Multicentre gene therapy for OTOF-related deafness followed up to 2.5 years
Yasunaga, S. et al. A mutation in OTOF, encoding otoferlin, a FER-1-like protein, causes DFNB9, a nonsyndromic form…
UTSW Spinout InterAct Therapeutics Selected by Cell & Gene Therapy Incubator Program » Dallas Innovates
Left: Daniel Hargrove, co-founder and CEO of InterAct Therapeutics; Right: Isaac Chan, MD, PhD, Co-founder and CSO of…
CRISPR gene editing therapy cuts LDL cholesterol in small study
A CRISPR-based gene editing therapy dramatically reduced levels of low-density lipoprotein, often called “bad cholesterol,” and triglycerides in…
uniQure Provides FDA Update on AMT-130; BLA Timing Unclear
uniQure (NASDAQ: QURE) on Nov. 3, 2025 announced FDA feedback…
New gene-editing method can correct many disease-causing mutations in mammalian cells
Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person’s genome, often…
Next-generation gene therapy tools built by UH scientist
Reading time: 2 minutesAssistant Professor Jesse Owens using a program to model the structure of his integrase protein.…
Huntington’s disease treated for first time using gene therapy
A brain scan of a person with Huntington’s disease, which causes a loss of brain volume as neurons…
U.S. Cell & Gene Therapy Contract Research Organizations
Dublin, Sept. 18, 2025 (GLOBE NEWSWIRE) — The “U.S. Cell & Gene Therapy Contract Research Organizations Market Size,…
Diversity challenges and reconciles genetics in facioscapulohumeral muscular dystrophy
Wang LH, Tawil R. Facioscapulohumeral dystrophy. Curr Neurol Neurosci Rep. 2016;16:66. Article PubMed Google Scholar Mul K, Lassche…
Personalized Medicine (Biologics, Small Molecules, Gene
Dublin, Sept. 08, 2025 (GLOBE NEWSWIRE) — The “Personalized Medicine: Market Share, and Growth Analysis by Product Type…