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.
It was a rollercoaster of a month for the CAR-T industry. At its low Juno announced the discontinuation of its lead product JCAR015, a decision which, following a series of patient deaths and the halt of its Phase 2 ROCKET study in 2016, is not entirely surprising. Juno will now focus on JCAR017, a product with a defined T cell composition, and one it feels has better prospects for approval overall. While potential safety issues with CAR-T products have always been acknowledged, the news will test investor resilience. At its peak, later in the month, Kite Pharma announced completion of submission of its US Biologics License Application for axicabtagene ciloleucel in patients with relapsed or refractory aggressive NHL. Now we play the waiting game, for the approval of the world’s first CAR-T product.
Gene therapy is an approach that seems naturally suited to developing treatments for rare, monogenic diseases. This month sees progress for a much more prevalent condition – Alzheimer’s disease – with encouraging preclinical data released by Sangamo Therapeutics using its ZFP technology. Another interesting announcement comes from Lysogene, which released baseline data from its observational study in MPS IIIA. Regulators have recently made clear their desire to see companies operating in rare diseases provide data on the natural history of the condition under study – Lysogene is clearly fulfilling the wishes of regulators in this regard. Not a month goes by without an announcement from bluebird bio, and this month is no different, with the announcement of positive data from the first patient treated with its lentiviral vector for severe sickle cell disease.
Citation: Cell Gene Therapy Insights 2017; 3(4), 211-226.