Arbor Biotechnologies Announces $73.9 Million Series C Financing to Advance Novel Gene Editing Therapeutics

Arbor Biotechnologies宣布完成7390万美元C轮融资，以推进新型基因编辑疗法的发展。

Series C led by ARCH Venture Partners and TCGX with significant participation from existing and new investors

C轮融资由ARCH Venture Partners和TCGX领投，现有投资者和新投资者均积极参与。

Financing extends Arbor’s cash runway into 2027 and supports the clinical development of lead program ABO-101 and continued advancement of a broader portfolio of CNS-targeted gene editing therapeutics and reverse transcriptase (RT)-based editing programs

融资延长了Arbor的现金跑道至2027年，并支持主导项目ABO-101的临床开发，以及持续推进更广泛针对中枢神经系统的基因编辑治疗和基于逆转录酶（RT）的编辑项目。

CAMBRIDGE, Mass., March 18, 2025-- Arbor Biotechnologies™, a biotechnology company discovering and developing the next generation of genetic medicines, today announced the closing of a $73.9 million Series C financing to support the advancement of its pipeline of novel gene editing therapeutics targeting diseases in the liver and central nervous system (CNS).

马萨诸塞州剑桥市，2025年3月18日——Arbor生物技术公司™，一家致力于发现和开发下一代基因药物的生物技术公司，今天宣布完成了7390万美元的C轮融资，以支持其针对肝脏和中枢神经系统（CNS）疾病的新基因编辑治疗管道的进展。

ARCH Venture Partners and TCGX led the financing, with participation from new investors QIA, Partners Investment, Revelation Partners, and Kerna Ventures and existing investors, including funds managed by abrdn Inc., Ally Bridge Group, Arrowmark Partners, Deep Track Capital, Piper Heartland Healthcare Capital, Surveyor Capital (a Citadel company), Temasek, T.

ARCH Venture Partners和TCGX领投了本轮融资，新投资者QIA、Partners Investment、Revelation Partners、Kerna Ventures以及现有投资者（包括由abrdn Inc.管理的基金、Ally Bridge Group、Arrowmark Partners、Deep Track Capital、Piper Heartland Healthcare Capital、Surveyor Capital（Citadel旗下公司）、淡马锡等）参与其中。

Rowe Price Associates and Vertex Pharmaceuticals Incorporated..

普信集团和福泰制药公司。

The proceeds will support clinical development of the company's lead therapeutic candidate, ABO-101, in primary hyperoxaluria type 1 (PH1) and progression to IND/CTA filing of its first-in-class programs, including an RT editing program for a rare liver disease and a program targeting amyotrophic lateral sclerosis (ALS)..

所得款项将用于支持公司主要治疗候选药物ABO-101在原发性高草酸尿症1型（PH1）中的临床开发，并推进其首创项目进入IND/CTA申请阶段，这些项目包括针对一种罕见肝病的RT编辑计划和针对肌萎缩侧索硬化症（ALS）的计划。

“This financing is a testament to the hard work of our team as well as our consistent focus and capital-efficient execution in developing a differentiated portfolio of gene editing therapeutics with the aim of realizing a new generation of potentially curative genetic medicines for patients,” said Devyn Smith, CEO of Arbor Biotechnologies.

“这笔融资证明了我们团队的辛勤工作，以及我们在开发差异化基因编辑治疗组合方面的持续专注和资本高效执行力，旨在为患者实现新一代潜在治愈性遗传药物，”Arbor生物技术公司首席执行官Devyn Smith表示。

“We are grateful for the support of this top-tier investor syndicate and their confidence in the Arbor team. With their backing, we are well positioned to make significant strides toward delivering novel gene editing therapeutics, including those targeting CNS diseases with high unmet need.”.

“我们感谢这一顶级投资者集团的支持，以及他们对Arbor团队的信任。在他们的支持下，我们有能力在提供新型基因编辑疗法方面取得重大进展，包括那些针对高度未满足需求的中枢神经系统疾病的疗法。”

The company’s pipeline is built upon a suite of proprietary, wholly owned genomic editors that enable a variety of functions, unlocking sophisticated and precise ways of editing the genome that offer unique properties, high specificity and broad therapeutic applications. Arbor is advancing its lead asset ABO-101—a liver-targeted gene editing therapeutic for the treatment of PH1—in RedePHine, a Phase 1/2, multi-center, open-label, dose-escalation clinical trial designed to evaluate its safety, tolerability, pharmacokinetics, pharmacodynamics, and biomarker activity in patients with PH1 (NCT06839235)..

公司管线基于一套专有的、完全自主的基因组编辑工具，能够实现多种功能，解锁复杂而精确的基因组编辑方式，具有独特的特性、高特异性和广泛的治疗应用。Arbor正在推进其领先资产ABO-101——一种针对肝脏的基因编辑疗法，用于治疗PH1——在一项名为RedePHine的1/2期、多中心、开放标签、剂量递增临床试验中进行评估，旨在评估其在PH1患者中的安全性、耐受性、药代动力学、药效学及生物标志物活性（NCT06839235）。

“Arbor is developing a differentiated portfolio with first-in-class potential to deliver on the promise of CRISPR-based genetic medicines,” said Keith Crandell, co-founder and partner at ARCH Venture Partners. “Arbor has established a track record of pipeline focus, coupled with execution and capital efficiency, to yield strong preclinical data supporting its pipeline.

