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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.
Kite Pharma delivered new pivotal data this month that showed that there was a limited drop in complete response rate between 3 and 6 months in ZUMA-1, a study investigating lead candidate axicabtagene ciloleucel in patients with aggressive NHL. While there was a substantial drop in complete response rate in the first 3 months of the study, this decline softened over time, adding some confidence the therapy can be a cure for this form of lymphoma. While more follow-up will be required to substantiate durability, it certainly strengthens the case for approval at the FDA. While Kite’s launch of its first autologous CAR product becomes imminent, other developments continue to appear in off-the-shelf approaches to cell-based immunotherapy.
Exciting news this month in the field of hereditary muscle diseases! A collaboration led by the University of Washington has demonstrated preclinical proof of concept for systemically delivered, AAV-based gene therapy in dogs with X-linked myotubular myopathy. In addition, there is good news for boys with Duchenne muscular dystrophy, with Exonics announcing the award of seed funding to support preclinical development of its CRISPR/Cas9 technology, which could offer a one-off, curative treatment for the disease. Other interesting developments include the award of Breakthrough Therapy designation by FDA for UniQure’s AAV-5 treatment for hemophilia B, based on emerging clinical data from its ongoing, dose-ranging Phase 1–2 study. It’s always encouraging to receive ‘validation’ of clinical data from a regulatory authority with this type of designation!
Citation: Cell Gene Therapy Insights 2017; 3(2), 83-94.