The ML4 Foundation says the FDA has allowed the first U.S. human trial of an AAV9 gene therapy for mucolipidosis type IV to proceed, opening clinical testing for an ultra-rare neurodegenerative disease with no approved disease-modifying therapy.
Teva’s TEV-408 met the primary endpoint in a 50-patient Phase 2a celiac disease trial, significantly reducing gluten-induced intestinal damage while also limiting inflammatory-cell increases and gastrointestinal symptoms.
uniQure files AMT-130 for accelerated FDA approval after three-year Huntington’s disease data showed a 75% slowing in progression.
Pyrotech Therapeutics has dosed the first participant in an 84-person Phase 1 study of PTT-121, an intravenous GSDMD inhibitor being developed initially for septic shock.
BioMarin Pharmaceutical has completed its acquisition of Alesta Therapeutics, adding Phase 1/2a candidate ALE1, an oral small molecule designed to target the metabolic abnormality driving hypophosphatasia.
Upstream Bio sets Phase 3 plans for quarterly verekitug in severe asthma and CRSwNP after FDA discussions. See what the pivotal strategy means.
Ocugen starts Phase 3 testing of one-time OCU410 gene therapy after a 31% reduction in geographic atrophy lesion growth in Phase 2.
Remibrutinib wins two Phase III multiple sclerosis trials as Novartis prepares global filings. See what the results mean for the BTK race.
Alumis’ 408-patient LUMUS study missed its primary and secondary endpoints in systemic lupus erythematosus, but prespecified interferon-high patients showed stronger responses that the company intends to discuss with regulators.
Ocugen has begun the 237-patient Phase 3 ArMaDa3 trial of OCU410, a one-time gene therapy for geographic atrophy after Phase 2 treatment reduced lesion growth by 31% at 12 months.