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基因编辑技术研究商Specific Biologics宣布完成3500万美元A轮融资,以推进肌萎缩侧索硬化症(ALS)基因编辑疗法的研发

Specific Biologics Announces Oversubscribed US$35 Million Series A Financing to Advance Gene-Editing Therapy for ALS

CISION 等信源发布 2026-11-02 23:00

可切换为仅中文


Financing led by Hatteras Venture Partners, with participation from foundational investors Lumira Ventures and adMare BioInnovations and a new syndicate of life sciences investors

本轮融资由哈特拉斯风险投资公司(Hatteras Venture Partners)领投,基石投资者卢米拉风险投资公司(Lumira Ventures)和adMare生物创新公司(adMare BioInnovations)以及一个新组成的生命科学投资者财团参与投资。

Proceeds will be used to advance lead Dualase® therapeutic candidate through initial clinical biomarker proof-of-concept in patients with C9ORF72-associated ALS

所得款项将用于推进领先的双酶(Dualase®)治疗候选药物,在携带C9ORF72基因突变的肌萎缩侧索硬化症(ALS)患者中开展初步临床生物标志物概念验证研究。

Gene-editing platform designed to precisely remove disease-causing DNA repeat expansions, with potential applications across more than 40 repeat expansion disorders

基因编辑平台,旨在精确去除致病性DNA重复扩增,在40多种重复扩增疾病中具有潜在应用前景

TORONTO

多伦多

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Nov. 2, 2026

2026年11月2日

/PRNewswire/ -- Specific Biologics, a biotechnology company pioneering a new generation of precision gene-editing therapies for serious genetic diseases, today announced the closing of an oversubscribed US$35 million Series A financing. The round was led by Hatteras Venture Partners, with participation from foundational investors Lumira Ventures and adMare BioInnovations, alongside new investors Genesys Capital, Business Development Bank of Canada's (BDC) Life Sciences Venture Fund, DeepWork Capital and Seido Capital. .

/美通社/ -- 专注于为严重遗传性疾病开发新一代精准基因编辑疗法的生物技术公司 Specific Biologics 今日宣布,已完成超额认购的3500万美元A轮融资。本轮融资由 Hatteras Venture Partners 领投,创始投资者 Lumira Ventures 和 adMare BioInnovations 参与跟投,新投资者 Genesys Capital、加拿大商业发展银行(BDC)生命科学风险投资基金、DeepWork Capital 以及 Seido Capital 也参与了本轮投资。

The financing will support development of Specific Biologics' lead Dualase® therapeutic candidate through initial clinical biomarker proof-of-concept in the first cohort of an adaptive Phase I/II clinical trial in patients with C9ORF72-associated amyotrophic lateral sclerosis (ALS), a neurodegenerative disease for which there are currently no approved therapies..

这笔融资将支持Specific Biologics公司的主要Dualase®治疗候选药物的开发,直至在C9ORF72基因相关肌萎缩侧索硬化症(ALS)患者开展的适应性I/II期临床试验的首个队列中完成初步临床生物标志物概念验证。肌萎缩侧索硬化症是一种神经退行性疾病,目前尚无获批的治疗药物。

'This financing represents a defining step toward our goal of bringing a potentially transformative medicine to people living with ALS,' said Brent Stead, Chief Executive Officer of Specific Biologics. 'Our goal is to address the disease at its genetic source to create a new therapeutic option for patients who have waited far too long.'.

“此次融资是我们向将一种可能具有变革性的药物带给肌萎缩侧索硬化症(ALS)患者这一目标迈出的关键一步,”Specific Biologics 首席执行官布伦特·斯蒂德表示。“我们的目标是从基因根源上应对这种疾病,为那些等待已久的患者创造一种新的治疗选择。”

'Gene editing has the potential to transform the treatment of genetic diseases, but realizing that promise requires overcoming longstanding challenges in precision, specificity and delivery,' said Ben Scruggs, Partner at Hatteras Venture Partners. 'Specific Biologics has developed a highly differentiated technology with significant therapeutic and commercial potential designed to address these challenges, supported by compelling preclinical evidence.'.

“基因编辑具有变革遗传性疾病治疗的潜力,但要实现这一前景,需要克服在精准性、特异性和递送方面长期存在的挑战,”Hatteras Venture Partners 的合伙人 Ben Scruggs 表示。“Specific Biologics 开发了一种高度差异化的技术,旨在应对这些挑战,该技术具有显著的治疗和商业潜力,并得到了令人信服的临床前证据的支持。”

'Our decision to invest meaningful additional capital in Specific reflects our growing conviction in the scientific differentiation, clinical potential and long-term value of the Dualase® platform,' said Nikhil Thatte, Partner at Lumira Ventures. 'We believe the opportunity to generate early clinical proof-of-concept in ALS patients represents an important inflection point for the company and a meaningful step toward realizing the broader promise of its technology.'.

Lumira Ventures 合伙人 Nikhil Thatte 表示:“我们决定对 Specific 进行大规模追加投资,反映了我们对 Dualase® 平台在科学差异化、临床潜力和长期价值方面的信心日益增强。我们认为,在肌萎缩侧索硬化症(ALS)患者中实现早期临床概念验证,为公司带来了重要的转折点,也是迈向实现其技术更广泛前景的重要一步。”

Specific Biologics' proprietary Dualase® platform is designed to overcome important limitations of conventional gene-editing technologies by precisely removing disease-causing DNA repeat expansions while preserving the normal sequences required for biological function. These abnormal stretches of repetitive DNA are implicated in more than 40 genetic disorders.

