Search
40+ results for “CRISPR ITGB2 leukocyte adhesion deficiency gene correction”
- DevelopmentBeam reports durable BEAM-302 AAT restoration, advances pivotal cohortUpdated Phase I/II data from Beam Therapeutics' (Nasdaq: BEAM) BEAM-302 program, presented at the European Respiratory Society Congress in Barcelona, show...
- DevelopmentSemaglutide clears obesity treatment threshold in children aged 6 to under 12Phase III data from Novo Nordisk's STEP Young trial show that once-weekly semaglutide (Wegovy, Ozempic, Rybelsus) reduced body mass index (BMI) in children...
- BusinessSernova, Seraxis merge to build integrated stem cell islet replacement platform for T1DCanada-based Sernova Biotherapeutics Inc. (TSX: SVA) and Germantown, Maryland-based Seraxis Holdings, Inc. have signed a definitive merger agreement to...
- Regulatory & PolicyIntellia’s one-shot CRISPR therapy gets March 2027 FDA decision dateCambridge, Massachusetts-based Intellia Therapeutics, Inc. (Nasdaq: NTLA) announced that the US FDA has accepted its Biologics License Application (BLA) and...
- DiscoveryGerman researchers show AI-designed protein vehicles outperform lipid nanoparticles in RNA deliveryResearchers at Helmholtz Munich and the Technical University of Munich, led by corresponding authors Christoph Gruber, Florian Giesert, and Wolfgang Wurst,...
- BusinessMoonwalk Bio raises USD 70m Series B for adipose-targeted obesity therapiesSouth San Francisco-based Moonwalk Biosciences has closed an oversubscribed USD 70 million Series B financing to advance a pipeline of adipose-targeted RNA...
- BusinessIntellia secures USD 400m non-dilutive debt facility ahead of lonvo-z commercial launchMassachusetts-based Intellia Therapeutics, Inc. (Nasdaq: NTLA) announced a non-dilutive senior secured term loan facility of up to USD 400 million with...
- BusinessNeuShen lands USD 80m+ Series B for CNS expansionWith three central nervous system (central nervous system, or CNS) clinical-stage programs under way up to Phase II, China-based NeuShen Therapeutics...
- DevelopmentArgo's siRNA therapy cuts HAE attack rates 83–96% in small Phase II study&x20;Argo Biopharmaceutical Co., Ltd. (Argo Biopharma) provided updated Phase II data for BW-20805, an investigational small interfering RNA (siRNA) therapy...
- DevelopmentStoke, Biogen report four-year durability for Dravet therapy zorevunersenFour-year open-label extension data for zorevunersen, the antisense oligonucleotide being developed by Massachusetts-based Stoke Therapeutics (Nasdaq: STOK)...
- DevelopmentSoufflé joins DUX4 race in FSHD with muscle-targeted siRNA conjugateMassachusetts-based Soufflé Therapeutics, Inc. has registered a Phase I/II clinical trial of SFL-0821, an antibody-siRNA conjugate designed to silence DUX4...
- DevelopmentFrance clears Sensorion's gene therapy SENS-601 into human testing for GJB2 hearing lossFrance's drug regulator has cleared Sensorion to begin human testing of SENS-601, its adeno-associated virus (AAV)-based gene therapy for GJB2-related...
- DevelopmentUltragenyx’s Angelman setback puts UBE3A strategy under scrutinyThe Phase III Aspire study of apazunersen (GTX-102) in Angelman syndrome failed to meet its primary endpoint, leaving the condition without an approved...
- DiscoveryGentiBio secures NIH backing for allogeneic engineered Treg therapy in type 1 diabetesCambridge, Massachusetts-based GentiBio, Inc. has received a USD 1.07 million NIH R44 SBIR Phase II grant from the National Institute of Diabetes and...
- BusinessBoehringer Ingelheim licenses Owkin's K Pro AI platform and immunology data Boehringer Ingelheim has licensed K Pro, the agentic AI Scientist platform developed by New York and Paris-based Owkin, along with multimodal patient data...
- BusinessTypewriter emerges with USD 56m to build non-viral in vivo CAR-T platformCambridge, Massachusetts-based Typewriter Therapeutics, Inc. has emerged from stealth with a USD 56 million Series A financing round to advance a non-viral,...
- DevelopmentAdicet preps off-the-shelf gamma delta CAR-T for first-in-human prostate cancer study Redwood City-based Adicet Bio (Nasdaq: ACET) has registered a Phase I/II trial of ADI-212, an allogeneic gamma delta chimeric antigen receptor (CAR) T cell...
- BusinessKorea's Pharos iBio, Kolon Pharma advance fourth-generation EGFR inhibitor into preclinical South Korea-based Pharos iBio and Kolon Pharma have signed a joint research and development agreement to co-develop PHI-701, a preclinical EGFR-targeting...
- Regulatory & PolicyuniQure files AMT-130 BLA after stop-start FDA pathuniQure N.V. (Nasdaq: QURE) has submitted a Biologics License Application (BLA) to the US FDA seeking accelerated approval of ifezuntirgene inilparvovec...
- DiscoverySeoul team extends prime editing to large-scale DNA replacementResearchers at Seoul National University College of Medicine have developed a prime editing-based system capable of replacing genomic sequences with DNA...
