March 14, 2025 -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the investigational medicine KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). “Receiving orphan drug designation from the FDA underscores the growing n

No significant clinical adverse event observed in Phase 1clinical study Pharmacokinetic profile established and range of doses selected for Phase 2a Clinical Efficacy Study First patient expected to be treated in the Phase 2a Clinical Efficacy Study on March 14, 2025 at the Montreal Heart Institute March 13, 2025 -- COSCIENS Biopharma Inc., a life science company which develops and commercializes a diversified portfolio of cosmeceutical, nutraceutical and pharmaceutical products, today announce

March 11, 2025 -- Medera Inc. (“Medera”), a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases by developing a range of next-generation therapeutics, today announced that patient dosing has been completed in its MUSIC-HFrEF Phase 1/2a clinical trial of the gene therapy candidate SRD-001 in patients with heart failure with reduced ejection fraction (HErEF). HErEF is a prevalent form of heart disease that accounts for half of an esti

Trial for this rare genetic disease to be conducted by NIH/NIAID in partnership with Corvus ITK inhibition intended to address dysregulation of T cells that causes ALPS and has been shown to be effective at treating the disease in preclinical models March 12, 2025 -- Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced that the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), has i

March 12, 2025 -- Portage Biotech Inc. (“Portage” or the “Company”) (NASDAQ: PRTG), a clinical-stage immuno-oncology company with a portfolio of innovative therapeutics, today announced the resumption of patient enrollment in the fourth and final cohort of the dose escalation stage for PORT-6, a highly selective A2A antagonist, within its ADPORT-601 Phase 1b clinical trial. Portage had previously paused this trial due to funding concerns; this resumption of the trial underscores the encouraging

March 12, 2025 -- Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression

Medigene AG , an oncology platform company focused on the research and development of T cell receptor (TCR)-guided therapies for the treatment of cancer, today announced the submission of a patent for a novel natural killer (NK) cell-specific TCR construct to the European Patent Office. With that, Medigene is advancing its TCR-guided strategy by expanding the application of its proprietary 3S (sensitive, specific, and safe) TCRs into NK cells. This patent application marks a major expansion of M

Kaerus Bioscience , a clinical-stage biopharmaceutical company created by Medicxi for the development of therapeutics for rare genetic syndromes of neurodevelopment, today announces the successful completion of its Phase 1 clinical trial of KER-0193, a novel BK channel modulator the company is developing for Fragile X syndrome (FXS) and other neurodevelopmental conditions. In the trial, KER-0193 was found to be safe and well-tolerated in both single ascending dose and multiple ascending dose co

2025年3月13日，再鼎医药TF ADC新药维替索妥尤单抗的上市申请获得NMPA受理。 今年1月，再鼎医药宣布维替索妥尤单抗治疗晚期宫颈癌的全球三期临床innovaTV 301研究中国亚组取得阳性主要结果。 Tivdak于2021年在美国获批上市，2024年销售额为1.3亿美元。 ADC为再鼎医药布局重点，除了TF ADC之外，DLL3 ADC、LRRC15 ADC、ROR1 ADC也在快速推进，其中DLL3 ADC将于今年启动小细胞肺癌关键临床。 内容来源于网络，如有侵权，请联系删除。

中国国家药监局药品审评中心（CDE）官网近日最新公示，百时美施贵宝公司申报的1类新药BMS-986489（BMS-986012 + nivolumab固定剂量复方）注射液获得临床试验默示许可，拟开发治疗小细胞肺癌（SCLC）患者。公开资料显示，这是一款抗Fucosyl-GM1单抗BMS-986012与抗PD-1单抗纳武利尤单抗的固定剂量组合，已经在国际范围内进入3期临床研究阶段。 神经节苷脂Focusyl-GM1 （FucGM1）是一种肿瘤相关抗原，在约50%~90%的SCLC肿瘤中表达，但在正常组织中表达有限，使其成为免疫肿瘤学中有希望的靶点。 根据百时美施贵宝公开资料介绍，BMS-986012是一种新型的、与FucGM1特异性结合的全人源IgG1抗体。在临床前研究中，该产品显示出对表达FucGM1的肿瘤细胞系的抗体依赖性细胞毒性（ADCC）。在补体依赖性细胞毒性（CDC）和抗体依赖性细胞吞噬（ADCP）实验中也观察到该产品介导的肿瘤细胞杀伤。当BMS-986012与抗PD-1抗体联合使用时，治疗效果明显改善。 BMS-986012联合纳武利尤单抗此前已经在针对SCLC的早期临