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.
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.
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.
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.
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.
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.
Evotec AG has expanded its capacity to discover new drugs through the use of adult stem cells that have been reprogrammed to produce any type of cell in the human body. These induced pluripotent stem (iPS) cells can be used to model disease and identify prospective treatments.
Another step towards developing a cell therapy for retinal disease was reported by UK-based ReNeuron Group Plc on 19 June, following approval by the US Food and Drug Administration of its formulation for cryopreserving cells.
Novartis’ candidate chimeric antigen receptor T (CAR T) cell therapy for cancer is scheduled to be reviewed on 12 July by the US Food and Drug Administration’s Oncologic Drugs Advisory Committee, according to Oxford BioMedica Plc, which is supplying lentiviral vector for the product.
A cartilage repair product developed by co.don AG of Germany received a positive opinion from the European Medicines Agency on 19 May, making it the ninth advanced therapy medicinal product (ATMP) to be assessed for marketing in Europe.