An outstanding biopharmaceutical company focused on developing innovative gene therapies for hematological conditions: Aruvant Sciences

Aruvant Sciences, a part of the Roivant family of companies, is a clinical-stage biopharmaceutical company committed to developing and commercializing transformative gene therapies for rare diseases. The company has a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an active research program with a lead product candidate in development for individuals suffering from sickle cell disease  (SCD). The company’s lead product candidate, ARU-1801, an investigational lentiviral gene therapy, is currently in a clinical trial as a potential one-time curative treatment for SCD. Preliminary clinical data from the ongoing Phase 1/2 study demonstrated durable efficacy in reducing the negative impacts of SCD. Aruvant is preparing for the launch of Aruvant’s pivotal clinical trial of ARU-1801 in severe SCD. 

The company’s second product, ARU-2801, is a one-time, adeno-associated virus (AAV) gene therapy designed to deliver potentially curative efficacy to patients with hypophosphatasia without the limitations of chronic administration. To support the development of Aruvant’s  pipeline products and help build awareness of patient needs, the company partners with patient advocacy groups. Aruvant’s mission is to bring hope to patients living with rare diseases by developing life changing and potentially curative gene therapies, with a near-term focus on sickle cell disease.

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

Aruvant’s 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.

Sickle Cell Disease

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 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.

Historically, the research community has not developed medicines quickly enough to meet the needs of sickle cell disease patients. Aruvant is excited to be part of a future for patients with sickle cell disease that includes more treatment options and potential cures. The team at Aruvant is committed to bringing transformative, cutting-edge treatment options to the sickle cell community, and others impacted by rare diseases.

The best way for us to succeed in developing these breakthrough treatments is by truly understanding the needs and desires of patients and their family members. Aruvant believes in its science, but science cannot improve lives without the trust of patients. Therefore, the company committed to forging important and unique collaborations with the sickle cell and rare disease community. It is through these collaborations that the company can become a part of the community and can do better by patients and their families.

Meet the leader behind the success of Aruvant Sciences

Dr. Will Chou M.D., CEO of Aruvant Sciences brings over 15 years of healthcare experience to Aruvant, and most recently served as vice president and global disease lead for Novartis’ Cell and Gene Therapy unit, where he led the global commercial launch of Kymriah, the first CAR-T cell therapy. Prior to that role, he led the Kymriah lymphoma clinical development program to approvals in the North America, Europe and Asia. Prior to joining Novartis, Dr. Chou worked at the Boston Consulting Group where he focused on commercial and clinical pharmaceutical strategy. Dr. Chou holds an M.B.A. from the Yale School of Management, an M.D. from the University Of Pittsburgh School Of Medicine, and an A.B. in politics and economics from Princeton University. Dr. Chou completed his residency in internal medicine at Yale New Haven Hospital and a fellowship in geriatrics at Yale University.

Ms. Clark-Schoeb is Senior Vice President (SVP), communications, leading Aruvant’s investor relations, public relations, advocacy relations and government affairs work. She has over two decades of experience helping healthcare companies develop unique and effective communications programs. She began working with Aruvant after spending almost five years with Zyla Life Sciences as SVP of communications. Prior to Zyla, Ms. Clark-Schoeb led her own consulting business, working with pharmaceutical and biotechnology companies such as Sanofi, Syndax and Horizon Therapeutics. Prior to that she was senior associate at W2O, director of investor relations at Burns McClellan, director of corporate finance at The Medicines Company and a healthcare investment banker at Donaldson, Lufkin and Jenrette. Ms. Clark-Schoeb received her bachelor’s degree from Harvard University and a master’s in healthcare innovation from the University of Pennsylvania. She is a founding member of the Society of Healthcare Innovation.

“Aruvant’s mission is to bring hope to patients living with rare diseases by developing life changing and potentially curative gene therapies, with a near-term focus on sickle cell disease”