Category Archives: Gene therapy

WASHINGTON--(BUSINESS WIRE)--Greenleaf Health, Inc., a leading Food and Drug Administration (FDA) regulatory consulting firm, today announces that the firm has expanded its portfolio of services to guide companies developing cell and gene therapy products. Greenleafs team of regulatory experts will be led by Karen Midthun, M.D., former Director of the FDAs Center for Biologics Evaluation and Research (CBER), and John Taylor, former FDA Counselor to the Commissioner and Principal Deputy Commissioner.

REGULATORY LANDSCAPE: Cell & Gene Therapy

The rapidly evolving fields of cell and gene therapy offer the possibility of novel treatments, and perhaps ultimately cures, for devastating and intractable illnesses. In response to what the FDA has called a "turning point in the development of these technologies and their application to human health, new policies have been introduced to address the development of safe and effective cell and gene therapies.

With innovation often comes uncertainty. In the case of cell and gene therapy products, the FDA has raised concerns about developers operating outside of the existing regulatory paradigm. To prevent this, the FDA has clarified the regulatory framework for regenerative medicine products and announced near-term enforcement actions aimed at ensuring compliance by companies developing and manufacturing cell and gene therapies.

Greenleafs expanded services support companies striving to introduce cell and gene therapy products to patients. The firms team of experts has a robust blend of technical skill and FDA institutional knowledge that spans all therapeutic areas and quality, manufacturing, and compliance systems. By working cross-functionally, Greenleaf ensures that clients have the comprehensive, specialized support needed to understand and navigate the complex regulatory landscape for cell and gene therapies.

FULL-SERVICE SUPPORT

Members of Greenleafs Drug and Biological Products Team work together with the firms Product Quality, Manufacturing, and Compliance Team to deliver guidance on cell and gene therapy products.

Product Development & Review

With the expert direction of Karen Midthun, M.D., Greenleafs team of advisors assists sponsors of cell and gene therapies by optimizing FDA interactions and submissions to support development and regulatory review. Greenleaf also helps sponsors understand and respond to the FDA requirements applicable to various cellular products, and provides guidance to sponsors of cell and gene therapies to treat rare and ultra-rare diseases on ways to maximize trial design using appropriate clinical endpoints and natural history study data to aid efficient product development.

Quality, Manufacturing & Compliance

Greenleafs Product Quality, Manufacturing, and Compliance Team, led by John Taylor and supported by the firms network of independent compliance experts, offers credible, informed guidance to help manufacturers of cell and gene therapies comply with the FDAs multiple current GXP regulations. Greenleaf experts provide strategic and technical support for establishing manufacturing and quality controls; pre- and postapproval inspection readiness; compliance assessments; evaluating and responding to FDA regulatory correspondence; and engaging with CBERs Advanced Technologies Team.

UNMATCHED EXPERTISE

Greenleaf is comprised of experts with a combined total of more than 250 years of FDA experience. The firms team of advisors demonstrates unmatched levels of skill in its specialties of drug and biological products and product quality, manufacturing, and compliance. Greenleafs Cell and Gene Therapy Team, led by Dr. Karen Midthun and John Taylor, is guided by decades of regulatory experience in senior FDA positions, global public health organizations, academia, and industry.

Karen Midthun, M.D.Principal, Drug & Biological Products

Dr. Midthun contributes specialized insight informed by her regulatory, research, and clinical experience to FDA-regulated entities developing cell and gene therapies. Dr. Midthun joined Greenleaf following a distinguished 28-year career in public health, of which 22 years were dedicated to the FDA.

An infectious disease physician by training, Dr. Midthun most recently served as the Director of the FDAs Center for Biologics Evaluation and Research (CBER). During her FDA tenure, Dr. Midthun played a critical role in facilitating policy and technology development in the areas of cell, tissue, and gene therapies, blood products, and vaccines.

