Actio Biosciences Secures $66 Million Series B Financing to Advance Genetics-Driven Pipeline of Small Molecule Therapeutics for Rare and Common Diseases

Actio Biosciences 获得 6600 万美元 B 轮融资，用于推进针对罕见和常见疾病的基因驱动小分子治疗药物管线。

Financing co-led by Regeneron Ventures and Deerfield Management will support advancement of lead programs, ABS-1230 and ABS-0871

再生元创投和鹿田管理共同领投的融资将支持主要项目ABS-1230和ABS-0871的进展。

Phase 1 trial initiation expected in second half 2025 for ABS-1230, a KCNT1 inhibitor for the treatment of KCNT1-related epilepsy; program granted rare pediatric and orphan drug designations by FDA

预计ABS-1230（一种用于治疗KCNT1相关癫痫的KCNT1抑制剂）的I期试验将于2025年下半年启动；该计划已获得FDA的罕见儿科疾病和孤儿药资格认定。

Enrollment ongoing in Phase 1 trial for ABS-0871, a TRPV4 inhibitor for the treatment of Charcot-Marie-Tooth disease type 2C

ABS-0871（一种用于治疗2C型Charcot-Marie-Tooth病的TRPV4抑制剂）的1期试验正在进行中。

SAN DIEGO, June 18, 2025-- Actio Biosciences, a clinical-stage biotechnology company leveraging a novel approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced the closing of a $66 million Series B financing.

圣地亚哥，2025年6月18日——Actio Biosciences是一家临床阶段的生物技术公司，利用一种新颖的遗传学和精准医学方法，开发针对罕见病和常见病共同基础生物学的新疗法，该公司今天宣布完成了6600万美元的B轮融资。

New investor Regeneron Ventures and current investor Deerfield Management co-led the financing, with participation from existing investors Canaan, Droia Ventures and Euclidean Capital..

新投资者再生元创投和现有投资者迪尔菲尔德管理公司共同领投融资，参与融资的还有现有投资者迦南、Droia Ventures和欧几里得资本。

Actio’s pipeline is led by ABS-1230, a KCNT1 inhibitor for the treatment of KCNT1-related epilepsy and other genetic epilepsies, and ABS-0871, a TRPV4 inhibitor currently being evaluated in a Phase 1 healthy volunteer clinical trial for the treatment of Charcot-Marie-Tooth disease type 2C (CMT2C) and overactive bladder..

Actio公司的主要研发管线包括ABS-1230，一种用于治疗KCNT1相关癫痫及其他遗传性癫痫的KCNT1抑制剂，以及ABS-0871，一种TRPV4抑制剂，目前正在针对治疗2C型夏科-马里-图斯病（CMT2C）和膀胱过度活动症的1期健康志愿者临床试验中进行评估。

“We have made tremendous progress across our pipeline – executing a precision medicine strategy that targets the root causes of disease through genetically informed drug development,” said David Goldstein, Ph.D., co-founder and CEO of Actio. “ABS-1230 and ABS-0871 have the potential to be transformative disease-modifying therapies in their respective rare indications and growing evidence supports expansion into broader indications.

“我们在整个研发管线中取得了巨大的进展——通过基因信息指导的药物开发，实施了一种针对疾病根本原因的精准医疗策略，”Actio的联合创始人兼首席执行官David Goldstein博士说道。“ABS-1230和ABS-0871有潜力在其各自的罕见病适应症中成为变革性的疾病修饰疗法，并且越来越多的证据支持将其扩展到更广泛的适应症。”

This new funding from industry-leading investors speaks to the value of our approach and provides us with important resources to continue advancing our programs.”.

“来自行业领先投资者的这笔新资金证明了我们方法的价值，并为我们提供了继续推进项目的重要资源。”

The Series B financing will support the advancement of Actio’s lead programs, ABS-1230 and ABS-0871.

B轮融资将支持Actio的主要项目ABS-1230和ABS-0871的进展。

ABS-1230 is a potential first-in-class oral, selective small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy, a rare and often fatal pediatric epileptic encephalopathy with a United States (U.S.) prevalence of approximately 2,500 individuals. In preclinical studies, ABS-1230 has been shown to inhibit all recurrently observed pathogenic mutations in the KCNT1 gene, making ABS-1230 broadly applicable to patients with KCNT1-positive epilepsy.

ABS-1230是一种潜在的首创口服、选择性小分子KCNT1抑制剂，用于治疗KCNT1相关癫痫。这是一种罕见且常致命的儿童癫痫性脑病，在美国（U.S.）的患病人数约为2,500人。在临床前研究中，ABS-1230已被证明能够抑制KCNT1基因中所有反复出现的致病变异，使ABS-1230广泛适用于KCNT1阳性癫痫患者。

Actio plans to initiate the healthy volunteer portion of a Phase 1 clinical trial of ABS-1230 in the second half of 2025 with plans to expand into a proof-of-concept Phase 1b study in KCNT1-related epilepsy patients in early 2026. ABS-1230 was recently granted both rare pediatric and orphan drug designations from the U.S.

