Regenerative Medicine

The first gene therapy for haemophilia to be submitted for regulatory review, valoctocogene roxaparvovec, has been given ‘priority review’status by the US Food and Drug Administration. Developed by BioMarin Pharmaceutical Inc, the therapy will be assessed for safety and efficacy for the treatment of patients with haemophilia A. Haemophilia A is the more prevalent of the two disease types, haemophilia A and B. Under a priority review, the FDA aims to issue a decision on marketing in six months rather than the usual 10, which means the outcome of the review should be known by 21 August.

A new gene therapy company, Ikarovec Ltd, has been launched in the UK to develop a treatment for diabetic macular oedema, the most common cause of sight loss in people with diabetes. The company’s founder Peter Widdowson was previously chief executive of Quethera, another UK ophthalmic gene therapy company which was sold to Astellas Pharma Inc in 2018.

Evotec SE, which claims to have one of the industry’s biggest platforms for developing induced pluripotent stem (iPS) cells, has further expanded its partnership activities in the field. Under a 2016 agreement with Celgene Corp, now part of Bristol-Myers Squibb Co, Evotec has been using iPS cells to identify prospective treatments for neurodegenerative diseases. This agreement has now been broadened to include additional cell lines. The cell lines were not identified.

The Roche group has acquired commercialisation rights, outside the US, for an experimental gene therapy for Duchenne muscular dystrophy, a rare degenerative neuromuscular disorder. The deal was announced on 23 December and is with Sarepta Therapeutics Inc. It comes only days after Roche received regulatory clearance to acquire the gene therapy company Spark Therapeutics Inc.

New long-term data for the cancer therapy Yescarta (axicabtagene ciloleucel) have shown that nearly half of patients treated with the cell-based gene therapy were alive three years after treatment. The long-term data were presented on 7 December at the American Society of Hematology meeting in the US, and are similar to outcome data reported in 2017.

Kiadis Pharma NV has changed course and will in future focus its cell therapy capabilities on the development of natural killer (NK) cell products for the treatment of liquid and solid tumours. The Netherlands-based company acquired a portfolio of NK cell assets with the takeover of CytoSen Therapeutics Inc of the US in April.

bluebird bio Inc and Novo Nordisk A/S have entered into a research collaboration to jointly  develop in vivo genome editing treatments for genetic diseases, including haemophilia. During a three year research collaboration, the two companies will focus on identifying a gene therapy candidate for haemophilia A.

The Italian biotech company MolMed SpA is withdrawing its cell therapy for use in transplantation procedures, Zalmoxis, from the market. Announcing the decision on 10 October, the company said the decision had been taken “for commercial reasons.”

Zalmoxis (allogeneic genetically modified T cells) was approved for reimbursement in Italy in 2017. It was not immediately clear whether the product was being financially supported by any other European healthcare authorities.

A new US company, Trucode Gene Repair Inc, has been launched with $34 million in financing from Kleiner Perkins and GV, the venture capital arm of Alphabet Inc. The company is advancing an in vivo gene editing technology designed to correct mutations that cause genetic disorders such as sickle cell disease and cystic fibrosis.

Emily Whitehead celebrated her 14^th birthday in May, which was also the seventh year she has been free of cancer. The complete remission of her cancer - acute lymphoblastic leukaemia – is the first of many successes of the immunotherapy, chimeric antigen receptor (CAR) T cells, which are experiencing an expositive growth around the world.