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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.
It was a busy start to 2017 with multiple new immunotherapy products en route to the clinic. Kite submitted an IND for KITE-718, a TCR technology targeted to MAGE antigens that will be deployed for multiple solid tumor indications. Adaptimmune had an IND accepted for a TCR, also targeted to MAGE. On this front GSK also nominated its second target from Adaptimmune (PRAME) under its collaboration agreement signed back in 2014. Adaptimmune will bring the technology to IND readiness, after which it will be handed off to GSK to complete development. Cellectis submitted an IND for a gene-edited CAR product (UCART123) that will be targeted to AML and BPCDN, the first IND filing for an off-the-shelf T-cell product in the USA. Cellectis’ product wasn’t the only first in the USA though, NantKwest made headlines delivering the first off-the-shelf, engineered NK cell product into the clinic (haNK), which, like Kite and Adaptimmune’s products, will be targeted to solid tumors. It’s clear the race this year will be to demonstrate efficacy data in solid tumors, rather than liquid.
This month sees several companies advancing their programs by achieving important regulatory milestones, including Rare Pediatric Disease status for Lysogene’s AAV-based treatment for GM1 gangliosidosis, Orphan Drug Designation in Europe for Abeona’s AAV-based treatment for San Filippo B, and Fast Track Designation in the USA for Fibrocell’s autologous, fibroblast-based gene therapy treatment for dystrophic epidermolysis bullosa. Spark Therapeutics continues to release positive news on an almost monthly basis, with the announcement that a $15 million milestone payment has been triggered, and Sarepta Therapeutics builds on the recent approval of its exon-skipping therapy for Duchenne muscular dystrophy with the announcement of a research collaboration with Nationwide Children’s Hospital, where they will work together on their microdystrophin program, as well as another form of gene therapy.
Citation: Cell Gene Therapy Insights 2017; 3(1), 1-7.