Bespoke Gene Therapy Consortium (BGTC)

The Problem
With the current commercial drug development model, companies cannot recover the costs required to develop gene therapies to treat rare and ultra-rare genetic diseases, because these diseases affect relatively few patients.
The Solution
This project will create tools to streamline the gene therapy development process, aiming to reduce associated costs and encourage companies to pursue gene therapies for rare genetic diseases.

 

Overview

The Accelerating Medicines Partnership® (AMP®) Bespoke Gene Therapy Consortium (BGTC) is the sixth AMP program and the first to focus on a therapeutic platform.  The AMP BGTC brings together partners from the public, private, and non-profit sectors to foster development of gene therapies intended to treat rare genetic diseases, which affect populations too small for viable commercial development. Building on the successful AMP model, this program focuses on generating a standard operational playbook for developing such gene therapies. This playbook, which will be established and piloted using four-to-six clinical trial test cases, will include streamlined templates, master regulatory files, and uniform manufacturing processes to create a pathway toward the commercial viability and sustainability of gene therapies for very rare diseases. This approach could have substantial positive impacts on the larger gene therapy field, especially as the field moves into the era of genome editing.

Increased demand for gene therapy manufacturing, and limitations in the development and regulatory approval process, have led to a bottleneck in delivering these important new therapies to patients. Unfortunately, there is a lack of scalability, reproducibility, or regulatory generalizability in the current environment, which makes access to these therapies inefficient for most patients.

The issues include determining the quantity of supportive preclinical evidence needed prior to patient treatment, understanding the clinical information that should be captured, and producing a quality product that is fit-for-purpose – all in the context of an appropriate regulatory paradigm that facilitates safe patient treatment with a high likelihood of effectiveness.

To address this unmet medical need the NIH, the FDA, and a wide range of industry and nonprofit partners have joined together to launch the BGTC, which is coordinated by the FNIH. The BGTC focuses on a single gene delivery technology, adeno-associated virus (AAV), one of the safest platforms for gene delivery to address a variety of human diseases. The program’s primary aims include pursuing an in-depth understanding of basic AAV life cycle biology, which will facilitate optimization of vector generation and delivery, as well as standardizing and streamlining regulatory requirements for approval of gene therapies for ultra-rare and bespoke diseases.

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 and regulatory advances that will ultimately benefit the entire field; 

  • Bring gene therapies to all individuals in need sooner

Design Phase Documents

Guidance Documents 

Committed Partners

Public-Sector Partners:

  • National Center for Advancing Translational Sciences (NCATS)
  • National Institute for Neurological Disorders and Stroke (NINDS)
  • National Institute of Mental Health (NIMH)
  • National Human Genome Research Institute (NHGRI)
  • National Heart, Lung and Blood Institute (NHLBI)
  • National Institute of Child Health and Human Development (NICHD)
  • National Eye Institute (NEI)
  • National Institute on Deafness and Other Communication Disorders (NIDCD)
  • National Institute of Dental and Craniofacial Research (NIDCR)
  • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
  • The Brain Research Through Advancing Innovative Neurotechnologies® (BRAIN) Initiative, a trans-NIH initiative involving 10 NIH Institutes and Centers
  • U.S. Food and Drug Administration (FDA)

Private-Sector Partners:

  • Alliance for Regenerative Medicine (ARM)*
  • American Society of Gene & Cell Therapy*
  • Biogen Inc.*
  • CureDuchenne*
  • Janssen Research & Development, LLC*
  • National Organization for Rare Disorders (NORD)*
  • Novartis Institutes for BioMedical Research*
  • Pfizer Inc.*
  • REGENXBIO Inc.*
  • Spark Therapeutics*
  • Takeda Pharmaceutical Company Limited*
  • Taysha Gene Therapies*
  • The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)*
  • Thermo Fisher Scientific*
  • Ultragenyx Pharmaceutical*

*Provided financial support for this program.

Read what the partners are saying

FNIH Contact

 

Media

FNIH Press Release (October 27, 2021): FNIH Launches a Transformative Partnership to Streamline Development of Treatments for Rare Diseases 

NIH Press Release (October 27, 2021) NIH, FDA and 15 private organizations join forces to increase effective gene therapies for rare diseases 

Scientific Publications

Bespoke Gene Therapy Consortium’ sets out to enable gene therapies for ultra-rare diseasesNature Reviews Drug Discovery

Creating drug master files for AAV vectors - BioCentury

Support

For more information about supporting or becoming a partner in this program, please contact Julie Wolf-Rodda, Senior Vice-President of Development, jwolf-rodda@fnih.org.

 

ACCELERATING MEDICINES PARTNERSHIP and AMP are registered service marks of the U.S. Department of Health and Human Services.