Tuesday, October 4, 2022


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Aruvant Sciences Announces Data Presentation at EHA 2022 Hybrid Congress

Stable transduction of fetal hemoglobin seen in sickle cell disease patients given ARU-1801 gene therapy in Phase 1/2 trial

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Aruvant Sciences (“Aruvant”), a private company focused on developing gene therapies for rare diseases, announced that Michael W. Kent, M.D., pediatric blood and marrow transplant physician at Atrium Health/Levine Children’s Hospital, is presenting new data on ARU-1801, a one-time gene therapy for individuals with severe sickle cell disease (SCD), at the European Hematology Association (EHA) 2022 Hybrid Congress.

The data is from the ongoing MOMENTUM Phase 1/2 study which is examining the potential for ARU-1801 to increase levels of fetal hemoglobin (HbF) to reduce or eliminate vaso-occlusive events (VOEs).

“The data shows that ARU-1801 offers potential long-term improvement of the primary symptoms seen with SCD without the toxicities and resource utilization associated with fully myeloblative chemotherapies,” said Dr. Kent. “The strong engraftment of ARU-1801 in patients with severe SCD is particularly notable since individuals were only given a single low dose of chemotherapy.”

The data from the ongoing MOMENTUM Phase 1/2 clinical trial examining the clinical benefit of ARU-1801 demonstrates:

Strong long-term hematopoietic stem cell (HSC) engraftment as demonstrated by high, stable ratios of perhiperhal blood (PB) to drug product (DP)VCN, achieved with only reduced intensity conditioning (RIC);
ARU-1801 can reach effective levels of anti-sickling hemoglobin at vector copy numbers (VCN) less than or equal to one, with approximately 40 percent HbFG16D expression per drug product VCN;
HbFG16D may have a more potent anti-sickling effect than endogenous HbF; and
ARU-1801 offers potential long-term amelioration of SCD phenotype without the toxicities and resource utilization required by other approaches that use myeloablative conditioning.

The EHA 2022 Hybrid Congress is taking place in Vienna, Austria from June 9 to June 12, 2022. The poster presentation is abstract #: EHA-2237.

The MOMENTUM study examines ARU-1801, an autologous lentiviral cell therapy with a modified, highly potent gamma-globin payload, in individuals with severe SCD. Unlike investigational gene therapies that require fully myeloablative conditioning, ARU-1801 is given with reduced intensity conditioning (RIC), which is a lower dose chemotherapy. ARU-1801 is designed to address the limitations of current curative allogeneic transplant options, such as low donor availability, the risk of graft-versus-host disease and toxicity from myeloablative conditioning chemotherapy. The data being presented highlights clinically meaningful reduction in participants’ vaso-occulsive events, which are the painful crisis that are commonly experienced by SCD patients.

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