A Phase 3 trial investigating a new small molecule drug for advanced fibrosis failed to meet its primary endpoint, sending the developer Gilead Sciences Inc back to the drawing board. The drug, selonsertib, was being investigated in patients with stage 3 fibrosis due to non-alcoholic steatohepatitis (NASH), a progressive liver disease characterised by the accumulation of fat and inflammation in the liver.
The US Patent and Trademark Office has issued a new patent to Emmanuelle Charpentier and colleagues for their work on the gene editing technology CRISPR-Cas9. The patent, granted to Dr Charpentier, the Regents of the University of California and the University of Vienna, focuses on systems and methods for using the CRISPR-Cas9 technology in a single guide format, including uses to target and edit or modulate genes, according to ERS Genomics Ltd, an Ireland-based company created to provide access to the technology.
Cambridge Innovation Capital Plc is leading an investment group that includes Genentech to provide seed capital to promising life science and healthcare companies in the UK and elsewhere. The seed capital will be issued by a new business accelerator called Start Codon located in Cambridge, UK.
Novartis’ new simplified structure was on display in the first quarter as the company finalised plans to spin-off its Alcon eye care division and reported financial results for its two core business units: innovative medicines and the Sandoz generics business.
A new player in the gamma delta T cell therapy arena has brought a cancer therapy to the clinic – this time for acute myeloid leukaemia. Glasgow, UK-based TC BioPharm Ltd has started a Phase 1 study of an allogeneic cell therapy consisting of activated and expanded gamma delta T cells.
Gamma delta T cells are a subset of lymphocytes which have both innate and adaptive immune properties. Besides TC BioPharm, at least two other companies are active in the field: GammaDelta Therapeutics Ltd of the UK and Gadeta BV of the Netherlands.
The first non-drug treatment for attention deficit hyperactivity disorder (ADHD) – a device that stimulates the trigeminal nerve – has been approved by the US Food and Drug Administration.
Called the Monarch external trigeminal nerve stimulation (eTNS) system, it is intended to be used in the home under the supervision of a caregiver. The prescription-only device is indicated for patients from seven to 12 years of age who have been diagnosed with ADHD but are not taking a drug treatment.
Hookipa Pharma Inc has priced its initial public share offering on the US Nasdaq market at $14 per share to raise a gross $84 million, excluding any exercise of the underwriters’options. The proceeds will finance the company’s portfolio of immunotherapies targeting infectious diseases and cancers.
The European Parliament has given its backing to Horizon Europe, a multi-year research programme for the sciences with a proposed budget of €100 billion. The programme is scheduled to run from 2021 to 2027, succeeding Horizon 2020.
It will finance collaborative research projects across the sciences including healthcare and medicine research and development. Included in the programme is a European Innovation Council that will finance the work of entrepreneurs who are exploring new areas of science. This council has already been successfully operating as a pilot project.
The Belgian cell therapy company Promethera Biosciences SA has reported a positive safety profile and early efficacy signals from a Phase 2a study of its lead therapy for liver disease, HepaStem. HepaStem is an allogeneic therapy derived from the stem cells of healthy donors.
The stem cells are administered by infusion into the bloodstream of patients where they circulate until reaching the liver, settle and support regeneration.
Catalent Inc, a service provider for the biotech industry, is to pay $1.2 billion to acquire Paragon Bioservices Inc a company with expertise in adeno-associated virus (AAV) vectors for gene therapy. The New Jersey, US-based company said it expects the market for gene therapies to grow by 25% in the coming years.