Bespoke Gene Therapy Consortium (BGTC)

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The Bespoke Gene Therapy Consortium (BGTC) is a developing public-private partnership dedicated to making gene therapy a reality for people with rare genetic diseases affecting populations too small to be viable from the current commercial perspective. Building on the successful Accelerating Medicines Partnership model, this program will focus on developing an operational playbook that invokes the use of streamlined templates, master regulatory files, and uniform production processes. It is anticipated that following a pilot phase of 4-6 test cases, a pathway toward the commercial viability of these therapies will be found. This may ultimately have a tremendously positive impact on the larger field of gene therapy if it moves more broadly into the era of genome editing.

Adeno Associated Virus (AAV)

The limited access to gene therapy, especially to populations in the ultra-rare or bespoke category, has been recognized by pharmaceutical, academic, NIH and FDA leadership. Ongoing discussions to address this issue have focused on a single delivery technology, Adeno Associated Virus (AAV), and the ability to facilitate and generalize access and processes for the development of bespoke therapies. An in-depth understanding of basic AAV life cycle biology, to provide greater clarity on means to optimize vector generation and delivery, and a standardization and streamlining of regulatory requirements are the areas of greatest need and largest potential impact for the BGTC.      

Watch this video of the Program Plan and Partnering Webinar from August 26

Access the webinar slides here

Goals

  • Make adeno-associated virus (AAV) technology more accessible to a broader range of diseases;
  • Accelerate the potential to streamline preclinical and product testing;
  • Facilitate scientific advances that will ultimately benefit the entire field;
  • Bring therapies to all individuals in need sooner

Concept Documents

Concept plan
Working Group participants

Guidance Documents 

Current FDA CBER Cellular & Gene Therapy Guidances

Support

For more information about supporting this program, please contact Joanne Morse.

Design phase partners

The support and expertise of the following partners has been instrumental in the design phase of this program:

Public Sector Partners
National Center for Advancing Translational Sciences (NCATS)
National Heart, Lung, and Blood Institute (NHLBI)
National Institute of Neurological Disorders and Stroke (NINDS)
National Institute of Mental Health (NIMH)
U.S. Food and Drug Administration (FDA)

Private Sector Partners
AveXis*
Bayer*
Bristol Myers Squibb*
Boehringer Ingelheim*
Eli Lilly and Company*
Pfizer*
Roche*
Sanofi*
UCB*
Ultragenyx*  

*Provided financial or in-kind support for this project.

FNIH Contact

Steve Hoffmann, Director, Inflammation & Immunity, shoffmann@fnih.org
Joe Menetski, Associate Vice President, Research Partnerships, jmenetski@fnih.org