PHILADELPHIA, Feb. 01, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of idiopathic inflammatory myopathies (IIM, or myositis).

费城，2024年2月1日（环球通讯社）--Cabaletta Bio，Inc.（纳斯达克：CABA），一家临床阶段生物技术公司，专注于开发和推出首个针对自身免疫性疾病患者的治疗性靶向细胞疗法，今天宣布，美国食品和药物管理局（FDA）已授予CABA-201孤儿药名称（ODD），一种含有4-1BB的全人CD19-CAR T细胞研究疗法，用于治疗特发性炎性肌病（IIM或肌炎）。

CABA-201 is in development as a potential treatment for autoimmune diseases driven by B cells. Four RESET™ (REstoring SElf-Tolerance) Phase 1/2 trials are advancing for the evaluation of CABA-201 across multiple autoimmune conditions, including the Phase 1/2 RESET-Myositis™ trial. “Myositis, believed to be driven by B cells, is a severe and potentially fatal autoimmune disease for which no curative therapy exists.

CABA-201正在开发中，作为由B细胞驱动的自身免疫性疾病的潜在治疗方法。四重复位™ （恢复自我耐受）1/2期试验正在推进，以评估CABA-201在多种自身免疫疾病中的作用，包括1/2期重置肌炎™ 审判。“肌炎被认为是由B细胞驱动的，是一种严重且可能致命的自身免疫性疾病，目前尚无治疗方法。

Current treatment options provide modest efficacy, with a significant portion of diagnosed patients having an inadequate response to treatment, thus, there is a clear need for innovative medicines that can meaningfully change the treatment paradigm,' said David J. Chang, M.D., Chief Medical Officer of Cabaletta.

Cabaletta首席医学官David J.Chang医学博士说，目前的治疗方案疗效不高，大部分确诊患者对治疗反应不足，因此，显然需要创新药物来有意义地改变治疗模式。

“CABA-201 is designed to deeply and transiently deplete CD19-positive B cells, which may enable an immune system reset, and has the potential to deliver durable remission off therapy in patients diagnosed with myositis and other autoimmune diseases where B cells play a role. Orphan Drug Designation is an important recognition for investigational therapies for rare diseases and provides us with potentially valuable benefits as we seek to make a difference in the lives of patients and develop the first targeted, and potentially curative, cell therapy fo.

“CABA-201旨在深度和短暂地消耗CD19阳性B细胞，这可能使免疫系统重置，并有可能为诊断为肌炎和其他自身免疫性疾病（B细胞起作用）的患者提供持久的缓解治疗。孤儿药物指定是对罕见疾病研究疗法的重要认可，并为我们提供潜在的有价值的好处，因为我们试图改变患者的生活，并开发出第一种有针对性的，潜在治愈性的细胞疗法。