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40+ results for “haploidentical HSCT leukocyte adhesion deficiency LAD-I”
- Regulatory & PolicyFDA approves Rocket Pharmaceuticals' gene therapy Kresladi for severe pediatric leukocyte adhesion deficiency-IThe US FDA has granted accelerated approval to KRESLADI (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy developed by...
- DevelopmentUltomiris misses Phase III endpoint in adult HSCT-TMA, leaving pediatric approval path intactAstraZeneca (LSE/STO/NYSE: AZN) reported that ravulizumab (Ultomiris) failed to meet the primary endpoint in a Phase III placebo-controlled trial in adults...
- DevelopmentBeam Therapeutics' BEAM-302 shows sustained protective AAT levels in alpha-1 antitrypsin deficiency trialBeam Therapeutics reported updated clinical data from its ongoing Phase I/II trial of BEAM-302 in alpha-1 antitrypsin deficiency, showing that a single 60...
- DiscoveryBIDMC wins NIH grant to study ADAMTS13 deficiency beyond rare clotting disorderBeth Israel Deaconess Medical Center (BIDMC) has received a USD 1.63 million NIH R01 grant from the National Heart, Lung, and Blood Institute to investigate...
- Regulatory & PolicyFDA Approves Loargys by Immedica Pharma for Hyperargininemia in Arginase 1 Deficiency PatientsThe US FDA has granted accelerated approval to Loargys (pegzilarginase-nbln) for the treatment of hyperargininemia in adult and pediatric patients aged two...
- DevelopmentiECURE's ECUR-506 achieves 57% reduction in hyperammonemic events in OTC deficiency trialiECURE reported statistically significant reductions in hyperammonemic events from the ECUR-506 OTC-HOPE trial, with the completed low-dose cohort showing a...
- Regulatory & PolicyFDA Approves Wellcovorin by GSK as First Treatment for Cerebral Folate Transport DeficiencyThe US FDA approved Wellcovorin (leucovorin calcium) tablets on March 10, 2026, for the treatment of cerebral folate deficiency in adult and pediatric...
- NewsUltragenyx gene therapy DTX301 cuts plasma ammonia in Phase III OTC deficiency trialNovato, California-based Ultragenyx Pharmaceutical announced Phase III results for DTX301 (avalotcagene ontaparvovec), an AAV8 gene therapy for ornithine transcarbamylase deficiency, reporting a…
- DevelopmentSanofi's efdoralprin alfa outperforms plasma therapy in Phase II alpha-1 antitrypsin deficiency trialSanofi (Nasdaq: SNY) reported Phase II data showing efdoralprin alfa, a recombinant human AAT-Fc fusion protein, outperformed standard-of-care...
- Regulatory & PolicyFDA Approves ACCRUFeR by Shield Therapeutics as First Prescription Oral Iron for Pediatric Iron DeficiencyThe US FDA has approved ACCRUFeR (ferric maltol) for the treatment of iron deficiency in children aged 10 years and older, Shield Therapeutics announced on...
- DevelopmentAirna's RNA-editing therapy targets PiZ mutation in alpha-1 antitrypsin deficiency via ADAR recruitmentAIRNA doses first patient in Phase 1 trial of AIR-001, an RNA-editing therapy for alpha-1 antitrypsin deficiency
- BusinessClaris Bio raises USD 118m to advance first-ever pharmacologic therapy for limbal stem cell deficiencyJersey City, New Jersey-based Claris Biotherapeutics, Inc. has closed a USD 118 million Series B financing to advance CSB-001 (oremepermin alfa ophthalmic...
- DevelopmentBioMarin faces uncertainty for ENPP1 enzyme replacement therapy after mixed Phase IIIBioMarin Pharmaceutical (Nasdaq: BMRN) reported that its Phase III ENERGY 3 trial evaluating BMN 401 in children aged 1 to 12 with ENPP1 deficiency met only...
- NewsMabwell launches Phase I/II trials for CDH17 ADCChina-based biotech Mabwell is initiating two Phase I/II clinical trials of its CDH17-targeted antibody–drug conjugate (ADC) 7MW4911. The China study (CTR20254163) will…
- DevelopmentNovartis's FAP-targeting radioligand enters Phase I for solid tumorsNovartis has initiated a first-in-human Phase I trial of [⁶⁸Ga]Ga-DFC413 (JFI447), a Gallium-68-labeled radioligand designed to image fibroblast activation...
- DevelopmentAstellas terminates Phase I for KaliVir-licensed oncolytic vaccinia virusAstellas Pharma Global Development, Inc. has terminated its Phase I clinical trial of ASP1012, an engineered oncolytic vaccinia virus, in adults with...
- DevelopmentHemispherian's GLIX1 initiates Phase I/IIa trial in recurrent glioblastoma patientsHemispherian AS, an Oslo-based oncology company, announced the initiation of a first-in-human Phase I/2a GLIX1 clinical trial in glioblastoma on April 8,...
- DevelopmentTenvie Therapeutics initiates Phase I trial of NLRP3 inhibitor in obesityTenvie Therapeutics, a Brisbane, California-based biotech, announced the dosing of the first subject in a Phase I/Ib clinical study of TNV262, a small...
- DevelopmentMETiS TechBio's enters MTS109 into Phase I for refractory autoimmune diseaseShanghai Changzheng Hospital has opened enrollment in the first-in-human MTS109 clinical trial (NCT07526350), testing an mRNA-encoded, lipid...
