Spur Therapeutics Ltd. has selected SPR-301 as lead development candidate from its gene therapy program for a genetically defined subset of Parkinson’s disease characterized by mutations in the GBA1 gene. The mutations cause a deficiency in the enzyme glucocerebrosidase (GCase), leading to the accumulation of α-synuclein and subsequent death of neuronal cells that are hallmarks of Parkinson’s disease.
Alveogene Ltd. has announced its novel inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency, AVG-002, has been awarded orphan drug designation by the FDA.
Researchers from SL Bigen Inc. and collaborators presented the preclinical characterization of BM-205, a novel entity of engineered MSCs designed to exert antitumor functions.
At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.
Voyager Therapeutics Inc. has selected a lead development candidate, VY-1706, for its tau silencing gene therapy program in Alzheimer’s disease. The company anticipates filing an IND application with the FDA and a clinical trial application (CTA) with Health Canada for VY-1706 in 2026.
Siren Biotechnology Inc. has unveiled its lead asset, SRN-101, for the treatment of high-grade gliomas. The FDA has granted orphan drug and rare pediatric disease designations to SRN-101 for high-grade gliomas and pediatric-type diffuse high-grade gliomas, respectively.
Belief Biomed Inc.’s gene therapy drug BBM-D101 has been awarded U.S. orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD).
Retinitis pigmentosa (RP) is the most common hereditary degenerative eye disease that leads to progressive vision loss, primarily caused by retinal degeneration.
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York.
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