifically can cause sticker shock for the patient. Exhibit A: Novartis Zolgensma, the spinal muscular atrophy (SMA) gene therapy treatment, priced at $2.125 million, is the worlds most expensive drug to date. But cell and gene therapies are their own animals; the differences between them and traditional biologics are too numerous to count. Their innovative and life-saving properties are unmatched and, make no mistake, these therapies are changing modern medicine. As a result, the often one-time, personalized therapies are expensive and resource-intense propositions. Attend any industry conference that broaches the pricing subject, and youre likely to hear the following: Competition will eventually drive down costs, or its simply too early to tell whats next. Both statements are true, but theyre not very actionable. Therefore, I turned to Eric David, CEO of Aspa Therapeutics; Paul Lammers, M.D., MSc, president, CEO, and director of Triumvira Immunologics; and Janet Lambert, president of the Alliance for Regenerative Medicine (ARM), for their take on current pricing models for cell and gene therapies and potential solutions to drive down costs for the patient.

THE STATE OF CURRENT PRICING MODELS

Prices for cell and gene products represent the value that the therapies bring to patients and to healthcare systems over time, and they help to drive further innovation across the sector. The therapies have the potential to provide a durable or possibly even a curative effect, often with a single administration, and represent a significant improvement in standard of care for many patients, says Lambert. She goes on to say that this method of treating disease by addressing its underlying cause has the potential to provide cost savings to the healthcare system through decreased direct medical costs as well as through improved patient quality of life, increased productivity, improved caregiver quality of life, improved social integration, and other indirect cost savings.

These therapies represent a substantial change in the healthcare reimbursement paradigm, as many treatments for serious conditions currently involve chronic palliative care, providing incremental improvements and/or temporary delays in the progression of disease. Current reimbursement systems are configured toward providing this type of chronic care, and may be unable to cope with the high up-front costs of cell and gene therapies, says Lambert. Coupled with the newness of these technologies and the lack of long-term follow-up data, these therapies can have an undesirably high potential risk profile for payors. Therefore, ensuring patient access to these therapies relies on the development and implementation of payment models that help payers to absorb the costs of these therapies as well as offset the perceived risks.

WHY COSTS WILL COME DOWN EVENTUALLY

At this years BIO conference in Philadelphia, I attended a session titled Gene Therapy 2.0: No Longer Science Fiction. I noticed that when the topic of pricing came up, some panelists at this session became noticeably quiet except for Eric David. In fact, his passion for the topic was so palpable, I decided to contact him after the event to pick his brain.

The challenges to the industry regarding gene therapy pricing are twofold, explains David. First and foremost, the cost to manufacture a gene therapy is significantly more than conventional biologics such as monoclonal antibodies and recombinant proteins. Cost of goods/manufacturing alone for a gene therapy can be between $500,000 and $1 million, and that does not include costs for R&D, the costs to run crucial clinical trials, or the costs to build the commercial infrastructure necessary to provide access to patients. In addition, for the foreseeable future, these therapies will be administered as one-time-only, and they will be administered to very small patient populations sometimes just a few hundred patients worldwide. Companies must be able to recoup their significant investments, or they will not be able to tackle these highly unmet needs.

David went on to explain that current pricing models spread out the payments for a gene therapy over several years, assuming efficacy remains durable. This ensures that payors do not have to bear the entire cost of a gene therapy up front. Over time, costs will come down significantly, as they did for monoclonal antibodies, driven by increased manufacturing capacity, greater commoditization of bioengineering and manufacturing resources, and efficacy-based pricing models, says David.

REIMBURSEMENT MODELS: PAYMENT-OVER-TIME AND PAY-FOR-PERFORMANCE

According to Lambert, two reimbursement models that are currently garnering attention and traction from industry and payors alike are payment-over-time and pay-for-performance. Payment-over-time models, which allow insurers to amortize the cost of therapies over several years, better reflect the value provided by cell and gene therapies, she says. Pay-for-performance models can be combined with amortization models, benchmarking future payments on positive health outcomes for patients, or they can be used stand-alone, providing rebates in cases in which the therapy was not as efficacious as expected. In either case, these models help to shift or share the risk from the payor to the developer, which may make payors more confident.

Other innovative models are already in place to help mitigate the costs of existing expensive therapies. For instance, re-insurance, a process by which risk is shared by multiple insurance companies, has been adopted by insurers in the U.S. to help pay for expensive solid organ and stem cell (bone marrow) transplantation procedures. Risk pools have been used successfully by private insurers in Canada and are the basis for the UKs government-sponsored Cancer Drugs Fund, Lambert explains. The so-called Netflix Model, which has been adopted by two state Medicaid programs [i.e., Louisiana and Washington] to pay for high-cost treatments for Hepatitis C can help normalize costs when demand for a therapy fluctuates significantly from year to year.

