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40+ results for “TYF-ITGB2 leukocyte adhesion deficiency gene therapy”
- 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...
- DevelopmentAirNexis’s AN01 improves lung function on top of bronchodilators in COPDA dual phosphodiesterase inhibitor delivered by dry powder inhaler met its primary endpoint in a Phase IIb randomized trial in chronic obstructive pulmonary...
- 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...
- DevelopmentBMS’s GPRC5D CAR-T clears registrational trial in quadruple-exposed multiple myelomaArlocabtagene autoleucel met the primary endpoint of overall response rate (ORR) and all key secondary endpoints in the registrational QUINTESSENTIAL Phase...
- DevelopmentInhibrx's OX40 agonist nearly doubles response rate versus Keytruda alone in head and neck cancerA hexavalent OX40 agonist that has struggled to find clinical traction across the broader class has now produced the first randomized efficacy signal for an...
- 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,...
- BusinessPDS Biotech raises up to USD 22.55m to advance IL-12 immunotherapy toward Phase III in CRCNew Jersey-based PDS Biotechnology Corporation (Nasdaq: PDSB) has raised up to USD 22.55 million in a private investment in public equity (PIPE) transaction...
- DevelopmentAmgen’s tarlatamab delivers Phase III OS win in first-line ES-SCLC maintenanceAdding tarlatamab to durvalumab maintenance produced a statistically significant and clinically meaningful improvement in overall survival in...
- DevelopmentAZ’s tozorakimab shows Phase III benefit across eosinophil levels in COPDFull Phase III results from the previously announced positive OBERON and TITANIA trials show that AstraZeneca's IL-33 antibody tozorakimab reduced...
- Regulatory & PolicyFDA approves AZ’s Etcamah for pre-progression switch in HR+ breast cancerAstraZeneca has received accelerated approval from the US FDA for Etcamah (camizestrant), an oral selective estrogen receptor degrader (SERD), in...
- DevelopmentNovartis’s pelacarsen misses Phase III endpoint despite deep Lp(a) loweringNovartis’s pelacarsen failed to reduce major cardiovascular events in the Phase III Lp(a)HORIZON trial despite substantially lowering lipoprotein(a)...
- DevelopmentNovartis’s Avidity-acquired del-desiran falls short in Phase III DM1 trialThe Phase III HARBOR study of delpacibart etedesiran (del-desiran) in myotonic dystrophy type 1 (DM1) has failed its primary endpoint, dealing a major...
- BusinessBrainChild Bio raises USD 116m to advance CAR T therapy for pediatric brain cancerSeattle-based BrainChild Bio, Inc., a clinical-stage company developing CAR T cell therapies for central nervous system tumors, has closed a USD 116 million...
- 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...
- Regulatory & PolicyAcepodia wins FDA clearance for dual-payload GPC3 ADC in liver cancerCalifornia- and Taiwan-based Acepodia (TWSE: 6976) has received US FDA clearance to begin Phase I testing of ACE723, a GPC3-targeted dual-payload...
- 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)...
- BusinessENA expands antiviral nasal spray INNA-051 into asthma patients with grant backingAustralia-based ENA Respiratory has received AUD 2.98 million (USD 1.9 million) from Biointelect Venturer to fund the first study of its INNA-051 antiviral...
- 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...
- Regulatory & PolicyFDA approves Zanvastro as first disease-modifying treatment for Alexander diseaseThe US FDA has approved Zanvastro (zilganersen), an antisense oligonucleotide (ASO) targeting glial fibrillary acidic protein (GFAP) mRNA, for the treatment...
- BusinessCipla joins Keytruda biosimilar race with Qilu dealIndia-based Cipla Limited (BSE: 500087; NSE: CIPLA) has secured exclusive US commercialization rights for QL2107, a biosimilar to pembrolizumab (Keytruda),...
- DevelopmentAbbVie’s monthly BCMAxCD3 bispecific clears Phase III hurdleAbbVie (NYSE: ABBV) has reported that etentamig, its investigational BCMA × CD3 bispecific T-cell engager (TCE), met both dual primary endpoints in the...
- 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...
- Regulatory & PolicyNMPA accepts Hansoh’s B7-H3 ADC HS-20093 in post-platinum SCLCChina's National Medical Products Administration (NMPA) has accepted a biologics license application from Hansoh Pharmaceutical Group Company Limited (HKEX:...
- BusinessGSK bets USD 1.3b on HutchMed's KRAS-EGFR conjugateGSK has agreed to pay USD 110 million upfront for exclusive rights to develop and commercialize HMPL-A830, a pre-clinical antibody-targeted therapy...
- 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...
- BusinessTScan restructures around in vivo TCR-T, pauses Phase III hematology programWaltham, Massachusetts-based TScan Therapeutics (Nasdaq: TCRX) announced a major restructuring that will shift the company away from near-term development...
- DiscoveryVincere Bio wins grant for USP30 inhibitor IND-enabling studies in acute kidney injuryVincere Biosciences, a small molecule drug developer, has received a USD 1.3 million SBIR Phase II grant from the National Institute of Diabetes and...
- 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...
- DevelopmentNorroy advances first domestically developed PSMA-targeted therapy into pivotal China trialChina-based Norroy Bioscience Co., Ltd. has initiated a pivotal Phase III trial of 177Lu-NYM032, a prostate-specific membrane antigen (PSMA)-targeted...
- 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...