Arbor Biotechnologies Nominates Three Gene Editing Development Candidates for ALS, with Potential to Address Up to 97% of People Living with the Disease

Programs span familial and sporadic ALS, advancing Arbor’s strategic focus on the disease

First CNS investigational new drug (IND) filings are anticipated in 2027

Milestone builds on clinical development expertise demonstrated with ABO-101 and reinforces versatility of proprietary editing technologies

CAMBRIDGE, MA – October 21, 2026 – Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today announced the nomination of three CNS-focused development candidates: ABO-201 for familial ALS, and ABO-202 and ABO-203 for sporadic ALS. Together, the three programs are Arbor’s first wave of CNS candidates.  As the only editing company focused on CNS, Arbor is extending its gene editing platform to a disease area that has remained largely out of reach for existing genetic medicines. Each DC is designed to be delivered intravenously in a single AAV capsid engineered to cross the blood-brain barrier.

“These three candidates mark our entry into the central nervous system,” said Devyn Smith, Ph.D., Chief Executive Officer of Arbor Biotechnologies. “ALS has defeated one conventional approach after another, in part because the available tools could not reach the tissue or the targets that matter. Compact editors and a capsid that crosses the blood-brain barrier change what is addressable, and we are advancing this wholly owned CNS pipeline toward the clinic with the same discipline we brought to ABO-101.”

Arbor’s CNS pipeline is initially focused on ALS, where the company is pursuing both genetically defined and broader sporadic patient populations, as well as opportunities to address related diseases with shared biological underpinnings, such as frontotemporal dementia (FTD). ABO-201 is designed to excise the pathogenic repeat expansion in C9ORF72, the most common genetic driver of ALS and FTD. ABO-202 and ABO-203 are intended to address TDP-43-related biology, present in approximately 97% of ALS cases, familial and sporadic, by restoring proper splicing and expression of STMN2 and UNC13A.

Delivery has been the central barrier to gene editing in neurodegenerative disease. Traditional CRISPR-Cas9 systems are large enough to crowd out the regulatory elements a single viral vector must also carry, pushing developers toward multi-vector systems or lipid nanoparticles that distribute predominantly to the liver. The nominated candidates deliver Arbor’s proprietary compact gene editors intravenously in a single blood-brain barrier-crossing AAV capsid and have achieved high levels of gene editing across critical CNS regions in preclinical models, including non-human primates.

“Our CNS strategy is deliberate: high-need diseases with strong biological rationale, the editing approach best suited to the underlying pathology, and programs advanced with clear development intent.  This allows us to open a new therapeutic area for gene editing therapies outside the liver,” added Smith. “We are moving ABO-201, ABO-202 and ABO-203 through IND-enabling activities now, with our first CNS IND filings anticipated in 2027.”

Arbor also announced that Albert Misko, M.D., Ph.D., a board-certified neurologist, has joined the company as Vice President, Head of Clinical Development, where he will lead clinical development for the CNS portfolio.

About Arbor Biotechnologies, Inc.

Arbor Biotechnologies, a clinical stage, next-generation gene editing company based in Cambridge, MA, is advancing a portfolio of first-in-class genomic medicines for serious diseases that have no existing functional cures, with a CNS pipeline initially prioritizing multiple ALS targets, and a co-developed clinical program, ABO-101, for the treatment of primary hyperoxaluria type 1. The company’s unique breadth of gene editing technologies goes beyond the limitations of early editing approaches to unlock new gene targets and expand the therapeutic reach of genomic medicines.

For more information, please visit: arbor.bio.

Media Contact:
Peg Rusconi
Deerfield Group
peg.rusconi@deerfieldgroup.com