Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease.
Zipbio has signed an exclusive license agreement with Meiragtx Holdings plc to advance an AAV gene therapy for geographic atrophy. Under the agreement, Meiragtx will receive exclusive rights to Zipbio’s first-in-class therapies targeting the complement pathway for geographic atrophy.
Genprex Inc. has released preliminary in vivo proof-of-concept data for GPX-002, the company’s diabetes gene therapy drug candidate, in a nonhuman primate (NHP) model of type 2 diabetes and in a mouse model of type 2 diabetes. GPX-002 is being developed for the potential long-term control of both type 1 and type 2 diabetes.
SYNGAP1-related disorders (SRDs) are rare neurodevelopmental conditions characterized by a wide range of symptoms, including intellectual disability, epilepsy, motor deficits and increased risk-taking behavior.
Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether.
Viatris Inc. has announced FDA clearance of its IND application for MR-146, an Enriched Tear Film (ETF) AAV gene therapy candidate for the treatment of neurotrophic keratopathy. The company plans to initiate a phase I/II trial in patients with neurotrophic keratopathy in the first half of next year.
Latus Bio Inc. has reported IND clearance by the FDA for LTS-101, a gene therapy candidate to treat the CNS manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. The FDA has also granted orphan drug, rare pediatric disease and fast track designations to LTS-101.
The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells.
Gemma Biotherapeutics Inc.’s GB-221, a novel gene therapy for spinal muscular atrophy type 1 (SMA1), has received clinical trial clearance from ANVISA, Brazil’s health regulatory agency.
The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells.
