Posted: Nov 30, 2023
A new age of precision gene therapy.
Axel Schambach et al. Lancet 2023 11
From the abstract: "Gene therapy has become a clinical reality as market-approved advanced therapy medicinal products for the treatment of distinct monogenetic diseases and B-cell malignancies. This Therapeutic Review aims to explain how progress in genome editing technologies offers the possibility to expand both therapeutic options and the types of diseases that will become treatable. "
Public Health Genetics, Gene Therapy, and Duchenne Muscular Dystrophy
CDC Webinar, December 18, 2023
From the website: "This webinar will review the population health impact of rare diseases such as DMD. Presenters will cover the genetics and clinical impact of DMD, the evolution of gene therapy, the development of FDA-approved gene therapy to treat the underlying protein deficiency that causes DMD, and the health equity challenges. "
Gene therapies for rare diseases are under threat. Scientists hope to save them- As industry steps aside, scientists seek innovative ways to make sure expensive treatments can reach people who need them.
H Ledford, Nature, October 6, 2023
From the article: "In the past two years, two gene therapies have been withdrawn from the European market for business reasons after earning regulators’ approval. Concern is mounting that other gene therapies for rare diseases will meet a similar fate, as might upcoming treatments that rely on the related technique of genome editing, which makes targeted DNA changes. "
Hemostasis - A Balancing Act.
H Marijke van den Berg et al. N Engl J Med 2023 8 (9) 853-856
From the paper: "It is reassuring that this first-in-class drug is effective in treating hemophilia A or B with inhibitors. Given its reported efficacy in both types of hemophilia without inhibitors, concizumab is evolving as an attractive therapeutic for all patients with hemophilia. In the bigger picture, these new treatment options need to be weighed against the possibility of cure of hemophilia through gene therapy: two adeno-associated virus (AAV) vector–based products have been approved for clinical use, and more are in the pipeline."