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Town Hall

Event Title
OTAT Town Hall: Clinical Development of Gene Therapy Products for Rare Diseases
February 7, 2023

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) hosted 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 was part of a series to answer questions from stakeholders about a variety of topics on which OTAT has regulatory oversight.

View the recording for the OTAT Town Hall on Clinical Development of Gene Therapy Products for Rare Diseases.

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.

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.

View previous OTAT Town Halls

•    September 2022: Gene Therapy Chemistry, Manufacturing, and Controls
•    December 2022: Cell Therapy Chemistry, Manufacturing, and Controls

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