John Taylor, J.D.President, Greenleaf Health, and Principal, Compliance & Regulatory Affairs

Taylor has held many high-profile positions at the FDA, as well as senior leadership roles within industry. Taylors wealth of regulatory experience, robust technical skills, and unique strategic perspective are unmatched. Clients working with Greenleafs Product Quality, Manufacturing, and Compliance Team benefit from Taylors vast FDA institutional knowledge.

Taylor joined Greenleaf following a distinguished 20-year career at the FDA, where he served in multiple leadership positions, including as the FDAs Acting Deputy Principal Commissioner, FDA Counselor to the Commissioner, Acting Deputy Commissioner for Global Regulatory Operations and Policy, and Associate Commissioner for Regulatory Affairs.

ABOUT GREENLEAF

Greenleaf Health provides strategic and technical guidance to pharmaceutical, biotechnology, and medical device companies researching, developing, and manufacturing innovative solutions to pressing global public health challenges.

The firm includes former leaders and regulatory professionals from the FDA, Capitol Hill, top global pharmaceutical and medical device companies, leading law firms, and the top U.S. biotechnology trade organization. Greenleafs blend of former FDA officials and industry experts provides a unique set of capabilities when advising entities regulated by the FDA.

For more information on Greenleafs cell and gene therapy services and Greenleaf Health, visit greenleafhealth.com.

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Greenleaf Health Expands Services to Support Cell & Gene Therapy - Business Wire

SAN RAFAEL, Calif., Sept. 26, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN), a pioneer in treatments for rare disease Phenylketonuria (PKU) and in gene therapies, today announced that it has submitted a Clinical Trial Application (CTA) with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. for BMN 307, an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to reduce blood phenylalanine (Phe) concentrations levels in patients with PKU. BMN 307 will be evaluated to determine whether a single dose of treatment can restore Phe metabolism in patients with PKU, normalize plasma Phe level, and enable a normal diet. The company expects to start enrolling patients in a Phase 1/2 trial early next year and is actively preparing regulatory submissions for other countries. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and its second gene therapy development program. BMN 307 follows BioMarin's first investigational gene therapy program, valoctocogene roxaparvovec for severe hemophilia A, currently in a Phase 3 study.

"This clinical trial application marks the latest milestone in BioMarin's 15-plus year commitment to the PKU community. BioMarin has brought the only two approved therapies for PKU to patients around the world," said Hank Fuchs, President, Worldwide Research and Development at BioMarin. "Leveraging our expertise in gene therapy, we are pleased to be adding a new gene therapy product, which is potentially a transformative solution, to build on our current achievements for the PKU community."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is found in most forms of protein. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychiatric-related issues, affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

Phase 1/2 Study

The Phase 1/2 study will evaluate the safety efficacy and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The Phase 1/2 study consists of a dose-escalation phase, followed by a dose expansion phase. In addition, there is also an observational study in PKU patients to measure markers of disease and clinical outcomes over time inpatients.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has treated approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, in addition to the investigational gene therapy BMN 307 in development. BioMarin has conducted 40 clinical studies in PKU and has sponsored 38 external clinical studies. BioMarin researchers have authored 54 publications in medical and scientific journals on PKU, and BioMarin has supported another 52 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a corrected copy of the defective gene. The functional gene is inserted into a vector, containing a small DNA sequence, that acts as a delivery mechanism, providing the ability to deliver the functional gene to cells. The cells can then use the information to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has constructed, commissioned, and validated one of the first gene therapy manufacturing facilities of its kind in the world, which is located inNovato, California. This facility can support up to 4,000 doses per year, and the production process was developed in accordance with International Conference on Harmonisation guidance to facilitate eventual worldwide registration with health authorities. In 2018, the International Society for Pharmaceutical Engineering (ISPE) selected the Company's gene therapy manufacturing facility as the Facility of the Year Category Winner for Project Execution. The BMN 307 PKU gene therapy clinical production occurred at commercial scale in this facility. Production of material at commercial scale in the state of the art facility significantly reduces risk associated with making process changes later in development and can speed overall development timelines.

