News

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.
Sickle cell gene therapy offers hope to N.J. teen
New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...
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 ...
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 ...
BioPharma Dive19h
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 ...
STAT18h
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 ...
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 ...
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, ...
BioSpace1d
uniQure’s Regulatory Progress Sparks Optimism for ‘Desperate’ Huntington’s Space
Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on ...
PharmiWeb3d
Navigating Cell and Gene Therapy Access Challenges
Cell and gene therapy (CGT) represents the pinnacle of biomedical innovation, offering unprecedented potential to treat, ...