News Medical

SP-101 gene therapy shows promise for cystic fibrosis treatment

Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis (CF) in the peer-reviewed journal Human Gene Therapy. They ...
Benzinga.com

Gene Therapy Developer Beam Therapeutics Reports One Patient Death In Early-Stage Sickle Cell Disease Trial

On Tuesday, Beam Therapeutics Inc. (NASDAQ:BEAM) announced that 35 patients have cleared screening and enrolled in the BEACON Phase 1/2 trial of BEAM-101, an investigational genetically modified cell ...
Business Insider

Atsena Therapeutics Receives Rare Pediatric Disease Designation from FDA for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)

DURHAM, N.C., Jan. 16, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent ...
Business Wire

AviadoBio Announces Presentation of Promising Preclinical Safety and Biodistribution Data of its Gene Therapy Candidate AVB-101 for FTD-GRN at the Annual European Society of ...

LONDON--(BUSINESS WIRE)--AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, announced today the ...