Oct 31, 2024
Spur Therapeutics announces positive preclinical results for SBT101, its gene therapy candidate designed to treat Adrenomyeloneuropathy (AMN). (Source)
Oct 30, 2024
Lexeo Therapeutics Reports Positive Interim Data for LX1001, a Novel Gene Therapy Targeting APOE4-Associated Alzheimer’s Disease, at CTAD. Gene therapy, Alzheimer’s disease, AAV (Source)
Oct 29, 2024
Abeona Therapeutics Resubmits Biologics License Application to the FDA for Prademagene Zamikeracel, a Gene Therapy Designed to Treat Recessive Dystrophic Epidermolysis Bullosa. (Source)
Oct 28, 2024
Genprex and the University of Michigan Rogel Cancer Center partner to study the gene used in Reqorsa gene therapy for translocated lung cancer. (Source)
CombiGene discontinues its CG01 epilepsy gene therapy program due to strategic evaluation and cost-cutting measures. (Source)
Oct 25, 2024
enGene Raises $60M in Private Placement to Advance Detalimogene, a Promising Gene Therapy for Bladder Cancer. (Source)
Oct 24, 2024
Poseida Therapeutics to present encouraging preclinical data for hereditary angioedema gene therapy P-KLKB1-101, including reduced kallikrein protein activity in mice and successful gene editing in primate livers. (Source)
BridgeBio Announces Positive Results from High-Dose Cohort of Phase 1/2 CANaspire Study for Canavan Disease Gene Therapy BBP-812 at ESGCT 2024. (Source)
Dyno Therapeutics and Roche Announce Strategic Partnership to Develop Advanced AAV Gene Therapy Vectors for Neurological Disorders. (Source)
Oct 23, 2024
Ikarovec secures additional £8M ($6.5M) to advance its lead sight loss program toward clinical trials. Gene therapy, geographic atropy, wet age-related macular degeneration (Source)
Oct 22, 2024
Sangamo Therapeutics plans to submit a Biologics License Application (BLA) for ST-920 in Fabry disease in 2025, aligned with the FDA’s accelerated approval pathway. (Source)
Ocuphire Pharma Acquires Opus Genetics, Expanding Gene Therapy Pipeline for Inherited Retinal Diseases. (Source)
Oct 21, 2024
REGENXBIO Shares Positive Phase II Results for ABBV-RGX-314 at AAO 2024. Gene therapy, wet age-related macular degeneration (AMD) (Source)
Oct 15, 2024
Beacon Therapeutics reports positive Phase 2 results for AGTC-501 (a gene therapy program) in X-linked retinitis pigmentosa. (Source)
MeiraGTx reports positive results from a randomized clinical trial comparing its AAV-GAD gene therapy for treating Parkinson’s disease. (Source)
Oct 12, 2024
Benitec Biopharma announced positive preliminary results from a Phase 1b/2a clinical trial evaluating the safety and efficacy of low-dose BB-301. Gene therapy, oculopharyngeal muscular dystrophy (OPMD), DNA-directed RNA interference (Source)
Oct 09, 2024
Purespring Therapeutics has secured £80 million (approximately $105 million) in a Series B financing round. The newly acquired funds will be used to accelerate the development of Purespring’s AAV gene therapy pipeline.
Oct 08, 2024
Astellas Pharma and AviadoBio have entered into an exclusive option and license agreement for AVB-101, a gene therapy candidate being developed to treat frontotemporal dementia and other potential indications. (Source)
Swiss biotech company Somagenetix AG secures a CHF 10 million ($11.7M) Series A funding round to develop a lentiviral gene therapy treatment for Chronic Granulomatous Disease. (Source)
Fiocruz and GEMMABio partnered to fund research on gene therapy treatments for Brazilians within the Unified Health System, SUS. (Source)
Oct 07, 2024
Kyowa Kirin‘s newly acquired subsidiary, Orchard Therapeutics, and Er-Kim have partnered to commercialize Libmeldy®, the first approved gene therapy for early-onset metachromatic leukodystrophy (MLD). autologous hematopoietic stem cell gene therapy (Source)
