Regenerative Medicine

Kite Pharma is to get its own chief executive under a reorganisation initiated by its parent company Gilead Sciences Inc, which has owned Kite since 2017. The move was disclosed on 2 May with Gilead’s first-quarter earnings announcement.

Daniel O’Day, the Gilead CEO, said in a conference call with analysts that Kite will become a separate business unit, according to Reuters.

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.

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.

The Dutch cell therapy company Kiadis Pharma NV has taken steps to strengthen its presence in the haematopoietic stem cell transplant sector with the agreed takeover of CytoSen Therapeutics Inc. The all-share transaction includes an upfront payment in stock as well as milestone payments contingent on the achievement of six clinical development and regulatory goals. CytoSen’s lead asset is a natural killer (NK) cell product designed to enable patients to receive a stem cell transplant from haploidentical donors. It is due to enter clinical development in 2020.

The ReNeuron Group Plc has entered into an exclusive licensing agreement with a subsidiary of the Shanghai Fosun Pharmaceutical (Group) Co Ltd of China for the development, manufacture and commercialisation of its two allogeneic cell lines which underpin the UK company’s candidate cell therapies for neurological disorders and diseases of the retina.

The UK gene therapy company Autolus Therapeutics Plc has raised $100.8 million in a US public share offering to support its portfolio of programmed T cell therapies for the treatment of cancer. This follows the issue of 4.2 million American Depositary Shares (ADSs) representing 4.2 million ordinary shares which were priced at $24 per ADS. In addition, Autolus has granted the underwriters a 30-day option to purchase up to an additional 630,000 ADSs at the public offering price.

An early stage chimeric antigen receptor (CAR) T cell therapy that targets an antigen expressed on myeloma cells has been singled out for a fast-track review by the European Medicines Agency. The cell-based gene therapy, JNJ-4528, is being co-developed by Nanjing Legend Biotech Co of China and Janssen Research & Development LLC.

A new gene therapy has been given a favourable scientific review in Europe paving the way for its use in patients with beta-thalassaemia as early as the second quarter. The treatment, Zynteglo, uses a lentiviral vector to deliver functional copies of a beta-globin gene into a patient’s haematopoietic stem cells in order to correct for a shortage of beta-globin. It was given a positive opinion by the European Medicines Agency on 29 March.

Pfizer Inc has entered into an option agreement to acquire Vivet Therapeutics SAS, a privately-held French company with a gene therapy for Wilson disease, a rare genetic disorder that prevents the body from regulating copper. The disease can lead to liver and brain damage.

Pfizer will initially take a 15% equity interest in Vivet with an exclusive option to acquire the remaining shares in the future. The exercise of the option is linked to the performance of the therapy in a Phase 1/2 trial.