Dyne Therapeutics’ DYNE-101 shows positive results in phase 1/2 ACHIEVE study. Read why DYN stock may rise with FDA approval and a $2.78B market opportunity.

Natural "off switch" could slow or even reverse the growth of aggressive tumors.

SKY-0515 is Skyhawk's investigational small molecule RNA splicing modifier developed through the company's novel RNA-splicing ...

Roche to present latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association 2025 conference: Basel Tuesday, March 18, 2025, 11:00 Hrs [IST] ...

LncRNAs and circRNAs are key regulators of gene expression, influencing disease mechanisms and offering potential as ...

QRL-101-03 (NCT06532396) is a randomized, double-blind, placebo-controlled, multiple-ascending dose clinical trial evaluating the safety, tolerability, and PK of QRL-101 in up to 60 healthy ...

We provide world-class development and manufacturing of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), messenger RNA (mRNA), viral vectors ... on its novel SKY-0515 small ...

A child with a rare genetic disorder — spinal muscular atrophy type I — was treated prenatally for the first time. Courtesy of Jakob Owens  via Unsplash During an amniocentesis test, a long, thin ...

A study reveals how cancer disrupts RNA splicing and presents a potential therapy using antisense oligonucleotides to target ...

Alternative RNA splicing is like a movie editor cutting and rearranging scenes from the same footage to create different ...