Arbor Biotechnologies
Cambridge, US Β· Founded 2016 Β· Delaware corporation Β· 102 employees on LinkedIn Β· 23 known investors
Arbor develops programmable DNA editors for genetic medicine, aiming to enable functional cures for patients by addressing genetic diseases through bespoke gene editing approaches. The company is developing a portfolio of DNA editing technologies capable of targeting over 90% of the human genome for therapeutic applications.
Also known as Arbor Bio Β· Arbor Biotechnologies, Inc.
Founders & leadership
Arbor Biotechnologies was founded in 2016 by DAVID ARTHUR SCOTT, WINSTON XIA YAN, and FENG ZHANG.
Board
Investors Β· 23
Also in the syndicate Β· 18
Funding
SEC filings, press & company announcements$230.6M disclosed across 2 of 5 rounds Β· 2017β2025
- Undisclosed amountSeries CMar 2025Source β
βΆ$215MraisedNov 2021 Β· 37 investors Β· BiotechnologyRule 506(b)
- DAVID CHENGExecutive Officer
- PAUL MEISTERDirector
- RAMI HARAWIDirector
- KEITH CRANDELLDirector
- KELLY MORGANExecutive Officer
- JOHN MURPHYExecutive Officer
- MARK ANGELINODirector
- PAM STETKIEWICZExecutive Officer
- DEVYN SMITHExecutive Officer, Director
- CHEN YUDirector
- DAVID WALTDirector
- Offering amount
- $215M
- Amount sold
- $215M
- First sale
- Oct 2021
- Incorporated
- Corporation, Delaware
- Federal exemptions
- 06b
βΆ$15.6MraisedAug 2017 Β· 10 investors Β· BiotechnologyRule 506(b)
- KEITH CRANDELLDirector
- DAVID R. WALTDirector
- DAVID ARTHUR SCOTTExecutive Officer
- WINSTON XIA YANExecutive Officer
- ANNIE HAZLEHURSTDirector
- FENG ZHANGDirector
- Offering amount
- $15.6M
- Amount sold
- $15.6M
- First sale
- May 2017
- Incorporated
- Corporation, Delaware, 2016
- Federal exemptions
- 06b
Source: SEC EDGAR Form D. Amounts as filed; amended filings shown once at their latest values.
Company profile
researched Aug 2026Arbor Biotechnologies is a Cambridge, Massachusetts biotechnology company discovering and developing next-generation genetic medicines based on programmable DNA editors. It was founded in 2016 by Feng Zhang, David Walt, David Scott and Winston Yan, and describes a wholly owned portfolio of proprietary editors capable of addressing more than 90% of the human genome, supporting gene knockout, excision, reverse transcriptase (RT) editing and large gene insertion.
The company's pipeline is focused on in vivo genetic medicines for liver and central nervous system diseases, with the aim of one-time dosing and durable effect. Its lead candidate, ABO-101, is a liver-directed therapy for primary hyperoxaluria type 1 (PH1) that consists of a lipid nanoparticle, licensed from Acuitas Therapeutics, encapsulating mRNA encoding a Type V CRISPR Cas12i2 nuclease plus an optimized guide RNA targeting the human HAO1 gene; knocking down HAO1 is intended to durably reduce oxalate overproduction. ABO-101 is being evaluated in RedePHine, a Phase 1/2 multi-center, open-label, dose-escalation trial (NCT06839235) assessing safety, tolerability, pharmacokinetics, pharmacodynamics and biomarker activity, and has received US FDA orphan drug and rare pediatric disease designations. Other disclosed programs include ABO-103 (undisclosed liver indication, RT editing, LNP) and CNS programs ABO-202 (ALS, STMN2), ABO-203 (ALS, undisclosed target), ABO-204 (ALS, SOD1) and ABO-206 (undisclosed, compact RT editing), all AAV-delivered. Arbor does not develop cell-based therapeutics internally, instead partnering with companies that have ex vivo expertise across autoimmune, oncology and other indications.
