News

BioSpace2d
As Gene Therapy Endures ‘Cold Winter,’ FDA Leaders Promise Support
Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the ...
The Manila Times1h
Medera and Novoheart Presented Breakthroughs in Human mini-Heart Platforms and Gene Therapy at ISSCR 2025
Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases ...
Pharmaceutical Technology on MSN1d
FDA approves Capsida’s CAP-003 gene therapy for Parkinson’s
The FDA has approved Capsida Biotherapeutics' investigational new drug (IND) application for CAP-003 to treat Parkinson’s ...
GEN - Genetic Engineering and Biotechnology News7d
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 ...
BioPharma Dive6d
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 ...
Labiotech.eu2d
Can gene therapy treat vision loss?
Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...
Pharmaceutical Technology on MSN1d
SpliceBio secures $135m for gene therapy development
SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007 for Stargardt disease.
STAT23h
FDA clears Capsida gene therapy for clinical trials
Health secretary Robert F. Kennedy Jr. yesterday appointed a new, smaller vaccine advisory committee at the CDC — just two ...
BioPharma Dive8d
FDA to use new review tool on Sarepta’s gene therapy work
The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...
BioSpace7d
Sarepta Wins FDA’s Platform Technology Designation, Marking ‘Critical Shift’ in Gene Therapy Development
The Platform Technology Designation, which predates the current FDA leadership, is designed to streamline the drug ...
STAT6d
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 ...
FDA Grants Platform Technology Tag to Sarepta's Gene Therapy Vector
With FDA platform technology designation for its viral vector, SRPT can fast-track other therapies using the same ...