Arbor Biotechnologies Presents Data Supporting Clinical Development of ABO-101 and Robust Potential of Platform to Enable Therapeutic Programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting
“This approach has fueled our robust pipeline of genetic medicines which address a range of liver and CNS indications, including PH1 and ALS.
- “This approach has fueled our robust pipeline of genetic medicines which address a range of liver and CNS indications, including PH1 and ALS.
- The data show highly specific targeting of the HAO1 gene in the liver and preservation of genomic integrity upon editing.
- Together, this strong preclinical data package and the ongoing IND-enabling studies support continued advancement of ABO-101 toward clinical evaluation.
- Details for the presentations are as follows:
Oral Presentation Title: Identification and Engineering of ABR-004, a Compact, High-fidelity Nuclease for Therapeutic Gene Editing