Founding story
Arbor was founded in 2016 by Feng Zhang, David Walt, David Scott and Winston Yan. Zhang is a core member of the Broad Institute of MIT and Harvard and serves as Arbor co-founder and Scientific Advisory Board chair; Walt is core faculty at the Wyss Institute at Harvard and a professor at Harvard Medical School and Brigham and Women's Hospital.
Business model
Arbor is a therapeutics developer that builds a wholly owned platform of CRISPR-based genomic editors and advances its own in vivo pipeline in liver and CNS diseases, while monetizing platform breadth through licensing and collaboration agreements with partners in areas it does not pursue internally, such as ex vivo cell therapy.
Sources describe partnership, licensing, co-development/co-profit-share and option agreements with companies including Vertex Pharmaceuticals, Lonza, Edigene, Ginkgo Bioworks, 4DMT, Allogene and Chiesi Group; no revenue figures are disclosed in the sources.
Traction
Arbor has raised a $15.6M Series A, a $215M Series B and a $73.9M Series C, with more than $300 million raised as of the Series B. ABO-101 has progressed through IND clearance, UK and EU regulatory clearances, FDA orphan drug and rare pediatric disease designations, and first patient dosing in July 2025 in the Phase 1/2 RedePHine trial (NCT06839235), which is planned to enroll roughly two dozen participants. The company has signed collaborations with Vertex, Lonza, Edigene, Ginkgo Bioworks, 4DMT, Allogene and Chiesi, and was named to Fierce Biotech's "Fierce 15" in 2022.
Latest developments
In March 2025 Arbor closed a $73.9 million Series C led by ARCH Venture Partners and TCGX, extending runway into 2027 and funding ABO-101 clinical development plus progression toward IND/CTA for an RT editing program in a rare liver disease and an ALS program. UK and EU clearances for ABO-101 followed in March/May 2025, first patient dosing occurred in July 2025, and in October 2025 the company established an exclusive collaboration with Chiesi Group to advance ABO-101 along with a multitarget option agreement for liver-targeted rare disease therapies.
βΈFull profile β market position, technology, go-to-market, geography, history, risks & controversies
Market position
Arbor competes in CRISPR-based genetic medicine. For PH1 it positions ABO-101 as a potential one-time treatment against approved RNA-based medicines requiring monthly or quarterly injections, Alnylam's Oxlumo and Novo Nordisk's Rivfloza. Its 2025 Series C was raised during a broad slowdown in gene and cell therapy venture funding; BioPharma Dive reported that among roughly two dozen venture firms it tracks, only $1.4 billion went into gene and cell therapy startups in 2024, the lowest total since at least 2022.
Arbor states that its suite of optimized editors goes beyond the limitations of early editing technologies, covering more than 90% of the human genome and multiple editing modalities, allowing selection of an editing approach per disease. Its lead asset is positioned as a durable, one-time alternative to chronic injectable therapies for PH1.
Technology
Arbor's discovery engine applies machine learning and AI to a protein database the company describes as containing billions of proteins, from which it has assembled a toolbox of wholly owned CRISPR genomic editors. Disclosed capabilities include nuclease knockdown and excision, reverse transcriptase editing, compact RT editing, large gene insertion and CRISPR transposases. Delivery approaches are lipid nanoparticles for liver programs (LNP licensed from Acuitas Therapeutics for ABO-101) and AAV vectors for CNS programs, with AAV capsid access obtained through a co-development agreement with 4DMT.
Go-to-market
The company advances internal in vivo liver and CNS programs through regulatory filings and clinical trials in the US, UK and EU, and partners externally for ex vivo cell therapy and additional liver targets, including an exclusive Chiesi Group collaboration on ABO-101 and a multitarget option agreement for liver-targeted rare disease therapies.
Patients with genetic diseases, initially primary hyperoxaluria type 1 and amyotrophic lateral sclerosis, with stated plans to expand to other CNS indications such as frontotemporal dementia and deeper-brain neurodegenerative diseases; pharmaceutical and biotechnology partners license Arbor's editors for ex vivo cell therapy and other applications.
Geography
Headquartered in Cambridge, Massachusetts. Regulatory and clinical activity spans the United States, the United Kingdom and the European Union; partners are based in the US, Europe and Asia.
