---
title: "Streamlined approaches to bespoke gene therapy development"
slug: "streamlined-approaches-to-bespoke-gene-therapy-development-1"
updated: 2025-05-12T18:22:51Z
published: 2025-05-12T18:22:51Z
canonical: "bgtcplaybook.document360.io/streamlined-approaches-to-bespoke-gene-therapy-development-1"
---

> ## 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.

# Streamlined approaches to bespoke gene therapy development

The BGTC seeks to overcome the current obstacles faced during the development of gene therapies by streamlining approaches in four different sectors:

1. Basic research
2. Clinical research
3. Manufacturing and production
4. Regulatory requirements

See below for an overview of the BGTC’s specific goals. [[1](/v1/docs/ref-intro-to-bgtc-playbook)]

![GTC logo for the AMP Bespoke Gene Therapy Consortium, with icons representing research, testing, data, and patient care](https://cdn.document360.io/a7cea773-7486-41ac-bd50-afa0255ce01f/Images/Documentation/image(79).png)

| **AAV accessibility** | Make adeno-associated virus technology (AAV) **more accessible** to a broader range of diseases - Optimized AAV vector production protocols - Improvements in AAV target gene expression |
| --- | --- |
| **Streamlined testing** | **Streamline** preclinical and product **testing** - Harmonized and validated sets of manufacturing and pre-clinical testing requirements |
| **Standardized submissions** | Facilitate **scientific and regulatory advances** that will ultimately benefit the entire field - Standardized regulatory submission package templates |
| **Timely therapy accessibility** | Bring gene therapies to all **affected populations sooner** - Clinical development manual to help advance all future AAV gene therapies for rare diseases |

This playbook is a manifestation of “streamlined testing” and “standardized submissions,” taking a comprehensive and systematic approach to AAV gene therapy development. It covers all aspects of the development process up to IND submission and first-in-human studies.

Supplementing future iterations of the playbook, we plan to also include templates and minimum requirements (e.g., manufacturing, pre-clinical, etc.) to distill a more repeatable regulatory process for AAV gene therapies (see table below). The minimum requirements as they apply to the BGTC approach will get to the core of what is considered necessary and sufficient for a safety and efficacy evidence package in your regulatory submission to the FDA. This means reducing redundancies and leveraging available information to increase efficiencies in a typically complex and inefficient process.

The BGTC approach highlights best practices and recommendations to enable you to *strike the right balance* between **meeting FDA data requirements** and **accelerating your submission** as much as possible. You may refer to the [Platform-based Approach for AAV Gene Therapies](/v1/docs/platform-based-approach-for-aav-gene-therapies) section for more information on how this approach further supports and defines what we collectively call “minimum requirements”.

### Overview of BGTC Regulatory Playbook contents

| **Templates** | Easy “**plug-and-play**” documents to check off **submission requirements** |
| --- | --- |
| **Illustrative callout boxes** | Highlights of **important information** to consider along the journey |
| **Strategic insights** | Based on **FDA guidances**, **IQVIA expertise**, and the **consortium’s work** |
| **Uniform regulatory requirements** | Established **minimum requirements** to optimize likelihood of approval |
| **Sample outlines** | **Summaries / frameworks** to construct and organize the sections |
| **Decision trees** | **Pathways / flowcharts** to guide the process to submission efficiently |
| **Do’s and Don’ts** | **Tables** summarizing ***What to include?*** and ***What not to include?*** |
| **User-friendly and interactive** | Content annotated with **icons** and **hyperlinks** to examples and external sources |

This playbook helps you prepare for each stage of development, as well as answer the question “Am I ready?” each step of the way. At key milestones, we’ve provided Readiness Assessments and guidance for applying the BGTC approach in preparing for the INTERACT and Pre-IND meetings (see [INTERACT Meeting](/v1/docs/interact-meeting) and [Pre-IND Meeting](/v1/docs/pre-ind-meeting) sections). This is intended to support you in reaching the **optimal state of** **preparation** at these milestones. We will guide you in applying one or more elements of the BGTC approach at critical timepoints throughout your regulatory journey.

Please read on to understand more about the BGTC approach as it relates to minimum pre-clinical and other requirements.

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.