Cell & Gene Therapy Commercial Insight – June 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.

Cell Therapy


Mark Curtis. Financial Portfolio Manager, Emerging Technologies, Lonza AG, Switzerland

The string of successful financings continued in the month of June with both Neon and Autolus closing IPOs totaling more than $250 million, and Carisma closing a Series A of $53 million. In other news, Brainstorm Cell Therapeutics was ultimately not able to provide access to its NurOwn technology through the recently enacted Right-to-Try (RTT) legislation signed into place by Trump on May 30 of this year. While Brainstorm was able to meet three of four criteria required to successfully utilize the RTT pathway, including tapping into experienced clinical trial sites, educating patients on benefits and potential risks, and limiting access to patients that do not meet clinical study inclusion criteria, the company was unable to identify an alternative funding source for patients unable to pay for the therapy themselves. While RTT is a step in the right direction in terms of addressing indications of high unmet need, one wonders how useful the pathway will prove to be given that most, if not all, technologies intended to be administered under RTT will be high cost by their nature.

Gene Therapy


Richard Philipson. Chief Medical Officer, Trizell Ltd, UK

This month sees two companies receive Regenerative Medicine Advanced Therapy (RMAT) designation for their products – Nightstar’s treatment for choroideraemia and Voyager’s treatment for Parkinson’s disease. The RMAT designation, which requires preliminary clinical evidence that indicates that a therapy has the potential to address unmet medical needs, was established in the 21st Century Cures Act and builds on other mechanisms to expedite drug development and approval such as Breakthrough Therapy and Fast Track designations. Elsewhere, some good news for Solid Biosciences with the lifting of the FDA’s clinical hold on its Phase I/II trial in Duchenne muscular dystrophy; the company is in a race with Sarepta and Pfizer, which are both developing competing mini- or micro-dystrophin gene therapy treatments, with preliminary data from Sarepta’s product already looking very encouraging.

DOI: 10.18609/cgti.2018.059
Citation: Cell Gene Therapy Insights 2018; 4(6), 607-626.

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