NEW YORK – The US Food and Drug Administration granted regenerative medicine advanced therapy designation to Editas Medicine for its investigational treatment for severe sickle cell disease, EDIT-301, the gene-editing company said this week. EDIT-301 contains patient-derived CD34+ hematopoietic stem and progenitor cells with edited gamma globin gene (HBG1 and HBG2) promoters and is designed to create a sustained increase in fetal hemoglobin production. The FDA previously awarded the Cambridge, Massachusetts-based firm orphan drug designation and rare pediatric disease designation for EDIT-301 as a treatment for sickle cell disease and beta thalassemia.
GenomeFrontier Therapeutics this week partnered with BioCina to develop viral vector-free chimeric antigen receptor T-cell products (CAR-T) for cancer treatment. GenomeFrontier will use BioCina's platform to develop CAR T-cell therapies using minicircle DNA and plasmid DNA vectors. BioCina will also support process development and good manufacturing practices for the DNA vectors and manufacture the CAR T-cell therapy for use in a Phase I clinical trial. GenomeFrontier has several CAR T-cell therapy candidates in preclinical development for the treatment of blood cancers and solid tumors.
Diakonos Oncology said this week that its dendritic cell vaccine, DOC1021, was granted fast-track designation by the US Food and Drug Administration. The designation was based on preliminary data from a Phase I trial evaluating DOC1021 in patients with glioblastoma multiforme. DOC1021 is a personalized vaccine that uses a patient's dendritic cells to promote an immune response and kill cancer cells. With fast-track designation, a sponsor can meet with the FDA more frequently to get advice on developing the fast-track designated agent, submit data on a rolling basis, and apply for accelerated approval or priority review.
AstraZeneca's Alexion this week said China's National Medical Products Administration has approved its C5 complement inhibitor Soliris (eculizumab) as a treatment for adults with neuromyelitis optica spectrum disorder, a rare autoimmune disease, and who are anti-aquaporin-4 (AQP4) antibody positive (Ab+). Soliris, which is administered intravenously every two weeks, is designed to inhibit the C5 protein in a part of the immune system known as the terminal complement cascade.
Soliris is already approved in China as a treatment for paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and refractory generalized myasthenia gravis (gMG).
The Abu Dhabi Department of Health and Abu Dhabi Health Data Services (ADHDS) this week announced a partnership, through which physicians will be able to access pharmacogenomic test reports for consenting participants enrolled in the Emirati Genome Program (EGP). The PGx reports will be accessible through Malaffi, the health information exchange platform operated by ADHDS. The EGP is a population genomic initiative aimed at increasing precision medicine access in Abu Dhabi. The national project is a key part of Abu Dhabi's National Genome Strategy, within which the government aims to improve healthcare by building "a comprehensive and sustainable genomic ecosystem" that leverages whole-genome sequencing and artificial intelligence technologies.
ITM and Canadian Nuclear Laboratories said this week that they have launched a new joint venture company, dubbed Actineer, to produce the therapeutic radioisotope actinium-225. The joint venture is meant to provide the alpha-emitting isotope for use in targeted cancer radiopharmaceuticals and to boost international supply through a dedicated large-scale infrastructure.
Seren and SerenOx Africa said last week that they opened a hematology clinic and laboratory in Tanzania to offer low-cost diagnostic testing to sub-Saharan Africa. The organizations said underdiagnosed diseases in the region, including sickle cell disease, can be cured or controlled with affordable therapies when patients have access to diagnostic testing. UK-based Seren is a University of Oxford spinout that applies technologies developed at the university to delivering rapid diagnosis of inherited blood disorders, infectious diseases, and early-stage cancers. Seren, Oxford, and the UK National Institute for Health and Care Research support SerenOx Africa, a Tanzania-based organization dedicated to advancing diagnostics in Africa through access to molecular testing.
University of Pennsylvania spinout FloBio said this week that the US Food and Drug Administration has granted breakthrough device designation for its rapid point-of-care test for determination of patient clotting status. The Philadelphia-based company's automated hemodynamic assay provides information on a patient's blood clotting status, including anticoagulation from taking a direct oral anticoagulant. The firm said it is developing a platform to help guide treatment and cost-effective management of blood clotting abnormalities and serious bleeding.
Oxford BioDynamics said this week that it and UK health insurer Bupa have entered a strategic agreement that gives Bupa patients access to Oxford's EpiSwitch CiRT (checkpoint inhibitor response test). Developed using the company's proprietary 3D genomic biomarker platform, the assay is intended to predict individuals' responses to this class of immunotherapy drugs, aiding oncologists in selecting appropriate treatment. Under the new agreement, the CiRT test will be available to doctors considering treating Bupa customers with an immune checkpoint inhibitor, where decisions need to be made in the face of potential serious illness and lack of response to treatment.
Burning Rock Biotech said this week that its OverC multi-cancer detection blood test has been granted breakthrough device designation by the China National Medical Products Administration. The test is intended for early detection of multiple cancer types in adults of either sex, aged 50-75 years old, who are at average risk for cancer. The assay demonstrated 69 percent sensitivity and 99 percent specificity in the company's case-control study, THUNDER, with up to 92 percent accuracy in predicting tumor tissue of origin.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared in Precision Medicine Online.