Spark Therapeutics Inc has set the price per patient of its new gene therapy for retinal dystrophy at $850,000 but at the same time opened negotiations with leading US insurers to link payments with the success of the treatment.
Following a positive opinion in Europe for its cell therapy for fistulas in Crohn’s disease, TiGenix NV has tightened its therapeutic focus to concentrate on allogeneic therapies and in particular, on inflammatory conditions associated with immune-mediated diseases.
Orchard Therapeutics Ltd has completed an oversubscribed $110 million Series B financing round to support its pipeline of gene therapies which is led by a clinical-stage product for the inherited disorder, adenosine deaminase severe combined immunodeficiency (ADA-SCID).
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.