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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.
Atlas Venture and Third Rock launched Magenta Therapeutics with a $50 million series A this past month, perhaps the culmination of a period of renewed interest and activity among industry in the area of stem cell transplant. The company’s mission will be to bring transplant to the masses. While bone marrow transplant has been practiced for more than 50 years, generally it has only been used in instances where the benefit of a cure outweighs the risk of GVHD, which is often fatal. It has predominantly been used as a cure for leukemias and lymphomas. However, transplant has great potential beyond oncology, and many multiples of patients with rare blood disorders and severe autoimmune disorders stand to benefit from a world of transplant without GVHD. Magenta will seek to develop technologies in three areas: patient conditioning, stem cell mobilization and stem cell expansion/modulation; all areas of critical significance to the advancement of transplant, and factors that can help reduce patient mortality. Removing GVHD from the picture with improved matching could make it possible to safely ‘reset’ a patient’s immune system, which means the next generation of transplant technology could be a real threat to in vivo gene therapy in diseases such as Type 1 diabetes, multiple sclerosis, sickle cell anemia and Crohn’s disease.
This month illustrates the challenges faced by companies developing and commercializing gene therapy products, with uniQure announcing workforce cuts and pipeline prioritization. Nevertheless, the field continues to march forwards with further exciting developments in CRISPR-Cas9, with the announcement of the first clinical trial using this technology in patients with metastatic non-small-cell lung cancer. There is also positive news from AveXis, who have announced that the US Food and Drug Administration (FDA) has accepted the use of natural history data for comparison in its pivotal single arm trial in SMA Type 1. The FDA is notoriously resistant to the use of natural history data, so this is a real achievement!
Citation: Cell Gene Therapy Insights 2016; 2(6), 635-647.