History
Following its 2016 founding, Arbor closed a $15.6 million Series A in May 2017 and published a series of CRISPR discovery results, including Cas13d as a compact RNA-targeting nuclease (2018), additional type V CRISPR-Cas subtypes (2018), a type V CRISPR-associated transposase system (2019) and engineered Cas12i2 for genome editing (2022). It signed a research collaboration and license agreement with Vertex Pharmaceuticals in December 2018, later expanded in 2021 (ex vivo cell therapies) and 2023 (RT editing). A $215 million oversubscribed Series B closed in November 2021, bringing total funds raised to more than $300 million at that time. Subsequent agreements included Lonza (2021), Edigene (2022), Ginkgo Bioworks (2023), 4DMT (2024), Allogene (2024) and the acquisition of Serendipity in May 2024. ABO-101 was selected as lead clinical candidate in October 2023, received IND clearance and rare pediatric disease designation in December 2024, orphan drug designation in February 2025, UK and EU clearances in spring 2025, and first patient dosing in July 2025. A $73.9 million Series C closed in March 2025, followed by an exclusive collaboration with Chiesi Group in October 2025.
Risks & controversies
In 2024 Arbor cut staff and trimmed early-stage research, which the CEO characterized as a shift from platform discovery to product development. The company operates amid a funding slump for gene and cell therapy developers, and sources note that genetic medicine developers have had difficulty winning market share on convenience, citing hemophilia gene therapies that have struggled commercially. ABO-101 remains investigational and is in early-stage clinical testing.
Compiled by commissioned research from 8 cited public sources β announcements, filings, and press listed under research sources below.
Key figures
latest reportedCompany-reported or press-reported figures, each dated to when it was claimed β not independently audited.
Founder mafia
2 people who came through Arbor Biotechnologies went on to found or lead other companies.
Competitors Β· 2
by search overlap
Companies competing with Arbor Biotechnologies for the same Google search keywords, organic and paid, via search-intersection analysis.
Timeline Β· 29
launches, deals, and filingsExclusive collaboration to support advancement of ABO-101 and a multitarget option agreement to develop novel liver-targeted therapies for rare diseases.
Series C led by ARCH Venture Partners and TCGX to fund clinical development of ABO-101 in PH1 and advance CNS and RT-editing programs; extends runway into 2027.
$73.9M source β
Company timeline lists UK and EU regulatory approvals cleared in March/May 2025.
ABO-101 granted orphan drug designation by the US FDA for treatment of PH1.
ABO-101 granted rare pediatric disease designation by the US FDA for treatment of PH1.
Arbor cut staff and trimmed early-stage research in 2024; CEO described the restructuring as a shift from platform discovery work to product development.
Oversubscribed and up-sized Series B led by Temasek, Ally Bridge Group and TCG Crossover; proceeds to advance liver and CNS programs into the clinic and expand the discovery engine. Chen Yu of TCG Crossover joined the board.
$215M source β
Arbor executed a research collaboration and license agreement with Vertex Pharmaceuticals.
Dated company events from announcements, filings, and press; legal rows summarize public dockets and regulator releases.
Legal entities Β· 1
corporate structureIn the news
βΈResearch sources Β· 8
primary sources listed
- Arbor Biotechnologiesarbor.bio Β· web
8 public sources were cited for this profile; the first-party ones are listed here.
Frequently asked questions
- What does Arbor Biotechnologies do?
- Cambridge, MA gene editing company developing CRISPR-based genetic medicines for liver and CNS diseases, led by ABO-101 for PH1.
- Who founded Arbor Biotechnologies?
- Arbor Biotechnologies was founded by DAVID ARTHUR SCOTT, WINSTON XIA YAN, FENG ZHANG in 2016.
- Who are Arbor Biotechnologies's investors?
- Arbor Biotechnologies's investors include ARCH Venture Partners, Illumina Ventures, Ono Venture Investment, Inc., TCG Crossover, Third Rock Ventures.
- How much funding has Arbor Biotechnologies raised?
- Arbor Biotechnologies has disclosed $230.6M raised across 2 of its 5 known rounds.
- Where is Arbor Biotechnologies headquartered?
- Arbor Biotechnologies is headquartered in Cambridge, US.