A STRATEGY TO BRING DOWN COSTS: MANUFACTURING IMPROVEMENTS

Dr. Lammers, of Triumvira Immunologics, says one strategy that could reduce the costs of these therapies involves fully automating T-cell therapy production. There is a strong movement in the T-cell manufacturing world to move toward the use of fully automated systems, (i.e., you put the patients leukapharesis material in, and 10-14 days later, the finished CAR-T or TAC-T cells are ready to be re-infused into the patient), says Dr. Lammers. Then, you insert a new cartridge, and the system is ready to process the next patients materials. These systems are small about the size of a small microwave and can be stacked, so the actual footprint needed would be far smaller than the current process used. Dr. Lammers and David note that increased manufacturing capacity also will drive down costs.

Eric DavidCEO, Aspa Therapeutics

INVOLVING THE PATIENT IN THE PROCESS

We all know that, from a patients perspective, it can be hard to swallow the published price of a therapy, But payors understand the why behind high costs of gene therapy as well as how few patients will be treated, and most patients and their families will never have to pay the whole price of a therapy, says David. In addition, the tremendous innovation occurring across scientific, regulatory, and commercial models is enabling investment in these areas of profound unmet medical need. Without this innovation, which is stimulated in part by the markets acceptance of the current pricing models, there would remain absolutely no investment in these diseases, as had been the case for decades previously.

Simply put, cell and gene therapies are expensive to develop, manufacture, and commercialize, and they will continue to be for the foreseeable future. As industry and payors continue to pursue innovative financing models, it is important for policymakers to identify and reduce the legal and regulatory barriers to the adoption of these programs, particularly for public payors.

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Potential Solutions To Current Pricing Models For Cell And Gene Therapies - Life Science Leader Magazine

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Verve Therapeutics, a next-generation cardiovascular company developing therapies that safely edit the adult human genome to permanently reduce a persons risk of coronary artery disease, today announced that Sekar Kathiresan, M.D., chief executive officer, will be speaking at the 2019 Jefferies Gene Therapy/Editing Summit.

Dr. Kathiresan will give a corporate overview presentation on Tuesday, October 8, 2019, at 12:00 p.m. ET at the Palace Hotel in New York City. He will also participate in a panel discussion on gene editing safety and clinical/regulatory insights, to be held on Tuesday, October 8, 2019, at 1:45 p.m. ET. Verve will also be holding one-on-one meetings at the conference.

About Verve TherapeuticsVerve Therapeutics is a biotechnology company created with a singular focus: to protect the world from heart disease. Verve is developing therapies to safely edit the genome of adults and confer lifelong protection from coronary artery disease, the most common form of heart disease and the leading cause of death worldwide. Founded by world-leading experts in cardiovascular medicine, human genetics and gene editing, Verve is backed by a top-tier syndicate of investors, including GV (formerly Google Ventures), ARCH Venture Partners, F-Prime Capital, and Biomatics Capital. Verve is headquartered in Cambridge, Massachusetts. For more information, visit http://www.VerveTx.com.

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Verve Therapeutics to Present at the 2019 Jefferies Gene Therapy/Editing Summit - Business Wire

By Rob Wright, Chief Editor, Life Science LeaderFollow Me On Twitter @RfwrightLSL

Matt Patterson

THAT WAS A TERRIFYING MOMENT,reflects Matt Patterson, cofounder, chairman, and CEO of Audentes Therapeutics, a genetic medicines company focused on the adeno-associated virus (AAV). It was the fall of 2012, and Patterson was preparing a final pitch to OrbiMed Advisors to lead a $25 million to $30 million Series A funding round for Audentes, which was then just an idea for a company. But pitching OrbiMed wasnt what was terrifying. After all, at the time Patterson was working as an entrepreneur in residence (EIR) for OrbiMed, so he knew the people in the room. However, this was his last pitch after his first few hadnt garnered much interest. You have to remember, in 2012 there werent a lot of gene therapy companies other than bluebird bio, he explains.

In his role as an EIR, Patterson had connected with a researcher at Wake Forest University working on X-linked myotubular myopathy (XLMTM), a severe neuromuscular disease with no available treatment. It took me about 72 hours to decide I wanted to start a company to develop something for these patients and their families, and I quickly came up with an initial vision for how to do a clinical trial, what endpoints to study, and what might be compelling to regulatory authorities, he notes.

But when he made that first pitch to OrbiMed, Audentes was considered extremely high risk, since in addition to being gene therapy, it was based on early-stage preclinical work and a single asset idea. To decrease that risk and make the company more compelling for his final pitch, Patterson added programs for Pompe disease and Duchenne muscular dystrophy through additional academic connections.

As an EIR, you are obligated to provide your employer [i.e., OrbiMed] the first opportunity to invest in any idea you have, though legally youre a consultant and can leave to do your own thing, he clarifies. And although he really wanted OrbiMeds support, he already had determined and communicated that if they werent interested by this point, hed seek funding elsewhere. This was the nerve-wracking part; not having OrbiMed would make raising funds much more difficult. How do you explain to other VCs why the firm you worked for didnt want to be part of the build? he asks.

