Skip to main content
Donate
Current Pipeline
CRISPR

Arbor Biotechnologies

Arbor Biotechnologies is exploring an investigational Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) approach to treating Angelman syndrome.

Discovery & Dev

Discovery & Dev

Pre-clinical

Phase 1

Phase 2

Phase 3

To patients

  • progress mobile progress mobile progress mobile progress mobile progress mobile progress mobile

Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.

Therapeutic Approach

A therapeutic approach with two parts: one that recognizes a specific section of DNA or RNA and one that uses an enzyme (CAS) to cut at a specific section in the DNA or RNA. A CRISPR could be designed to cut the UBE3A-ATS and potentially allow the paternal UBE3A gene to be turned on.

Recent Updates

  • May 2025

    Arbor Biotechnologies to Present Data on CNS Programs and Platform Advances at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting

    Read More

Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.