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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Commercial Insights

Cell & Gene Therapy Commercial Insight – January 2019

Mark Curtis & Richard Philipson

CELL THERAPY:

On the deal front the major news in January was the Obsidian-Celgene collaboration, which will give Celgene access to Obsidian’s Destabilizing Domain (DD) Technology. The platform, an enabling technology for enhancing cell therapies, allows for the regulation of the expression of immunomodulatory factors. Celgene will gain exclusive, world-wide rights to cell therapies incorporating DD-regulated IL12 and CD40L. As part of the upfront payment Celgene also took an equity stake in Obsidian. Enabling technologies, like Obsidian’s, which allow for precise control of engineered components of adoptive cell therapies, will play an increasingly important role in T cell immunotherapy in the future.

GENE THERAPY:

This month sees plenty of activity in licensing and collaborations, with Parkinson’s disease continuing to be a target of great interest to biotechs. Deals between Sirion and Denali, and Voyager and Neurocrine, both have Parkinson’s disease as a target of interest. The challenges of getting gene therapies to sites of interest in the CNS are formidable, but Voyager has already plotted a course to the clinic, with encouraging long-term data emerging from its phase Ib study in Parkinson’s, which uses stereotactic surgery to get the vector to the putamen. The news is less good for Translate Bio and its mRNA treatment for ornithine transcarbamylase (OTC) deficiency, which was placed on clinical hold by FDA at the end of last year. It’s not the first time Translate has faced a clinical hold; MRT5005, its treatment for cystic fibrosis, was placed on clinical hold in December 2017, taking 4 months before activities could be restarted. Let’s hope the current hold won’t take as long to lift.

DOI: 10.18609/cgti.2019.023
Citation: Cell Gene Therapy Insights 2019; 5(1), 149-164.
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