- February 7, 2023
- 11:30 AM - 1:00 PM ET
The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) is hosting a virtual town hall to answer stakeholder questions related to the clinical development of gene therapy products for rare diseases (excluding hematology/oncology products). This event is part of a series to answer questions from stakeholders about a variety of topics on which OTAT has regulatory oversight.
Focus for This Town Hall: Gene Therapy Products Intended to Treat Rare Diseases
There are around 7,000 known rare diseases affecting more than 30 million people in the United States. Many rare diseases are life-threatening, and only a fraction of rare diseases have an available treatment. Developing safe and effective gene therapy products to treat rare diseases can be challenging; despite these challenges, gene therapy-related research and development continues to grow rapidly. To support continued growth in the gene therapy field, the FDA developed a guidance document in January 2020 to provide recommendations to sponsors developing gene therapy products intended to treat rare diseases.
- Date: Tuesday, February 7, 2023
- Time: 11:30 a.m. to 1:00 p.m. ET
- Location: The webinar will be held via Zoom.
- Registration: Registration is required. Please register for the event now.
Submit a Question
Questions can be submitted during the registration process. Please submit questions by January 11, 2023. Attendees will also be able to submit questions during the live event.
Please note, the FDA is not able to comment on or answer questions regarding specific investigational products or drug applications during the town hall. Further, questions considered inappropriate or out of scope for the event will not be addressed.
About the OTAT Town Hall Series
OTAT launched its virtual town hall series to engage with product development stakeholders and discuss topics related to OTAT-regulated products. The town halls have a question-and-answer format with the goal of providing regulatory information to stakeholders to advance drug development. Learn more about OTAT and view our available resources.