Actio计划在2025年下半年启动ABS-1230的1期临床试验健康志愿者部分，并计划在2026年初扩展到针对KCNT1相关癫痫患者的1b期概念验证研究。ABS-1230最近获得了美国授予的罕见儿科疾病和孤儿药双重认定。

Food and Drug Administration (FDA)..

食品和药物管理局（FDA）。

ABS-0871 is a potential first-in-class oral, small molecule TRPV4 inhibitor for the treatment of CMT2C, a rare inherited disorder that affects both motor and sensory functions with a U.S. prevalence of approximately 2,500 individuals. Preclinical evaluation of this program in novel construct valid CMT2C rare disease models has demonstrated marked improvements in motor function and mobility, compared to untreated controls.

ABS-0871是一种潜在的首创口服小分子TRPV4抑制剂，用于治疗CMT2C型疾病，这是一种影响运动和感觉功能的罕见遗传性疾病，在美国的患病人数约为2500人。该药物在新型构建的有效CMT2C罕见病模型中的临床前评估显示，与未治疗的对照组相比，运动功能和活动能力显著改善。

Given its mechanism of action targeting TRPV4, ABS-0871 may also have therapeutic potential for the treatment of overactive bladder. Actio is currently advancing ABS-0871 in the healthy volunteer portion of a Phase 1 clinical trial with plans to expand into a Phase 1b study in patients with TRPV4+ CMT2C in 2026.

鉴于其作用机制针对TRPV4，ABS-0871也可能对治疗膀胱过度活动症具有治疗潜力。Actio公司目前正在健康志愿者参与的I期临床试验部分推进ABS-0871，并计划于2026年扩展到TRPV4+ CMT2C患者中的Ib期研究。

ABS-0871 was granted rare pediatric drug, orphan drug and fast track designations from the FDA..

ABS-0871获得了FDA授予的罕见儿科药物、孤儿药和快速通道资格。

“Actio has done an exceptional job of identifying serious unmet needs in rare disease and advancing solutions that may also be applicable to patients with more common diseases,” said Jason Fuller, Ph.D., Partner, Deerfield Management. “We’re pleased to support the company, encouraged by its progress, and excited by the team’s potential to make a difference for patients.”.

“Actio 在发现罕见病领域中未被满足的严重需求方面做出了卓越的工作，并且正在推进的解决方案也可能适用于更常见疾病的患者，”Deerfield Management 合伙人 Jason Fuller 博士表示。“我们很高兴能够支持这家公司，对其取得的进展感到鼓舞，并对团队为患者带来改变的潜力感到兴奋。”

About Actio Biosciences

关于Actio生物科学公司

Actio Biosciences is a clinical-stage company leveraging advances in precision medicine to develop new therapeutics that target shared genetics in rare and common diseases—bringing meaningful medicines from one to many. Applying its expertise in genetics, drug discovery and data sciences, Actio seeks to identify programs where both biological and technical risk can be minimized to streamline the drug development process and bring forward exceptionally potent and precisely targeted therapeutics.

Actio Biosciences是一家临床阶段的公司，利用精准医学的进展来开发针对罕见病和常见病中共有遗传因素的新疗法——将有意义的药物从个体扩展到广泛群体。通过运用其在遗传学、药物发现和数据科学方面的专业知识，Actio旨在识别那些可以最大限度降低生物学和技术风险的项目，从而简化药物开发流程，并推动高效且精准靶向的治疗方法问世。

Actio is advancing two lead rare disease programs – ABS-0871 and ABS-1230 – with first-in-class potential for the treatment of Charcot-Marie-Tooth disease, type 2C (CMT2C) and KCNT1-related epilepsy, respectively. Founded in October 2021, the San Diego-based company is guided by leaders in genetics and drug development and backed by top healthcare investors.

Actio公司正在推进两个主要的罕见病项目——ABS-0871和ABS-1230，这两个项目分别具有治疗2C型Charcot-Marie-Tooth病（CMT2C）和KCNT1相关癫痫的首创新药潜力。该公司成立于2021年10月，总部位于圣地亚哥，由遗传学和药物开发领域的领军人物指导，并受到顶级医疗保健投资者的支持。

For more information, please visit ActioBiosciences.com and follow the company on LinkedIn and X..

如需更多信息，请访问 ActioBiosciences.com 并在 LinkedIn 和 X 上关注该公司。

Contact:

联系人：

Katie Engleman, 1AB

凯蒂·恩格尔曼，1AB

katie@1abmedia.com

凯蒂@1abmedia.com