Gene-Therapy Treatment Of Infant With Rare Genetic Condition Successful In World First » TwistedSifter
Pexels When baby KJ was born with a rare disease, which could cause a potentially fatal amount of…
Opus Genetics’ BEST1 Gene Therapy IND Cleared by FDA
Opus Genetics (Nasdaq: IRD) has received FDA clearance for its…
New generation of CRISPR shows safer path to treating genetic diseases
A new generation of CRISPR technology developed at UNSW Sydney offers a safer path to treating genetic diseases…
Join Sept. 18 with George Church and this year’s top biotech startups
I asked AI the othÂer day how many biotechs have been co-foundÂed by HarÂvard’s George Church. About 50…
Opus Genetics Announces Financial Results for Second Quarter 2025 and Provides Corporate Update
Opus Genetics, Inc. – Positive 12-month Phase 1/2 clinical data in adult cohort and early pediatric clinical data…
Ousted FDA vaccine chief Vinay Prasad returning to agency
WASHINGTON (AP) — A Food and Drug Administration official is getting his job back as the agency’s top…
Genetic insights into the origin, admixture, and migration of the early Austronesian peoples
Shutler R, Marck JC. On the dispersal of the Austronesian horticulturalists. Archaeology Phys Anthropol Ocean. 1975;10:81–113. Google Scholar …
When ganglioside pathways go awry: congenital disorders and experimental insights
Thudichum JLW. A treatise on the chemical constitution of the brain: Bailliere, Tindall, and Cox, London; 1884. Chiricozzi…
FDA vaccine chief Vinay Prasad leaves following criticism from Laura Loomer
WASHINGTON (AP) — The Food and Drug Administration’s polarizing vaccine chief is leaving the agency after a brief…
Rocket’s PKP2-Arrhythmogenic Cardiomyopathy Gene Therapy RP-A601 Snags RMAT Designation
Rocket Pharmaceuticals’ RP-A601, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat plakophilin-2 related Arrhythmogenic Cardiomyopathy…
NGLY1 deficiency – clinical features and therapeutic strategy
Suzuki T. Catabolism of N-glycoproteins in mammalian cells: molecular mechanisms and genetic disorders related to the processes. Mol…
As Sarepta, FDA tangle over Duchenne therapy, families are caught in the middle
Jennifer Hill Blair recognized the Cincinnati number on her buzzing phone and slipped out of the school meeting,…