Ultimately, OrbiMed provided Audentes with $1 million in seed funding and a soft commitment to support the Series A. It wasnt the robust financial infusion Patterson had hoped for, but it was enough to get started.

FROM THE CLEANROOM TO THE BOARD ROOM

Though Patterson studied science in college, he says he didnt know much about biotech until 1993. A college friend, also a biochemistry major, got a lab job at Biogen Idec, he shares. The friend encouraged Patterson to look into the same work, and eventually he landed a manufacturing tech job at Genzyme. I was a protein purification technician in a GMP facility, he recounts proudly. In other words, his days involved gowning in and then spending 6 to 8 hours working away in a lab coat, hairnet, and safety glasses. It has played an important role for me as a CEO, because I can relate well to the day-to-day challenges faced by people working in manufacturing and laboratory areas, he says.

After nearly five years at Genzyme, during which time he transitioned to working in regulatory affairs, he moved to California and joined BioMarin as employee number 15. Now in a more senior role, he had the opportunity to work with and learn from folks like Emil Kakkis, M.D., Ph.D., current president and CEO of Ultragenyx Pharmaceuticals. At BioMarin I realized rare diseases was where I wanted to work for the long term, and after leading the regulatory group though approval of our first product, they offered me the opportunity to expand into other more business-oriented roles, he says. This eventually led to Patterson joining Amicus Therapeutics in 2004 as chief business officer and employee number eight. Less than two years later, he was picked to be COO, and less than five years after that he found himself in an even bigger role. John Crowley, Amicus chairman and CEO, was considering alternative careers at the time and had decided to step down, so I became acting CEO. Being a CEO hadnt been a driving force in my early days, but rather something that became more real over time as I found myself in ever-expanding roles. However, Crowley ended up returning as Amicus CEO, and Patterson decided it was time to consider a new opportunity.

In late 2011 he joined OrbiMed as an EIR, where he was working closely with General Partner Jonathan Silverstein, who had brought Patterson to the firm. One of the benefits of being at a VC is there is an amazing number of technologies and company profiles that flow through these firms, he elaborates. It also seemed an interesting way to see a wide variety of prospects, to learn, and eventually land a CEO opportunity. During this time, Patterson became reacquainted with gene therapy. Amazed by the amount of progress being made, especially in the monogenic rare disease targets where he was so personally passionate, he began reaching out to various academic contacts he had made during his time at BioMarin and Amicus. This was how he connected with the researcher at Wake Forest working on XLMTM. The early research was extremely compelling and had all the hallmarks of past successful programs I had worked on, meaning there was significant medical need, a clear understanding of the scientific basis of the disease, compelling early data from two animal models (i.e., mouse and dog), and a motivated patient and medical community wanting to see research advance. All of these things were exciting to Patterson, and he became convinced that he could help.

In preparing for the previously mentioned pitches to OrbiMed, Patterson worked up a vision for how much he needed to raise to accomplish milestones meaningful to VC investors. This is an important part of building early-stage businesses, because when you think about financing, you have to think about how much money is needed to comfortably get to milestones that will produce additional value, as VCs look at it from the perspective of what they are going to get for their money and in what time frame, he explains. This is where working at OrbiMed helped refine Pattersons knowledge, making it easier for him to think about how to pitch an opportunity. Nonetheless, even with this wisdom and pitching to people he knew it remained a challenging conversation.

By the fall of 2012, Patterson had become even more passionate about the company, which by then even had a name Audentes Therapeutics. For those Latin nerds, Audentes means those who have courage, those who have boldness or daring, he notes. Patterson says he consulted his mother (a former Latin teacher) on the name to make sure his grammatical use of the word was accurate. This also explains how the company eventually came up with its NASDAQ ticker symbol, BOLD. The courage theme always resonated with me when I think about the patients and families within the rare disease communities, so I wanted a word that captured that.

THE BUILD ITS NEVER AS EASY AS YOU THINK

After officially founding the company in November 2012, Patterson worked out of his apartment in Manhattan for about six months before moving to San Francisco. I always knew that if I wanted to build an innovative biotech, I needed to put it where I could hire really talented people, and for me, at the time, that was either Boston or San Francisco. Having worked in both regions, Patterson also figured personal connections might prove important to getting those first few staffers on board. People say they like the idea of going to a cool, small startup, but the reality is its not for everyone, he states. Once prospects begin to kick the tires, its personal relationships and a history of working together that tend to make the difference.

But there was a lot to be done before he could begin recruiting. For example, he needed to have weekly calls with the scientists on both U.S. coasts and in France to make sure they were making progress that would keep Audentes on track from a development standpoint. He had to put sponsored-research agreements in place to help fund some of the work that needed to happen to continue to prove the company and its compounds had potential. There were other licenses that were needed to enable the technology to go forward and make the investment story more compelling, he elaborates. So, Patterson started a conversation with another company that had IP related to the vector Audentes wanted to use and convinced them not to force him to pay up front for a license. I pitched the company and my background, and thankfully they thought it credible, he shares. The two agreed on terms that deferred payment until Patterson landed the Series A funding. This, along with securing licenses for any IP developed by the academic scientists he was working with, was pivotal, because now he could comfortably say to VCs that he had key IP agreements in place. The clock was ticking, and all these things needed to happen in parallel, because the reality is a million bucks wasnt going to last very long.

