Company Formed to Advance Sickle Cell Gene Therapy

Sickle cell illustration (National Heart, Lung, and Blood Institute)

27 Nov. 2018. A Swiss medical technologies company and Cincinnati Children’s hospital in Ohio are starting a new enterprise to commercialize research on gene therapies for inherited blood disorders. Aruvant Sciences is a joint venture between Roivant Sciences in Basel, Switzerland and Innovation Ventures, the the arm of Cincinnati Children’s Medical Center taking its research discoveries to market.

Aruvant Sciences plans to develop discoveries from the lab of Punam Malik, a hematologist at Cincinnati Children’s studying gene therapies to treat sickle cell disease and a related rare blood condition called beta thalassemia. Malik is director the Cincinnati Comprehensive Sickle Cell Center, and chairs the medical center’s gene and cell therapy program. Research in her lab led to discovery of a gene therapy treatment for sickle cell disease and beta thalassemia code-named RVT-1801, now the lead product of Aruvant Sciences.

Sickle cell disease is a genetic blood disorder affecting hemoglobin, a protein in blood that delivers oxygen to cells in the body. People with sickle cell disease have hemoglobin molecules that cause blood cells to form into an atypical crescent or sickle shape. That abnormal shape causes the blood cells to break down, lose flexibility, and accumulate in tiny capillaries, leading to anemia and periodic painful episodes. Sickle cell disease is prevalent worldwide, and affects 70,000 to 80,000 people in the U.S., including about 1 in 500 people of African descent. People with beta-thalassemia have lower production of hemoglobin in their blood.

RVT-1801 delivers a modified healthy fetal hemoglobin gene to a patient’s own blood-forming stem cells. Research by Malik and others found people with sickle cell disease have a malfunctioning gene that switches off production of healthy fetal hemoglobin, even well into adulthood. With RVT-1801, a healthy gene is delivered with a benign virus to the patient’s blood-forming stem cells taken from bone marrow. The stem cells are cultured and transformed into healthy blood-forming cells with functioning genes producing fetal hemoglobin, later transplanted back into the patient. Cincinnati Children’s holds a U.S. patent on the technology, with Malik listed as its inventor.

An early- and mid-stage clinical trial, led by Malik, is testing the gene therapy among 10 participants with sickle cell disease. The study is assessing the feasibility of the process for 1 year, and safety of the treatments for as long as 15 years. Malik plans to present early results from the first 2 participants in the trial on 3 December at a meeting of American Society of Hematology in San Diego.

Aruvant Sciences is the latest in a string of companies formed by Roivant Sciences to develop biomedical technologies. Roivant calls the individual companies “vants” with each of the companies focusing on a particular disease or technology type. Roivant Sciences says it now has 35 therapies in various stage of preclinical development or clinical trials. With the start of Aruvant Sciences, Cincinnati Children’s is receiving an initial payment, eligible for milestone payments and royalties on sales, and gaining shares of the company’s stock, but no further financial details were disclosed.

In addition to starting Aruvant Sciences, Roivant is also forming a not-for-profit Roivant Foundation to improve access to to sickle cell therapies in developing countries. More than 70 percent of people with sickle cell disease are believed to be in sub-Saharan Africa.

More from Science & Enterprise:

• Biotechs Collaborate on More Precise Gene, Cell Cancer Therapies
• FDA Issues Draft Gene Therapy Regulatory Guidance
• Gene Therapy Shown to Reduce Extreme High Cholesterol
• Long-Term Effects Shown from Hemophilia Gene Therapy
• Gene Therapy Safety Study for Heart Failure Underway

*     *     *