October 27, 2023 marked the second anniversary of the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC), another year in which public and private partners continued their groundbreaking efforts to make gene therapy more accessible by creating a platform approach to deliver novel therapies for many different rare genetic disorders.

Please see the adjoining timeline for a few highlights from year two.

Breaking: BGTC Accepting New Members!

The AMP® BGTC is now welcoming new members to apply to join the consortium! Apply to join now and contribute to a brighter, healthier future! The deadline for verbal commitments is January 31st, 2024. For additional information, please contact Courtney Silverthorn, Associate Vice President, Science Partnerships, Translational Science and Director, Accelerating Medicines Partnership Program.

Year Two Highlights
  • February 28 – The BGTC team celebrated Rare Disease Day, premiered two brand new videos (see below), and participated in Rare Disease Day activities at NIH

  • May 16 – The BGTC announced its initial clinical trial portfolio on the first day of the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Los Angeles, CA; the portfolio consists of eight diseases: Charcot-Marie-Tooth disease type 4J, congenital hereditary endothelial dystrophy, Morquio A syndrome, multiple sulfatase deficiency (MSD), NPHP5 retinal degeneration, propionic acidemia (PCCB), retinitis pigmentosa 45 and spastic paraplegia 50

  • May 20 – The BGTC hosted its first annual in-person meeting on the final day of the ASGCT annual meeting, giving conference attendees the chance to learn more about the program and participate in a Q&A session with consortium steering committee members

  • Summer – The BGTC premiered three videos that explored the consortium’s participation in the ASGCT annual meeting; the videos in particular highlighted the clinical trial portfolio announcement and the first annual meeting

Year Two Videos & Other Media Highlights

In its second year, the BGTC team published several videos promoting the ongoing work of the consortium, touching on the goals of the program and AAV technology upon which the program’s platform is based. Most importantly, the videos aimed to highlight the patients and families the BGTC strives to support with its research.

After the ASGCT annual meeting in May, the consortium also shared three videos recapping the events of conference. These videos included the announcement of the program’s initial cohort of rare diseases to be investigated in upcoming clinical trials as well as the BGTC’s first annual meeting on the final day of the conference.

As the BGTC moves into year three, these videos showcase not only the work that has already been accomplished, but preview the important work to come.

BGTC Overview Short Film
AAV Animation
ASGCT Video Playlist

As a result of the clinical trial portfolio announcement in May, the BGTC received extensive media coverage, highlighting the portfolio and the potential breakthroughs it promises for rare disease patients.

The announcement was covered by publications such as USA Today, Yahoo!News, Fierce Biotech, Endpoints News, RareDiseaseAdvisor, and BioCentury, to name a few. The news also resulted in a podcast interview with Xtalks Life Science, featuring FNIH’s Courtney Silverthorn.

AAV Biology Subteam Updates

To enhance the fundamental knowledge of AAVs, BGTC released two Request for Proposals (RFPs) – one focused on investigating AAV gene expression/transduction biology and the second on investigating and/or developing ways to improve recombinant AAV vector production. Through these funding mechanisms, BGTC is supporting eight discovery research projects, six on gene expression/transduction biology and two on vector production. The knowledge generated from these projects will be publicly available and will serve as a foundation to overcome key bottlenecks in AAV gene therapy development.

Pre-Clinical Subteam Updates

The Pre-Clinical Subteam continued their work this year focusing on two major objectives.  First, they evaluated the non-clinical portions of the proposals for BGTC support, summarizing how much proof-of-concept data had been generated to date and forwarding their perspectives of the programs to the Clinical Subteam.  Second, the team drafted a briefing book to the FDA in support of identifying a minimally acceptable IND-enabling toxicology program that would eventually accompany each of the core programs at submission. A meeting was held with FDA on June 27th with active dialogue between BGTC and FDA leading to guidance on general approaches we could take with each program.  The Pre-Clinical Subteam looks forward to working with the Coordination Center and sponsors of each program in the coming year to design and implement nonclinical packages for the initial clinical trials to be conducted.


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