This regulatory playbook is designed with usability in mind to ensure it is easy to navigate regardless of your level of expertise. It is organized into sections that cover specific aspects of AAV gene therapy development to take you from your first pre-clinical studies all the way through to IND submission. The BGTC’s goal is to provide you with a workflow and guidance for your regulatory submissions to ensure seamless execution and increase your candidate’s likelihood of IND acceptance.
Now, consider the regulatory process as a “mountain,” and this playbook as your most trusted “navigator” to get you to the summit – “Study may proceed”! See the figure below.
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Role of the BGTC Playbook
This playbook provides detailed guidance on the FDA’s regulatory requirements, best practices, and key considerations for successful AAV gene therapy development. You will find tips for preparing and conducting various FDA meetings, as these are key milestones in your development path.
We have also included considerations to inform your clinical trial design, rare disease patient engagement, as well as study site management, IND maintenance and long-term follow-up. These aspects are particularly challenging when pursuing ultra-rare diseases and so we have included some industry considerations to support your planning of these components.
For easier filing, we have created a portfolio of templates focused on AAV gene therapies which are annotated with industry expertise and SME guidance from members across the BGTC. These templates will be plug-and-play, and you will be able to utilize them for the different sections as we will highlight throughout this playbook. Don’t worry, we will tell you when to use which template and link these templates within the playbook as well!
Now, we’ll get into a high-level overview of what you can expect in each section of this playbook. Click on the sections in the figure below to skip to the specific content and choose your own adventure!
Playbook sections
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Additional sections
Overall, this playbook is designed to be an essential tool for the AAV gene therapy community in streamlining the navigation of the US regulatory landscape. Please note, while the playbook is focused on a successful submission to the US FDA, sponsors should also communicate with regulatory agencies besides the US, where they consider conducting their clinical trial(s). We have designed the playbook to be flexible and adaptable to the specific needs of your asset. By following the guidance and best practices outlined in the playbook, you can address the safety, efficacy, and regulatory compliance of your gene therapies in development, while also doing your part to help advance the field of bespoke gene therapy.