Next, Patterson set about trying to improve his PowerPoint slide deck for the 2013 J.P. Morgan (JPM) healthcare conference in San Francisco. As an executive of Amicus for many years, the JPM environment wasnt new. But instead of getting a room at the Westin St. Francis [the host hotel for the annual gathering] and getting one-on-ones all day with investors, it was me lugging around my laptop and meeting with VC contacts in the lobby of the nearby Hilton with dozens of people around us screaming their own investment stories, he relates. It was an experience that helped him better understand what did and did not resonate with investors which in this case was primarily gene therapy. I heard a lot of different excuses for why people didnt want to invest in gene therapy, but essentially most of them were just saying it was too risky.

Over the next four to five months, Patterson spoke with about 25 VCs with little progress. In retrospect, given the profile of what I was trying to do, the best use of my time would have been with firms that had a history of investing in innovative science at an early stage, because at that time there was really only a handful of VCs interested in early stage, with most of them clearly being more comfortable with programs already having proof-of-concept. But where he did make progress was when he finally connected with Kush Parmar, M.D., Ph.D., at 5AM Ventures. He was a relatively new member of the 5AM team at the time, but lucky for me, he was personally interested, as he had been doing some work on gene therapy and was aware of the advancements made in academic research as well as its potential in rare diseases, Patterson grins. With Parmar as a champion, 5AM was immediately interested, which helped bring OrbiMed further along, as the two had a history of working together. And while OrbiMed, led by Silverstein, stepped up to lead the Audentes Series A, at a $30 million round, they wanted Patterson to find yet one more firm. He continued his dialogue with multiple firms, but with the credibility of OrbiMed and 5AM backing, it suddenly got easier. Once theres momentum in a deal, VCs will often follow each other, the CEO notes. In the end, we were glad to add Versant Ventures as our third, and that became the Series A, closing in July 2013, with OrbiMed investing $15 million, and 5AM and Versant splitting the other $15 million, and all three joining the Audentes board.

HOW A TOTAL FAILURE BECAME A SOURCE OF SUCCESS

Another round of financing was completed in the fall of 2014. At the time, gene therapy was gaining more acceptance as a powerful treatment option for a range of rare diseases, so Patterson started planning to expand the companys pipeline to help mitigate risk and increase the impact of their therapies. And then, the unthinkable happened. We had a total failure of the manufacturing of the lead product in late 2014, which was being done at an academic center, the executive explains.

When he started the company, Patterson felt confident he understood the manufacturing component. After all, biologics manufacturing at Genzyme and BioMarin was where he got his start. Turns out I didnt know as much as I thought, because on the surface gene therapy looks a lot like a traditional biologics manufacturing processes, but the manufacture of AAV gene therapy products was far more complex scientifically than I had initially appreciated, and that was a tough lesson to learn, he states. Further, there was no one in the world that knew how to do it at the scale necessary to run a clinical development program and eventually be commercial. Obviously, the company needed to find a new way. The initial thought was to come up with a small-scale process and work with a CMO. But heres the problem. First, there were no CMOs that knew how to make AAV gene therapy products at the time. Second, if the company did decide to go the CMO route, it would be a long-term collaboration with Audentes essentially teaching the CMO gene therapy process development and scale up. I realized this was a really important turning point in building the company, he relates. Because how to manufacture this type of product, do it well, and at large scale was something that was going to become a big issue, especially considering we were seeing more gene therapy companies being started.

Instead of lamenting over this setback, Patterson viewed it as an opportunity, akin to the biotechs of the 1980s and 90s working on innovative technologies and having to build their own internal manufacturing capabilities. Similarly, we realized this was our moment to be a leader, and we recognized the importance of owning that manufacturing capability, he shares. But there was just one problem he had to convince a board composed primarily of VCs that the money they had just invested should go toward building an internal, large-scale GMP manufacturing facility. That wasnt the plan during the fundraising round, and it wasnt a plan normally supported by investors. Still, Patterson was able to convince them of the incredible importance of having such capabilities to the eventual success of the business. It was the most important strategic decision weve ever made as a business and probably my greatest success as a CEO, he smiles.

The company found an old decommissioned GMP plant in South San Francisco that was really just a section of a warehouse that had been used for biologics manufacturing. We invested $15 million into the facility and leased the adjacent space to be able to expand capacity down the road, Patterson says. Taking such a staged approach made the move more financially feasible for the business. It also made it easier to pitch to the board of directors, who thankfully had the courage to support it, he adds. Its a tired phrase in biologics that the process is the product, but it is very much the case in gene therapy.

