FDA Biologics

⏱️REdI 2026 is approaching! Don't miss your opportunity to learn from FDA experts across all three medical product centers. Build your regulatory knowledge in drugs, devices, and biologics. 🗓️ Mark your calendar and register today ➡️ fda.gov/news-events/re…

This week, @FDACBER and @FDA_Drug_Info held an all-hands discussion on FDA's Plausible Mechanism Framework, featuring expert guests from @Penn and @broadinstitute. Topics included an update on Baby KJ, whose personalized CRISPR therapy inspired the framework, and researcher experiences with the IND & approval process. Watch the recording here: youtube.com/live/1OdI5_ls7…

⏱️REdI 2026 is approaching! Don't miss your opportunity to learn from FDA experts across all three medical product centers. Build your regulatory knowledge in drugs, devices, and biologics. 🗓️ Mark your calendar and register today ➡️ fda.gov/news-events/re…

We will also discuss an update on Baby KJ, whose personalized CRISPR-based gene-editing therapy for a severe urea cycle disorder inspired the Plausible Mechanism Framework, and share researcher experiences on IND clearance and the FDA approval process.

The program will include special guests Rebecca Ahrens-Nicklas, MD, PhD, University of Pennsylvania, Lindsey A. George, MD, University of Pennsylvania, Kiran Musunuru, MD, PhD, University of Pennsylvania, and Sonia Vallabh, PhD, Broad Institute of MIT and Harvard.

Join FDA via livestream for a discussion on the plausible mechanism guidance and its applicability to both CDER and CBER products: youtube.com/live/1OdI5_ls7…

Today FDA approved the first-ever gene therapy for the treatment of genetic hearing loss under the National Priority Voucher Program. ✅6th approval under the National Priority Voucher Program ✅Approved 61 days after filing fda.gov/news-events/pr…

New phenol-free formulation promises field deployment for disease surveillance and vaccine trials, eliminating cold-chain requirements for neglected tropical disease affecting 90+ countries. sciencedirect.com/science?_ob=Ga…

This guidance will provide sponsors with clear, scientifically grounded recommendations to bring effective treatments to patients sooner.

Today, the FDA issued a draft guidance that when finalized, will provide recommendations for standardized methods for sponsors developing human gene therapy products that incorporate genome editing technologies. fda.gov/news-events/pr…

⏱️REdI 2026 is approaching! Don't miss your opportunity to learn from FDA experts across all three medical product centers. Build your regulatory knowledge in drugs, devices, and biologics. 🗓️ Mark your calendar and register today ➡️ fda.gov/news-events/re…

Today, the FDA issued a draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations. fda.gov/news-events/pr…

To further expedite product development, the FDA announced a more flexible approach to the agency’s oversight of chemistry, manufacturing and control requirements for cell and gene therapies. fda.gov/news-events/pr…

Today, the FDA approved the first cell-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS). fda.gov/news-events/pr…

FDA approved the first hematopoietic stem cell transplant (HSCT) therapy to treat patients with severe aplastic anemia (SAA). Learn more: fda.gov/news-events/pr…

Today, the FDA approved the first Chimeric Antigen Receptor (CAR) T-cell therapy in the U.S. for treatment of adults with marginal zone lymphoma (MZL) who have failed treatment with or relapsed after two or more prior lines of therapy. fda.gov/news-events/pr…

Today, the FDA approved a nerve scaffold for the treatment of sensory nerve discontinuity in adults and pediatric patients aged one month and older. The treatment has also been approved for larger sensory nerve, motor, and mixed nerve discontinuities. fda.gov/news-events/pr…

Step into the forefront of novel endpoint development for rare disease drug development! CBER invites you to submit a proposal to the rare disease endpoint advancement (RDEA) Pilot Program, a collaboration with @FDA_Drug_Info. Deadline is 12/30/25! bit.ly/48UBUx4

Today, the FDA approved a gene therapy for the treatment of spinal muscular atrophy in adult and pediatric patients 2 years of age and older with confirmed mutation in the survival motor neuron 1 gene. fda.gov/news-events/pr…

Join CBER’s Office of Vaccines Research and Review (OVRR) on October 23, 2025, for up-to-date information about the diagnosis of allergic contact dermatitis using patch tests. Register: fda.gov/AllergicContac…