Arbor Biotechnologies Achieves Unprecedented Precise In Vivo Reverse Transcriptase Editing in the Brain Using a Single AAV Vector, Opening a New Path to Treatments for Neurological Diseases
- Oral presentation at ASGCT showcases breakthrough technology enabling precise in vivo genome editing in the CNS using a single AAV vector
- Two poster presentations describe methods that support accelerated development of Arbor’s proprietary gene editing platform
CAMBRIDGE, MA – May 12, 2026 – Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today announces the presentation of data demonstrating the first precise in vivo genome editing in the brain using a compact reverse transcriptase (RT) editor delivered in a single adeno‑associated virus (AAV) at the American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting in Boston, Massachusetts.
“This work highlights a major advance in genome editing, overcoming a size and delivery barrier that has limited the application of next-generation genome editing technologies to address diseases beyond the liver. These data demonstrate that precise genome editing in the brain is achievable using a single, clinically relevant AAV vector,” said John Murphy, CSO at Arbor. “By dramatically reducing editor size while retaining in vivo activity, we’ve opened the door to treating a wide range of diseases that were previously out of reach, including neurodegenerative diseases in the central nervous system.”
Arbor’s novel genome editor comprises a miniature nickase and RT engineered to be highly active while remaining under 1,200 amino acids in length. The editor and its corresponding guide RNA were packaged into AAV9, a capsid with established CNS tropism and a strong clinical precedent.
Data from the oral presentation show that in adult mice, systemic delivery of the compact RT editor resulted in up to 40% precise editing in bulk liver tissue when making an edit within the DNMT1 locus. Intracerebroventricular (ICV) treatment of neonatal mice with the same editor yielded up to 25% precise editing in the brain.
Arbor will also present two posters showcasing novel approaches that support accelerated development of gene editors. The first poster describes an approach that enables high resolution mapping of in vivo genome editing outcomes in the CNS. The technique allows for quantification of productive editing events in target cells by measuring editing in nuclei isolated by cell type specific sorting. The second poster details a guide activity prediction tool enabling the rapid identification of potent and specific guide candidates, reducing screening timelines and accelerating the development of Cas12i2-based therapeutics.
Details for the oral presentation are as follows:
Title: In vivo editing targeting neurodegenerative diseases in the mouse brain using an all-in-one AAV compact RT editor
Abstract Number: 99
Session: New base and prime editing technologies
Session Date and Time: Tuesday, May 12, 2026, 4:00 PM
Location: Room MCEC Room 205ABC (Level 2)
Presenter: Kyle E. Watters, PhD
Details for the poster presentations are as follows:
Title: Editing activity predictions can accelerate the selection of potent and specific guide RNAs for Cas12i2-based therapeutics
Abstract Number: 1336
Session: Tuesday Poster Reception
Session Date and Time: Tuesday, May 12, 2026, 5:00-6:30 PM EST
Location: MCEC Exhibit and Poster Hall (Halls B2-C, Exhibit Level)
Presenter: Robert A. Morgan
Title: High‑resolution mapping of in vivo genome editing utilizing Fluorescence-Activated Nuclear Sorting for Gene Editing (FANgS)
Abstract Number: 1475
Session: Tuesday Poster Reception
Session Date and Time: Tuesday, May 12, 2026, 5:00-6:30 PM EST
Location: MCEC Exhibit and Poster Hall (Halls B2-C, Exhibit Level)
Presenter: Parnian Dolati
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 addressing serious diseases for which there are no existing functional cures, with the lead program, ABO-101 for the treatment of primary hyperoxaluria type 1, in clinical studies and a CNS pipeline initially prioritizing multiple ALS targets. The company’s unique breadth of gene editing technologies goes beyond the limitations of early editing approaches to unlock access to new gene targets and expand the therapeutic reach of genomic medicines. For more information, please visit: arbor.bio.
Media Contact:
Peg Rusconi
Deerfield Group
prusconi@nulldeerfieldgroup.com