Arbor Biotechnologies to Present Expansion of CNS Editing Platform Capabilities at the American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting
Oral presentation highlights novel compact RT editor demonstrating first preclinical in vivo proof of concept for precise gene editing in the CNS delivered via a single AAV
- Poster presentations showcase deepening platform capabilities to develop genomic medicines for neurodegenerative diseases and beyond
CAMBRIDGE, MA – April 27, 2026 – Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today announced three upcoming presentations at the 2026 American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting, taking place May 11-15 in Boston, Massachusetts.
Data presented at ASGCT will showcase Arbor’s expertise in developing novel editing technologies to address intractable CNS diseases with high unmet need and continued advancement of a leading technology platform addressing critical challenges in genomic medicine development.
In an oral session, Arbor will present data demonstrating first-in-class in vivo CNS gene editing with a proprietary compact RT editor, composed of a novel nickase and novel reverse transcriptase (<1200 amino acids), which can be delivered via a single AAV. This represents a step forward for the industry, where currently effective delivery of RT editing technology to the CNS requires multiple AAVs. Arbor’s compact RT editing technology unlocks opportunities to expand its pipeline of gene editing therapeutics to address diseases of the CNS as well as other extrahepatic tissues where AAV is the only effective means of delivery.
Arbor will also present two posters showcasing novel approaches that support accelerated development of gene editors: one providing high resolution mapping of in vivo genome editing outcomes in the CNS, and the second poster detailing a novel guide activity prediction technology to enable rapid identification of potent and specific guides.
Details for the presentations are as follows:
Oral Presentation Title: In vivo editing targeting neurodegenerative diseases in the mouse brain using an all-in-one AAV compact RT editor
Session: New Base and Primer Editing Technologies
Session Date and Time: Tuesday, May 12, 2026, 3:30-5:00pm EST
Location: MCEC Room 205ABC
Presenter: Kyle Watters, PhD
Poster Title: Editing activity predictions can accelerate the selection of potent and specific guide RNAs for Cas12i2-based therapeutics
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
Poster Title: High‑resolution mapping of in vivo genome editing utilizing Fluorescence-Activated Nuclear Sorting for Gene Editing (FANgS)
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 ALS. 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