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40+ results for “base editing ITGB2 leukocyte adhesion deficiency”
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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,...
- 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...
- 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...
- 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...
- 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...
- BusinessSamsung Biologics raises USD 2.2b to fund PolyPeptide acquisition and capacity expansionSouth Korea-based Samsung Biologics (KRX: 207940.KS) announced a rights offering to raise KRW 3 trillion (approximately USD 2.2 billion), with the bulk of...
- Regulatory & PolicyPfizer and BioNTech's XFG-adapted Comirnaty given FDA clearance for fall 2026 rolloutThe US FDA has approved a supplemental Biologics License Application (sBLA) for Comirnaty XFG (COVID-19 vaccine, mRNA), the 2026–2027 XFG-adapted...
- 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 & 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...
- DevelopmentIvonescimab Phase III PFS benefit gets peer-reviewed backing ahead of FDA decisionPublication of the global Phase III HARMONi trial data in The Lancet Oncology gives ivonescimab its first peer-reviewed, globally conducted evidence base...
- 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...
- 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...
- 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...
- Regulatory & PolicyFDA approves Genglycos as first gene therapy for glycogen storage disease type IaThe US FDA has granted accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), an AAV8 gene therapy developed by Novato, California-based...
- DevelopmentEnhertu becomes first HER2-directed therapy to beat chemoimmunotherapy in 1st-line lung cancerTrastuzumab deruxtecan (Enhertu) has become the first HER2-directed therapy to demonstrate a progression-free survival benefit over the global standard of...
- BusinessBioMarin pays USD 275m upfront for early-stage oral HPP bet ALE1BioMarin Pharmaceutical (Nasdaq: BMRN) has agreed to acquire Alesta Therapeutics for USD 275 million upfront plus up to USD 215 million in development and...
- BusinessSlate Medicines goes public via Fulcrum merger to challenge PACAP migraine fieldRaleigh, North Carolina-based Slate Medicines, Inc. will gain a Nasdaq listing through an all-stock reverse merger with Cambridge, Massachusetts-based...
- DiscoveryMyeloid cell transplantation rescues Friedreich's ataxia through mitochondrial transfer in miceHematopoietic transplantation can rescue neurological and cardiac deficits in a mouse model of Friedreich's ataxia (FA) — not by replacing diseased neurons...
- BusinessSilence Therapeutics completes private fund raise at USD 201.3m for divesiran trial UK-based Silence Therapeutics plc (Nasdaq: SLN) announced the closing of an upsized public offering of American Depositary Shares (ADSs), raising...
- DevelopmentCRISPR Therapeutics takes one-shot gene editing into hypertensionCRISPR Therapeutics AG (Nasdaq: CRSP) has initiated a first-in-human clinical trial of CTX340, an in vivo gene editing therapy designed to permanently...
- BusinessIASO Bio acquires MediSix to expand CAR-T reach into T-cell malignanciesChina-based IASO Biotechnology ("IASO Bio") has completed the acquisition of MediSix Therapeutics, a Singapore-based immune cell engineering company, adding...
- BusinessPTC, Lilly scoop up Sangamo gene therapies in bankruptcy auctionRichmond, California-based Sangamo Therapeutics (OTC: SGMOQ) has concluded a court-supervised Section 363 asset auction, selecting Eli Lilly and Warren, New...
- BusinessSilence upsizes USD 175m offering after divesiran Phase II winUK-based Silence Therapeutics plc (Nasdaq: SLN) announced the pricing of an upsized underwritten public offering of American Depositary Shares (ADSs),...
- BusinessAbCellera offers up USD 200m in stock to support NK3 menopause antibody Canada-based AbCellera Biologics Inc. (Nasdaq: ABCL) announced a proposed underwritten public offering of common stock and pre-funded warrants targeting USD...
- BusinessSentynl gains US rights to Mereo's Phase III alvelestat for alpha-1 lung diseaseMereo BioPharma (Nasdaq: MREO) and Solana Beach, California-based Sentynl Therapeutics have entered an option and license agreement granting Sentynl...
- BusinessEpicrispr raises USD 90m to advance first-in-class epigenetic therapy into pivotal FSHD studiesSouth San Francisco-based Epicrispr Biotechnologies has closed a USD 90 million oversubscribed Series C financing round to advance its lead epigenetic gene...