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40+ results for “marnetegragene autotemcel LAD-I gene therapy”
- Regulatory & PolicyFDA approves Rocket Pharmaceuticals' gene therapy Kresladi for severe pediatric leukocyte adhesion deficiency-IThe US FDA has granted accelerated approval to KRESLADI (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy developed by...
- NewsVectorY Therapeutics’ VTx-002 gene therapy enters Phase I/II ALS trialNetherlands-based VectorY Therapeutics has initiated a first-in-human clinical trial of VTx-002, an investigational gene therapy targeting amyotrophic lateral sclerosis (ALS) in a…
- DevelopmentVoyager reports durable six-month preclin safety data for Alzheimer's gene therapy ahead of Phase IVoyager Therapeutics (Nasdaq: VYGR) reported six-month preclinical toxicology data for VY1706 Alzheimer's gene therapy candidate at the Alzheimer's...
- BusinessApertura Gene Therapy and TSC Alliance collaborate on AAV gene therapy for tuberous sclerosis complexApertura Gene Therapy has entered a collaboration agreement with the TSC Alliance, a Silver Spring, Maryland-based patient advocacy organization founded in...
- BusinessApertura Gene Therapy and Viralgen partner to manufacture blood-brain barrier-crossing AAV capsid for CNS gene therapyApertura Gene Therapy and Viralgen, a contract development and manufacturing organization based in San Sebastián, Spain, have entered a manufacturing...
- NewsBreakthrough Prize 2026 honors gene therapy pioneers, CRISPR innovators, and ALS gene discoveryThe Breakthrough Prize Foundation announced the 2026 Breakthrough Prize winners on April 18, with the Life Sciences awards—presented alongside prizes in fundamental…
- BusinessOptimeos secures backing for non-viral nano gene therapy deliveryPrinceton, New Jersey-based Optimeos Life Sciences has announced an investment from Hatch BioFund, an early-stage life sciences venture capital firm...
- DiscoveryEpiCure Therapeutics secures NIH grant for SLC6A1 gene therapy developmentEpiCure Therapeutics, Inc., a Seattle, Washington-based Allen Institute spinout, has received a USD 1.5 million NIH R44 grant to advance an AAV gene therapy...
- BusinessREGENXBIO raises USD 100m amid pivotal year for gene therapy pipelineRockville, Maryland-based REGENXBIO Inc. (Nasdaq: RGNX) announced a proposed underwritten public offering of common stock, intending to raise $100 million...
- NewsUltragenyx files gene therapy for US approval in glycogen storage diseaseUS-based biotech Ultragenyx Pharmaceutical Inc., (NASDAQ: RARE) announced that the US FDA has accepted a Biologics License Application (BLA) for DTX401 (pariglasgene…
- NewsUltragenyx gene therapy shows long-term promise in rare neurological disorderUltragenyx Pharmaceutical announced positive clinical trials for UX111 (rebisufligene etisparvovec), an investigational AAV9 gene therapy targeting Sanfilippo syndrome Type A, or mucopolysaccharidosis…
- DiscoveryPhoenix Nest gets NIH backing for Sanfilippo syndrome gene therapy manufacturingPhoenix Nest, a Brooklyn, New York-based biotechnology company, has received a USD 1.49 million NIH SBIR grant to fund manufacturing and analytical...
- NewsRegenxbio’s gene therapy RGX-202 posts positive early data in Duchenne muscular dystrophyRegenxbio Inc., a Maryland-based gene therapy company, announced new interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202 in patients…
- BusinessNeurogene raises USD 125m to accelerate Rett syndrome gene therapy toward approvalNew York-based Neurogene Inc. (Nasdaq: NGNE) announced the pricing of a USD 125 million public offering of common stock and pre-funded warrants on July 1,...
- BusinessLEO Pharma bags Replay's HSV gene therapy platform for USD 50m upfront Denmark-based LEO Pharma has entered into a definitive agreement to acquire San Diego-based Replay, a private gene therapy company, for USD 50 million...
- Regulatory & PolicyRegenxbio's Navsunli clears FDA path toward first gene therapy for Hunter syndromeRockville, Maryland-based REGENXBIO Inc. (Nasdaq: RGNX) has announced alignment with the US FDA on a path forward for resubmission of the Biologics License...
- BusinessGenetix expands gene therapy footprint into Middle East with SPIMACO manufacturing dealSomerville, Massachusetts-based Genetix Biotherapeutics has struck an exclusive licensing and commercialization agreement with Saudi-based Saudi...
- DevelopmentRegenxbio's hits Phase III endpoints with gene therapy for Duchenne muscular dystrophyRegenxbio (Nasdaq: RGNX), the Rockville, Maryland-based gene therapy company, reported positive topline data from the pivotal Phase III portion of its...
- BusinessMeiraGTx secures USD 400m from Oberland Capital to fund gene therapy commercializationUK-based MeiraGTx Holdings plc (Nasdaq: MGTX) announced a strategic investment of up to USD 400 million from Oberland Capital Management LLC, structured to...
- DiscoveryNorthwestern University receives USD 1.84m NIH grant for tauopathy gene therapy developmentNorthwestern University has received a USD 1.84 million NIH RF1 grant from the National Institute of Neurological Disorders and Stroke to advance...
