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40+ results for “CRISPR ITGB2 leukocyte adhesion deficiency gene correction”
- 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...
- 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…
- NewsUltragenyx gene therapy DTX301 cuts plasma ammonia in Phase III OTC deficiency trialNovato, California-based Ultragenyx Pharmaceutical announced Phase III results for DTX301 (avalotcagene ontaparvovec), an AAV8 gene therapy for ornithine transcarbamylase deficiency, reporting a…
- DiscoveryBIDMC wins NIH grant to study ADAMTS13 deficiency beyond rare clotting disorderBeth Israel Deaconess Medical Center (BIDMC) has received a USD 1.63 million NIH R01 grant from the National Heart, Lung, and Blood Institute to investigate...
- BusinessScribe Therapeutics files for Nasdaq IPO with clinical-stage cardiovascular CRISPR pipelineScribe Therapeutics Inc. (Nasdaq: SCTX), a clinical-stage gene editing company co-founded by Nobel Laureate Dr. Jennifer Doudna and backed by more than USD...
- NewsIntellia’s CRISPR therapy Phase III to continue after FDA lifts clinical holdIntellia Therapeutics revealed that the US FDA has lifted the clinical hold on its MAGNITUDE-2 Phase III trial for nexiguran ziclumeran (nex-z),…
- DiscoveryYale University receives USD 1.38m NIH grant for CRISPR-based Alzheimer's tau therapyYale University has received a USD 1.38 million NIH cooperative agreement from the National Institute of Neurological Disorders and Stroke to develop a...
- Regulatory & PolicyFDA Approves Loargys by Immedica Pharma for Hyperargininemia in Arginase 1 Deficiency PatientsThe US FDA has granted accelerated approval to Loargys (pegzilarginase-nbln) for the treatment of hyperargininemia in adult and pediatric patients aged two...
- DiscoveryUPenn picks up USD 1.1m NHLBI grant for CRISPR base-editing CVD programThe University of Pennsylvania's Kiran Musunuru has received an R35 award from the National Heart, Lung, and Blood Institute totaling USD 1,137,435 in...
- DevelopmentiECURE's ECUR-506 achieves 57% reduction in hyperammonemic events in OTC deficiency trialiECURE reported statistically significant reductions in hyperammonemic events from the ECUR-506 OTC-HOPE trial, with the completed low-dose cohort showing a...
- BusinessAurigene secures non-exclusive CRISPR/Cas9 license from ERS Genomics for drug discoveryAurigene Pharmaceutical Services, a contract research and manufacturing subsidiary of Dr. Reddy's Laboratories (NYSE: RDY), has secured a non-exclusive...
- Regulatory & PolicyFDA Approves Wellcovorin by GSK as First Treatment for Cerebral Folate Transport DeficiencyThe US FDA approved Wellcovorin (leucovorin calcium) tablets on March 10, 2026, for the treatment of cerebral folate deficiency in adult and pediatric...
- DevelopmentSanofi's efdoralprin alfa outperforms plasma therapy in Phase II alpha-1 antitrypsin deficiency trialSanofi (Nasdaq: SNY) reported Phase II data showing efdoralprin alfa, a recombinant human AAT-Fc fusion protein, outperformed standard-of-care...
- DiscoveryScribe takes USD 25m in CIRM funding to push CRISPR CVD programs into clinicScribe Therapeutics has received more than USD 25 million from the California Institute for Regenerative Medicine (CIRM) to advance two preclinical...
- NewsSNIPR Biome posts positive early data for CRISPR-armed phages against E. coli infectionDenmark-based SNIPR Biome announced publication of final Phase I results for SNIPR001, a CRISPR–Cas-armed bacteriophage cocktail designed to selectively kill Escherichia coli…
- DevelopmentBeam Therapeutics' BEAM-302 shows sustained protective AAT levels in alpha-1 antitrypsin deficiency trialBeam Therapeutics reported updated clinical data from its ongoing Phase I/II trial of BEAM-302 in alpha-1 antitrypsin deficiency, showing that a single 60...
- DevelopmentFull Phase III data reinforce Intellia's lead in race to first In Vivo CRISPR approvalFull Phase III data for lonvoguran ziclumeran (lonvo-z) — published simultaneously in the New England Journal of Medicine and presented at the European...
- Regulatory & PolicyFDA Approves ACCRUFeR by Shield Therapeutics as First Prescription Oral Iron for Pediatric Iron DeficiencyThe US FDA has approved ACCRUFeR (ferric maltol) for the treatment of iron deficiency in children aged 10 years and older, Shield Therapeutics announced on...
- BusinessSyntax Bio raises USD 14.4m to scale CRISPR-programmed cell therapy for type 1 diabetesChicago-based Syntax Bio has closed an expanded Series A totaling USD 14.4 million, bringing total funding to more than USD 25 million, as the synthetic...
- DevelopmentAirna's RNA-editing therapy targets PiZ mutation in alpha-1 antitrypsin deficiency via ADAR recruitmentAIRNA doses first patient in Phase 1 trial of AIR-001, an RNA-editing therapy for alpha-1 antitrypsin deficiency
- Regulatory & PolicyIntellia starts FDA submission for in vivo Crispr therapy lonvo-z as one-time angioedema treatmentIntellia Therapeutics has initiated a rolling submission of a Biologics License Application (BLA) to the US FDA for Lonvoguran ziclumeran (lonvoguran...
- BusinessClaris Bio raises USD 118m to advance first-ever pharmacologic therapy for limbal stem cell deficiencyJersey City, New Jersey-based Claris Biotherapeutics, Inc. has closed a USD 118 million Series B financing to advance CSB-001 (oremepermin alfa ophthalmic...
- 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...
- NewsSatellite Bio wins FDA Rare Pediatric Disease designation for SB-101 in urea cycle disordersSatellite Biosciences announced receipt of a Rare Pediatric Disease (RPD) designation from the FDA for SB-101, an allogeneic off-the-shelf hepatocyte cell therapy,…
- DevelopmentBioMarin faces uncertainty for ENPP1 enzyme replacement therapy after mixed Phase IIIBioMarin Pharmaceutical (Nasdaq: BMRN) reported that its Phase III ENERGY 3 trial evaluating BMN 401 in children aged 1 to 12 with ENPP1 deficiency met only...
- 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...
- DiscoveryMSK developing machine learning models to map gene networks in RAMemorial Sloan-Kettering Cancer Center has received a USD 1.12 million NIH supplemental award to build machine learning models of gene regulatory networks...
- 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...
- NewsVertex expands label for Casgevy to young children with sickle cell diseaseThe US FDA has supplementally approved Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with sickle cell disease (SCD) with…
- NewsPrime Medicine prevails in Beam dispute, retaining exclusive rights to AATD therapy PM647An arbitration tribunal has confirmed that Massachusetts-based Prime Medicine (Nasdaq: PRME) holds exclusive development rights over PM647, its investigational prime editing therapy…
- BusinessScribe Therapeutics raises USD 128.7 million in IPO priced at top of rangeScribe Therapeutics Inc. (Nasdaq: SCTX), a clinical-stage biotechnology company co-founded by Nobel laureate Jennifer Doudna, priced an upsized initial...
- 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...