Cell & Gene Therapy Commercial Insight – November 2017

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.


This past month the FDA made progress in formalizing the development and commercialization of cell therapy and regenerative medicine products in the USA with the release of new guidance describing the regenerative medicine advanced therapy (RMAT) designation and how it relates to other regulatory support mechanisms. The RMAT, which is reserved for therapies that are intended to treat, modify, reverse or cure, serious conditions, will include all of the benefits of the existing breakthrough therapy designation and fast track designation, including discussions on surrogate endpoints. Companies seeking a RMAT designation will only need to show that a therapy has the potential to address an unmet medical need in order to receive support. The policies now in place will no doubt stimulate the development of novel regenerative medicine therapies. What will be critical now is the development of a reimbursement environment conducive to the uptake of these therapies as they make their way to market.


This month sees plenty of good news in the clinical development arena. Two companies have announced positive progress in their early clinical development programs: Ultragenyx has successfully completed the first cohort of three patients in its Phase 1 study in OTC deficiency, and Abeona has enrolled the first patient with MPS IIIA at its site in Spain, adding to already active sites in the USA and Australia. Ziopharm has released new data showing encouraging median overall survival in patients with recurrent glioblastoma treated with its IL-12-based gene therapy, and AveXis has achieved a notable milestone with the publication in the New England Journal of Medicine of positive clinical data from its ongoing trial in spinal muscular atrophy. There’s also an announcement from BioCanCell that clinical data on its lead product, BC-819, in development for non-muscle invasive bladder cancer, will be presented at the upcoming Genitourinary Cancers Symposium in February 2018.

DOI: 10.18609/cgti.2017.086
Citation: Cell Gene Therapy Insights 2017; 3(10), 891-901.

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