About Phenylketonuria

PKU, or PAH deficiency, is a genetic disorder affecting approximately 50,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most adult patients to adhere to the strict diet to the extent needed for achieving adequate control of blood Phe levels.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin's BMN 307 program generally, BioMarin's planned submissions to regulatory authorities for BMN 307, BioMarin's gene therapy manufacturing capabilities, and the timing and results of BioMarin's planned Phase 1/2 trial of BMN 307. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others:results and timing of current and planned preclinical studies and clinical trials of BMN 307; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; the risks related to commercialization of BMN 307 and our ability to manufacture sufficient quantities of BMN 307; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2019 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

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BioMarin, Pioneer in Phenylketonuria (PKU) Therapies, Submits Clinical Trial Application (CTA) in UK for Investigational Gene Therapy for PKU -...

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Newswise The Childrens Cancer Institute at the Joseph M. Sanzari Childrens Hospital at Hackensack Meridian Health Hackensack University Medical Center and the John Theurer Cancer Center have announced they are participating in a multicenter Phase I/II clinical trial of an investigational gene therapy from bluebird bio, Inc. This trial is specifically for adolescents and adults with severe sickle cell disease (SCD) who cannot be effectively treated using standard therapies such as antibiotics, vitamins, blood transfusions or any pain relieving medications. The study is evaluating the safety and effectiveness of LentiGlobin for sickle cell disease, a gene therapy produced using the patients own modified stem cells to treat their sickle cell disease.

By using the patients own cells to produce functional hemoglobin that can prevent sickling of their red blood cells, LentiGlobin for SCD offers patients the opportunity to treat their disease without the need to have a matched bone marrow donor. The John Theurer Cancer Center is one of a limited number of centers internationally, and the Joseph M. Sanzari Childrens Hospital is the only pediatric site in New Jersey, where the study, which is enrolling patients age 12-50, is taking place.

Sickle cell affects 100,000 Americans. It affects one in every 365 African American births and one in every 16,000 Hispanic American births, said Alfred P. Gillio, M.D., director, Childrens Cancer Institute and section chief, Pediatric Stem Cell Transplantation and Cellular Therapy Program, Joseph M. Sanzari Childrens Hospital at Hackensack University Medical Center. This trial is for patients who have severe sickle cell disease and seek advanced treatment options but do not have a well-matched stem cell donor. Only 15% of sickle cell patients have a matched sibling donor and only 25 percent of patients have a matched unrelated volunteer donor.

Sickle cell affects every organ in a patients body, said Stacey Rifkin-Zenenberg, D.O., FAAP, pediatric hematologist/oncologist, Childrens Cancer Institute, and section chief, Pain and Palliative Care, Joseph M. Sanzari Childrens Hospital at Hackensack University Medical Center. This disease really has a tremendous effect not only on the patient, but also the family.

Sickle cell disease is an inherited disease caused by a mutation in the beta-globin gene, resulting in abnormal hemoglobin and sickle-shaped red blood cells. Symptoms and complications of the disease include anemia, infections, stroke, poor quality of life and early death. To date, the only cure for sickle cell disease is receiving a stem cell transplant from a matched donor, but this is not a therapeutic option for many patients. Supportive care including hydroxyurea and blood transfusions can ameliorate symptoms of the disease. To date, without a marrow donor, there has been no alternate curative therapy. Life expectancy of a person with sickle cell disease is 20 to 40 years of age. In some cases, patients using disease modifying medications can live to 50 or 60.

This therapy may be a major advance for sickle cell patients and so far, the results look very promising, said Scott D. Rowley, M.D., FACP, hematologist, medical director, Stem Cell Transplantation and Cellular Therapy and medical director, BMT Cell Lab, John Theurer Cancer Center, Hackensack Meridian Health Hackensack University Medical Center, who is enrolling adult patients. This investigational treatment, which is a one-time therapy, may be an option for our patients who have no other treatment options.

