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Tim Hunt, CEO of the Alliance for Regenerative Medicine, explains “the incredibly promising but nonlinear world of cell and gene therapies.” Skip to Main Content Manage alerts for this article ...
Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root ...
One gene therapy trial in OA is under way and another in RA is close to reaching human study, but that’s a year or more away. The road for gene therapies in RA is complicated, costly and fraught ...
LONDON, May 27, 2025 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced the publication of preclinical proof-of-concept data for FLT201, its adeno-associated virus (AAV) gene therapy candidate ...
Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of ...
Baby saved by gene-editing therapy 'graduates' from hospital, goes home. KJ Muldoon was diagnosed with a rare, deadly genetic disease after he was born. By Sony Salzman. June 4, 2025, 11:09 AM.
Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene ...
UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...
A gene therapy offered new hope for children suffering from a rare and potentially fatal immune disorder, according to a recent study published in the New England Journal of Medicine. The study ...
The gene therapy technology was licensed by UC San Diego to Eikonoklastes Therapeutics in 2021. Eikonoklastes was granted Orphan Drug Designation (ODD) by the FDA for the use of the patented gene ...
K.J. Muldoon, an infant in Philadelphia, has been treated with a personalized Crispr gene-editing therapy for a rare and deadly disease. Photo: Muldoon family.