FDA guidance could shape rare disease gene therapy development

FDA guidance could shape rare disease gene therapy development

The U.S. Food and Drug Administration (FDA) has issued draft guidance meant to help developers of human gene therapies involving genome editing bring treatments to patients more efficiently, including those with rare and life-threatening diseases. Some recommendations also may apply to other cell and gene therapy products, including adeno-associated viral vectors, or AAV vectors. While the guidance is not specific to aromatic L-amino acid decarb…