News

Kennedy says ‘charlatans’ are no reason to block unproven stem cell treatments
The HHS secretary said people should have access to experimental therapies including unregulated uses of stem cells. But some ...
STAT11h
FDA reassures rare disease advocates that ‘being flexible’ is plan for gene therapy
Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...
BioPharma Dive12h
FDA meeting gives window into gene therapy field’s angst
Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...
Precision Medicine Online12h
RFK Jr., FDA Officials Voice Support for Gene and Cell Therapies, Vow Flexibility in Rare Diseases
Trump administration health officials assured drugmakers and researchers at a roundtable hosted by the FDA that they want to ...
Precision Medicine Online13h
FDA Grants Platform Technology Designation to Viral Vector Used in Sarepta's LGMD Gene Therapy
Sarepta is the first company to publicly disclose that it's received the designation, for which the FDA had issued a draft guidance last year.
BioSpace16h
RFK Jr. Joins Deputies in Voicing Support for Regulatory Flexibility for Cell and Gene Therapies
In a roundtable event on Thursday, HHS Secretary Robert F. Kennedy Jr. said his office will work to eliminate barriers that ...
GEN - Genetic Engineering and Biotechnology News1d
Cell and Gene Therapy Leaders Tell FDA: “Believe in American Solutions”
Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...
The FDA granted Sarepta’s rAAVrh74 Viral Vector a Platform Technology Designation
The FDA has designated the viral vector rAAVrh74, which Sarepta Therapeutics, Inc. (NASDAQ:SRPT) uses in its gene therapy ...
Fierce Biotech1d
As the investor pendulum swings away from cell therapy, Immatics CEO focuses on raising the bar
As favor in the general market swings away from cell therapy, Immatics’ CEO is choosing to focus on what he can control: ...
EurekAlert!1d
Research alert: Alzheimer's gene therapy shows promise in preserving cognitive function
By reprogramming brain cells, a new gene therapy approach for Alzheimer's developed by UC San Diego researchers could address ...
Baby released from Philadelphia hospital after receiving personalized gene editing therapy
After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...
Baby released from CHOP after receiving personalized gene editing therapy
After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...