Mark Curtis & Richard Philipson
Advanced Cell Therapies – Progress Towards the Clinic
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.
Industry and investors alike have placed their bets on autologous T-cell immunotherapies as the future of oncology. Some have deviated from the pack and made advances on the allogeneic side, namely Cellectis, which is developing gene-edited donor-derived T cells that evade immune detection. Although progress with directed differentiation of pluripotent cells to T cells is a threat that could disrupt the current trajectory. Fate Therapeutics announced a deal with Memorial Sloan Kettering Cancer Center (MSKCC) this past month that will focus on the development of a truly scalable platform for the production of engineered T cells from pluripotent sources. While directed differentiation has a complex IP space, Fate will lay exclusive claim to patents recently generated at Sloan Kettering for the production of both T and NK cells.
This month sees exciting developments in the clinical arena, with Bluebird Bio announcing the initiation of a Phase 3 clinical trial in transfusion-dependent beta-thalassemia, and Alnylam announcing positive initial data from its Phase 1 study in healthy volunteers, investigating its RNAi therapeutic targeting glycolate oxidase for the treatment of Primary Hyperoxaluria Type 1. Alnylam has a rich pipeline of RNAi therapies and, with its announcement of plans for a European hub in the UK, is clearly confident in its future value and success. Balancing this positive news is the announcement of the disbandment of Novartis’s cell and gene therapy unit. Although the company has committed to continuing to develop CAR-T therapies, this can only be seen as a blow to the field, and is reflected in the negative effects on the valuations of fellow CAR-T players Kite and Juno.
Read full article »