NGNE to update on a potential registrational phase 3 study, using NGN-401, for the treatment of patients with Rett Syndrome.

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Gene was suffering from severe heart disease and advanced Alzheimer's disease, while Betsy suffered from hantavirus, which is spread through rodent droppings Gene Hackman's wife Betsy Arakawa ...

Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces that the first patient in the TREE study ...

Enhancers are genomic elements critical for regulating gene expression. In this Review, the authors discuss how sequence-to-function models can be used to unravel the rules underlying enhancer ...

Feb. 26, 2025 — Drawing inspiration from the tardigrade, researchers developed a new strategy that may protect cancer patients from the side effects of radiation ... Comprehensive Resource ...

Acadia Pharmaceuticals Inc. today announced that the journal Med published results from the open-label study, DAFFODILtm, evaluating the safety, tolerability and exploratory efficacy of DAYBUE® ...

After that, the next step in CRISPR gene editing is usually to alter that piece of DNA. However, CRISPR has also been adapted to do other things too, such as turning genes on or off without ...