The first gene therapy for patients with an inherited retinal disease was authorised by the US Food and Drug Administration on 19 December, bringing the number of approved gene therapies in the US up to three. Luxturna (voretigene neparvovec-rzyl), was developed by Spark Therapeutics Inc.
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.
Bruno Strigini, chief executive of Novartis Oncology is retiring from the company in early 2018, the Swiss multinational announced on 15 December. Mr Strigini joined Novartis in 2014 and has led the oncology division since 1 July 2016. A successor has not yet been named.
Kåre Schultz has announced a sweeping restructuring programme for Teva Pharmaceutical Industries Ltd that will see its workforce reduced by more than 25% and substantial changes to the company’s generic portfolio in the US.
A new oncolytic virus therapy that is intended to treat solid tumours is poised to enter clinical development triggering a $15 million milestone payment from Bristol-Myers Squibb Co to the developer PsiOxus Therapeutics Ltd of Oxford, UK.