Regenerative Medicine

It is one thing to research and develop an advanced therapy medicinal product (ATMP), but quite another to bring it successfully to market. Among the nine ATMPs approved in Europe since 2009, only five are still on the market.

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A Phase 3 trial of a candidate gene therapy for retinal disorder has been highlighted in The Lancet as a possible standard for future trials of genetic medicines. The trial was conducted by Spark Therapeutics Inc whose therapy, voretigene neparvovec, is under review at the US Food and Drug Administration.

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The US Food and Drug Administration has taken steps to curb the activities of unlicensed stem cell clinics while preparing for the roll-out in the autumn of a new regulatory framework for developers of cell and tissue-engineered products.

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Gilead Sciences Inc has made a dramatic entry to the field of regenerative medicine with an agreed bid for Kite Pharma Inc which has a portfolio of engineered cell therapies, the most advanced of which is under review in both the US and the EU.

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An experiment in which CRISPR-Cas9 was used to repair a gene mutation in a human embryo appears to have solved two safety problems associated with this technology while raising ethical questions about its long-term application.

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UK-based Nightstar Ltd has recruited David Lubner to its board of directors less than a month after raising $45 million in a Series C financing round to advance its lead gene therapy product for choroideremia, an inherited cause of progressive blindness.

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A gene therapy developed by Spark Therapeutics Inc for an inherited retinal disease has received a paediatric designation from the US Food and Drug Administration. The decision is the agency’s way of highlighting the treatment’s potential for children and young adults.

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Syncona Investment Management Ltd, an investment company in which the Wellcome Trust has a significant holding, reported total net assets of £895 million at the end of its fiscal year on 31 March, up from £472 million a year earlier. This follows its merger in November 2016 with BACIT Ltd, an investor in diversified portfolios based in the Guernsey Islands.

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Shire Plc is advancing a candidate gene therapy acquired from its 2016 purchase of Baxalta, with plans to take the product into a first human trial in patients with haemophilia A. The product is a factor VIII gene therapy delivered in an adeno-associated virus (AAV) vector.

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TiGenix has joined the growing number of European companies with a presence in the US to open an office in Cambridge, Massachusetts and prepare for the registration of its lead stem cell therapy product intended to treat fistulas in patients with Crohn’s disease.

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