Since our founding, we have developed a wholly owned portfolio of editing technologies that can be used to create in vivo targeted genetic medicines, beginning with the liver and CNS. Our technology can also be used to enable cell-based therapeutics, and while we are not developing these internally, we have partnered with leading companies who have expertise in ex vivo applications.
The liver has become an important target for genetic medicines, resulting in validated delivery approaches, clear regulatory and clinical paths for products and validated biology—all of which were key factors in our decision to prioritize liver disease as initial clinical indications in which to apply our novel technology. We have selected liver diseases with high unmet need where we can provide differentiated therapies for patients. This approach allows us to de-risk elements around our technology for our lead indications.
Our lead programs are in primary hyperoxaluria (PH) and transthyretin amyloidosis (ATTR). Using proven and effective delivery systems such as lipid nanoparticles (LNPs), we are able to package our editing cargo to deliver to the liver. Arbor’s gene editing approach provides the potential for a one-time dose of disease-modifying therapies that are durable for the duration of a patient’s lifetime.
Beyond the liver, we are also focused on the CNS, where there are many devastating diseases of genetic origin. Until the discovery of gene editing, the technology to effectively target the underlying genetic mutations within these diseases had not existed. This presents an exciting new opportunity for genomic medicine in the CNS. We have selected our initial diseases for targeting in the CNS and our mode of delivery will be via adeno-associated virus or AAV, which is powered to target select regions of the brain that will provide the most meaningful benefit to patients.