That same year, the company began to consider another big move. It was clear we were building a capital-hungry enterprise, which meant going public so we would have the funds needed to continue to build the business the way we knew it needed to be built, he states. After a mezzanine financing round, Patterson and his team began the laborious task of choosing banks for the IPO. It was important to have the panache of at least one larger bank, but I also wanted to make sure it was a bank that would fight for us if times got tough, he says. He also wanted a couple of smaller banks that he felt would work hard to get the deal done, as there were signs the markets would be a little more uncertain in 2016. The syndicate ended up consisting of Bank of America (BofA), Merrill Lynch, Cowen and Company, and Piper Jaffray as joint book-running managers for the offering, and Wedbush PacGrow as acting comanager.

The first quarter of 2016 turned out to be the worst quarter in biotech in many years. But the additional private financing gave Audentes the runway to be patient, and it was finally able to complete the IPO in the summer of 2016.

The banks, of course, wanted Patterson to immediately embark on a road show to announce the deal, but they were surprised to see three weeks blocked off in June on his calendar. He explained he was getting married and didnt believe cancelling his honeymoon in the interest of the IPO would be good for his well-being. Thankfully, all the banks agreed, he shares. So, it turned out to be a pretty fun and exciting six-week window, both personally and professionally.

Today, Audentes Therapeutics is a publicly traded company valued at nearly $1.8 billion.

However, there are other measures of success. For example, in September 2017, Audentes initiated a clinical trial of its lead program, AT132 for XLMTM, and positive results to date led them to announce plans to file for approval of that product in mid-2020. In the meantime, the company has expanded its pipeline of neuromuscular targeted programs, going after Pompe disease, Duchenne muscular dystrophy, and myotonic dystrophy and has grown to more than 250 employees. Throughout the last seven years, weve gone from humble beginnings to where we are today, and while Im sure we have a long road ahead of us, I am very proud of what it has become, Patterson concludes.

When Matt Patterson was at OrbiMed Advisors working as an entrepreneur in residence (EIR), he became enamored with the idea of founding a gene therapy company. He admits, though, that there were plenty of other opportunities that he could have pursued considering the amazing number of technologies and company profiles that flowed through OrbiMed. I had looked at a couple of later-stage companies, as OrbiMed wanted me to be pitching a company that was already in the clinic with some data, he says. But he had made up his mind that he was only interested in finding an opportunity where he could work as CEO. He says that if a company was in the clinic with data that looked good, and they were looking for a CEO, then something else must have been wrong. A fixer-upper wasnt interesting to me, and I didnt want to be a hatchet man whose job it was to fire most of the team and refocus the company, he explains.

He did find some interesting rare-disease assets at some larger companies that he thought were, frankly, not well-suited to advance, and so he set out to try to convince those companies that those assets would be better off in his hands. For example, when Bristol-Myers Squibb (BMS) bought Amylin Pharmaceuticals, Amylin had an orphan drug product called metreleptin for a genetic disorder. I tried to convince BMS that they should out-license that program to me. The conversation lasted about three days, meaning two interactions of, Thats interesting. Well get back to you. and then, No. In the end, he stayed committed to his original plan and ended up starting a new company, Audentes, around the X-linked myotubular myopathy (XLMTM) program he discovered at Wake Forest University.

Throughout the course of growing Audentes Therapeutics, Matt Patterson admits to taking the concept of culture very seriously, but he wasnt overly formal about defining it especially in those early years. Ive always been fascinated by the fact that, in biotechnology, one could have a brilliant scientific hypothesis and vision, but the idea goes nowhere because you failed to hire and retain a talented team, he mentions. Its critical that people enjoy their work and are inspired to put in the effort needed to be successful. I was always paying attention to our culture. We had a high rate of employee retention, and everything seemed to be going well, he elaborates. But when the company reached the 100-employee mark, he felt culture was something to be discussed a little bit more formally. We spoke about it at a leadership team meeting and attempted to engage the employee base more broadly in the issues they found important, so theyd have a voice, and we could learn.

The company also did various surveys and small group meetings that were really productive, but in the end, Patterson says neither fundamentally changed the companys overarching vision or mission as a business. All of those exercises led to the creation of three core values, although Patterson admits he was skeptical when they began creating them. Ive been at companies where they went through that exercise, and it became something like a 3x5 card that sat on the desk of every employee but was never truly embraced. He recalls starting the first meeting by saying, I dont think this is going to be a good use of time, and the last thing I want is to come up with a few catchy phrases that management thinks sound good but really dont resonate, as that seems silly. Id rather publish nothing than have that outcome. But as the program evolved and the group began getting into the brass tacks of core values and how to make them real, he became more enthusiastic. We came up with three that we felt were meaningful, could be acted upon, and made an example of on a daily basis.

