News

Managed Healthcare Executive1d
Gene Therapy Shows Promise for Fatal Heart Condition Tied to Friedreich Ataxia
Lexeo Therapeutics’ gene therapy provides a healthy version of the frataxin gene, which is critical for energy production in ...
Applied Clinical Trials Online2d
The Hard Truth About Rare Disease and Gene Therapy Drug Development
Since the Orphan Drug Act went into effect in 1983, more than 600 drugs have been approved for rare diseases, according to ...
Labiotech.eu2d
Cystic fibrosis: is the next blockbuster a gene therapy?
Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...
Fierce Biotech9d
Atsena raises $150M for ocular gene therapies with Bain, Sofinnova in tow
The $150 million will also go toward Atsena’s preclinical pipeline and expand the use of the biotech’s spreading AAV.SPR ...
11 Best Gene Therapy Stocks to Buy According to Analysts
Here is our list of the 11 Best Gene Therapy Stocks to Buy According to Analysts.
BioSpace16d
AccurEdit Therapeutics’ LNP-based In Vivo Gene Editing Product ART001 Obtained Orphan Drug Designation from the FDA
CAMBRIDGE, Mass., March 25, 2025 /PRNewswire/ -- AccurEdit Therapeutics today announced that its innovative in vivo gene editing product, ART001, has obtained Orphan Drug Designation (ODD) from the ...
Le Lézard17d
AccurEdit Therapeutics' LNP-based In Vivo Gene Editing Product ART001 Obtained Orphan Drug Designation from the FDA
CAMBRIDGE, Mass., March 25, 2025 /PRNewswire/ -- AccurEdit Therapeutics today announced that its innovative in vivo gene editing product, ART001, has obtained Orphan Drug Designation (ODD ...