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USFDA Guidance: Leveraging Prior Knowledge in Genome Editing Gene Therapy Development
As genome editing technologies continue to advance, sponsors are increasingly developing innovative gene therapies targeting rare, serious, and life-threatening diseases. These programs often involve complex manufacturing processes, extensive nonclinical studies, sophisticated analytical testing, and lengthy clinical development timelines. To support more efficient development pathways, the FDA released the draft guidance, Leveraging Prior Knowledge in the Development of Huma
Sharan Murugan
Jun 63 min read
USFDA Guidance: Safety Assessment of Genome Editing in Human Gene Therapy Products Using NGS and Expanded Access to Investigational Drugs for Treatment Use-Q&A
The regulatory landscape for advanced therapies and patient access in the United States continues to evolve, with the FDA issuing updated guidances in April 2026 that address both cutting-edge genome editing safety and patient access to investigational treatments . Together, these guidances highlight the balance between innovation, safety evaluation, and ethical access to therapies across the product lifecycle. The guidance Safety Assessment of Genome Editing in Human Gene
Sharan Murugan
Apr 203 min read
USFDA Guidance: Human Gene Therapy Products Incorporating Human Genome Editing & Development of Chimeric Antigen Receptor (CAR) T Cell Products
On Monday (29 January 2024) the U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation released two...
Sharan Murugan
Jan 31, 20242 min read