US based pharma company Roivant Sciences and Cincinnati Children’s Hospital Medical centre has announced the launch of Aruvant Sciences, a biopharmaceutical company focused on developing innovative gene therapies for hematological conditions.

Roivant also announced the formation of a new non-profit foundation with the mission of improving access to medical care for patients with sickle cell disease in the developing world.

The launch of Aruvant builds on a partnership between Roivant and Innovation Ventures which is the commercialization arm of Cincinnati Children’s Hospital Medical centre.

The lead candidate in Aruvant’s pipeline, RVT-1801, is an investigational gene therapy for sickle cell disease and β-thalassemia.

RVT-1801 utilizes proprietary technology intended to increase functioning red blood cells by inserting a modified fetal hemoglobin gene into autologous stem cells through a lentiviral vector.

Studies have indicated that sickle cell patients with elevated levels of fetal hemoglobin have fewer vaso-occlusive crises and hospitalizations, due in part to improved characteristics of fetal hemoglobin relative to adult hemoglobin. RVT-1801 is the only known clinical-stage gene therapy to deliver the gene encoding fetal hemoglobin, which has been modified to optimize oxygen carrying capacity and anti-sickling properties.

Myrtle Potter, Vant Operating Chair at Roivant Pharma, will serve as Chairman of Aruvant.

RVT-1801 was developed in the laboratory of Dr. Punam Malik, Director of the Cincinnati Comprehensive Sickle Cell centre at Cincinnati Children’s.

“Treating sickle cell anemia and β-thalassemia has been my passion since I first trained as a physician in India,” said Dr. Malik.

“I look forward to sharing important data next week at ASH on RVT-1801, which has been designed to improve patient experience and access through our RIC regimen. I am excited to work with the team at Roivant to rapidly advance the development of this potentially transformative therapy. A critical reason why we chose to work with Roivant on this program was their authentic commitment to patients globally”, Dr Malik added.

Dr. Mayukh Sukhatme, President of Roivant Pharma said, “We are excited to develop this novel modified fetal hemoglobin gene therapy for sickle cell disease. Our partnership with Cincinnati Children’s represents Roivant’s commitment to the development of novel, transformational therapies, including gene therapies that have the potential to shift treatment paradigms for serious diseases.”

Under the terms of the agreement, Cincinnati Children’s will receive an upfront payment and shares of Aruvant and will be eligible for milestone payments and tiered royalties.

In addition to establishing Aruvant, Roivant has created a nonprofit organization, the Roivant Foundation, focused on improving access to therapies for sickle cell disease for patients in the developing world. Over 70% of individuals with sickle cell disease live in Sub-Saharan Africa.

Vivek Ramaswamy, Founder and CEO of Roivant said, “As a native of Cincinnati, I am particularly excited about Roivant’s sustained partnership with Cincinnati Children’s, which dates back to our first year of operations. I am also proud of our creative efforts to improve access to care in the developing world through the work of the Roivant Foundation.”

Dr. Peggy Hostetter, Chief Medical Officer of Cincinnati Children’s and Director of the Cincinnati Children’s Research Foundation said, “Roivant has been a valued partner of Cincinnati Children’s for several years, and we are delighted to work together on the launch of Aruvant, a new company which will have staff based right here in Cincinnati. An important goal of our research enterprise is to facilitate the translation of promising discoveries from bench to bedside, and the closing of this deal represents a significant step in ensuring this program has the potential to impact patients here in Cincinnati and around the world.”