从2017年首款CAR-T疗法获批以来，FDA累计已经批准6款CAR-T疗法，分别是诺华的Kymriah、传奇/强生的Carvykti、吉利德的Yescarta和Tecartus、百时美施贵宝的Breyanzi和Abecma。2024年上半年，6款CAR-T疗法合计销售额20亿美元，预计全年将超过40亿美元。 CAR-T疗法在血液瘤取得了里程碑般的临床突破，但复杂的个体化制备过程和高额的制备成本，严重限制了CAR-T疗法的普及，在支付天花板较低的国内市场就更是如此。在这种背景下，通用型CAR-T替代自体CAR-T的系统性优势就尤为明显，可以批量化制备，成本更低且患者无需等待。 然而尽管优势明显潜力巨大，通用型CAR-T疗法的道路却充满挫折，这其中宿主抗移植物反应 （HvGR）是亟待解决的痛点，它会导致通用型CAR-T缓解持久度差的问题。具体而言，通用型CAR-T由于采用了异体来源的T细胞，给药治疗后会引起患者的T细胞、NK细胞攻击异体的CAR-T细胞，导致通用型CAR-T被排异，从而影响疗效。 多家生物技术公司通过结合多种不同策略来尝试攻克HvGR，目前仍在探索中。大多公司的uC

Oct. 31, 2024 -- Tizona Therapeutics, Inc., a privately held, clinical-stage company developing cancer immunotherapies, today announced the initiation of two randomized arms within the ongoing Phase 1b clinical trial of TTX-080, a novel, first-in-class antibody targeting HLA-G. Patients with biomarker-defined metastatic colorectal cancer (mCRC) will be enrolled to receive either TTX-080 plus cetuximab and FOLFIRI or cetuximab and FOLFIRI alone in the second-line setting. “Although screening an

Oct. 31, 2024 -- Werewolf Therapeutics, Inc. (the “Company” or “Werewolf”) (Nasdaq: HOWL), an innovative biopharmaceutical company pioneering the development of conditionally activated therapeutics engineered to stimulate the body’s immune system for the treatment of cancer and other immune-mediated conditions, today announced the addition of WTX-921, a novel IL-10 INDUKINE development candidate for Inflammatory Bowel Disease (IBD) and potentially other inflammatory diseases, to the Company’s p

Participants in the Phase 2a study experienced rapid and meaningful decreases in depressive symptoms Suicidal ideation decreased by 80% SPN-820 was well-tolerated with few adverse events SPN-820 is a novel, first-in-class intracellular modulator of mTORC1 for the treatment of depression Oct. 31, 2024 -- Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, announce

Acquisition strengthens Lyell’s clinical pipeline with the addition of IMPT-314, a dual-targeting CD19/CD20 CAR T-cell product candidate expected to initiate pivotal development in 2025 in patients in the 3rd line setting with B-cell non-Hodgkin lymphoma who have not yet been exposed to CAR T-cell therapy Sumant Ramachandra, M.D., Ph.D., MBA, appointed to Lyell’s Board of Directors Oct. 31, 2024 -- Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical-stage T-cell reprogramming company advancing

11月1日，南京宁丹新药技术有限公司（简称“宁丹新药”）刚刚宣布完成数亿元人民币的C轮融资。本轮融资由南京江宁高新区科创投和华兴康平共同完成，募集资金将主要用于加速该公司核心管线的开发与上市进程、自主创新平台建设及公司全球化布局。 这也是宁丹新药在今年内完成的又一轮融资。今年1月，宁丹新药宣布超募完成B（含B+轮）融资，合计募集人民币资金逾2亿元，由腾讯投资、康哲药业之子公司、中信建投投资、华兴康平等新股东共同投资，原股东恒荣投资继续加持。 宁丹新药是一家聚焦于中枢神经系统疾病（CNS）领域新药研发和产业化的创新型公司，一直致力于为广大CNS疾病患者寻找更优的疾病解决方案。该公司在研管线主要用于治疗卒中、颅内肿瘤、认知障碍、情感障碍、神经病理性疼痛、脑小血管病等多个高发中枢相关疾病，目前分别处于临床中和临床前研究阶段。 根据宁丹新药公开资料，该公司进展最快的项目Y-2是一款脑卒中改良型创新药物。这是先声药业与宁丹新药合作开发的依达拉奉右莰醇舌下片，为一种脑细胞保护剂，含依达拉奉和右莰醇两种活性成分，具有抗氧化、抗炎和协同增效作用，能够显著减少AIS导致的脑细胞损伤。该药还具有独特

11月1日，中国国家药监局药品审评中心（CDE）官网最新公示，由艾司林科（苏州）生物医药有限公司申报的AL01211胶囊拟纳入突破性治疗品种，针对适应症为法布雷病。公开资料显示，这是AceLink Therapeutics在研的一款口服非脑渗透性GCS（葡萄糖神经酰胺合酶）抑制剂。它有望为目前需频繁静脉注射酶替代疗法的法布雷病患者提供一种口服小分子疗法的潜在选择。 法布雷病是一种罕见的X连锁遗传性溶酶体蓄积病，患者因为α-半乳糖苷酶A基因突变，基因产物结构和功能异常，使得代谢底物鞘氨醇三聚己糖苷和相关鞘糖脂在心、肾、肺、眼、脑和皮肤等多种器官的神经及血管组织细胞中堆积，导致心室肥厚、肾衰、脑卒中、外周神经疼痛等病症发生。目前，该疾病的传统治疗方案为酶替代疗法，患者需要频繁输入酶替代疗法（ERT）且终身治疗，对其正常生活、工作、学习影响很大。 AceLink Therapeutics成立于2018年，该公司对糖脂生物学有着深刻理解，并以此为基础开发糖脂代谢类疾病突破性疗法。AL01211是该公司主要产品，为一种口服糖基神经酰胺合成酶（GCS）抑制剂，拟开发治疗法布雷病和1型戈谢病。

Preclinical data from ex vivo human islet experiments showed that icovamenib (BMF-219) was able to enhance the activity of glucagon-like peptide-1 (GLP-1)-based therapies, potentially leading to increased insulin secretion and improved glycemic control in patients with diabetes Phase II study (COVALENT-211), combining icovamenib with a GLP-1-based therapy, planned to begin in 2025 BMF-650, an investigational next-generation, oral small-molecule GLP-1 receptor agonist (GLP-1 RA), demonstrated pos

The Data Monitoring Committee recommended to continue the clinical trial without modification of the current protocol, based on the pre-planned review of safety data. The recommendation was based on the unblinded review by the DMC of safety data from more than 1000 patients randomized in the main and exploratory cohorts, including over 800 and 170 patients that have been treated for more than 24 and 72 weeks, respectively. This fifth DMC review confirms the good safety profile of lanifibranor. N

Dose-dependent increase in neuroprotective APOE2 expression in all participants with ongoing durability at 12 months Consistent reductions across CSF tau biomarkers and tau PET in majority of participants LX1001 well tolerated across all dose cohorts with no reports of amyloid-related imaging abnormalities (ARIA) Company to host webcast today at 7:00 AM ET Oct. 30, 2024 -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering treatments for g