Regenerative Medicine

The Zolgensma gene therapy for spinal muscular atrophy, which was approved by the US Food and Drug Administration in May 2019, has now received a positive review by the European Medicines Agency. Pending approval by the European Commission, it is expected to be available to treat babies and young children across the EU soon. Spinal muscular atrophy is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.

Llft BioSciences Ltd of the UK has teamed up with King’s College London to create a new version of its cell therapy for cancer which uses a special type of neutrophil to attack solid tumours. The cell therapy is still in preclinical development, but the company will work with the university to develop a new version using induced pluripotent stem cells (iPSCs). It believes the iPSC version will be easier to manufacture and will deliver significant cost savings to patients.

Human macrophages that were genetically engineered with chimeric antigen receptors (CARs) have demonstrated an anti-cancer effect in mice, suggesting a new application for cell therapy in solid tumours. Results from preclinical studies of the new molecule were published in Nature Biotechnology on 23 March 2020.

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.