Editas Medicine, Inc. (Nasdaq: EDIT), announced that the gene editing therapy EDIT-301 is promising in the first patient with sickle cell disease (SCD). The experimental treatment is given to the patient according to the Phase 1/2 RUBY clinical trial. The EDIT-301 therapy uses a proprietary enzyme called AsCas12a and noted that is the first time has been used for genetic editing of human cells in a clinical trial. The first patient dosed in the RUBY clinical trial has shown signs of engraftment determined by analyses of the patient’s neutrophils and platelets. Editas Medicine, also announced that the U.S. Food and Drug Administration (FDA) has removed a partial hold on the RUBY trial, given the go-ahead for planned assessments of efficacy. “Dosing and successful engraftment of the first patient coupled with the FDA’s removal of the partial clinical hold on the RUBY trial are important steps toward our goal of bringing this new and promising treatment to people living with sickle cell disease,” Gilmore O’Neill, Editas president and CEO, said. For more information press release.

Vertex Pharmaceuticals Inc. (VRTX) and CRISPR Therapeutics (CRSP) presented new data on exa-cel (CTX001™) at the European Hematology Association (EHA) Congress. Forty-four patients with transfusion-dependent β-thalassemia (TDT) and thirty-one with severe sickle cell disease (SCD) were monitored from 1.2 to 37.2 months after exa-cel dosing. TDT patients had substantial mean increases in fetal hemoglobin (HbF) and corresponding increases in mean total hemoglobin (Hb), while all severe SCD were free of VOCs and mean HbF of approximately 40% by month 3 and 4 respectively. The data from 75 patients demonstrate that exa-cel has the potential to be a one-time functional cure, with safety profile be consistent with myeloablative conditioning and autologous stem cell transplant. For more information, press release.

SOMERVILLE, Mass .(BUSINESS WIRE) and Bluebird bio, Inc. (BLUE) announced the support of U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) for betibeglogene autotemcel (beti-cel) for the treatment of people with β-thalassemia who require regular red blood cell transfusions. The advisory committee’s recommendation is based on the Biologics License Application (BLA) currently under priority review by the FDA with a decision goal date set for August 19, 2022. The BLA is based on data from bluebird bio’s Phase 3 studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), the Phase 1/2 HGB-204 (Northstar) and HGB-205 studies, and the long-term follow-up study LTF-303 as of March 2021. Additionally, as of the latest data cutoff date (August 2021), data from bluebird bio’s clinical development program represent 240 patient-years of experience with beti-cel and the longest available follow-up data in beta-thalassemia patients requiring regular RBC transfusions treated with a one-time gene therapy. If approved, beti-cel will be the first potentially curative gene therapy option for people with beta-thalassemia who require regular red blood cell transfusions and the first ex-vivo LVV gene therapy available in the U.S. For more information, here.

Global Blood Therapeutics, Inc. (GBT) announced the presentation of five abstracts, related to the Sickle Cell Disease (SCD) programs, at the European Hematology Association (EHA) 2022 Hybrid Congress which will be held from June 9-12 in Vienna, Austria. All presentations are related to the Sickle Cell Disease (SCD) programs and include new real-world evidence data on Oxbryta® (voxelotor), data from the Phase 1 study of the next generation sickle hemoglobin (HbS) polymerization inhibitor GBT021601 (GBT601), initial findings from the Sickle Cell Health Awareness, Perspectives and Experiences (SHAPE) survey and patient and caregiver perspectives on the unmet needs in SCD. “Data presented at this year’s Congress will contribute to the growing body of evidence that could transform the treatment of sickle cell disease,” said Kim Smith-Whitley, M.D., executive vice president and head of research and development at GBT. For more information: Global Blood Therapeutics, press release.