The prospect of being able to re-dose a gene therapy has come into the fore with the launch on 4 January of Generation Bio Inc, a company with technology for delivering DNA directly to the liver using lipid nanoparticles rather than a viral vector.
Japan’s Takeda Pharmaceutical Company Ltd has announced plans to acquire the Belgium-based cell therapy company TiGenix NV for €520 million, a company with which it already has a licensing deal for the company’s lead product.
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.