The first is to be bold, find a way. This means striving to really figure out how to solve a tough challenge, whether scientific, clinical, or other. The second is to care deeply about patients, the work, and the team. The third, which is about being focused and working hard every day to achieve the companys goals, is #GSD, which stands for get stuff done, though Patterson shares that they tend to use a four-letter word in place of stuff internally. We felt those three captured who we were and who we wanted to be, he says. When someone does something outstanding to solve a challenge or to help a teammate, Audentes might recognize them with a Boldy, a symbolic award that includes a small financial component.

They implemented the three core values about 18 months ago, and ever since then they seem to come up all the time. When employees use them casually and regularly without management telling them to, thats the most important validation, he contends. And though this CEO went into meetings about culture and core beliefs as a bit of a skeptic, he says he came out a believer.

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Building A 1 Billion Gene Therapy Company In 6 Years - Life Science Leader Magazine

Gene therapy for HIV Ravi Gupta, Cambridge University; Michael Pepper, University of Pretoria

Around the world, almost 40 million people are living with HIV. It grows in and progressively destroys the immune system, leaving victims highly susceptible to what would normally be trivial infections. Now, after decades of effort, scientists are finally beginning to cautiously use the C word: cure - and gene therapy is likely to be central to the approach theyre taking. Infectious diseases specialist Ravi Gupta from Cambridge University and University of Pretoria physician andmolecular cell biologist Michael Pepper told Chris Smith and Katie Haylor about their research in this area. First up, Katie asked Ravi, what does HIV actually target when it gets into the body...

Ravi - So HIV is what we call a retrovirus which means that it infects cells, as many other viruses do, except this time rather than just making copies of itself, it actually integrates into the genes or chromosomes of the individual so it's there permanently.

So this is why HIV is a disease that doesn't go away and could not be cured until recently because of this latent phase that we refer to. Now this happens primarily in white blood cells that are there to protect you. They're called lymphocytes and they have a protein called CD4 and this is a protein that HIV absolutely requires to gain entry to a cell. So that's why it's only able to infect a CD4 positive T cell.

Chris - And by growing in those cells and destroying them in the process, it's going to leave that person with a dwindling population of the cells that are a lynchpin part of the immune system?

Ravi - That's right, CD4 cells orchestrate the entire immune system and so once they start disappearing you get susceptibility to not only infections but cancers.

Chris - And how did you cure, in inverted commas, your patient?

Ravi - We were able to identify an individual who unfortunately due to advanced HIV infection developed Hodgkin's lymphoma which is a recognized complication, because our immune systems defend us against cancer in our everyday lives.

And so this individual had end-stage cancer that was not responsive to any chemotherapy that we used and the only option left for him was a transplant using cells from a donor who was already immune to HIV. And we know that certain individuals are immune.

Chris - Why was that person immune to HIV, the donor person?

Ravi - Around two decades ago, we identified a second receptor or protein that HIV absolutely requires. This is called CCR5. So you need both CCR5 and CD4 for the virus to enter cells.

Chris - So that's sitting on the outside of the cell. It's almost like a stepping stone for the virus to be able to grab hold of and then get into the cell and if that's not there the virus can't invade.

Ravi - Absolutely. And so we realized that around 1 percent of individuals have two mutated copies of CCR5 in their genes and therefore they cannot be infected.

Chris - And hence if you put that bone marrow into your patient and they then build a new immune system from that person's HIV resistant cells, they can't then mount an ongoing HIV infection.

Ravi - Absolutely.

Chris - And that's what you believe has occurred in this patient?

Ravi - That's right. There was a patient who needed a transplant from a donor and for that to work you need to give high doses of chemotherapy to clear the patient's own cells, to allow the incoming cells that are resistant to HIV to then take hold and to populate the blood.

Chris - I suppose Michael that the problem with the strategy that Ravi is outlined here, is that, as he says, only a tiny minority of people naturally have a bone marrow with that particular genetic configuration that's resistant to HIV. So this wouldn't be a practical solution for the 40 million or so people who are currently infected with HIV.

Michael - Absolutely Chris. The problem is amplified here in sub-Saharan Africa where we have a huge genetic diversity. And to find somebody who has an adequate match and is also deficient in CCR5 is really very very difficult. So our approach is to try and engineer cells that we're going to give to patients in order to make them resistant to the virus.

Chris - So it's a similar sort of strategy in the sense that Ravi is putting into a patient a set of cells that are resistant to HIV, albeit from a donor. You're saying can I take a person's own cells or even get donor cells and change them in some way to make them resistant, so when they go in that person's immune system can be rebuilt from those cells and their own virus can attack them.

Michael - The idea is to take the person's own cells, engineer them outside of the body so that they don't express CCR5, and then create space in the bone marrow so that when you give them back to the patient they can take up residence and start producing an immune system which is resistant to HIV.

There are many techniques that are being used to do this. One of probably the most topical at the moment is gene editing, to edit out CCR5 from the cells that you're going to give back to the patient. And then there are other techniques, such as the one that we're using, which is to try and prevent the protein from being expressed and therefore the docking element on the surface of the cell would be absent.

