We are a preclinical stage company pioneering a new class of rationally designed gene therapies with potentially curative benefit in patients with both rare and prevalent devastating diseases. Our rationally designed gene therapy candidates are methodically created to address the key limitations of current approaches and to meet the specific needs of a disease. We created our proprietary Affinia Rationally-designed Therapies (ART) platform to develop these novel therapies, focusing on the three key components of a gene therapy: capsids, which are the outer shells of a virus used to deliver the genetic code to tissues to treat a disease; promoters, which control how the cells read the genetic code that is delivered; and manufacturing approaches, which determine the quality and cost of these potential medicines. Our goal is to realize the full promise of gene therapy by addressing key limitations of current approaches, including tissue targeting specificity, cell expression and manufacturability. The potential of our platform has attracted a team of seasoned executives with extensive experience in advancing transformational gene therapy technologies from discovery through approval and commercialization.

Our lead product candidate, AFTX-001, is Anc80L65-ARSA for the treatment of metachromatic leukodystrophy (MLD). MLD is a rare, rapidly progressive and fatal genetic disease that results from the deficiency of functional arylsulfatase A (ARSA), an enzyme that is critical for neuronal survival. Patients with the most common form of MLD, called late infantile MLD, typically die before the age of eight years.  We anticipate an IND submission for AFTX-001 in the first half of 2023.

Our second product candidate, AFTX-002, is Anc80L65-trastuzumab for the treatment of brain metastases secondary to human epidermal growth factor receptor 2 overexpressing (HER2+) breast cancer (BMBC). BMBC is a common and deadly complication of HER2+ breast cancer with a median survival of only 18 months. Trastuzumab is an approved monoclonal antibody for the treatment of HER2+ breast cancer; however current methods of administration are impractical for achieving sustained therapeutic levels in the brain sufficient to be effective against BMBC. We anticipate an IND submission for AFTX-002 in the second half of 2023.