Specific Biologics 公司专有的 Dualase® 平台旨在克服传统基因编辑技术的重要局限性,通过精准切除致病性 DNA 重复扩增序列,同时保留生物功能所需的正常序列。这些异常的重复 DNA 片段与 40 多种遗传性疾病相关。

Specific has demonstrated editing in preclinical studies in multiple neuromuscular repeat expansions..

Specific 已在多种神经肌肉重复扩增疾病的临床前研究中证实了其编辑效果。

'Specific Biologics has assembled a dynamic and experienced team in the field of genome editing and an excellent syndicate of investors with the strategic vision to translate breakthrough science into clinical proof of concept,' said Paul Hastings, Board Chair of Specific Biologics. 'Their technology has the potential to redefine what is possible in gene editing and open the door to therapeutic approaches for devastating genetic conditions that have historically been beyond reach.'.

Specific Biologics 董事会主席保罗·黑斯廷斯表示:“Specific Biologics 已组建了一支在基因组编辑领域充满活力且经验丰富的团队,并汇聚了一批具备战略视野的优秀投资者联盟,致力于将突破性科学成果转化为临床概念验证。他们的技术有望重新定义基因编辑的可能性,并为那些历来难以企及的毁灭性遗传疾病开辟新的治疗途径。”

About ALS and Dualase®

关于ALS和Dualase®

ALS is a progressive and ultimately fatal neurodegenerative disease characterized by the loss of motor neurons responsible for voluntary muscle movement, including speaking, swallowing and breathing. An expansion of repeated DNA sequences in the C9ORF72 gene is the most common known inherited genetic cause of ALS and is also associated with frontotemporal dementia..

肌萎缩侧索硬化症(ALS)是一种进行性且最终致命的神经退行性疾病,其特征是负责随意肌运动(包括说话、吞咽和呼吸)的运动神经元丧失。C9ORF72基因中重复DNA序列的扩增是已知最常见的ALS遗传病因,也与额颞叶痴呆相关。

Specific Biologics' lead Dualase® candidate is designed to precisely shorten the pathogenic C9ORF72 repeat expansion at the DNA level without off-target effects, offering the potential for durable therapeutic benefit by addressing the underlying genetic driver of disease rather than solely its downstream effects.

Specific Biologics 的主要候选药物 Dualase® 旨在从 DNA 水平精准缩短致病的 C9ORF72 重复扩增,且无脱靶效应,通过针对疾病的根本遗传驱动因素而非仅其下游效应,有望提供持久的治疗获益。

Unlike larger gene editing systems, its compact size fits within a single adeno-associated virus (AAV) to be delivered directly to the central nervous system to reach affected motor neurons..

与较大的基因编辑系统不同,其紧凑的尺寸可容纳于单个腺相关病毒(AAV)载体中,从而能够直接递送至中枢神经系统,以靶向受影响的运动神经元。

In preclinical dose-response studies using patient-derived ALS cell models, Dualase® achieved nearly complete collapse of the pathogenic C9ORF72 repeat expansion. Dualase® also collapses repeat-expanded C9ORF72 in disease-relevant animal models with correction of downstream hallmarks of the disease and no detectable off-target effects..

在使用患者来源的肌萎缩侧索硬化症(ALS)细胞模型的临床前剂量反应研究中,Dualase® 几乎完全消除了致病的 C9ORF72 重复序列扩增。Dualase® 还在疾病相关的动物模型中消除了 C9ORF72 的重复序列扩增,纠正了该疾病的下游标志性病理特征,且未检测到脱靶效应。

About Specific Biologics

关于特定生物制品

Specific Biologics is a venture-backed biotechnology company pioneering next-generation precision gene-editing therapies for serious genetic diseases. Its proprietary two-site Dualase® platform is designed to overcome key limitations of existing gene-editing technologies through efficient, precise and highly specific .

Specific Biologics 是一家获得风险投资支持的生物技术公司,致力于开创针对严重遗传疾病的下一代精准基因编辑疗法。其专有的双位点 Dualase® 平台旨在通过高效、精准且高度特异性的方式,克服现有基因编辑技术的关键局限性。

in vivo

体内

editing of challenging genetic targets. The versatile Dualase® platform supports both targeted DNA removal and repair using single AAV delivery or lipid nanoparticle-delivered mRNA, creating opportunities across multiple therapeutic areas to unlock new treatment possibilities for patients with devastating genetic disorders..

针对具有挑战性的基因靶点进行编辑。多功能 Dualase® 平台支持通过单次腺相关病毒(AAV)递送或脂质纳米颗粒递送的 mRNA,实现靶向 DNA 的移除与修复,从而在多个治疗领域创造机遇,为患有严重遗传性疾病的患者开启新的治疗可能。

For more information, visit

欲了解更多信息,请访问

www.specificbiologics.com

www.specificbiologics.com

or follow Specific Biologics Inc. on LinkedIn at

或在 LinkedIn 上关注 Specific Biologics Inc.,网址为

www.linkedin.com/company/specific-biologics

www.linkedin.com/company/specific-biologics

SOURCE Specific Biologics Inc.

来源:Specific Biologics Inc.