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.
CureVac AG has expanded its therapeutic reach to include projects targeting genetic diseases in addition to its portfolio of candidate drugs for cancer and infectious disease. This follows an agreement with Arcturus Therapeutics Ltd to develop a product to treat ornithine transcarbamylase (OTC) deficiency.
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.
A new Belgian start-up has been launched with Series A funding of €15.2 million to develop re-myelinating therapies for patients with multiple sclerosis and other myelin-related diseases. Called Rewind Therapeutics SA, the company is located in Leuven.
Addex Therapeutics Ltd has leveraged more than a decade of research into addiction with a partnership that will see it co-develop a portfolio product with Indivior Plc, a London-listed and US headquartered specialty pharmaceutical company.
Belgium-based iTeos Therapeutics SA has regained rights to an early-stage cancer drug from Pfizer Inc which was being investigated in a Phase 1 trial as a potential single agent for treating patients with malignant gliomas.
BioNTech AG, Europe’s largest privately-held biotech company, has raised $270 million in a Series A financing round to advance a portfolio of oncology drugs tailored to individual cancers. The round was led by the Redmile Group and joined by the Struengmann Family Office, a founding investor.
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.