Ramaswamy's latest 'vant' to focus on gene therapies for blood disorders

red blood cells
Punam Malik, M.D., of Cincinnati Children's developed Aruvant’s lead candidate, known as RVT-1801. (Arek Socha)

Another day, another “vant”—this time, Vivek Ramaswamy’s Roivant and Cincinnati Children’s Hospital Medical Center are launching Aruvant Sciences, which will focus on developing gene therapies for blood diseases, starting with sickle cell disease and beta-thalassemia. 

Punam Malik, M.D., director of the Cincinnati Comprehensive Sickle Cell Center at Cincinnati Children's, developed Aruvant’s lead candidate, known as RVT-1801. The gene therapy is designed to increase the number of functioning red blood cells in a patient by inserting a modified fetal hemoglobin gene into the individual's own stem cells. 

Treating sickle cell and beta thalassemia with fetal hemoglobin is not a new idea. Both disorders are caused by mutations in the beta-globin gene, which result in missing or defective hemoglobin. Fetal hemoglobin, found in newborn babies and later replaced by adult hemoglobin, can be protective in adults who have blood disorders. This is because fetal hemoglobin has a different component that is not affected by the mutations that cause sickle cell and beta-thalassemia. 

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Bluebird Bio is also working on a lentiviral vector-based gene therapy for patients with beta-thalassemia. Earlier this year, the company announced that its treatment eliminated or reduced the need for blood transfusions in patients with beta-thalassemia who relied on transfusions to survive. And in August, CRISPR Therapeutics and Vertex Pharmaceuticals started the first company-sponsored clinical trial of a CRISPR-edited treatment in the indication as Bluebird: transfusion-dependent beta-thalassemia. 

RELATED: CRISPR Therapeutics, Vertex start first company-backed human CRISPR trial 

Malik will present early data from a phase 1/2 study of RVT-1801 in sickle cell patients on Monday, at the Annual Meeting and Exposition of the American Society of Hematology. 

Under the deal, Cincinnati Children’s will receive an undisclosed upfront payment, as well as shares in Aruvant. Down the line, the hospital stands to pick up milestone payments and tiered royalties. 

Roivant has also set up a nonprofit, the Roivant Foundation, which aims to broaden access to treatments for sickle cell patients in developing countries. 

RELATED: After gene therapy pivot, Axovant bolsters its executive ranks 

The announcement comes the same day that Roivant’s neuro-focused company, Axovant, added five new executives to its leadership. After a disastrous foray into Alzheimer’s disease, the biotech shifted gears in June, when it licensed a gene therapy for Parkinson’s disease from Oxford Biomedica. 

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