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40+ results for “marnetegragene autotemcel LAD-I 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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 & 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...
- 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...
- 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...
- 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...
- 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...
- 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:...
- 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...
- 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...
- 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...