News

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 ...
Falmouth teen begins human gene therapy for sickle cell. 'High risk and it's scary'
A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.
BioSpace17h
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 ...
BioPharma Dive2d
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 ...
BioSpace1d
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 ...
Zacks Investment Research on MSN1d
FDA Grants Platform Technology Tag to Sarepta's Gene Therapy Vector
Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, ...
Zacks Investment Research on MSN2d
BEAM Up on FDA's Orphan Drug Status for Gene-Editing Therapy
Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing ...
Baby saved by gene-editing therapy 'graduates' from hospital, goes home
KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...
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 ...
STAT2d
After groundbreaking CRISPR therapy, a homecoming
After 307 days at CHOP, KJ was dressed in what early studies suggest are the world’s smallest and cutest graduation ...