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Find Clinical Drug Pipeline Developments & Deals by Aruvant Sciences
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 myeloablative chemotherapies.
Data demonstrate that ARU-1801 gene therapy may not only be able to reduce severe vaso-occlusive events but also reduce days in hospital for SCD patients, which could provide a clinically meaningful benefit for patients and help reduce health care costs.
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 chemotherapy.
The agreement aims to support ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). Aruvant has chosen Lonza to help develop and manufacture ARU-1801 for its upcoming pivotal trial.
Preliminary clinical data from Aruvant's ongoing Phase 1/2 study that demonstrates ARU-1801, administered with only reduced intensity conditioning, can achieve durable reductions in disease burden.
Data presented at ASH is from the MOMENTUM study, an open label Phase 1/2 clinical trial examining ARU-1801 as a one-time potentially curative gene therapy for individuals with sickle cell disease (SCD).
In partnership with SCDAA, Aruvant is working to educate patients about gene therapy, while gaining critical insights from the patient community for its ARU-1801 SCD development program.
Aruvant's ARU-1801, received FDA Orphan Drug designation for its efficacy to deliver a highly potent gene payload with enhanced anti-sickling properties, for a Reduced Intensity Conditioning regimen.