We are pleased to announce the organization of the ITHANET virtual information day and invite you to attend on Sep 29th 14:00-17:00 GMT+3. During the event, the recent developments and collaborations of the ITHANET community portal for haemoglobinopathies will be presented. The event’s poster and agenda can be found here. Please register here by Sep 25th.

The U.S. Food and Drug Administration (FDA) announced the approval of Zynteglo (betibeglogene autotemcel), for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell transfusions. Ζynteglo the first cell-based one-time gene therapy product administered as a single dose. It is very important that Zynteglo is a customized treatment created using the patient’s bone marrow stem cells that are genetically modified to produce functional β-globin. Of 41 patients receiving Zynteglo, 89% achieved transfusion independence, since they maintained a pre-determined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. The most common adverse reactions associated with Zynteglo included reduced platelet and other blood cell levels. Also, there is a potential risk of blood cancer associated with this treatment therefore patients who receive Zynteglo should have their blood monitored for at least 15 years for any evidence of cancer. No cases of blood cancer have been seen in studies of Zynteglo. The FDA granted approval of Zynteglo to bluebird bio, Inc. More information can be found here.

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.

Pfizer Inc. and Global Blood Therapeutics, Inc. (GBT) announced the companies’ agreement under which Pfizer will acquire GBT, a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities, starting with sickle cell disease (SCD). Under the terms of the transaction, Pfizer will acquire all the outstanding shares of GBT for $68.50 per share in cash, for a total enterprise value of approximately $5.4 billion, including debt and net of cash acquired. GBT developed Oxbryta® tablets, a first-in-class medicine that directly targets the root cause of SCD. Oxbryta was approved in the United States in November 2019 and is also approved in the European Union, United Arab Emirates, Oman and Great Britain. In addition, GBT is developing GBT021601, an oral, once-daily, next-generation sickle hemoglobin (HbS) polymerization inhibitor in the Phase 2 portion of a Phase 2/3 clinical study. GBT601 has the potential to be a best-in-class agent targeting improvement in both hemolysis and frequency of vaso-occlusive crisis (VOC). GBT’s promising pipeline also includes inclacumab, a fully human monoclonal antibody targeting P-selectin which is being evaluated in two Phase 3 clinical trials as a potential quarterly treatment to reduce the frequency of VOCs and to reduce hospital readmission rates due to VOCs. Both GBT601 and inclacumab have received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration. The acquisition complements and further enhances Pfizer’s more than 30-year heritage in rare hematology and reinforces the company’s commitment to SCD by bringing expertise and a leading portfolio and pipeline with the potential to address the full spectrum of critical needs in this underserved community. Pfizer intends to continue to build on the companies’ shared commitment to and engagement with the SCD community. 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.

Page 1 of 49