News

GEN - Genetic Engineering and Biotechnology News6d
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 ...
The National on MSN3h
Emirati man with rare blood disorder benefits from new $2 million gene therapy in the US
An Emirati man with a rare inherited disorder has become only the second patient in the world to benefit from a pioneering $2 ...
Pharmaceutical Technology5h
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.
STAT5d
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 Dive5d
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 ...
FDA Will Use AI to Approve Drugs and Devices Faster
Learn more about the generative AI technology that will be used at the Food and Drug Administration (FDA) to speed up reviews ...
pharmaphorum1d
SpliceBio gathers $135m for eye disease gene therapies
SpliceBio's lead in-house pipeline project is in Stargardt disease, an IRD with no approved therapies that is caused by ...
Healio1d
VG801 gene therapy in Stargardt disease receives rare pediatric disease designation
This is a developing news story. Please check back soon for updates. The FDA granted VG801 rare pediatric disease designation ...
Zacks Investment Research on MSN7d
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 ...
BioPharma Dive7d
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 ...
Labiotech.eu1d
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 ...
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.