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.
Asterias provided an update this month on progress in its SCiStar study investigating embryonic stem cell-derived oligodendrocyte progenitors (AST-OPC1) in patients with spinal cord injury. Several months ago the company reported on positive clinical benefit to patients through improved motor function. New data generated by magnetic resonance imaging shows that patients who received AST-OPC1 cells did not form lesion cavities in their spinal columns. Lesions, which typically fill with liquid, prevent recovery of motor and sensory function. This is a significant milestone in the development of treatments for spinal cord injury because it provides a mechanism for spinal cord recovery that directly results from engraftment of cells into the spine.
This month sees several companies release positive news at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting. Agilis Biotherapeutics (MA,USA), Solid Biosciences (MA, USA) and Intellia Therapeutics (MA, USA) all presented data showing advancement of their programs, and it’s good to see 5-year follow-up data from Agilis showing evidence of persistent clinical benefit in patients with AADC deficiency treated with a single dose of the CNS-delivered, AAV-based therapy. On the regulatory front, Spark Therapeutics (PA, USA) has completed its rolling BLA submission and two products – Xylocor’s (PA, USA) angiogenesis gene therapy for chronic, refractory angina and Sangamo’s (Canada) cDNA gene therapy for haemophilia A – have received fast track designation from FDA. Finally, news from Editas Medicine (CA, USA) that the planned IND for its CRISPR-based Leber congenital amaurosis treatment is delayed by at least 6 months shows the vulnerability of companies to challenges in manufacturing gene therapies.
Citation: Cell Gene Therapy Insights 2017;3