Read the full article »
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.
We saw some activity on the financing front this past month. Juno closed a public financing, raising gross proceeds of over US$300 million, which will give the company the financial power it needs to continue progressing what has become a substantial pipeline, while CAR-T developer, Autolus, closed a series C financing of US$80 million. Adaptimmune came upon some cash as well after GSK exercised its option to an NY-ESO T cell program. Adaptimmune received $48 million in total, which it will channel into other, wholly owned programs, including MAGE-A4, MAGE-A10 and AFP.
Alnylam’s pipeline update this month shows the perils and challenges of developing treatments that act on something as fundamental as a cell’s protein synthesis machinery. Whilst its RNAi treatment for acute intermittent porphyria (Givosiran) is progressing to Phase 3 in a study agreed with FDA, the news for Fitusiran is less encouraging, with the announcement of a patient death due to an initiating event of cerebral venous sinus thrombosis in its haemophilia A program. The approval by FDA of Biogen’s nusinersen for spinal muscular atrophy last year was an important milestone for RNA therapeutics, but this month’s news shows that the path for these treatments is never straightforward. The news is not all gloomy, as this month sees Agilis Biotherapeutics on the cusp of a BLA for its treatment for AADC deficiency and Dimension Therapeutics crossing the line to get its treatment for OTC deficiency into Phase 1.
Citation: Cell Gene Therapy Insights 2017; 3(8), 663-676.