NEW YORK and BASEL, Switzerland, April 27, 2021 /PRNewswire/ -- Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, today announced that data on ARU-1801, a potentially curative gene therapy for sickle cell disease (SCD), will be delivered in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. Dr. Punam Malik, director of the Cincinnati Comprehensive Sickle Cell Center and program leader of the Hematology and Gene Therapy Program at the Cincinnati Children's Hospital Medical Center, will present data from the ARU-1801 Phase 1/2 MOMENTUM clinical trial during ASGCT, which will take place virtually from May 11 to May 14, 2021.
"The oral presentation at ASGCT represents tremendous progress in our mission to find a cure for sickle cell disease patients," said Will Chou, M.D., chief executive officer of Aruvant. "The data we have shown thus far demonstrates real promise for the initial concept that Dr. Malik developed many years ago. The data she will be presenting is even more important today given the need for treatment options that can be delivered without exposing the patients to high intensity chemotherapy. Our preliminary results demonstrate that with ARU-1801, it is possible to successfully provide a gene therapy for SCD using only reduced intensity conditioning."
Dr. Malik's oral presentation will review early results demonstrating the safety and efficacy of ARU-1801 with reduced intensity conditioning (RIC). ARU-1801 is a lentiviral gene therapy for those living with severe SCD that is administered once and designed to address the limitations of current curative treatment options, such as low donor availability and toxicity from conditioning chemotherapy.
The MOMENTUM study is evaluating ARU-1801 in patients with SCD. The data being presented at ASGCT is from the first three patients in the ongoing study. Through 24 months post ARU-1801 treatment, Patients 1 and 2 saw 93% and 85% reductions in the number of vaso-occlusive events (VOEs), respectively, and Patient 3 had no VOEs through 10 months of follow-up, a 100% reduction.
Session: Gene Therapies for Hemoglobinopathies
Title: Early Results from a Phase 1/2 Study of ARU-1801 Gene Therapy for Sickle Cell Disease (SCD): Safety and Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Date and Time: Wednesday May 12, 5:30 p.m. EDT Abstract: The abstract was published today at the ASGCT website.
About Aruvant Sciences Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an active research program with a lead product candidate, ARU-1801, in development for individuals suffering from sickle cell disease (SCD). ARU-1801, an investigational lentiviral gene therapy, is being studied in a Phase 1/2 clinical trial, the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. For more information on the clinical study, please visit www.momentumtrials.com and for more on the company, please visit www.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciences and on Instagram @Aruvant_Sciences.
About Roivant Roivant's mission is to improve the delivery of healthcare to patients by treating every inefficiency as an opportunity. Roivant develops transformative medicines faster by building technologies and developing talent in creative ways, leveraging the Roivant platform to launch Vants—nimble and focused biopharmaceutical and health technology companies. For more information, please visit www.roivant.com.