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40+ results for “base editing ITGB2 leukocyte adhesion deficiency”
- 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...
- 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...
- NewsBeam secures USD 500m financing as Pfizer licenses in vivo base editing candidateBeam Therapeutics announced a USD 500 million strategic financing alongside Pfizer’s opt-in to an exclusive global license for a liver-targeted in vivo…
- 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
- DevelopmentEli Lilly posts first data from Verve acquired in vivo base editor in hypercholesterolemia trialEli Lilly (NYSE: LLY) reported Phase Ib data from the Heart-2 trial showing that a single infusion of VERVE-102, an in vivo PCSK9 base editor, reduced LDL...
- 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...
- DiscoveryBroad Institute advances gene editing to permanently lower prion protein and halt neurodegenerationThe Broad Institute has received a USD 11.3 million NIH U19 renewal to advance gene editing approaches aimed at permanently lowering prion protein (PrP)...
- NewsRznomics’ RZ-001 RNA-editing therapeutic earns FDA RMAT designation for hepatocellular carcinomaRznomics (KOSDAQ: 476830), a clinical-stage biopharmaceutical company based in South Korea, announced that the US FDA has granted Regenerative Medicine Advanced Therapy…
- 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...
- 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...
- 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...
- 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…
- DevelopmentWave Life's RNA editing oligonucleotide restores wild-type M-AAT in early AATD trialWave Life Sciences (Nasdaq: WVE), based in Cambridge, Massachusetts, reported updated data from its Phase Ib/IIa WVE-006 RestorAATion-2 trial showing that...
- NewsSeamless Therapeutics, Lilly form USD 1.1b partnership on recombinase gene editing for hearing lossGermany-based biotech Seamless Therapeutics and Eli Lilly and Co., entered into a global strategic research collaboration and licensing agreement to jointly develop…
- 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...
- BusinessEli Lilly commits USD 1.9b to access Ascidian's RNA exon editing platform for kidney diseasesEli Lilly (NYSE: LLY) has committed up to USD 1.9 billion to access Boston-based Ascidian Therapeutics' RNA exon editing platform for inherited kidney...
- DiscoveryARPA-H awards USD 160m to accelerate rare disease gene editing through umbrella clinical trialsARPA-H has announced up to USD 160 million in funding over five years for seven performer teams under its THRIVE (Treating Hereditary Rare Diseases with In...
- 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...
- 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...
- NewsGSK hands back RNA drug from collaboration with Wave Life SciencesMassachusetts-based biotech Wave Life Sciences revealed it has regained full global rights to WVE-006, a GalNAc-conjugated RNA editing therapeutic candidate for alpha-1…
- DiscoveryJAX joins Broad-led consortium on USD 34.5 million ARPA-H rare disease gene-editing awardThe Jackson Laboratory (JAX), in collaboration with the Broad Institute and partners across 12 institutions, has been selected for an up to USD 34.5 million...
- 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...
- 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…
- DevelopmentAnixa's FSHR-directed CAR-T shows extended survival in recurrent ovarian cancerSurvival data from a follicle-stimulating hormone receptor-targeted CAR-T therapy in recurrent ovarian cancer are adding to a limited evidence base for cell...
- 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...
- 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...
- 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...
- DevelopmentScribe's epigenetic silencer targeting PCSK9 for single-dose LDL reduction enters first human trialScribe Therapeutics, an Alameda, California-based biotechnology company, has received clearance from the Australian Therapeutic Goods Administration (TGA)...
- NewsFDA expands Octapharma’s Wilate into pediatric von Willebrand disease prophylaxisThe US FDA issued an approval to expand the label of Wilate (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) for routine prophylaxis…
- BusinessMerck KGaA, Versant launch Saturnus Bio in USD 50m rare cardiology ventureSaturnus Bio, a precision cardiology company founded by Versant Ventures, has launched with USD 50 million in upfront funding from Merck as part of a...
- 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...
- BusinessLyora Therapeutics launches with USD 2.5m seed funding for inherited retinal disease genetic medicinesProvidence, Rhode Island-based Lyora Therapeutics has emerged from stealth with USD 2.5 million in pre-seed financing and a two-program pipeline of genetic...
- BusinessCEL-SCI raises USD 7.2m in public offering to fund Multikine cancer immunotherapy developmentA pattern of recurring equity raises at Vienna-based CEL-SCI Corporation reflects the cash demands of a pre-revenue clinical-stage company advancing a...
- NewsScancell Holdings’ iSCIB1+ DNA immunotherapy gains FDA Fast Track for advanced melanomaScancell Holdings plc (LSE: SCLP), a clinical-stage biotechnology company headquartered in Oxford, United Kingdom, announced receipt of US FDA Fast Track Designation…
- NewsEli Lilly and Profluent form USD 2.25b partnership on AI-designed recombinases for genetic medsEmeryville, California-based Profluent and Indianapolis-based Eli Lilly and Company (NYSE: LLY) have announced a multi-program strategic research collaboration to design and commercialize…
- DiscoveryUniversity of Delaware receives USD 1.36m NIH grant for ephrin signaling researchThe University of Delaware has received a USD 1.36 million R01 research grant from the National Institute of General Medical Sciences to investigate how a...
- 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 & PolicyPolaryx Therapeutics Secures FDA Fast Track Designation for PLX-200 Targeting Rare CLN2 DiseasePolaryx Therapeutics, Inc. (Nasdaq: PLYX), a clinical-stage biotechnology company headquartered in Paramus, New Jersey, announced that the U.S. Food and...
- 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...
- BusinessHemab Therapeutics moving towards Nasdaq IPOHemab Therapeutics Holdings, Inc. filed a Form S-1 registration statement with the U.S. Securities and Exchange Commission for an initial public offering of...