A new study in the journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 ...

A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick ...

A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 (NPC1) who ...

Reported positive initial data from INSPIRE DUCHENNE trial of next-generation Duchenne gene therapy candidate, SGT-003; In mid-2025, Company plans to request a U.S. Food and Drug Administration ...

The global gene therapy market is projected to grow from $8.85 billion in 2024 to $36.55 billion by 2032, at a CAGR of 19.4%.

Reported positive initial data from INSPIRE DUCHENNE trial of next-generation Duchenne gene therapy candidate, SGT-003; In mid-2025, ...

Viral vectors expressing proneural genes can stimulate adult mammalian retinal glia to become immature neurons. These glia-derived neurons share properties with the bipolar, amacrine and retinal ...

The axolotl (Ambystoma mexicanum) has extraordinary regenerative abilities—the salamander can regrow lost limbs and repair ...

The global viral vectors for non-human primates market was valued at USD 145.8 Mn in 2023 and is projected to grow at a CAGR ...

AI-assisted AAV capsid engineering has transformative potential in terms of accelerating and optimizing the development of gene therapies.

“We are thrilled to welcome these industry-leading experts to our Clinical and Scientific Advisory Board,” said Dr. Nicole K. Paulk, CEO, Founder, and President of Siren Biotechnology. “Their deep ...

Right now, the IND-enabling studies are made to test the safety, tolerability, vector biodistribution, transgene expression, ...