The results from early clinical studies are encouraging, said Dr. Gillio. With this treatment, the patient is their own donor and we are modifying their own cells to add copies of a functional beta globin gene.

In the current study:

About Hackensack Meridian Health Hackensack University Medical Center

Hackensack Meridian Health Hackensack University Medical Center, a 781-bed nonprofit teaching and research hospital located in Bergen County, NJ, is the largest provider of inpatient and outpatient services in the state. Founded in 1888 as the countys first hospital, it is now part of the largest, most comprehensive and truly integrated health care network in New Jersey, offering a complete range of medical services, innovative research and life-enhancing care, which is comprised of 34,100 team members and more than 6,500 physicians. Hackensack University Medical Center is ranked #2 in New Jersey and #59 in the country in U.S. News & World Reports 2019-20 Best Hospital rankings and is ranked high-performing in the U.S. in colon cancer surgery,lung cancersurgery,COPD, heart failure, heart bypass surgery, aortic valve surgery,abdominal aortic aneurysm repair, knee replacement and hip replacement. Out of 4,500 hospitals evaluated, Hackensack is one of only 57 that received a top rating in all nine procedures and conditions. Hackensack University Medical Center is one of only five major academic medical centers in the nation to receive Healthgrades Americas 50 Best Hospitals Award for five or more years in a row. Beckers Hospital Review recognized Hackensack University Medical Center as one of the 100 Great Hospitals in America 2018. The medical center is one of the top 25 green hospitals in the country according to Practice Greenhealth, and received 26 Gold Seals of Approval by The Joint Commission more than any other hospital in the country. It was the first hospital in New Jersey and second in the nation to become a Magnet recognized hospital for nursing excellence; receiving its sixth consecutive designation in 2019. Hackensack University Medical Center has created an entire campus of award-winning care, including: John Theurer Cancer Center, a consortium member of the NCI-designated Georgetown Lombardi Comprehensive Cancer Center; the Heart & Vascular Hospital; and the Sarkis and Siran Gabrellian Womens and Childrens Pavilion, which houses the Joseph M. Sanzari Childrens Hospital and Donna A. Sanzari Womens Hospital, which was designed with The Deirdre Imus Environmental Health Center and listed on the Green Guides list of Top 10 Green Hospitals in the U.S. Hackensack University Medical Center is the Hometown Hospital of the New York Giants and the New York Red Bulls and is Official Medical Services Provider to THE NORTHERN TRUST PGA Golf Tournament. It remains committed to its community through fundraising and community events especially the Tackle Kids Cancer Campaign providing much needed research at the Childrens Cancer Institute housed at the Joseph M. Sanzari Childrens Hospital. To learn more, visit http://www.HackensackUMC.org.

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Joseph M. Sanzari Childrens Hospital and John Theurer Cancer Center Launch Clinical Trial Evaluating Gene Therapy for Severe Sickle Cell Disease in...

Company to Participate in the Alliance for Regenerative Medicine Cell & Gene Meeting on the Mesa, Chardan Genetic Medicines Conference and Jefferies Gene Therapy Summit

DURHAM, N.C., Sept. 26, 2019 (GLOBE NEWSWIRE) -- Precision BioSciences, Inc. (DTIL), a genome editing company dedicated to improving life through the application of its pioneering, proprietary ARCUS platform, today announced that members of its senior leadership team will participate in three industry and investor conferences focused on gene editing and gene therapy in October. Presentations will consist of corporate overviews and a panel discussion. Participation at the Jefferies Gene Therapy Summit will be limited to private meetings.