“Arbor正在开发一个具有首创潜力的差异化产品组合，以实现基于CRISPR的基因药物的承诺，”ARCH Venture Partners的联合创始人兼合伙人Keith Crandell表示。“Arbor建立了专注于管线的良好记录，同时兼具执行能力和资本效率，从而获得了支持其管线的强有力临床前数据。”

We are impressed with the team’s progress to date and are proud to support the advancement of these programs.”.

我们对团队迄今为止的进展印象深刻，并为支持这些项目的推进感到自豪。

About ABO-101

关于ABO-101

ABO-101 is a novel, investigational gene editing medicine designed to be a one-time liver-directed gene editing treatment that results in a permanent loss of function of the HAO1 gene in the liver to reduce primary hyperoxaluria type 1 (PH1)-associated oxalate production. PH1 is a rare genetic disorder in which enzyme deficiencies in the liver lead to the overproduction and buildup of oxalate, resulting in kidney stones eventually leading to end stage kidney disease and systemic oxalosis.

ABO-101 是一种新型的在研基因编辑药物，旨在通过一次性靶向肝脏的基因编辑治疗，永久性破坏肝内 HAO1 基因的功能，从而减少原发性高草酸尿症 1 型（PH1）相关的草酸生成。PH1 是一种罕见的遗传性疾病，由于肝脏中的酶缺乏导致草酸过量产生和积累，引发肾结石，最终导致终末期肾病和全身性草酸中毒。

ABO-101 is designed to knock down HAO1 gene expression in the liver, thereby providing durable reduction in oxalate production. ABO-101 consists of a lipid nanoparticle (LNP), licensed from Acuitas Therapeutics, encapsulating messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA which specifically targets the human HAO1 gene.

ABO-101旨在抑制肝脏中HAO1基因的表达，从而持久降低草酸盐的产生。ABO-101由一种脂质纳米颗粒（LNP）组成，该颗粒从Acuitas Therapeutics获得许可，内含表达新型V型CRISPR Cas12i2核酸酶的信使RNA以及优化的引导RNA，专门靶向人类HAO1基因。

ABO-101 is currently under evaluation in RedePHine, a Phase 1/2, multi-center, open-label, dose-escalation clinical trial designed to study its safety, tolerability, pharmacokinetics, pharmacodynamics, and biomarker activity in patients with PH1 (NCT06839235). ABO-101 has been granted orphan drug designation (ODD) and rare pediatric disease designation (RPDD) by the US FDA for the treatment of PH1..

ABO-101目前正在RedePHine进行评估，这是一项1/2期、多中心、开放标签、剂量递增的临床试验，旨在研究其在PH1患者中的安全性、耐受性、药代动力学、药效学及生物标志物活性（NCT06839235）。ABO-101已被美国FDA授予孤儿药资格（ODD）和罕见儿科疾病资格（RPDD），用于治疗PH1。

About Arbor Biotechnologies, Inc.

关于Arbor生物技术公司

Arbor Biotechnologies™, a next-generation gene editing company based in Cambridge, MA, is advancing a pipeline of novel gene editing therapeutics to address a wide range of genetic conditions – from the ultra-rare to the most common genetic diseases. The company’s unique suite of optimized gene editors, which is capable of approaches ranging from gene knockout, excisions, reverse transcriptase editing, and large gene insertion, goes beyond the limitations of early editing technologies to unlock access to new gene targets and has fueled a robust pipeline of first-in-class assets focused on diseases of high unmet need.

Arbor Biotechnologies™ 是一家位于马萨诸塞州剑桥市的下一代基因编辑公司，正在推进一系列新型基因编辑治疗药物的开发，以应对从极罕见到最常见的遗传疾病等广泛遗传条件。该公司独特的优化基因编辑器组合能够实现多种方法，包括基因敲除、切除、逆转录酶编辑和大片段基因插入，突破了早期编辑技术的局限性，解锁了对新基因靶点的访问，并推动了一个专注于高未满足需求疾病的首创资产的强大管线。

With Arbor’s lead program, ABO-101 for the treatment of primary hyperoxaluria type 1, in clinical development, the company continues to focus their research and development efforts on genomic diseases of the liver and CNS for which there are no existing functional cures. For more information, please visit: arbor.bio..

随着Arbor公司主导项目ABO-101（用于治疗原发性高草酸尿症1型）进入临床开发阶段，该公司继续将其研发努力集中在目前尚无功能性治愈方法的肝脏和中枢神经系统的基因组疾病上。欲了解更多信息，请访问：arbor.bio。

Contact:

联系人:

Media Contact:

媒体联系人：

Peg Rusconi

佩格·鲁斯科尼

Deerfield Group

德菲尔德集团

prusconi@deerfieldgroup.com

prusconi@deerfieldgroup.com