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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
USFDA : CMC Guidance Reformulating Drug Products That Contain Carbomers & Potency Assurance for Cellular & Gene Therapy Products
The U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research...
Sharan Murugan
Dec 30, 20232 min read
USFDA Guidance: Manufacturing Changes & Comparability for CGT Products, Good Cause for Noncompliance
Last Week (13th & 14th July 2023) the United States Food & Drug Administration's Center for Drug Evaluation and Research, and Center for...
Sharan Murugan
Jul 15, 20232 min read
ICH/ USFDA Guidance: S12 Nonclinical Biodistribution Considerations for Gene Therapy Products
On 27th April, 2023 the United States Food & Drug Administration Center for Drug Evaluation and Research, and Center for Biologics...
Sharan Murugan
Apr 29, 20232 min read
USFDA Guidance: Multiple Biologics related Guidance - M10 Bioanalytical Method Validation
This week USFDAs Center for Biologics Evaluation and Research updated and released multiple guidances related to Gene therapy, Oncology,...
Sharan Murugan
Nov 4, 20221 min read
USFDA Guidance: Multiple Endpoints in Clinical Trials & Gene Therapy for Neurodegenerative Diseases
Earlier on 21 October 2022, the USFDA released two finalized guidances "Multiple Endpoints in Clinical Trials" and "Human Gene Therapy...
Sharan Murugan
Oct 23, 20221 min read
FDA’s finalized “Interpreting Sameness” Gene Therapy Products Guidance
USFDA on 29 September 2021, finalized "Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations". This guidance...
Sharan Murugan
Oct 3, 20211 min read