Cell & Gene Therapy Commercial Insight – April 2018

In by

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.


April was a month of healthy financing activity for the T-cell industry. Just when we thought a series A couldn’t get any larger, Allogene hit the ground running with a $300 million financing to get deep into clinical development with a series of allogeneic CAR-T therapies. The company will be led by ex-Kite CEO Arie Belldegrun. Pfizer will retain a 25% equity interest in the company in exchange for the portfolio of assets it will contribute. The deal immediately positions Allogene as a market leader in allogeneic CAR-T therapy. Allogene’s primary competitor, Cellectis, successfully executed an IPO to haul in $165 million to continue clinical development of its universal T-cell therapies. Cellectis will use $100 million of its IPO proceeds to construct a gene editing facility to support its clinical programs. The advent of gene editing approaches to make allogeneic T-cell immunotherapy possible have propelled the technology and made the allogeneic versus autologous debate more relevant than ever.


This month sees Pfizer continuing to advance its rare disease and gene therapy pipeline, with the Phase 1 start of its AAV-based gene therapy for Duchenne muscular dystrophy, a program that comes from its acquisition of Bamboo Therapeutics in 2016. Pfizer leads the pack of big pharma companies advancing pipelines in rare diseases, following GSK’s recent exit from the field, and seems fully committed to getting novel therapies approved. In the opposite corner to the Pfizer leviathan, the small biotech company GenSight Biologics is making the best of results from its Phase 3 clinical trial in Leber’s hereditary optic neuropathy, which missed its primary endpoint of improvement in visual acuity; the company is pinning its hopes on differences in retinal ganglion cell macular volume in treated versus untreated eyes, but this is unlikely to be an endpoint acceptable to regulators, and it clearly hasn’t yet translated to a clinically meaningful effect on vision. It looks like more hard work ahead to get this treatment approved.

DOI: 10.18609/cgti.2018.040
Citation: Cell Gene Therapy Insights 2018; 4(4), 385-395.

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