ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced that the U.S. Food and ...

ATSN-201 was given regenerative medicine advanced therapy designation to treat X-linked retinoschisis, for which there ...

This blog delves into the rise of rare disease drug approvals and the critical importance of regulatory guidance in shaping ...

Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...

TLX101 therapy was efficacious in recurrent high-grade glioma, a type of brain cancer, according to preliminary results from ...

Lexeo Therapeutics’ gene therapy provides a healthy version of the frataxin gene, which is critical for energy production in ...

Odylia Therapeutics event brings together leaders across biotech, pharma, venture capital, patient advocacy, and research ...

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 ...

Atsena Therapeutics has completed an oversubscribed Series C financing round, raising $150m to support the advancement of ...

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 ...

Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the U.S. Food and Drug ...

Durham-based Atsena Therapeutics has raised $150 million to advance gene therapies for preventing or reversing types of ...