The European Medicines Agency has issued a positive opinion for an allogeneic stem cell therapy to treat complex perianal fistulas in patients with Crohn’s disease. The product, Alofisel (darvadstrocel), was developed by TiGenix NV of Belgium.
Orchard Therapeutics Ltd has obtained rights to technology developed by the French research institution Généthon to continue development of a new gene therapy for the immune deficiency, X-linked chronic granulomatous disease.
Bluebird bio Inc has teamed up with the Edinburgh, UK-based TC BioPharm Ltd to create new immunotherapies for cancer based on gamma delta T cells, a small subset of T cells involved in the initiation and propagation of immune responses.
Pending regulatory approval, a first clinical trial of a gene edited treatment for patients with beta-thalassemia, an inherited blood disorder, will start in Europe in early 2018. The therapy has been developed by CrisprTherapeutics AG and Vertex Pharmaceuticals Inc.
The Italian reimbursement authority has reached an agreement with MolMed SpA under which it will reimburse the company’s cell therapy for patients who have received a stem cell transplant at €149,000 per infusion. Treatment can require up to four infusions.
Fate Therapeutics Inc has directed more resources toward NK cell-based immunotherapy with a new research deal covering potential allogeneic therapies for cancer. The agreement, announced on 6 December, is with the University of California at San Diego, US.
Semma Therapeutics Inc, a company founded by the Harvard University Professor Douglas Melton, has raised $114 million in an oversubscribed Series B financing round to bring a stem cell therapy for Type1 diabetes into the clinic.
The US Food and Drug Administration has approved a second gene therapy to treat cancer, giving a further boost to developers of the new technologies. The approval is for Yescarta (axicabtagene ciloleucel), a chimeric antigen receptor (CAR) T cell therapy to treat adults with certain types of large B-cell lymphoma.
GlaxoSmithKline Plc, which has a gene therapy on the market, is broadening its portfolio of advanced therapy medicinal products to include potential new treatments derived from induced pluripotent stem cells (iPSC). iPSCs are a type of pluripotent stem cell that can be generated directly from adult cells.
Spark Therapeutics Inc’s gene therapy for the treatment of inherited retinal dystrophy has received the unanimous backing of an advisory committee of the US Food and Drug Administration ahead of the agency’s decision on marketing in early 2018.