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Sangamo Therapeutics announced updated preliminary proof-of-concept efficacy and safety data from the Phase 1/2 PRECIZN-1 study of SAR445136, a zinc finger nuclease gene-edited cell therapy candidate in development with Sanofi for the treatment of sickle cell disease (SCD). The therapeutic product is manufactured by ex vivo gene editing of a patient's own (autologous) hematopoietic stem cells using non-viral delivery of zinc finger nuclease technology targeting the BCL11a gene erythroid-specific enhancer (ESE) to increase endogenous fetal hemoglobin (HbF) production. None of the four treated patients required blood transfusions post engraftment, while total hemoglobin stabilized by Week 26 after treatment with SAR445136 for all patients. Fetal hemoglobin level increased from 0.1-11% at screening to 14-39% by Week 26 in all four patients and was 38% in the longest-treated patient at 91 weeks. Percent F cells increased to 64-96% by 39 weeks of follow-up in all four patients, persisting at 99% in the patient with 91 weeks of follow-up. The SAR445136 investigational drug product had on-target BCL11A gene modification (61-78%) in all four patients. Most adverse events reported in the screening, mobilization, apheresis and conditioning periods were SCD-related events. No adverse events related to SAR445136 were assessed. The study is ongoing. For more information: press release

Bluebird Bio announced updated results from its Phase 1/2 HGB-206 study of lovotibeglogene autotemcel (lovo-cel; formerly LentiGlobin® for SCD, bb1111) gene therapy for sickle cell disease (SCD) following enhancements to the manufacturing protocols and treatment process. The therapy achieves stable production of gene therapy-derived anti-sickling hemoglobin and continued complete resolution of severe vaso-occlusive crisis (VOCs) up to 36 months follow-up (n=2). Data were presented in two oral sessions at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition ( December 11-14, 2021, Atlanta) an published in the The New England Journal of Medicine (NEJM) (December 12, 2021, DOI: 10.1056/NEJMoa2117175). For more information: press release

Beam Therapeutics Inc. (Beam), announced recent business and pipeline updates, as well as third quarter 2021 financial results, on 8 of November. Beam announced that its Investigational New Drug (IND) application for BEAM-101 for the treatment of sickle cell disease was cleared by the U.S. Food and Drug Administration (FDA). BEAM-101, the company’s lead ex vivo base editing product candidate, is a patient-specific, autologous hematopoietic investigational cell therapy which incorporates base edits that mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin (HPFH) to potentially alleviate the effects of mutations causing sickle cell disease or beta-thalassemia. This is the first open IND for base editing technology, a next-generation form of CRISPR capable of making single base changes without creating double strand breaks in the DNA. Beam is preparing to initiate a Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM-101 for the treatment of sickle cell disease, which Beam refers to as the BEACON-101 trial. More info: Beam Therapeutics, press release.

Bluebird bio, Inc. (BLUE) reported the acceptance of the Biologics License Application (BLA) for betibeglogene autotemcel (beti-cel) for priority review, from the U.S. Food and Drug Administration (FDA), on November 22, 2021. The BLA for beti-cel 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. Beti-cel is a potentially transformative gene therapy for adult, adolescent and pediatric patients with β-thalassemia across all genotypes who require regular red blood cell (RBC) transfusions. If approved, beti-cel will be the first one-time treatment that addresses the underlying genetic cause of disease for patients living with β-thalassemia offering an alternative to regular RBC transfusions and iron chelation therapy. The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of May 20, 2022. More info: bluebird bio, press release.

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), announced that Health Canada has approved FERRIPROX® (deferiprone) for the treatment of iron overload in patients with sickle cell disease (SCD) or other anemias. FERRIPROX® was previously approved in Canada for the treatment of iron overload in patients with thalassemia major when current chelation therapy is inadequate. The decision comes about five months after the FDA approved FERRIPROX® for the same indication in the U.S. and one month after its approval in Brazil. Read here for more information.

A new treatment, called Adakveo (crizanlizumab), is now available to treat patients with sickle cell disease (SCD) through the United Kingdom’s National Health Service (NHS). Adakveo will be available in England and Wales to eligible patients, 16 years and older, to help prevent recurrent sickle cell crises (pain episodes). It can be given as an add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate. This is the first time in decades that a new therapy for SCD has been made available on the NHS. Read here for more information.

  1. The investigational gene editing therapy GPH101 for sickle cell disease is cleared for clinical testing
  2. Oxbryta™ (Voxelotor) is granted priority review for the treatment of SCD in children ages 4 to 11
  3. A published perspective from the ClinGen Hemoglobinopathy Variant Curation Expert Panel on ACMG/AMP rule specification
  4. Bluebird submits BLA to FDA for β-thalassaemia gene therapy

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