Developing gene therapies for rare diseases
Lead Product Candidate ARU-1801
A potentially curative lentiviral gene therapy, ARU-1801 is in development for individuals living with sickle cell disease (SCD). ARU-1801 is administered once and designed to address the limitations of current treatment options in development, such as low donor availability and toxicity from conditioning chemotherapy. ARU-1801 is in an ongoing and actively recruiting clinical trial, and has demonstrated durable, meaningful clinical responses to date for patients with SCD, while only requiring reduced intensity conditioning (low dose chemotherapy).
Product candidate ARU-2801
Our second product candidate, ARU-2801, is a one-time, adeno-associated virus (AAV) gene therapy designed to deliver potentially curative efficacy to patients with hypophosphatasia (HPP) without the limitations of chronic administration. Preclinical research shows treatment with ARU-2801 results in sustained elevation of tissue non-specific alkaline phosphatase (TNAP), the missing enzyme in HPP, at levels that ameliorate disease symptoms.
Fetal Hemoglobin Plays an Important Role in SCD
Sickle Cell Disease (SCD) is an inherited disease that affects the production of hemoglobin, a protein in red blood cells that carries oxygen throughout the body. An inherited mutation causes people with SCD to not have normal, healthy adult hemoglobin in their red blood cells and instead have an abnormal hemoglobin called sickle hemoglobin. The result is rigid, crescent-shaped, or “sickled” red blood cells that can clump together in blood vessels and block the flow of blood throughout the body. Consequently, SCD can cause frequent episodes of severe pain, weakness and other serious complications. Aruvant’s clinical study, the MOMENTUM study, is evaluating an investigational therapy (given only one time) that allows the body to produce fetal hemoglobin.
Did you know?
Fetal hemoglobin is an “anti-sickling” hemoglobin that is present before birth in the red blood cells. After birth, the gene that makes fetal hemoglobin turns off, which mostly stops the production of fetal hemoglobin. More fetal hemoglobin in the blood can mean fewer episodes of sickling and pain.
Clinical Studies are Essential to Finding New Treatments
The MOMENTUM study is an ongoing phase 1/2 trial examining ARU-1801 as a potentially curative gene therapy for individuals living with sickle cell disease. Clinical trial volunteers are critical to making new treatments a reality. For more information on the study, visit http://sicklecellstudies.com or view the patient brochure here.
Our product candidate ARU-2801, an AAV gene therapy, is in pre-clinical development for hypophosphatasia, a devastating, ultra-orphan disorder with multi-organ damage and high mortality when left untreated. Early data from pre-clinical studies with ARU-2801 shows durable improvement in disease biomarkers and increased survival.