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40+ results for “AAV9 FOLR1 gene therapy cerebral folate deficiency”
- 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...
- DevelopmentEyePoint's Duravyu misses wet AMD primary endpoint, hinges on second trial for FDA pathEyePoint Pharmaceuticals (Nasdaq: EYPT) reported topline data from LUGANO, the first of two pivotal Phase III trials evaluating vorolanib (Duravyu)...
- DevelopmentCan-Fite's namodenoson boosts gemcitabine potency in pancreatic cancer preclinical modelsNew preclinical data from Can-Fite BioPharma Ltd. (NYSE American: CANF) show that namodenoson, an oral A3 adenosine receptor (A3AR) agonist, enhances the...
- BusinessJ&J hands C-CAR039 back to AbelZeta amid pivot toward in vivo CAR-TAbelZeta Pharma reclaimed full global rights to its anti-CD19/CD20 bispecific CAR-T cell therapy prizloncabtagene autoleucel (C-CAR039) from Janssen Biotech...
- BusinessBioCytics and LIDE Biotech partner to validate immune cell therapies in patient-derived tumor modelsHuntersville, North Carolina-based BioCytics, Inc. and Shanghai-based preclinical oncology contract research organization LIDE Biotech have announced a...
- 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...
- DiscoveryPitt, Magee-Womens team turns cancer dormancy into a druggable vulnerabilityA preclinical study from the University of Pittsburgh and Magee-Womens Research Institute & Foundation identifies a two-drug strategy that selectively kills...
- DiscoveryCell study uncovers polyamines as natural ferroptosis defensePolyamines — small, positively charged metabolites long studied for roles in cell growth and translation — directly chelate labile intracellular iron and...
- 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...
- Developmentargenx's Vyvgart Hytrulo delivers first Phase III success in IMNMEfgartigimod alfa and hyaluronidase-qvfc met the primary endpoint of a Phase III trial in autoimmune myositis, delivering the first statistically...
- DevelopmentTagrisso plus Orpathys extends OS in MET-resistant EGFR-mutated lung cancerThe SAFFRON Phase III trial has delivered the first global Phase III evidence that a targeted oral combination can improve both progression-free and overall...
- 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...
- DevelopmentVoyager registers first-in-human trial of tau-silencing gene therapy VY1706 in Alzheimer’sVoyager Therapeutics (Nasdaq: VYGR) has registered a Phase I VY1706 clinical trial in early Alzheimer's disease, marking the first time an adeno-associated...
- BusinessRedx takes control of Skye in Nasdaq combination backed by USD 125m financingSan Diego-based Skye Bioscience, Inc. (Nasdaq: SKYE) announced a definitive agreement to acquire UK-based Redx Pharma Limited alongside USD 125 million in...
- DevelopmentFate takes off-the-shelf CAR T into potentially registrational lupus nephritis trial Fate Therapeutics (Nasdaq: FATE) has dosed the first patient in RECLAIM-LN, its Phase II potentially registrational trial of FT819, an off-the-shelf...
- DiscoveryMGH, Harvard researchers restore PV neuron function, suppress seizures in NDD mouse modelA single transcription factor delivered to a specific class of inhibitory neurons in adult mice rescues seizures, restores hippocampal network oscillations,...
- DiscoverySynthetic glycan opens vaccine route against Candida aurisA single chemically synthesized sugar molecule can both vaccinate against and detect Candida auris, the multidrug-resistant fungal pathogen responsible for...
- DiscoveryOculogenex secures NEI grant as AMD gene therapy nears INDOculogenex, a La Habra, California-based gene therapy company, has received a USD 1.09 million R44 SBIR grant from the National Eye Institute (NEI) to...
- Regulatory & PolicyBiogen's felzartamab wins first global approval in China for multiple myelomaChina's National Medical Products Administration (NMPA) has approved Jingshu (felzartamab), an anti-CD38 monoclonal antibody, in combination with...
- DevelopmentPhenoNet revives inhaled cromolyn for Phase III Alzheimer’s testThe FDA's clearance of a Phase III trial for an inhaled reformulation of a decades-old anti-allergy compound in Alzheimer's disease puts a privately held...
- Regulatory & PolicyBMS scores first CELMoD approval as FDA embraces MRD surrogateBristol Myers Squibb has received US FDA accelerated approval for Zenbexus (iberdomide), the first cereblon E3 ligase modulating drug (CELMoD) to reach the...
- DevelopmentJW sets up epaminurad partnering push with Phase III gout winA Phase III trial of epaminurad, a selective oral uricosuric agent for gout, has met its primary endpoint and demonstrated statistical superiority over an...
- 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...
- Regulatory & PolicyYoucare takes Factor XI siRNA into US anticoagulation raceChina-based Youcare Pharmaceutical Group (SSE: 688658) received US FDA clearance to proceed with clinical trials of YKYY033 injection, a GalNAc-conjugated...
- DiscoveryNCI backs Minnesota's off-the-shelf cancer-killing Treg platformUniversity of Minnesota has received a USD 2.08 million National Cancer Institute P01 renewal to advance off-the-shelf allogeneic immune effector cell...
- DevelopmentRegeneron quietly takes SF3B1-mutant therapy into first-in-human trialRegeneron Pharmaceuticals (Nasdaq: REGN) has registered a first-in-human clinical trial of REGN17235, an investigational agent targeting clonal...
- Regulatory & PolicyAC Immune takes Parkinson's vaccine into US trials as Phase II data nearSwitzerland-based AC Immune SA (Nasdaq: ACIU), a clinical-stage biopharmaceutical company focused on neurodegenerative diseases, announced receipt of US FDA...
- 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...
- DevelopmentZipalertinib Phase III win sets up first-line Rybrevant challengeTaiho Oncology, Japan-based Taiho Pharmaceutical Co., Ltd., and Cambridge, Massachusetts-based Cullinan Therapeutics (Nasdaq: CGEM) announced that...
- BusinessMerck KGaA partner Remepy raises USD 36m Series A for 'hybrid drug' platformIsrael-based Remepy, a biotechnology company developing Hybrid Drugs — a class that combines conventional prescription pharmaceuticals with personalized,...
- Regulatory & PolicyIvonescimab expands China label as Summit awaits US testChina's National Medical Products Administration (NMPA) has approved a supplemental New Drug Application for ivonescimab (Aqissimab) in combination with...
- DevelopmentDefinium's LSD formulation scores second Phase III win in GADNew York-based Definium Therapeutics (Nasdaq: DFTX) reported positive topline results from the Phase III Voyage study of lysergide orally disintegrating...
- DevelopmentBioRay moves dual-payload TROP2 ADC into the clinicChina-based BioRay Pharmaceutical Co., Ltd. has registered a first-in-human Phase I study of BR113, a dual-payload antibody-drug conjugate (ADC) targeting...
- DiscoveryDaraxonrasib study reveals resistance mechanisms in pancreatic cancerResearchers at Redwood City, California-based Revolution Medicines and Dana-Farber Cancer Institute have published the first systematic characterization of...
- 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),...
- DevelopmentLaekna takes aim at Truqap as China accepts afuresertib NDAChina's drug regulator has accepted a new drug application for afuresertib, the pan-AKT inhibitor developed by Shanghai-based Laekna (HKEX: 2105.HK),...
- DevelopmentKeymed/Belenos’s twice-yearly TSLP × IL-13 bispecific clears Phase IIChina-based Keymed Biosciences Inc. (HKEX: 02162) announced that its Phase II clinical trial of CM512, a recombinant bispecific antibody targeting both...
- 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...