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

Financing led by new investor Venrock Healthcare Capital Partners, with participation from new and existing investorsProceeds support the ...

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 ...

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 ...

In just the past few years, researchers have found a way to use a naturally occurring bacterial system known as CRISPR/Cas9 to inactivate or correct specific genes in any organism. CRISPR/Cas9 gene ...

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 ...

Rice University researchers have developed a CRISPR-based gene activation strategy that boosts mitochondrial production in damaged heart cells, improving cardiac function after heart attacks in both ...