The Sickle Cell Disease Association of America (SCDAA) and Aruvant Sciences are proud to announce a new partnership to create educational programs to increase awareness of gene therapy as a potential curative treatment option for sickle cell disease patients. This collaboration will help SCDAA continue to deliver on its mission, while assisting Aruvant in learning more about the needs of sickle cell disease (SCD) patients. Under the agreement, Aruvant will collaborate with SCDAA to host local and national educational events and develop materials for a public-awareness campaign.

"In partnership with SCDAA, we are working to educate patients about gene therapy, while gaining critical insights from the patient community for our ARU-1801 SCD development program," said Will Chou, M.D., chief executive officer (CEO) of Aruvant. "Now is a perfect time to work with SCDAA to educate the community about gene therapy since we have an open and enrolling phase 1/2 clinical trial for our potentially curative experimental gene therapy, ARU-1801."

Sickle cell disease affects 100,000 individuals in the United States, disproportionately affecting African Americans with one in 500 African Americans suffering from the disease. This inherited disease affects the production of hemoglobin, a protein in red blood cells that carries oxygen throughout the body. The disease occurs when people inherit a mutation from each of their parents which 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. SCD can cause frequent episodes of severe pain, weakness and other serious complications. 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.

"In partnership with Aruvant, we can provide the critical education needed for our community to understand gene therapy and how these promising new treatments work to treat and maybe cure this genetic disease that impacts so many in our community," said Beverley Francis-Gibson, SCDAA president and CEO. "Partnering with companies like Aruvant is critical to help us support the research that could change the lives of many sickle cell disease patients."

Aruvant and SCDAA's educational events will review gene therapy and ongoing research, including discussion around Aruvant's MOMENTUM study. This clinical trial is examining a one-time investigational treatment, ARU-1801, to increase levels of fetal hemoglobin in patients with severe sickle cell disease, with the hope of fewer episodes of sickling and pain. Aruvant provided funding for SCDAA's 48th Annual National Convention 2020 which begins tomorrow, October 13, and will continue through October 17. To register, please visit https://bit.ly/SCDAA2020Convention.

The MOMENTUM Study

Aruvant is currently conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time only potentially curative gene therapy for patients with SCD. The MOMENTUM study is currently enrolling, and more information may be found at www.momentumtrials.com.

About Sickle Cell Disease Association of America

Sickle Cell Disease Association of America advocates for people affected by sickle cell conditions and empowers community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. The association and more than 50 member organizations support sickle cell research, public and professional health education and patient and community services. Visit www.sicklecelldisease.org.

About Aruvant Sciences

Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases, with an initial focus on helping patients suffering from sickle cell disease. The company's lead candidate, ARU-1801, is an investigational lentiviral gene therapy for sickle cell disease. ARU-1801 incorporates a patented modified gamma-globin into autologous stem cells, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only reduced intensity conditioning (RIC). Preliminary clinical data from an ongoing Phase 1/2 study in patients with sickle cell disease demonstrated continuing durable reductions in disease burden. For more information on the clinical study, please visit www.momentumtrials.com and for more on the company, please visit www.aruvant.com.