The U.S. FDA has cleared Genetic Leap’s IND application for GL-IL2-138, a small-molecule RNA drug that modulates natural IL-2, allowing for downregulating or upregulating of the immune system to fight diseases.
The U.S. FDA has cleared Aavantgarde Bio Srl’s IND application for AAVB-039, the company’s gene therapy program for Stargardt disease, the most common inherited form of macular degeneration.
Palatin Technologies Inc. has announced preclinical results for PL-7737, an oral selective melanocortin MC4 receptor (MC4R) agonist, showing effectiveness in rodent models of obesity.
The U.S. FDA has granted orphan drug designation to Klotho Neurosciences Inc.’s secreted-Klotho (s-KL) promoter, gene and delivery system (KLTO-202 or s-KL-AAV.myo) for the treatment of amyotrophic lateral sclerosis (ALS).
Eluciderm Inc. has received clearance from the U.S. FDA for its IND to conduct a phase I/IIa open-label study evaluating the safety and efficacy of ELU-42, a topical spray-on solution for open wound healing, in patients with diabetic foot ulcers (DFUs).
Sensorium Therapeutics Inc. has reported that the U.S. FDA has cleared the IND application for SNTX-2643 (SENS-01), its lead anxiety program. First-in-human dosing begins in Q3 2025.
Solid Biosciences Inc. has announced approvals of its IND application and CTA by the U.S. FDA and Health Canada, respectively, for SGT-501, a novel gene therapy approach for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), a highly malignant, arrhythmogenic channelopathy caused by mutations in the RYR2 and CASQ2 genes.
Brightpath Biotherapeutics Co. Ltd.’s iPS cell-derived BCMA CAR-natural killer T cell therapy candidate has been awarded orphan drug designation by the FDA for the treatment of multiple myeloma.
The U.S. FDA has cleared Rocket Pharmaceuticals Inc.’s IND application for RP-A701, an AAVrh.74-based gene therapy candidate for the treatment of BAG3-associated dilated cardiomyopathy (BAG3-DCM), a severe form of heart failure.
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