Research Horizons


Aruvant to Present New Data on Sickle Cell Gene Therapy

A gene therapy for sickle cell disease based on discoveries by experts at Cincinnati Children’s continues to show promise, according to an announcement from Aruvant Sciences, a company launched in 2018 to further develop and test the therapy in a Phase 1/2 clinical trial.

As of July 28, 2020, data from three patients treated with ARU-1801 are available, including one patient treated under an enhanced manufacturing process.

“The clinical results thus far demonstrate that ARU-1801 holds significant promise for achieving durable responses with a reduced intensity conditioning approach to gene therapy,” says Michael Grimley, MD, Division of Bone Marrow Transplantation and Immune Deficiency at Cincinnati Children’s and a principal investigator in the MOMENTUM study.

Detailed findings will be presented Dec. 7, 2020, at the American Society of Hematology (ASH) Annual Meeting and Exposition. An abstract was published online on Nov. 4, 2020. Findings also will be published in the upcoming supplemental issue of Blood.

“Given the impact chemotherapy toxicity has on physician and patient decision making around treatment options, ARU-1801 has the potential to be a unique option for SCD patients seeking gene therapy.” Grimley says.

ARU-1801 is based on years of study led by Punam Malik, MD, Director of the Comprehensive Sickle Cell Center at Cincinnati Children’s. In November 2018, Cincinnati Children’s and Roivant Sciences co-launched Aruvant Sciences to continue developing the treatment.