Areas of Focus
Since our founding, we have developed a wholly owned portfolio of editing technologies to create targeted in vivo genetic medicines that offer potential for one-time dosing with durable effects that span a patient’s lifetime. We have focused our efforts on developing treatments for diseases with high unmet need where we can provide differentiated therapies for patients.
The liver has become an important target for genetic medicines, resulting in validated delivery approaches, clear regulatory and clinical paths for products and well-established biology. Our lead program in primary hyperoxaluria (PH), ABO-101, leverages proven and effective delivery systems to route our editing cargo to the liver.
We are focused on the central nervous system (CNS), where there are many devastating diseases that lack effective treatment options. Advancements in gene editing technologies and delivery mechanisms unlock exciting new opportunities to effectively target the underlying drivers of many CNS diseases. We are starting with ALS as our initial indication for CNS targeting and are developing assets suitable for delivery via adeno-associated virus (AAV) vectors to target regions of the brain that will provide the most meaningful benefit to patients. As delivery mechanisms continue to mature, we plan to expand into other brain region-specific indications such as frontal temporal dementia and subsequently into neurodegenerative diseases located in deeper brain structures.