- DevelopmentAmgen terminates Phase I trial of AMG 378 in strategic decisionAmgen Terminates Phase I Trial of AMG 378 Before Primary Completion
- DevelopmentGrünenthal advances NOP agonist into efficacy testing after clear Phase IGermany-based Grünenthal Group has reported the successful completion of a Phase I trial for its proprietary nociceptin receptor (NOP) agonist, an...
- DevelopmentSarepta's siRNA conjugate SRP-1005 enters Phase I for Huntington's disease Sarepta Therapeutics has registered a first-in-human Phase I trial of SRP-1005 (formerly ARO-HTT), an siRNA conjugate designed to silence the huntingtin...
- NewsMerryLife Biomedical’s TML-6 shows promise in Phase I Alzheimer’s trialTaiwan-based MerryLife Biomedical announced positive Phase I results for TML-6, an innovative oral Alzheimer’s therapy designed to target multiple neurological pathways simultaneously.…
- NewsSNIPR Biome posts positive early data for CRISPR-armed phages against E. coli infectionDenmark-based SNIPR Biome announced publication of final Phase I results for SNIPR001, a CRISPR–Cas-armed bacteriophage cocktail designed to selectively kill Escherichia coli…
- NewsFDA expands Octapharma’s Wilate into pediatric von Willebrand disease prophylaxisThe US FDA issued an approval to expand the label of Wilate (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) for routine prophylaxis…
- DevelopmentCSPC's tissue factor-targeting ADC enters Phase I for advanced solid tumorsCSPC Pharmaceutical Group (HKEX: 1093) has initiated a first-in-human Phase I clinical trial of SYS6051, a tissue factor (TF)-targeting antibody-drug...
- DevelopmentChina's SmartNuclide puts GPA33-directed pretargeted radioimmunotherapy into Phase I for CRCA first-in-human trial of SNA028, a two-step pretargeted radioimmunotherapy targeting the GPA33 antigen, has been registered at Tianjin Medical University...
- DevelopmentBioNTech advances mRNA therapeutic HIV vaccine into randomized Phase I/II trialBioNTech has registered a randomized, placebo-controlled Phase I/II clinical trial of BNT168, an mRNA vaccine designed to elicit HIV-specific immune...
- DevelopmentJade Biosciences' anti-BAFF-R antibody entering Phase I for rheumatoid arthritisJade Biosciences, a NASDAQ-listed clinical-stage company (ticker: JBIO), has registered a first-in-human Phase I trial of JADE201, an afucosylated,...
- Development Janux Therapeutics' masked bispecific T cell engager enters Phase I for mCRPCJanux Therapeutics, the San Diego-based NASDAQ-listed biopharmaceutical company (ticker: JANX), has initiated a first-in-human Phase I clinical trial of...
- DevelopmentKestrel's pan-KRAS inhibitor swoops into Phase I for advanced solid tumorsKestrel Therapeutics, a Watertown, Massachusetts-based biotechnology company, has opened a first-in-human Phase I clinical trial for KST-6051, an oral...
- DevelopmentAlar's ketamine therapy meets safety goals in treatment-resistant depression Phase I Alar Pharmaceuticals, a Taiwan-based clinical-stage company listed on the TPEx exchange, reported Phase I results for ALA-3000, a subcutaneous...
- NewsAspen’s sasineprocel shows motor improvements in Phase I/IIa Parkinson’s disease trialAspen Neuroscience, a San Diego–based clinical-stage regenerative medicine company, announced 12-month data from its Phase I/IIa ASPIRO clinical trial evaluating sasineprocel (ANPD001),…
- NewsBioNTech initiates first-in-human Phase I Trial for HIV antibody BNT351BioNTech SE has launched a first-in-human (FIH) Phase I clinical trial (NCT07392372) to evaluate the safety, pharmacokinetics, and antiviral activity of BNT351,…
- NewsPfizer calls halt to Phase I development of Seagen-acquired CD228 ADCPfizer has terminated its Phase I PF-08046031 clinical trial in adults with advanced melanoma and other solid tumors, according to an update…
- DevelopmentAlnylam puts siRNA with undisclosed-target into Phase I for obesity managementAlnylam Pharmaceuticals (Nasdaq: ALNY) has initiated a first-in-human Phase I trial of ALN-6222, an investigational siRNA therapeutic administered...
- NewsVectorY Therapeutics’ VTx-002 gene therapy enters Phase I/II ALS trialNetherlands-based VectorY Therapeutics has initiated a first-in-human clinical trial of VTx-002, an investigational gene therapy targeting amyotrophic lateral sclerosis (ALS) in a…
- Regulatory & PolicyCHMP backs leriglitazone for early-stage cerebral adrenoleukodystrophy in childrenLeriglitazone (NEZGLYAL) has received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use, positioning it...
- DevelopmentSanofi and Kymera's second-generation IRAK4 degrader enters Phase I for hidradenitis suppurativaSanofi has initiated a first-in-human study of SAR447971 (also known as KT-485), an oral small-molecule IRAK4 targeted protein degrader discovered by...
- DevelopmentShanghai Henlius's HLX3902 trispecific antibody enters Phase I trial for advanced solid tumoursShanghai Henlius Biotech, the HKEX-listed Chinese biopharmaceutical company, has registered a first-in-human Phase I clinical trial for HLX3902, a...