Regenerative Medicine

UK-based Oxford Biomedica Plc has secured a new agreement to supply lentiviral vectors for gene therapies, this time with Juno Therapeutics, a member of the Bristol-Myers Squibb group. Announced on 18 March, the non-exclusive licence gives BMS access to the UK company’s lentiviral vector platform and is coupled with a five-year clinical supply agreement.

Positive long-term data have been reported for a cell therapy being tested in patients with retinitis pigmentosa, a group of hereditary diseases of the eye that can lead to a progressive loss of vision and blindness. Results from the ongoing US Phase 1/2a study of the human retinal progenitor cell therapy showed a meaningful clinical effect at all points in time out to 12 months after treatment.

GlaxoSmithKline Plc has expanded further into cell therapy with a partnership aimed at generating drug candidates for solid tumours. The collaboration is with Immatics Biotechnologies GmbH of Germany which has been working for several years with the MD Anderson Cancer Center in Texas, US on engineering T cells to fight cancer.

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.

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.