Rise Therapeutics LLC has gained IND clearance from the FDA to initiate a phase I trial of R-5780 in patients with cancer.

Ips Heart Inc. has been awarded orphan drug designation by the FDA for its GIVI-MPC stem cell therapy for Becker muscular dystrophy. GIVI-MPC has the unique ability to create new muscle with full length dystrophin in Becker muscular dystrophy.

In a recently published article, researchers from the University of Central Florida and collaborators reported developing a novel multiorgan microphysiological system (MPS) to simulate acute opioid overdose and recovery, addressing key challenges in opioid crisis research.

Recent work proposes a promising therapeutic approach for dysferlinopathies.

Rappta Therapeutics Oy has signed an exclusive global license agreement with Springworks Therapeutics Inc. for RPT-04402, a first-in-class molecular glue of specific protein phosphatase 2A (PP2A) complexes. Springworks will be responsible for global development and commercialization of RPT-04402, now named SW-3431, and expects to file an IND application by year-end.

Pulsesight Therapeutics SAS has submitted a clinical trial authorization (CTA) to the French authority ANSM seeking to conduct a first-in-human phase I trial of PST-611 for dry age-related macular degeneration (AMD)/geographic atrophy (GA).