Chris - So you're saying you manipulate the cells in a dish, having collected them from the patient. So you've got HIV uninfected cells and you manipulate them to remove from the cell that lynchpin protein that Ravi was talking about, the CCR5 that the virus would normally need to get in, and then you can put those cells back into the person and they then become the source of their immune system?

Michael - That's correct Chris. I think that's the technique that everybody is working on at the moment all over the world. In sub-Saharan Africa the question is going to be one of capacity and of course cost. So it was very exciting to hear Sue and Steve speaking earlier about their approach, which is to directly introduce the material that is going to do the gene therapy into the patient's bloodstream and that either the virus or the DNA would then have its effect on the target cells. And the hope is that in the long run, particularly in this part of the world, that we'll be able to do away with engineering the cells outside of the patient's body, and simply add the virus which is carrying the machinery necessary to engineer the cells or the DNA directly into the bloodstream of the patient.

Chris - Have you got evidence that this will actually work in a patient yet though?

Michael - So we have evidence in mice that have a human immune system, and we can achieve a functional cure in these mice. There are people working in other parts of the world that have done the same thing and there have recently been some publications from other people who've showed that gene engineered cells do persist in the body of people in whom CCR5 has been removed from the target cells.

Chris - Ravi what do you think? Does this sound plausible to you?

Ravi - Certainly I think that the theory is there. The problem that is going to emerge is that without use of chemotherapy to essentially remove existing cells, it's a question of a relatively small number of engineered cells being introduced or being modified. The problem is that HIV can then just go into the cells that have not been modified. And so that's the big problem we have.

Chris - So I guess what you're alluding to is what Michael was saying about making space in the bone marrow. So Michael presumably you've got to give patients bone marrow toxic drugs to wipe out some of their normal bone marrow, to make space for your modified cells to come in. And I should say it's probably a bit of an ethical dilemma isn't it? Because we're quite good at treating HIV with drugs at the moment. And you're saying give people more poisonous drugs and a risky procedure, when they're not actually ill at the moment.

Michael - Hopefully there are agents other than toxic chemotherapeutic agents that will be useful in the future, to open up a niche in the bone marrow. There is work going on in several areas around the world.But there is an alternative. And that is to use T cells. So you may have heard about CAR T cells which have been used very successfully for the treatment of leukemia and lymphoma. And people are now moving in the direction of creating CAR T cells that would be used for HIV.

Chris - This is the Chimeric Antigen Receptor T cells it isn't. It's where we modify the cells to endow them with a very specific, targeted, receptor that recognizes one thing we've programmed them to go after.

Michael - That's correct. Should this be successful, it would no longer be necessary to open up a niche in the bone marrow. One would simply remove the T cells from the patient, engineer them and give them back. And these cells are pretty long lasting.

Chris - Ravi? Your thoughts?

Ravi - Yes I think there is huge amounts of effort going into different approaches for modification and of course knocking out various populations of cells. So I think it is a very exciting field at the moment. I think what's incredible is that infectious diseases and cancer, for example, are sharing a lot of technologies and there are more similarities than we ever really appreciated in the past. I think that's a wonderful thing.

Chris - Ravi, we've talked a lot about deleting this CCR5 gene that HIV uses to clamber inside the cells it wants to hit. If you take that away, does that not render a person at any kind of disadvantage or less healthy than people who have that gene? Presumably it's there for a reason.

Ravi - That's a really good question. I think that it's been uncertain for a long time. We postulate that this mutation emerged as a natural or a process of natural selection, potentially because of infectious diseases such as smallpox or one of the other postulated things was the plague.

But for whatever reason this mutation has persisted in the population without apparent deleterious or harmful effects. On the other hand, a recent study published in Nature Medicine suggested that people with the double deletion in both copies of the gene lived on average a year and a half less than those who didn't have it. Which throws into question whether it's going to be safe or not.

Chris - And Michael, returning to the therapies that you're alluding to, both doing these genetic manipulations and putting stem cells in, and also using these CAR T cells, these modified T cells to go after HIV. It sounds wonderful and we know that we can do this for certain diseases, but can we afford it? Because there are 40 million people with HIV. They're not rich people. They're not in rich countries.

Michael - Chris that's the key question for this part of the world, where a large part of health budgets go towards providing antiretrovirals for the 7 million or so people in South Africa that are affected.

Gene therapy is expensive. If you do a health economics analysis though, the cost of HIV is enormous. And I'm not including the cost of the antiretrovirals, Im including the cost of the complications that arise such as cancer and infectious diseases. The cost to society where we have child-headed households in South Africa, and all of the social complications that come as a result of that.

I think as the procedures become refined, and as we can move to cheaper alternatives, such as for example not having to engineer cells but giving vectors or DNA directly to patients, the cost will come down. And of course the more we do, there'll be economies of scale. And so hopefully that this will bring the cost down.

But I think a case can be made for a once off, fairly costly, form of treatment as opposed to the lifelong cost of someone who is HIV positive.

