Antion Biosciences SA

We are pleased to announce our partnership with Noble Capital Markets Inc., a U.S. investment bank with extensive expertise in biotech financing. This collaboration aligns with PolTREG’s strategic plans for growth in the United States. “PolTREG is financially stable, with secured funding for our priority projects in the coming quarters. Over the years, we have developed a robust platform for multiple cell therapies, including polyclonal TREGs, CAR-TREGs, allogeneic TREGs, antigen-specific TREGs, and TCR TREGs. To fully unlock our potential, we are focusing on the U.S. market, which offers tremendous opportunities for capital access and collaborations with global pharmaceutical leaders. After numerous discussions with investment banks and financial advisors, we chose Noble Capital Markets for their deep knowledge and experience in the biotech sector.” – Prof. Piotr Trzonkowski, CEO & Co-founder of PolTREG S.A. Our plans for growth in the U.S. also include recently signed agreements: ✅ In March, PolTREG established a strategic collaboration with Antion Biosciences (Switzerland) to develop the next generation of allogeneic TREG therapies ✅ In February, we began cooperation with Kinexum Services LLC (U.S.) for strategic regulatory advisory regarding TREG therapy registration in the United States. We are also engaged in discussions with leading U.S. clinical centers to explore potential collaborations.

We are proud to announce our collaboration with Antion Biosciences, a Swiss biotechnology company at the forefront of gene silencing technology, to develop next-generation cell therapies that have the potential to transform the treatment landscape for patients worldwide. The expected results of the cooperation will be engineered allogeneic off-the-shelf CAR-Tregs products with enhanced immunomodulatory activity in the treatment of autoimmune and/ or neurodegenerative diseases. “The collaboration with Antion Biosciences places PolTREG among an elite group of companies developing allogeneic cell therapies. Treatments based on cells derived from a donor other than the patient offer greater scalability and accessibility, making them highly attractive to the big pharma sector. This strategic partnership significantly strengthens our capabilities in developing next-generation cell therapies. PolTREG is the only company in the world that combines long-term clinical data confirming the safety and efficacy of polyclonal Treg therapies with a broad portfolio of next-generation technologies, including allogeneic Tregs, CAR-TREGs, antigen-specific Tregs, and TCR TREGs” – said Prof. Piotr Trzonkowski, CEO of PolTREG. The partnership will harness Antion’s cutting-edge microRNA (miRNA)-mediated gene silencing platform to enhance PolTREG’s extensive pipeline of CAR-Treg-based therapies. Antion’s proprietary technology enables highly efficient, tunable, and multiplex engineering of cell and gene therapy products, allowing for unprecedented precision in controlling gene expression within complex cellular environments. “We are excited to partner with PolTREG, a global leader in Treg-based therapies, to combine our innovative miRNA-mediated gene silencing platform with their unmatched expertise and clinical experience in Tregs cell therapy development. This partnership represents a powerful synergy, enabling us to push the boundaries of allogeneic cell therapy innovation and redefine what’s possible in the treatment of autoimmune and / or neurodegenerative diseases”- said Antonija Sakic, Chief Business and Corporative Development Officer of Antion.

