Overview

October 27, 2024 marked the third anniversary of the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC), another year in which public, private, and patient partners continued their 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 three.

 

“It is truly heartening to see the amount of time, thought, and experience that BGTC members contribute to this program, especially considering that many of those involved are serving as volunteers, in addition to their “day jobs”. I suspect that this is due to the unique focus of the BGTC mission on streamlining the pathway to bringing potentially high impact therapies to patients for diseases of no (current) commercial interest. Many thanks to all of the BGTC members for their time and effort.”

 

P.J. Brooks, PhD
Deputy Director, Division of Rare Diseases Research Innovation
National Center for Advancing Translational Sciences (NCATS)

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Year Three Highlights

  • February 6 – The BGTC team announced the publication of the first version of a regulatory playbook, providing a roadmap for streamlining product development and navigation of the regulatory pathway for AAV gene therapies, with the goal of getting new treatments to patients sooner

  • February 29 – Attended Rare Disease Day at the NIH

  • May 7-11 – Numerous members of the BGTC attended the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting in Baltimore, Maryland; the FNIH and many of its partners held booths in the exhibit hall, and several key members of BGTC leadership presented on panels and fireside chats throughout the weeklong meeting

  • May 8 – The BGTC hosted its second annual in-person meeting during 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; the audience participated in an engaging feedback session regarding the regulatory playbook, and FNIH and BGTC patient ambassadors shared their stories and their determination in finding effective therapies for rare diseases

  • November – The BGTC conducted the first of many roundtable discussions with one of the BGTC rare disease patient organizations, to learn how to best serve and engage with the priorities of the patients they represent

Year Three Videos & Other Media Highlights

At this year’s ASGCT annual meeting in May, several consortium members were interviewed about the first version of the regulatory playbook, and also shared progress updates on the BGTC’s clinical trial portfolio. The resulting videos highlight the current status of the program, but also stress the fact that there is much work to be done for patients that are waiting for effective and safe gene therapies.

ASGCT 2024 Recap
 BGTC Regulatory Playbook
 ASGCT Video Playlist

As a result of the regulatory playbook announcement in February, the BGTC received extensive media coverage, highlighting the streamlined pathways and downloadable templates it promises for AAV gene therapy development and approval.

The announcement was covered by publications such as BioWorld, Noah News, RARE Daily, CRISPR Medicine News, and AgencyIQ.

In addition, Nature Reviews Drug Discovery published a commentary about the BGTC the same week the regulatory playbook was announced.

2024 Impact of BGTC

Regulatory Approvals – CMT4J IND submission received safe to proceed notice (December 2024)

Sustainability – All BGTC portfolio diseases submitted and received Orphan Drug and Rare Pediatric Disease Designations

Regulatory Dissemination – Published v1 of the BGTC Regulatory Playbook, a guide for navigation of the AAV regulatory pathway, including downloadable templates

Clinical Protocols – Development of standardized immunosuppression protocols for ocular, neurological, and systemic AAV clinical trial protocols

 

AAV Subteam Updates

The AAV Biology projects currently supported by the BGTC have had a productive year, meeting a total of six project milestones and deliverables. Thank you to all the project investigators and AAV Biology subteam members for driving the fundamental science to help improve AAV production and transduction properties.

Contact
Brad Garrison, MBA, PMP

Senior Project Manager, Translational Science

[email protected]
Kira Gillett, MS

Program Manager, Translational Science

[email protected]
Megan Watts, MPH

Associate Project Manager, Cell and Gene Therapy

[email protected]

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