Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC)

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

• Concept plan
• Design Phase Working Group participants
• Program Plan and Partnering Webinar video
• Program Plan and Partnering Webinar slides

Guidance Documents 

• Current FDA CBER Cellular & Gene Therapy Guidances

Media

Interview with Courtney Silverthorn (April 27, 2022): Foundation for the National Institutes of Health Accelerates Biomedical Research and Strategies Around the World, AscellaHealth Developments in Specialty Pharmacy 

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 

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