Tag: therapy

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.

NEW YORK, June 25, 2026 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, held today its shareholders general meeting at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4^th floor, 75013 Paris, France.

LEXINGTON, Mass. and AMSTERDAM, June 25, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the closing of its previously announced underwritten public offering of 5,686,813 ordinary shares at a public offering price of $45.50 per share, which includes 741,758 ordinary shares issued pursuant to the exercise in full by the underwriters of their option to purchase additional ordinary shares in the offering. The aggregate gross proceeds to uniQure from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by uniQure, were approximately $259 million. All securities in the offering were sold by uniQure.

 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).

Hannover, Germany, June 25, 2026 (GLOBE NEWSWIRE) -- EMBARC Announces Its Intention to Collaborate with Insmed on Landmark Interventional Study Evaluating Disease Modification Potential of Brensocatib in Bronchiectasis
—Long-Term, Open-Label Study to Enroll Approximately 3,000 Patients Across Europe—