- BusinessRay Therapeutics raises USD 125m Series B for optogenetic gene therapy programsRay Therapeutics, a Berkeley, California-based clinical-stage biopharmaceutical company developing optogenetic gene therapies for retinal degenerative...
- NewsIDefine and UT Southwestern collaborate on EHMT1 gene therapy for Kleefstra syndromeIDefine – The Kleefstra Syndrome Foundation, an Atlanta-based nonprofit patient advocacy organization, and UT Southwestern Medical Center announced a two-year pre-clinical research…
- NewsOcugen’s OCU410 modifier gene therapy shows Phase 2 efficacy in geographic atrophyOcugen reported positive preliminary 12-month data from its Phase 2 ArMaDa clinical trial of OCU410 (AAV5-RORA), a modifier gene therapy being developed…
- NewsuniQure receives FDA pushback on approval pathway for Huntington’s disease gene therapyIn a notice with implications for the broader Huntington’s disease development landscape, uniQure N.V. (NASDAQ: QURE) announced that it received final meeting…
- Regulatory & PolicyRegeneron files Otarmeni gene therapy for EU approval in genetic hearing lossRegeneron Pharmaceuticals (Nasdaq: REGN) has received acceptance from the European Medicines Agency for a Marketing Authorization Application for Otarmeni...
- NewsOS Therapies seeks FDA alignment on survival endpoint for osteosarcoma gene therapyOS Therapies, Inc. (NYSE American: OSTX) has requested a Type B meeting with the US FDA to resolve a central unresolved question…
- NewsFDA approves Regeneron’s Otarmeni as first gene therapy for genetic hearing lossThe US FDA has approved Otarmeni (lunsotogene parvec-cwha), the first gene therapy for genetic hearing loss, granting Regeneron Pharmaceuticals accelerated approval for…
- NewsEC approves Novartis’s Itvisma as first gene therapy for older SMA patientsThe European Commission has approved Itvisma (onasemnogene abeparvovec) for the treatment of children aged two years and older, teenagers, and adults with…
- Regulatory & PolicyChengdu Origen and Vanotech receive FDA clearance for KHN921 gene therapy in hypertrophic cardiomyopathyChengdu Origen Biotechnology and Vanotech have received US FDA clearance to proceed with a Phase I/II clinical trial of KHN921, an AAV9-based gene therapy...
- DevelopmentNeurogene's gene therapy shows sustained milestone gains in Rett syndrome through 30 monthsNew York-based Neurogene (Nasdaq: NGNE) reported updated Phase I/II data for NGN-401, an AAV9 gene therapy delivering a full-length human MECP2 transgene,...
- DiscoveryWeill Cornell Medicine receives USD 3.25m NIH award for AAV gene therapy Alzheimer's trialWeill Cornell Medicine has received a USD 3.25 million UG3 award from the National Institute on Aging to advance an AAV-mediated gene therapy into Phase I...
- DevelopmentAlar's ketamine therapy meets safety goals in treatment-resistant depression Phase I Alar Pharmaceuticals, a Taiwan-based clinical-stage company listed on the TPEx exchange, reported Phase I results for ALA-3000, a subcutaneous...
- BusinessApertura partners with NIH on TfR1 capsid approach for Niemann-Pick gene therapyNew York-based Apertura Gene Therapy has entered a Cooperative Research and Development Agreement with two NIH institutes to advance preclinical...
- BusinessAstellas Pivots Away from XTANDI, Betting Big on KRAS Degraders and Gene TherapyAstellas Pharma's Q4 FY2025 earnings call, held April 27, 2026, revealed a company accelerating its transition away from XTANDI dependence through a...
- DevelopmentAmlogenyx presents preclinical data for AM805 gene therapy targeting amyloid in Alzheimer's diseaseAmlogenyx Reports Preclinical Data for AM805, an AAV9 Gene Therapy Designed to Degrade Amyloid in Alzheimer's Disease
- Regulatory & PolicyMeiraGTx's AAV2-hAQP1 gene therapy earns FDA breakthrough designation for radiation-induced xerostomiaMeiraGTx Holdings plc (Nasdaq: MGTX), a clinical-stage genetic medicines company headquartered in London and New York, announced that the FDA has granted...
- DiscoverySiren Bio advances AAV immune-gene therapy into clinical trials for recurrent glioblastomaSan Francisco-based Siren Biotechnology has received a USD 8 million CLIN2 grant from the California Institute for Regenerative Medicine (CIRM) to advance...
- Regulatory & PolicyFDA accepts Ultragenyx's UX111 gene therapy resubmission for Sanfilippo syndrome Type A treatmentUltragenyx Pharmaceutical has submitted a resubmitted Biologics License Application to the US FDA for UX111 (rebisufligene etisparvovec), an AAV9 gene...
- BusinessLatus Bio closes USD 97m Series A financing for AAV gene therapy pipelinePhiladelphia-based Latus Bio has closed a USD 97 million Series A financing round to advance its AAV gene therapy pipeline, with lead programs targeting...
- BusinessEditas prices USD 319m offering as EDIT-401 gene therapy for hyperlipidemia progressesCambridge, Massachusetts-based Editas Medicine (Nasdaq: EDIT) has announced the pricing of an underwritten public offering of common stock and accompanying...