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.
Boehringer Ingelheim GmbH has more than doubled the size of its corporate venture capital fund to €250 million in order to take advantage of new investment opportunities in start-up companies developing cell and gene therapies and products for infectious disease and cancer.
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.
Netherlands-based ISA Pharmaceuticals BV has secured its first industrial collaboration for a product developed from its synthetic long-peptide portfolio, a deal which both validates the technology and illustrates the trend in immuno-oncology towards the development of combination therapies.
Roche has reached an agreement to acquire California, US- based Ignyta Inc for $1.7 billion, a company with small molecule therapies targeting kinases which have been activated by gene fusions – a class of oncogenes associated with both blood cancers and solid tumours.
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).
A Phase 2 study of a new antibody treatment for Alzheimer’s disease has failed to meet its primary endpoint using an adaptive trial design that was intended to show efficacy at 12 months. Biogen Inc and Eisai Co Ltd said the blinded study will continue until the second half of 2018.
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.