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40+ results for “haploidentical HSCT leukocyte adhesion deficiency LAD-I”
- BusinessYarrow raises USD 150m to advance first-in-class Graves' disease antibody into Phase IIConnecticut-based Yarrow Bioscience, Inc. (Nasdaq: YARW) announced the pricing of an underwritten public offering of 5,769,231 shares of common stock at USD...
- DevelopmentGan & Lee advances once-weekly oral GLP-1 peptide into Phase IChina-based Gan & Lee Pharmaceuticals Co., Ltd. has dosed the first participant in a Phase I study of GZC8072, an oral peptide glucagon-like peptide-1...
- 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...
- 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...
- BusinessCerenome secures USD 20m royalty-based financing to scale CNSide diagnostics platformHouston-based Cerenome, Inc. (Nasdaq: CNSY) has secured a senior secured financing facility of up to USD 20 million from 3i, LP, structured with...
- DevelopmentNodThera rebrands as Rezera as ruvonoflast meets Phase II endpoint, eyes Phase III in PADRuvonoflast (NT-0796), an oral NLRP3 inflammasome inhibitor developed by Boston-based Rezera Inc. (formerly NodThera), met its primary endpoint in the...
- BusinessCytonics crowdfunds USD 4.8m to advance A2M biologic into Phase Ib/IIa osteoarthritis studyJupiter, Florida-based Cytonics Corporation has completed a USD 4.8 million gross-proceeds financing campaign, bringing total capital raised to USD 47...
- 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 & 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...
- DiscoveryMcGill and Mount Sinai researchers identify PRMT7 as suppressor of CD8+ T cell expansionA protein arginine methyltransferase acts as a previously unrecognized suppressor of cytotoxic T cell proliferation, offering a potential new target for...
- DevelopmentBioNTech ends autogene cevumeran Phase II after OS imbalance favors controlThe BNT122-01 Phase II trial of autogene cevumeran in resected colorectal cancer has been terminated after an independent data safety monitoring board...
- BusinessLee's Pharm picks up 1cBio's preclinical PARP1 inhibitor for China and SE AsiaLee's Pharmaceutical Holdings (HKEX: 00950) has licensed exclusive rights to develop, manufacture, and commercialize OC-3, a preclinical selective PARP1...
- 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...
- Regulatory & PolicyAlebund gains FDA IND clearance for IgA-cleaving AP308 in IgA nephropathyAlebund Pharmaceuticals (HKEX: 09637) received US FDA clearance of its investigational new drug (IND) application for AP308, an engineered recombinant IgA...
- Regulatory & PolicyInnoCare gains China IND clearance for PSMA/STEAP1 bispecific ADC ICP-B381China's drug regulator has cleared InnoCare Pharma (HKSE: 9969) to begin clinical testing of ICP-B381, a bispecific antibody-drug conjugate (ADC) designed...
- BusinessKura launches Caspian with USD 50m to advance menin inhibition in diabetesA USD 50 million financing round has backed the launch of Caspian Therapeutics, a newly formed company spun out of San Diego-based Kura Oncology (Nasdaq:...
- DevelopmentLeyden Labs advances RIG-101 after Phase I shows safety and on-target immune activationPhase I data from Netherlands-based Leyden Laboratories B.V.'s intranasal innate immune modulator RIG-101 showed acceptable safety and on-target...
- DevelopmentPrana reports prolonged Type 2 biomarker suppression with IL-4Rα/TSLP bispecificPhase I data from a placebo-controlled study in healthy adults suggest that PRA-216, a bispecific antibody that simultaneously blocks IL-4Rα and TSLP, can...
- 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...
- BusinessKorsana goes public with USD 380m raise and Alzheimer's antibody poised for Phase IWaltham, Massachusetts-based Korsana Biosciences (Nasdaq: KRSA) has completed its reverse merger with Cyclerion Therapeutics and closed a USD 380 million...
- DevelopmentEvommune's EVO756 suffers second Phase IIb setback in two months with AD missCalifornia-based Evommune, Inc. (NYSE: EVMN) revealed EVO756, an oral Mas-related G protein-coupled receptor X2 (MRGPRX2) antagonist, failed to meet its...
- 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...
- 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...
- 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)...
- DevelopmentInnovent takes once-weekly oral GLP-1 into Phase IInnovent Biologics (HKEX: 01801) has registered a Phase I study of IBI3042, an oral small-molecule glucagon-like peptide-1 receptor (GLP-1R) agonist...
- BusinessSuperluminal raises USD 60m Series B to take rare obesity drug into Phase IBoston-based Superluminal Medicines has closed an oversubscribed USD 60 million Series B financing round to advance its lead small-molecule melanocortin-4...
- 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...