Alliance for Regenerative Medicine Cell & Gene Meeting on the MesaDate: October 2-4, 2019Presentation Time: October 3, 4:15 p.m. PTPanel Time: October 4, 9:45 a.m. PTLocation: Carlsbad, CASpeaker: Michael Dombeck, Senior Vice President, Corporate Development

Chardan Genetic Medicines ConferenceDate: October 7-8, 2019Presentation Time: October 8, 8:30 a.m. ETLocation: New York, NYSpeaker: Matt Kane, Chief Executive Officer

Jefferies Gene Therapy SummitDate: October 8, 2019Location: New York, NYMatt Kane, Chief Executive Officer (investor meetings)

A live audio webcast of the Chardan presentation will be available here (webcast link) and up-to-date details for each event can be found on the "Events & Presentations" page in the Investors section of the Precision BioSciences website (www.precisionbiosciences.com). A replay of the Chardan presentation will also be archived for 30 days on the Events & Presentations page.

About Precision BioSciences, Inc.Precision BioSciences is dedicated to improving life (DTIL) through its proprietary genome editing platform, ARCUS. Precision leverages ARCUS in the development of its product candidates, which are designed to treat human diseases and create healthy and sustainable food and agriculture solutions. Precision is actively developing product candidates in three innovative areas: allogeneic CAR T immunotherapy,in vivogene correction and food. For more information regarding Precision, please visitwww.precisionbiosciences.com.

Investor Contact:Jason WongBlueprint Life Science GroupTel. (415) 375-3340 Ext. 4jwong@bplifescience.com

Media Contact:Cory TrombleeScient Public RelationsTel. (617) 571-7220cory@scientpr.com

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Precision BioSciences to Present at Upcoming Industry and Investor Conferences - Yahoo Finance

CAMBRIDGE, Mass., Sept. 25, 2019 /PRNewswire/ --BlueRock Therapeutics, today announced that Emile Nuwaysir, Ph.D., Chief Executive Officer of BlueRock and Vice Chairman of the Alliance for Regenerative Medicine (ARM), will present at the Cell & Gene Meeting on the Mesa, to be held Oct. 2-4 in Carlsbad, Calif.

The following are specific details regarding BlueRock's participation at the conference: Event: BlueRock Therapeutics Company PresentationDate: Wednesday, Oct. 2Time: 11:45 a.m. - 12:00 p.m. PDTLocation: Cognate Bioservices Ballroom,Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

Event: Panel: Cell and Gene Therapy for Neurological Product IndicationsDate: Wednesday, Oct. 2Time: 4:00 - 5:00 p.m. PDTLocation: BlueRock Therapeutics Ballroom, Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

A live video webcast of all company presentations and workshops will be available at: http://www.meetingonthemesa.com/webcast and will also be published on the conference website shortly after the event.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 80 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers.

About BlueRock Therapeutics BlueRock Therapeutics, a wholly-owned and independently operated subsidiary of Bayer AG, is a leading engineered cell therapy company with a mission to develop regenerative medicines for intractable diseases. BlueRock Therapeutics'CELL+GENE platform harnesses the power of cells for new medicines across neurology, cardiology and immunology indications. BlueRock Therapeutics' cell differentiation technology recapitulates the cell's developmental biology to produce authentic cell therapies, which are further engineered for additional function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore or regenerate lost function. BlueRock's culture is defined by scientific innovation, highest ethical standards and an urgency to bring transformative treatments to all who would benefit. For more information, visit http://www.bluerocktx.com.

Company Contact:Candice Ellis, 978-879-7273Associate Director, Investor Relations and Corporate Communicationscellis@bluerocktx.com

Media Contacts: David Schull or Maggie BellerRusso Partners, LLC(646) 942-5631Maggie.beller@russopartnersllc.com

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BlueRock Therapeutics to Present at 2019 Cell & Gene Meeting on the Mesa - P&T Community

Collaboration between Baylor College of Medicine, The Methodist Hospital and Texas Children's Hospital. Clinical research in the areas of stem cell transplantation, cellular therapy, and gene therapy.

The Center fosters a multidisciplinary approach to new research as well as collaborative research endeavors in the area of gene therapy. The Vector Core manufactures several recombinant viral vectors.

The research is focused on various aspects of gene therapy, such as understanding basic virology, efficient gene delivery into the nucleus of cells, and incorporation of these genes into the genome.

