Spinoff from Novartis AG's acquisition of Avidity Biosciences advances precision cardiology programs using targeted RNA delivery platform Lead candidates ATR 1072 ...

In recent years, mRNA in lipid nanoparticles (mRNA–LNPs) has emerged as a promising strategy for treating numerous conditions ...

Four months after Avidity’s $11.4 billion buyout, Atrium debuted with $270 million in cash and plans to pick up its ...

How innovative technologies like AI and gene editing are advancing rare disease drug discovery and development.

Reflecting on the strength of the community and engagement, Dr. Chaubey said, “Symposium 2026 is a living example of turning possibility into reality – a community united by the desire to be among the ...

Alpha-1 Antitrypsin Deficiency (AATD) is a rare genetic disorder caused by SERPINA1 gene mutations, leading to conditions like pulmonary emphysema and liver dysfunction. The AATD market is in its ...

European regulators have approved Moderna Inc.’s novel combination flu and Covid shot, handing a victory to the embattled ...

Onvuzosiran achieved significant and durable reductions of plasma kallikrein levels with a favorable safety profile in Phase 1/2 – SAN DIEGO, Feb. 27, 2026 (GLOBE NEWSWIRE) -- ADARx Pharmaceuticals, ...

九州大学は、極限環境がリボ核酸(RNA)の前生物化学に与える影響を調査し、「アデノシン一リン酸」(AMP)がRNA鎖へ繋がる重合反応を起こす証拠は発見できず、逆に多様な有機化合物へ分解・再編成されることを発見したと発表した。

The Irish start-up ecosystem had a busy start to the year, with many announcing significant funding rounds. Chief among the raises is quantum start-up Equal1, which announced a $60m round to help with ...

New FDA guidelines for personalized genomic drug development are a step forward in advancing new therapies for ultrarare diseases, but industry leaders need further clarification.

Ionis Pharmaceuticals is a leader in RNA-based therapies. Its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The ...