- DiscoveryUniversity of Toronto, SickKids develop chemically modified tRNA platform for cystic fibrosis nonsense mutationsA chemically modified, lipid nanoparticle-delivered suppressor transfer RNA platform has restored functional cystic fibrosis transmembrane conductance...
- DevelopmentBigHat's AI-designed CDH17 antibody enters human testing for gastric cancerSan Mateo, California-based BigHat Biosciences has dosed its first patient with BHB810, a cadherin-17 (CDH17)-directed antibody-drug conjugate (ADC) for...
- DevelopmentOcugen launches Phase III trial of one-time gene therapy for geographic atrophyOcugen (Nasdaq: OCGN) has dosed the first patient in ArMaDa3, a global Phase III registrational trial of OCU410, an AAV5-based modifier gene therapy for...
- DevelopmentEnvudeucitinib misses Phase IIb lupus endpoints, subgroup signal keeps program aliveThe Phase IIb LUMUS trial of envudeucitinib in systemic lupus erythematosus (SLE) failed to meet its primary and secondary endpoints in the overall patient...
- DevelopmentSavolitinib plus Tagrisso hits first-line PFS goal in MET-overexpressing EGFR-mutated lung cancerThe SANOVO Phase III trial has moved savolitinib (Orpathys) plus osimertinib into the first-line EGFR-mutated lung cancer setting, showing a statistically...
- BusinessRedHill exits Talicia for USD 18m upfront to fund GI portfolio expansionRedHill Biopharma (Nasdaq: RDHL) has divested its 70% stake in Talicia, an FDA-approved H. pylori eradication therapy, to a subsidiary of Canada-based...
- Regulatory & PolicyHutchMed’s Atled wins China approval for FGFR2-driven bile duct cancerChina's NMPA has conditionally approved Atled (fanregratinib), an oral selective fibroblast growth factor receptor (FGFR) 1/2/3 inhibitor developed by...
- BusinessAusperBio raises USD 120m Series C for late-stage hepatitis B antisense therapy AusperBio Therapeutics, a biopharmaceutical company focused on oligonucleotide therapies for chronic hepatitis B (CHB), has closed a USD 120 million Series...
- Regulatory & PolicyEC approves Acadia's Daybu as first-ever Rett syndrome treatment in the EUThe European Commission has granted marketing authorization for Daybu (trofinetide), making San Diego-based Acadia Pharmaceuticals (Nasdaq: ACAD) the first...
- Regulatory & PolicyTakeda adds Japan approval for narcolepsy treatment Orzeyful Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Orzeyful (oveporexton) for the treatment of narcolepsy type 1 (NT1) in adults, marking a...
- Business4basebio and Genezen integrate synthetic DNA platform into viral vector manufacturing workflowUK-based 4basebio PLC (AIM: 4BB) and Indianapolis-based Genezen, a gene therapy contract development and manufacturing organization (CDMO), announced a...
- BusinessAirway Therapeutics raises USD 50m to advance late-stage zelpultide alfa trial in BPDAtlanta-based Airway Therapeutics, Inc. has closed a USD 50 million equity round to fund the late-stage clinical development of zelpultide alfa, a...
- DiscoveryMoffitt researchers identify ULK3 dependency in multiple myeloma, report preclinical inhibitor efficacyA serine/threonine kinase largely overlooked in myeloma biology drives autophagy-dependent tumor cell survival and proteasome inhibitor resistance,...
- Regulatory & PolicyRhythm's Imcivree becomes first approved therapy for acquired hypothalamic obesity in JapanJapan's Ministry of Health, Labour and Welfare (MHLW) has granted marketing authorization to Imcivree (setmelanotide) for acquired hypothalamic obesity...
- DiscoveryUCM and MIT research shows transient CB2R modulation locks breast cancer cells into less aggressive stateA brief pulse of cannabinoid receptor 2 (CB2R) activation is sufficient to lock breast cancer cells into a less aggressive, luminal-like identity that...
- Regulatory & PolicySpinal MRI findings trigger FDA hold, derail RegenxBio’s RGX-121 filingThe FDA's clinical hold on Navsunli (clemidsogene lanparvovec-sngl [RGX-121]), an investigational adeno-associated virus (AAV) gene therapy for...
- Regulatory & PolicyADARx's onvuzosiran secures FDA Fast Track for twice-yearly HAE prophylaxis approachSan Diego-based ADARx Pharmaceuticals, Inc. received US FDA Fast Track Designation for onvuzosiran (ADX-324), a prekallikrein (PKK)-targeting small...
- Regulatory & PolicyFDA extends deramiocel review three months as Capricor narrows indication to upper limb functionThe US FDA has extended the Prescription Drug User Fee Act (PDUFA) target action date for deramiocel (CAP-1002), San Diego-based Capricor Therapeutics'...
- BusinessLilly's RNA partner Rznomics joins Lilly Gateway Labs South Korea-based Rznomics Inc. (KOSDAQ: 476830) has joined Lilly Gateway Labs in San Diego, deepening a relationship with Eli Lilly and Company that began...
- BusinessVectorBuilder, Lir apply AI to optimize AAV capsids for manufacturingChicago-based VectorBuilder and Ireland-based Lir Therapeutics have announced a joint platform collaboration to engineer adeno-associated virus (AAV)...