Sionna Therapeutics Inc. has received FDA clearance of its IND application for SION-638, a small molecule designed to target the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A phase I study is now dosing healthy volunteers.
Sparingvision SAS has obtained FDA clearance for its IND application for SPVN-06, its lead gene-independent therapy for the treatment of retinitis pigmentosa (RP). SparingVision has also submitted a clinical trial authorization (CTA) application to the French regulator (ANSM), which is currently under review.
Opus Genetics Inc. has received FDA clearance for its IND application for a first-in-human phase I/II trial of OPGX-001 in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5). The trial is due to start in the U.S. early next year (ClinicalTrials.gov Identifier NCT05616793).
CSPC Pharmaceutical Group Ltd. has received IND approval from the FDA for a phase I study in the U.S. of its antibody-drug conjugate CPO-204 in patients with locally advanced or metastatic urothelial cancer.
The FDA has awarded orphan drug designation to Sensorion SA's OTOF-GT, a dual vector AAV gene therapy, for the treatment of otoferlin gene-mediated hearing loss.
The FDA has awarded orphan drug designation to Tenaya Therapeutics Inc.'s gene therapy product candidate, TN-401, for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC). TN-401 is an adeno-associated virus (AAV)-based gene therapy being developed for the treatment of genetic ARVC caused by plakophilin-2 (PKP2) gene mutations.
The FDA has awarded orphan drug designation to Canbridge Pharmaceuticals Inc.'s CAN-106, a clinical-stage long-acting recombinant humanized monoclonal antibody targeting C5, for the treatment of myasthenia gravis.
Gmax Biopharm LLC has received FDA clearance of its IND filing for GMA-131 injection, an ETa receptor-specific monoclonal antibody, for a phase Ib study in diabetic kidney disease (DKD).
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