Nominated KRRO-121 development candidate for the potential treatment of hyperammonemia in patients with urea cycle disorders and hepatic ...

Advancing AX-0810 through ongoing Phase 1 dosing, with target engagement data expected 1H 2026Selected Development Candidates AX-2402 for Rett ...

Parkinson's disease (PD) is a neurodegenerative disorder in which the α-synuclein protein abnormally aggregates within brain cells, causing neuronal damage. Through international collaboration, ...

Wave Life Sciences (NASDAQ:WVE) highlighted upcoming clinical catalysts across its obesity and RNA editing programs during a discussion hosted by Oppenheimer biotech analyst Cheng Li following the ...

A research team led by Rice's Yang Gao has uncovered new insights into the molecular mechanisms of ADAR1. ADAR1 converts adenosine to inosine in double-stranded RNA, a process essential for preventing ...

Eli Lilly And Co (NYSE:LLY) on Thursday entered into a strategic global research collaboration and licensing agreement with Rznomics Inc., a South Korea-based biopharmaceutical company specializing in ...

DNA is the backbone of the genetic code so, logically, decoding and understanding the genetic sequence along with epigenetic modifications came first. Yet, RNA and the emerging field of ...

All starting from the same DNA, neurons ultimately take on individual characteristics in the brain and body. Differences in which genes they transcribe into RNA help determine which type of neuron ...

ProQR Therapeutics is advancing its Axiomer ADAR-mediated RNA editing pipeline, aiming to file a Clinical Trial Application for its lead program AX-0810, targeting NTCP for cholestatic diseases, by Q2 ...

The ability to correct disease-causing genetic mistakes using genome editors holds great promise in medicine, but it is not without risk. When this type of "genetic surgery" is performed on DNA, for ...