---
title: "Introduction to the BGTC Regulatory Playbook"
slug: "introduction-to-the-bgtc-regulatory-playbook"
updated: 2026-08-24T16:30:26Z
published: 2026-08-24T16:30:53Z
canonical: "bgtcplaybook.document360.io/introduction-to-the-bgtc-regulatory-playbook"
---

> ## Documentation Index
> Fetch the complete documentation index at: https://bgtcplaybook.document360.io/llms.txt
> Use this file to discover all available pages before exploring further.

# Introduction to the BGTC Regulatory Playbook

The AMP BGTC recognized the need for a comprehensive playbook that would serve as a guiding framework for the development and regulatory submission of AAV gene therapies for rare diseases. Building out this playbook to support the key processes up to a Sponsor’s first-in-human (FIH) trial required a collaborative and modular approach. Version 1.0 of the playbook is designed to serve as a one-stop-shop guide and roadmap to IND submission for these innovative gene therapies.

To ensure the playbook’s credibility and integrity, the BGTC embarked on an ambitious journey to consolidate external information with its own internal expertise. The goal was to generate an operational playbook that would serve AAV drug developers of all backgrounds, but primarily those who could benefit from simplified language, guidelines, and templates for this complex process (e.g., family groups, non-profits, patient foundations, academic research labs, small biotechs, but not necessarily large pharmaceutical companies).

The process for developing this playbook started with collating source documents, such as FDA regulatory guidances, publications, and other publicly available resources. These foundational guidelines were supplemented with the BGTC’s extensive expertise and experience in gene therapy regulatory processes. The initial playbook structure was framed around key milestones common to a sponsor’s typical regulatory journey, from research and development (R&D), to pre-clinical, through clinical development. The BGTC plans to enhance the playbook in future versions by incorporating learnings from standardized outputs, leveraging BGTC subject matter experts (SMEs), defining efficiencies, platform strategies, and accrued knowledge from FDA interactions. These invaluable insights formed the bedrock of the playbook, ensuring that it would encapsulate the latest advancements and emerging best practices.

As the playbook took shape, it became evident that this collaborative endeavor had the potential to revolutionize the field of gene therapy, particularly through its continued evolution, capturing learnings from the BGTC projects and advances across healthcare and drug development. Not only would it provide researchers and developers a unified resource to navigate the intricate regulatory landscape of AAV gene therapies, but it would also facilitate harmonization and streamlining among various stakeholders, including researchers, regulatory bodies, clinicians, and ultimately, benefiting patients with these rare diseases.

The BGTC Regulatory Playbook is a collective effort of the BGTC SMEs, setting the stage for a paradigm shift in rare disease gene therapy development. With the BGTC leading the charge and drawing upon the collective experience, expertise, and passion of the scientific community, this playbook aims to serve as a guiding light, enabling researchers and developers to bring safe, effective, and transformative gene therapies to patients in need.

### Acknowledgements

We would like to thank all BGTC consortium members for their valuable review and contributions to the playbook.

### BGTC Regulatory Playbook contact

For questions, email Marissa Cabay, Project Manager, Rare Diseases Translational Science at [mcabay@fnih.org](mailto:mcabay@fnih.org).

Accelerating Medicines Partnership (AMP) - A public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), multiple biopharmaceutical and life science companies, non-profit and other organizations to transform the current model for developing new diagnostics and treatments.

Bespoke Gene Therapy Consortium (BGTC) - An AMP that aims to develop platforms and standards that will speed the development and delivery of customized or 'bespoke’ gene therapies that could treat the millions of people affected by rare diseases.

Adeno-associated virus (AAV) - A member of the parvovirus family of single-stranded small DNA viruses that require a helper virus such as adenovirus or herpes simplex virus for replication. AAV vectors are the leading platform for gene delivery for the treatment of a variety of human diseases.

Person or entity who takes responsibility for and initiates a clinical investigation. The sponsor may be an individual or pharmaceutical company, governmental agency, academic institution, private organization, or other organization. Sponsor does not actually conduct the investigation unless the sponsor is a sponsor-investigator.

Investigational New Drug Application (IND) - An Investigational New Drug Application (IND) is a request for authorization from the Food and Drug Administration (FDA) to administer an investigational drug or biological product to humans.

An IND must be authorized prior to interstate shipment and administration of any new drug or biological product that is not the subject of an approved New Drug Application or Biologics/Product License Application.