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.
Gene Therapy For Inherited Disease In The Unborn Child
In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...
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 ...
STAT7h
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 Dive8h
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 ...
Gene Therapy For Inherited Disease In Infants
As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...
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 ...
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 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 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 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 ...
Science News3d
Personalized gene editing saved a baby, but the tech’s future is uncertain
The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.