Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program ...

Rett syndrome is a rare progressive neurodevelopmental ... the loss of independence as patients are unable to perform daily activities or communicate needs. They usually require 24/7 care and ...

Axiomer ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Company expect ...

Expansion of collaboration with Rett Syndrome Research Trust ... Net cash used in operating activities during the full year ended December 31, 2024 was € 36.4 million, compared to € 21.5 ...

The $9.2 million increase was driven by Good Manufacturing Practices batch activities for the intended commercial ... gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a ...

Patient Advocacy Initiative staff speak on Notre Dame’s Rare Disease Day event and impact for the rare disease community.

Our growing discovery pipeline, now including CNS applications, leading intellectual property portfolio, and strategic partnerships - such as our $3.9 billion collaboration with Eli Lilly and ...

The ASX had a good run on Thursday,  with the market lifting 0.2% as key players like Coles Group (ASX:COL) and Qantas (ASX:QAN) reported some impressive numbers.

ProQR Therapeutics (PRQR) announced an expansion of its collaboration with the Rett Syndrome Research Trust ... Net cash used in operating activities during the three-month period ended September ...