What AMP BGTC Partners are Saying

Alliance for Regenerative Medicine (ARM)

“The Bespoke Gene Therapy Consortium aligns squarely with the Alliance for Regenerative Medicine's (ARM) mission: accelerating the development and delivery of transformative therapies to patients in need. Not only could this partnership help patients with ultra-rare diseases, who often have few, or no, viable treatment options, but it could also help us streamline the regulatory process for future gene therapies and provide these durable, and possibly curative, medicines to much larger patient populations.”

-Janet Lambert, Chief Executive Officer, ARM

American Society of Gene & Cell Therapy

“We’re honored and excited to be a part of the Bespoke Gene Therapy Consortium. It is integral to our mission to do all we can to help alleviate disease leveraging gene therapies regardless of commercial viability. The standardized models created by the BGTC, specifically surrounding Adeno-associated virus, represent an extraordinary opportunity to accelerate the journey of rare disease treatments from the lab to the clinic.”

-David M. Barrett, J.D., Chief Executive Officer of the American Society of Gene & Cell Therapy

Biogen Inc.

“As a pioneer in neuroscience, Biogen is applying gene therapy approaches with the goal of fighting some of the most debilitating neurological disorders. We are proud to collaborate with the Foundation for the National Institutes of Health and the Bespoke Gene Therapy Consortium to help accelerate the development of gene therapies that we hope will improve the lives of people living with rare diseases. By working together to address the genetic cause of disease, we hope this collaboration can help develop transformative medicines that may potentially prevent, halt or cure disease in the future.”

-Junghae Suh, Ph.D., Head of Biogen’s Gene Therapy Accelerator Unit


“CureDuchenne is delighted to be a partner in the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium and has great expectations for future gene therapy research and treatments emerging from this effort. The goals of this initiative have the potential to transform the field of gene therapy and align with both the vision and mission of CureDuchenne. We are committed to making gene therapy more accessible to a wider array of diseases and larger number of individuals. The learnings gained from this initiative will not only inform best practices, but also lower the barriers for future gene therapy programs.”

-Debra Miller, Founder and Chief Executive Officer, CureDuchenne

Janssen Research & Development, LLC

“Janssen is proud to participate in the AMP Bespoke Gene Therapy Consortium, which aims to streamline and enhance gene therapy development processes to help bring therapies faster to patients. This mission of advancing innovative science to meet the unmet needs of patients fully aligns with our values and our Credo.” 

-Rich Tillyer, Ph.D., Global Head, Discovery, Product Development & Supply, Janssen Research & Development

National Organization for Rare Disorders (NORD)

“The Bespoke Gene Therapy Consortium is a strong, collaborative initiative centered around patients and families. NORD is thrilled to be on the forefront of gene therapy development alongside this coalition, and we look forward to contributing to research and data collection that will truly make a difference for the nearly 30 million Americans living with a rare disorder.”

-Ed Neilan, M.D., Ph.D., Chief Scientific and Medical Officer, NORD

Pfizer Inc.

“Collectively, people living with a rare disease represent one of the largest underserved patient communities in the world. At Pfizer, we believe these patients, along with their family members and caregivers who support them, deserve better. That’s why we’re proud to collaborate in the Bespoke Gene Therapy Consortium, working in tandem with our partners to help advance this important field of research and help accelerate the discovery and development of potentially transformative therapies for patients in need.”

-Seng H. Cheng, Ph.D., Senior Vice President and Chief Scientific Officer, Rare Disease, Pfizer Inc.


“REGENXBIO is committed to improving lives through the curative potential of gene therapy. We are proud to join the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium to advance the field of AAV-gene therapy through collaboration with industry, NIH, FDA, and not-for-profit partners. We believe that by sharing expertise and insight, the learnings achieved in this partnership will ultimately further the development of therapies for patients with significant unmet needs.”

-Kenneth T. Mills, President and Chief Executive Officer, REGENXBIO Inc.

Spark Therapeutics

“Spark is proud to join the BGTC and work alongside others who share our commitment to advancing novel gene therapies for patients. Tackling rare genetic disorders and progressing the potential of gene therapy requires meaningful collaboration across communities. We commend the FNIH for establishing this important partnership, and we look forward to contributing toward productive outcomes that will help more people and families affected by rare and ultra-rare diseases.”

-Gallia G. Levy, M.D., Ph.D., Chief Medical Officer, Spark Therapeutics

Takeda Pharmaceutical Company Limited

“Takeda is committed to developing gene therapies that can provide functional cures to patients while enabling broader geographic access to ensure more patients can benefit from these potentially transformative treatments. The Bespoke Gene Therapy Consortium offers an unprecedented opportunity to advance the development of safe and effective gene therapies for patients with ultra-rare orphan diseases. We hope our expertise in global drug development will contribute to the Consortium’s goal to build an efficient development, manufacturing and regulatory framework for bespoke gene therapy drug discovery efforts.”

-Vivian Choi, Head of Global Gene Therapy Research, Takeda

Taysha Gene Therapies

“Taysha is proud to participate in the Bespoke Gene Therapy Consortium to help accelerate the development of gene therapies for the treatment of rare diseases through collaboration with NIH, FDA, and leading industry and non-profit partners. We believe this consortium can leverage each partners’ insights and experience to improve upon the development process for gene therapies and we are excited to contribute to this initiative. We are looking forward to working with other partners and collaborators through the BGTC to help patients and families afflicted by rare and severe conditions.”

-Suyash Prasad, MBBS, MSc, MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development, Taysha Gene Therapies

Thermo Fisher Scientific 

“Thermo Fisher Scientific is proud to support this important national initiative. This public-private partnership will standardize viral vector manufacturing and make gene therapies more accessible to patients in need.”

-Richard Snyder, Vice President, Science and Technology, Pharma Services, Thermo Fisher Scientific

Ultragenyx Pharmaceutical

“At Ultragenyx, we recognize both the life-changing potential that one-time gene therapies can have on people with rare diseases and the challenges associated with developing and manufacturing these innovative therapies. That’s why we are participating in the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium. As the goal of this partnership aligns with our mission to deliver safe and effective new treatments to patients with rare diseases, we look forward to collaborating with our peers and sharing our experience and insights in gene therapy in order to streamline the overall process and bring new bespoke gene therapies to patients with rare genetic diseases faster, safer and more efficiently.”

-Reed Clark, Ph.D., Senior Vice President of Research, Ultragenyx Gene Therapy

This page last reviewed on October 29, 2021