Mark Curtis & Richard Philipson
Providing a critical overview of the sector’s commercial developments – M&As, licensing agreements & collaborations, financial results, IPOs and clinical/regulatory updates, with commentary from our Expert Contributors.
Interest in pluripotent stem cell (PSC)-derived products remains strong and we continue to make ground on the creation of off-the-shelf designer cells. Fate Therapeutics, through its partnership with Sloan Kettering, has made some particularly interesting advancements. Recently, at the American Society for Hematology Annual Meeting in December, the company announced that it has successfully created T cells with multiple modifications executed through a single CRISPR/Cas9-mediated engineering event. The cells Fate created were derived from a clonal iPSC line that was modified to introduce a chimeric antigen receptor (CAR) and remove T-cell receptor (TCR) expression, giving them the ability to target cancer while effectively making them ‘invisible’ to the immune system. Fate also reported on the generation of iPSC-derived NK cells, which will be used for IND enabling work.
This month sees the outstanding achievement of Spark Therapeutics in the landmark approval by FDA of LUXTERNA™ for the treatment of RPE65 mutation-associated retinal dystrophy – the first FDA-approved gene therapy for a genetic disease. December always sees a rash of news from the American Society of Hematology annual meeting, and this year is no different. News from BioMarin on progress with its treatment for hemophilia A will certainly keep Spark on its toes, as it looks like BioMarin is ahead of its competitor in the race to be the first to market for this serious hereditary bleeding condition. A third key player in the hematology field, bluebird bio, also keeps up the positive news flow with updates suggesting favorable clinical outcomes in patients with sickle cell disease or beta-thalassemia receiving treatment with its lentivirus-based therapy. All in all, a great way to end the year!
Citation: Cell Gene Therapy Insights 2018; 4(1), 9-21.