Tag: safety profile

SAN DIEGO, June 26, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced positive five-year data from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference, taking place June 25-27, 2026, in Orlando, Florida, alongside previously reported results from the Company’s HOPE-3 Phase 3 trial.

 ABO-101 is an investigational gene editing therapy designed to address the underlying disease biology of PH1 by reducing hepatic oxalate production

STOCKHOLM, Sweden, June 25, 2026 – AnaCardio, a clinical stage biopharmaceutical company developing novel drugs to treat heart failure, today announced the publication in The Lancet of results from its Phase 1b/2a GOAL-HF1 study evaluating AC01, a first-in-class, oral calcium-sensitising contractile agent, in patients with heart failure with reduced ejection fraction (HFrEF). The results showed a favourable safety and tolerability profile, dose-proportional pharmacokinetics with confirmed target engagement. In exploratory efficacy analyses, numerical rapid and sustained improvements in haemodynamics and cardiac structure and function were observed on top of optimised guideline-directed medical therapy (GDMT).

— Registration-enabling study demonstrated high clinically meaningful objective responses, achieving rapid and durable disease control —

Energy Safety Response Group testing found no thermal runaway, no sustained fire, and no propagation under direct flame impingement and overcharge abuse, supporting safe siting of long-duration storage in dense urban environments