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40+ results for “TYF-ITGB2 leukocyte adhesion deficiency gene therapy”
- BusinessIMUNON partners with National Cancer Institute to expand IL-12 DNA plasmid beyond ovarian cancerNew Jersey-based IMUNON, Inc. (Nasdaq: IMNN) entered a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part...
- BusinessExegenesis and Modalis combine muscle-targeting AAV with epigenome editing for preclinical DMD candidateExegenesis Bio and Japan-based Modalis Therapeutics Corporation (Tokyo: 4883) signed a research collaboration and license agreement to advance MDL-201, a...
- DiscoveryJohns Hopkins tests pancreatic TLR4–hyperglycemia axis as driver of necrotizing enterocolitisJohns Hopkins University has received a USD 2.5 million NIH R01 grant from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) to...
- DevelopmentOMass enters human testing with MC2R antagonist OMS1620 for congenital adrenal hyperplasiaUK-based OMass Therapeutics has registered a Phase I trial of OMS1620, an oral small-molecule melanocortin-2 receptor (MC2R) antagonist being developed for...
- Regulatory & PolicyFDA grants Fast Track Designation to Sobi's pacritinib for VEXAS syndromeSwedish Orphan Biovitrum AB (Stockholm: SOBI), operating through its North American affiliate Sobi North America, announced that the US FDA has granted Fast...
- BusinessEncoded Therapeutics raises USD 275m to push ETX101 toward pivotal Dravet syndrome trialSouth San Francisco-based Encoded Therapeutics has closed a USD 275 million Series F financing round to advance its lead gene therapy candidate through...
- DevelopmentAbbVie's atogepant meets primary endpoint in Phase III menstrual migraine trialAtogepant (Qulipta/Aquipta) met the primary endpoint and all eight ranked secondary endpoints in the Phase III LUNA trial evaluating preventive treatment of...
- BusinessViaNautis expands into spinal cord injury with SCI Ventures backing for polyNaut platformCambridge, UK-based ViaNautis Bio has secured investment from SCI Ventures to expand its non-viral genetic medicine delivery platform into chronic spinal...
- BusinessMindera Health partners with UPenn to investigate quinacrine mechanism in lupusMindera Health is collaborating with University of Pennsylvania researchers to investigate why quinacrine helps some patients with cutaneous lupus—and...
- DevelopmentSeismic launches global Phase III trial of istaroxime in cardiogenic shockDurham, North Carolina-based Seismic Pharmaceuticals has launched a global Phase III trial of istaroxime in cardiogenic shock, a life-threatening condition...
- BusinessInsightec spinout Lotus Neuro launches brain drug delivery platformLotus Neuro, a clinical-stage biotechnology company spun out of focused ultrasound specialist Insightec, launched publicly with a platform designed to...
- DevelopmentInsilico’s rentosertib becomes first AI-discovered drug to enter Phase IIIInsilico Medicine (HKEX: 3696) has dosed the first patient in GENESIS-IPF-3, the Phase III trial of rentosertib (ISM001-055 / INS018\055) in idiopathic...
- Regulatory & PolicyRoche moves Enspryng toward first approved MOGAD therapyRoche (SIX: RO; OTCQX: RHHBY) announced a US FDA Priority Review award for a supplemental Biologics License Application (sBLA) for Enspryng (satralizumab),...
- Regulatory & PolicyBayer’s Hyrnuo joins Boehringer’s Hernexeos in first-line HER2-mutant NSCLCThe US FDA has granted accelerated approval to Hyrnuo (sevabertinib) for the first-line treatment of adults with locally advanced or metastatic non-squamous...
- DevelopmentIndependent research teams validate Apeximmune's PLA2G2D target for overcoming anti-PD-1 resistanceA Nature publication has given South San Francisco-based Apeximmune Therapeutics substantial external validation, with two independent research teams...
- BusinessStanford spinout TwoStep raises USD 62.5m as lead peptide-drug conjugate clears FDA INDSan Carlos, California-based TwoStep Therapeutics has raised USD 62.5 million in an oversubscribed Series A financing round, coinciding with US FDA...
- DevelopmentIDEAYA, Servier expand darovasertib into Phase III adjuvant uveal melanomaSan Francisco-based IDEAYA Biosciences (Nasdaq: IDYA) and France-based Servier announced that the first patient has been dosed in OptimUM-11, a global Phase...
- BusinessHarbour Bio partner Solstice raises USD 225m to advance CTLA-4 antibody in neoadjuvant cancerBoston-based Solstice Oncology launched with a USD 225 million Series A financing to advance porustobart (HBM4003), an Fc-enhanced CTLA-4 antibody that...
- DevelopmentLancet data show durable responses with PeproMene's BAFF-R CAR T after CD19 CAR T failurePhase I data for PMB-CT01, a B-cell activating factor receptor (BAFF-R)-targeted autologous chimeric antigen receptor (CAR) T-cell therapy, have been...
- DevelopmentPharvaris’s deucrictibant XR cuts HAE attack rate 83% in Phase III, advancing dual oral strategyPharvaris N.V. (Nasdaq: PHVS) has reported an 83% reduction in monthly hereditary angioedema (HAE) attack rate with deucrictibant extended-release (XR)...
- 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)...