Ravi - Yes I would echo those opinions because there was a time when people thought antiretrovirals were too expensive for Africa. And things change. So I think that not pursuing certain things in medical science, because of cost, is a mistake. We need to do the best science, to show we can do it, and then deal with the cost.

Read more:
Getting to Grips with Gene Therapy - The Naked Scientists

Were experiencing an exciting time in the region, with wave upon wave of innovations, collaborations, acquisitions, tech transfer and growth, said MTC CEO Martin Rosendale. Companies from all over the country are moving here to experience all that Maryland and the region have to offer. This conference provides the perfect backdrop to showcase it all and discuss how to further propel us to the No. 1 spot among life sciences hubs.

This years keynote speakers include, John Tisdale, MD, Chief, Cellular and Molecular Therapeutics Branch National Heart, Lung, and Blood Institute, National Institutes of Health, and Michelle McMurry-Heath, MD, PhD, Vice President, External Innovation, Global Leader for Regulatory Science and Executive Director of Scientific Partnerships for JLABS @ Washington, DC. Maryland Secretary of Commerce Kelly M. Schulz will give opening remarks, and Montgomery County Executive Marc Elrich will help close out the conference.

The conference will also highlight the cell and gene therapy and biomanufacturing revolution taking place in Maryland and throughout the region. Notable speakers in the cell and gene therapy/biomanufacturing space include:

Were pleased that the Maryland Tech Council selected Montgomery County as the location for this important conference, said County Executive Elrich. The life sciences sector is thriving here, so its fitting that the conference is taking place in our county. My administration is focused on creating more opportunities for new and emerging businesses to grow and locate here.

This year for the first time, attendees will be able to simplify the process of searching for, identifying, and meeting with potential partners and business development executives with the BIO One-on-One Partnering system.

Maryland is continuing to make major strides in cell and gene therapy, as well as biomanufacturing, said Secretary Schulz. This conference offers a great opportunity to bring together our best and brightest industry leaders to take a closer look at how the industry is evolving and how Maryland is leading the way.

For more information or to register, visit: marylandlifesciences.com/conference/about/.

About Maryland Life Sciences

Maryland Life Sciences (MDLS), a division of the Maryland Tech Council, is a regional association for the life sciences community. We support our member companies who are driving innovation through advocacy, education, workforce development, cost savings programs and connecting entrepreneurial minds. MDLS represents biotechnology, clinical and research data, therapeutic, genetic, medical device, pharmaceutical and service companies that support Marylands thriving industry. The valuable resources we provide to our members help them reach their full potential making Maryland a global leader in the life science industry. For more information: mdtechcouncil.com/communities/life-sciences/.

About Maryland Tech Council

The Maryland Tech Council (MTC) is a collaborative community that is actively engaged in building strong technology and life science industries by supporting the efforts of our individual members. We are the largest technology and life sciences trade association in the state of Maryland, and we provide value by giving members a forum to learn, share, and connect. MTC brings the regions community together into a single, united organization that empowers our members to achieve their business goals through advocacy, networking and education. The vision for the Maryland Tech Council is to propel Maryland to become the number one innovation economy for life sciences and technology in the country. For more information: mdtechcouncil.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191003005202/en/

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Maryland Life Sciences Bio Innovation Conference Highlights Region's Growth with Emphasis on Cell and Gene Therapy, Biomanufacturing - BioSpace

The Ministry of Food and Drug Safety inspected six gene therapy companies as a follow-up measure to the Invossa issue, a wire report said Thursday.

Invossa, a gene therapy for joint inflammation treatment by Kolon Life Science, was stripped of its sales approval in May this year after the company belatedly reported a mislabeling of the key cell used in the drug.

Invossa obtained the MFDS approval in July 2017 and started commercial sale in November of the same year.

While attempting to make inroads into the US market, KLS said it realized that what the company had thought was the source cell of Invossa had in fact been kidney-derived cell, not the cartilage-derived cell indicated in its documents earlier submitted for approval in Korea.

This mix-up had a devastating effect on the company, as the kidney-derived 293 cell in question was suspected to have potentially cancerous properties.

KLS countered the suspicion saying that the radiation treatment of the 293 cell made it safe.

The company said it will run a 15-year free medical check-up for patients who have taken the Invossa shot while trying to restart the phase 3 clinical trials for the drug in the US.

The US Food and Drug Administration recommended KLS replace the kidney-derived cell to cartilage-derived cell.

Kolon TissueGene, the US-based affiliate responsible for the research and development of Invossa, was tentatively delisted from the Kosdaq bourse in August.

Kolons demise has prompted the Drug Ministry to run a good manufacturing practice inspection on the 10 other gene therapy companies in Korea.

GC Green Cross Cell, Medipost, Tego Science and Corestem were among the six that were inspected in late August and early September.

Based on the inspection results, follow-up measures will take place this month at the earliest.

By Lim Jeong-yeo (kaylalim@heraldcorp.com)

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Invossa aftereffects course over to other gene therapy companies - The Korea Herald