Research in the laboratory has centered on the molecular biology of adeno-associated virus (AAV) in order to exploit the unique features of this virus to develop an efficient viral vector system for use in human gene therapy.

The Harvard Gene Therapy Initiative is headed by Dr. Richard Mulligan with the objective of promoting the use of gene therapy and to conduct research developing new gene delivery vector technologies.

Diseases of the lung, cardiovascular system, muscles, brain, and skin are focus areas of research as well as the development of gene therapy vectors and the identification of disease-causing genes.

A multidisciplinary team of scientists and physicians work together to realize the full potential of virus, gene and cell therapies from basic science discovery to clinical translation.

Oncolytic virotherapy, gene therapy for diabetes and cardiovascular diseases, virus-based gene therapy vectors.

The program has brought together regulatory, quality, product development, manufacturing and facilities engineering expertise to enable the translation of novel, experimental research into medicine for use in human clinical trials.

Penn Vector offers a variety of services associated with the development and production of both non-viral vectors and viral vectors including those derived from adeno-associated virus (AAV), adenovirus, and lentivirus.

The primary mission is to merge molecular genetics research and health care delivery by developing new therapeutic strategies for the treatment of human diseases that involve gene transfer.

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Gene Therapy Research Institutes and Universities

Patients cant wait, and neither will we

At Axovant, we operate with a sense of urgency to develop and deliver innovative gene therapies that transform the treatment of serious neurological and neuromuscular diseases. In this critical journey, we are inspired by breakthrough science and driven to challenge the status quo on behalf of patients and their families. By harnessing the transformative power of gene therapies, our aim is to fill their unmet medical needs with one-time therapies that deliver lifelong benefits.

Axovant is an agile organization with a sharp focus on the rapid delivery of lasting, transformative gene therapies. Our decision-making is guided by our compassion for patients and their urgent need for better solutions. To that end, we approach gene therapy development by selecting the product candidates that have great potential for transformative impact. We continue to tailor these therapies to precisely address the needs of the diseases and patients we serve. Our agile operations and leading manufacturing partnerships enable us to meet our goals for rapid clinical execution.

We have assembled a leadership team of the foremost experts in gene therapy development, manufacturing and commercialization. Our growing team is committed to answering the call of patients and has proven expertise in driving scientific breakthroughs into the clinic, navigating the regulatory environment, and ensuring patients have access to life-saving therapies. For us, every day matters because every life matters.

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About Us | Axovant Gene Therapies

April 30, 2019

Children born with a rare genetic disorder called X-linked severe combined immunodeficiency (X-SCID) dont have a functioning immune system. As a result, they cant fight off infections. Without treatment, an infant with X-SCID will usually die within the first year or two of life.

The best option for treatment of newly diagnosed infants with X-SCID has been stem-cell transplantation from a genetically matched sibling. But less than a quarter of children with X-SCID have a matched donor available. For those without a matched donor, standard treatment has been a half-matched bone marrow transplant from a parent. But most infants receiving this type of transplant only have part of their immune system, called T lymphocytes, restored. These infants will need lifelong injections of protective antibodies. In addition, as they grow into young adulthood, they may have chronic medical problems that affect growth, nutrition, and quality of life.

To develop a better approach to fix the immune systems of children with X-SCID, researchers have used gene therapy to alter patients own blood stem cells. An engineered virus brings a healthy copy of the gene into the stem cells to replace the mutated gene that causes the disease.

Early results from trials of gene therapy for X-SCID resulted in life-saving correction of T lymphocytes. But similar to bone marrow transplant from a parent, the immune restoration was incomplete. In addition, in those first gene therapy studies, almosta third of the children developed leukemia. The approach accidentally stimulated cells to grow uncontrollably. In later studies, improved design of the engineered virus didnt cause cancer, but also didnt fully restore a healthy immune system.

