Stuart Orkin and Swee Lay Thein shared a Breakthrough Prize in Life Sciences for their research on genetic causes of sickle ...

Three 2026 Breakthrough Prize winners reflect on developing Luxturna, a gene therapy that treats blindness caused by rare ...

Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, and ...

The agency's decision is based on results from a clinical trial in which the treatment improved hearing in 80 percent of the ...

Adeno-associated virus (AAV) has emerged as a reliable vehicle for delivering therapeutic DNA into patient cells, with eight AAV-based gene therapies approved worldwide as of mid-2025. 1 Yet questions ...

Researchers say a gene therapy allowed deaf children and adults as old as 32 to hear for the first time. The benefits have ...

CRISPR gene therapy increases haemoglobin and fetal haemoglobin in sickle cell disease, reducing vaso occlusive events in ...

The therapy is part of a plan to develop gene therapies that can be efficiently delivered at lower doses, enabling them to be used against more than just “ultra-rare” conditions.

Viralgen, a leading contract development and manufacturing organization (CDMO) specializing in recombinant adeno-associated ...

The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...