▎药明康德内容团队编辑 日前，邦耀生物（BRL Medicine）宣布其所开发的新一代同种异体通用型CAR-T疗法TyU19用于治疗自身免疫疾病的研究成果发布于国际顶级学术期刊《细胞》上。分析显示，TyU19能够在两周内使得3名难治性自身免疫疾病患者的B细胞耗竭，并逆转了两名弥漫性皮肤系统性硬化症（SSc）患者体内器官的大面积纤维化损伤。根据新闻稿，TyU19是在治疗自身免疫疾病研究中获得积极结果的全球首个同种异体通用型CAR-T疗法。 自身免疫疾病患者的免疫耐受受损，临床上常使用免疫抑制剂来治疗自身免疫疾病，但这类疗法存在治疗周期长、易复发、感染及肿瘤风险增加，及部分患者不能耐受或疗效不佳的情况。B细胞靶向单抗提供了新的治疗选择，但依然存在感染、停药后复发，及部分患者疗效不佳等问题。鉴于B细胞在自身免疫疾病中的重要作用，已有不少研究人员尝试将CAR-T疗法应用于自身免疫疾病，并展现出优异的安全性和有效性。然而，无论是针对肿瘤还是自身免疾病适应症，已上市或在研产品仍集中于自体来源的CAR-T疗法，这类疗法的生产较为耗时，且患者多需进行深度的清淋或者免疫抑制处理。 这次所公布的试验是一项由研究者发起的研究，旨在评估TyU19用于治疗复发性或难治性自身免疫疾病患者的疗效与安全性，预计招募包含系统性红斑狼疮（SLE）、干燥综合征、系统性硬化症、炎性肌病、ANCA相关系统性血管炎、抗磷脂综合征患者。在这次所发布的结果当中，一名难治性免疫介导的坏死性肌病（IMNM）患者和两名系统性硬化症患者接受了TyU19单次输注。 分析显示，CAR-T细胞在输注超过3个月后仍持续存在于患者体内，并在2周内实现了完全的B细胞耗竭。在6个月的随访期间，通过对两种疾病的临床缓解指数评分进行分析，所有三名患者均达到疾病深度缓解。 IMNM受试者为42岁的女性。影像及病理检查显示，她在接受TyU19治疗后肌肉炎症得到显著缓解，严重骨骼肌损伤获得改善，其总改善评分（TIS）迅速（2个月）从基线的72.5上升至100，并在随后的随访期间持续保持这一水平。 另外2名患有严重系统性硬化症的受试者，在接受TyU19细胞治疗后，所有患者的CRISS评分在几个月内得到显著提高；且在皮肤纤维化方面，2名患者修改后的Rodnan皮肤评分（mRSS）均显著下降。此外，患者的重要器官纤维化损伤也均得到了逆转，并在6个月的监测期内持续改善。 ▲接受TyU19治疗的IMNM与SSc患者均获得缓解（图片来源：参考资料[2]） 整个治疗过程耐受性良好，在三位患者中均未观察到细胞因子释放综合征（CRS）、移植物抗宿主病（GvHD）、免疫效应细胞相关神经毒性综合征（ICANS）。该结果显示，通用型CAR-T细胞在治疗严重难治性自身免疫疾病方面具有高度的安全性，并具潜在的免疫调节效果。 TyU19（BRL-301）是通过邦耀生物专有TyUCell通用型细胞平台所开发的同种异体CD19靶向CAR-T疗法。该平台通过CRISPR-Cas9技术，对来自健康供体的细胞进行多基因、选择性编辑，以预防异体细胞移植中可能存在的移植物抗宿主病风险，并避免患者免疫系统对异体细胞产生排异现象。该疗法无需对患者进行HLA分型筛选，或进行如自体CAR-T疗法患者所接受的深度清淋预处理（仅接受常规甚至强度更低的清淋方案），就可以完成在患者体内的快速扩增和长期维持。 ▲TyU19细胞疗法设计示意图（图片来源：参考资料[2]） 除了邦耀生物，许多生物医药公司也投入开发用于治疗自身免疫疾病同种异体通用型CAR-T疗法的行列。今年4月，Caribou Biosciences为其同种异体CD19靶向CAR-T疗法CB-010递交的IND申请获得美国FDA许可，用于治疗狼疮性肾炎（LN）和肾外狼疮（ERL），预计该1期临床试验GALLOP将于今年底前启动。今年3月，Atara Biotherapeutics也宣布其在研同种异体、CD19靶向CAR-T疗法ATA3219用于治疗LN的IND申请已经获得美国FDA的许可。Allogene Therapeutics利用其AlloCAR-T平台所开发、用于治疗SLE的首款CD19/CD70靶向CAR-T疗法ALLO-329也预计于2025年初进入1期临床试验。 此外，Kyverna Therapeutics与Intellia Therapeutics也正在共同开发同种异体CD19靶向CAR-T细胞疗法KYV-201用于治疗多种自身免疫疾病，该疗法目前于临床前阶段接受评估。Antion Biosciences公司也在近期宣布将开发新一代同种异体CD19靶向CAR-T疗法，用于治疗B细胞驱动的自身免疫疾病。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] Wang X. et al. Allogeneic CD19-targeted CAR-T therapy in patients with severe myositis and systemic sclerosis. (2024) Cell. DOI:https://doi.org/10.1016/j.cell.2024.06.027 [2] 国际首次！邦耀生物UCAR-T研究成果荣登Cell，聚焦自免疾病治疗取得重大突破. Retrieved July 16, 2024 from https://www.prnasia.com/story/454188-1.shtml [3] Universal CAR-T Cells (BRL-301) in Relapse or Refractory Autoimmune Diseases. Retrieved July 16, 2024 from https://clinicaltrials.gov/study/NCT05859997?term=NCT05859997&rank=1 [4] POS0462 PRECLINICAL DEVELOPMENT OF KYV-201, AN INVESTIGATIONAL ALLOGENEIC ANTI-CD19 CAR T CELL FOR THE TREATMENT OF AUTOIMMUNE DISEASE. Retrieved July 16, 2024 from https://ard.bmj.com/content/83/Suppl_1/1129 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新