In recent months, news came out about several distressing cases that have underscored the risks of gene therapy, which ...
Gene therapy cut severe infections 93% in Wiskott-Aldrich syndrome, using patients’ own modified stem cells to treat a rare ...
New data showing rapid and durable full-length MECP2 expression in vivo with single-stranded AAV support improvements ...
OTXL-101, a promising therapy in development for a severe, rare pediatric metabolic condition, becomes the first pipeline ...
Gene therapy is no longer the future of medicine—it's reality. Treatments that replace, silence or edit genes are already ...
Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today announced two poster presentations at the upcoming 19th ...
Morning Overview on MSN
A gene therapy for Sanfilippo syndrome is approved, the first one for a disease that steadily damages children’s brains
The Food and Drug Administration approved Fayuvi (rebisufligene etisparvovec-hopf), made by Ultragenyx Pharmaceutical, on September 17, 2026, as the first gene therapy for pediatric patients with ...
Addition continues to progress a novel pipeline of non-viral, LNP-delivered, RNA-only gene-addition therapies for severe, ...
“It’s something that we would have never really expected to happen in our career, for kids to be able to hear without the ...
MarketsandMarketsâ„¢ projects the global gene therapy market, valued at USD 7.21 billion in 2023 and USD 8.85 billion in 2024, ...
Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...
Gene therapy is still in development to test its safety and effectiveness, but it may be a promising new treatment for rheumatoid arthritis (RA). Gene therapy alters certain genes to help treat ...
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