In 2010, Dr. Harry Malech of NIHs National Institute of Allergy and Infectious Diseases (NIAID) and Dr. Brian Sorrentino of St. Jude Childrens Research Hospital reported a new and safer version of gene therapy for X-SCID. They designed a harmless engineered virus (called a lentivector) that could deliver genes into cells without activating other genes that can cause cancer. Before the altered stem cells were returned to their bodies, patients were given low doses of the chemotherapy drug busulfan. This made it easier for the new stem cells to grow in the bone marrow. In young adults and children treated at the NIH Clinical Center, the new therapy proved to be both safe and effective at restoring the full range of immune functions.

Based on this work, a team led by Dr. Ewelina Mamcarz of St. Jude Childrens Research Hospital began treatment in 2015 of newly diagnosed infants with X-SCID using the lentivector and busulfan. The work was funded in part by NHLBI. The team described the treatment of eight infants with the disorder on April 18, 2019, in the New England Journal of Medicine.

By 3 to 4 months after infusion of the repaired stem cells, 7 of the 8 infants had normal levels of multiple types of immune cells in their blood. The last infant required a second stem-cell infusion, after which his immune-cell levels rose to a normal range.

The infants new immune systems were able to fight off infections that the researchers had detected before the gene therapy. Four of the eight discontinued immune-system boosting medications that theyd previously needed. Of those four, three developed antibodies in response to vaccination, indicating a fully functional immune system.

A year and a half after gene therapy, all children were healthy and growing normally.

The broad scope of immune function that our gene therapy approach has restored to infants with X-SCID as well as to older children and young adults in our continuing study at NIH is unprecedented, Malech says.

The researchers will continue to follow the participants over time. They plan to track how the childrens immune systems develop and look for any late side effects.

References:Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408. PMID: 30995372.

Funding:NIHs National Institute of Allergy and Infectious Diseases (NIAID); National Heart, Lung, and Blood Institute (NHLBI); and National Cancer Institute (NCI); American Lebanese Syrian Associated Charities; California Institute of Regenerative Medicine; and Assisi Foundation of Memphis.

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Gene therapy reverses rare immune disorder | National ...

Homology Medicines gene therapy approach utilizes our proprietary AAVHSC vectors to deliver a functional gene to a cell where there is a missing or mutated gene. Once delivered, the functional gene may lead to therapeutic protein expression. With gene therapy, the genes do not integrate into the genome so this approach can be curative in slow- or non-dividing cells (e.g., adult liver or central nervous system).

Our gene therapy construct includes a functional copy of the gene and a promotor sequence that is designed to enable the gene to be turned on in the cell and ultimately transcribed to express a therapeutic protein without integrating into the genome.

Our unique vectors have demonstrated significant systemic biodistribution to multiple tissue types in preclinical studies, including liver, central nervous system (CNS), muscle (skeletal and cardiac) and eye*. This enables us to potentially address a broad range of monogenic diseases.

Our lead development program is an AAVHSC-mediated gene therapy treatment for adults with the rare disease phenylketonuria. Learn more about our pipeline and therapeutic focus.

*Homology data on file; Ellsworth JL, Smith LJ, Rubin H, et al. Widespread transduction of the central nervous system following systemic delivery of AAVHSC17 in non-human primates. American Society of Gene & Cell Therapy Annual Meeting. May 2017.

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Gene Therapy : Homology Medicines

Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.

A gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they can't cause disease when used in people. Some types of virus, such as retroviruses, integrate their genetic material (including the new gene) into a chromosome in the human cell. Other viruses, such as adenoviruses, introduce their DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome.

The vector can be injected or given intravenously (by IV) directly into a specific tissue in the body, where it is taken up by individual cells. Alternately, a sample of the patient's cells can be removed and exposed to the vector in a laboratory setting. The cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the vector will make a functioning protein.

Researchers must overcome many technical challenges before gene therapy will be a practical approach to treating disease. For example, scientists must find better ways to deliver genes and target them to particular cells. They must also ensure that new genes are precisely controlled by the body.

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How does gene therapy work? - Genetics Home Reference - NIH