Regenerative Medicine

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.

The US Centers for Medicare and Medicaid Services (CMS) has proposed to reimburse cancer treatments with chimeric antigen receptor (CAR) T cell therapies under a policy called ‘Coverage with Evidence Development.” Currently there is no national Medicare policy for covering CAR T cell therapy so local Medicare contractors will have discretion over whether to pay for it. The proposed coverage would require Medicare to cover the therapy when it is offered in a CMS-approved registry or clinical study in which patients are monitored for at least two years after treatment.

A male patient who was diagnosed with HIV infection in 2003 has experienced a remission from his disease after a haematopoietic stem cell transplant. The stem cell transplant was undertaken in order to treat a cancer. At the time the patient was on also on antiretroviral therapy to treat his HIV. The patient remained on antiretroviral drug therapy for 16 months after the transplant at which point the physicians stopped the drug therapy. Remarkably, the patient remained free of HIV.

Biogen Inc is to acquire Nightstar Therapeutics Plc, a gene therapy company spun out from the University of Oxford and financed by Syncona, which has a product in Phase 3 for the treatment of choroideremia, a rare retinal disorder. The US biotech company will pay $25.50 in cash for each share of Nightstar, valuing the company at approximately $800 million.

Sangamo Therapeutics Inc is readying a second generation of its zinc finger nuclease technology to use in an in vivo genome editing trial later this year. This is expected to provide further support for the company’s strategy of using engineered nucleases to correct DNA in patients with Hunter syndrome and other genetic diseases. Hunter syndrome is a rare genetic disorder that can lead to tissue and organ damage.

The Roche group has moved into gene therapy and simultaneously strengthened its franchise in haemophilia with the acquisition of Spark Therapeutics Inc, one of a small number of gene therapy companies in the world with a product on the market. The all-cash transaction is valued at approximately $4.3 billion, representing a substantial premium to Spark’s current stock price. Nonetheless, it is below the $8.7 billion that Novartis paid for AveXis Inc and the $9 billion Celgene Corp paid for Juno Therapeutics in 2018 – both gene therapy companies.

A gene therapy intended as a one-time treatment for dry age-related macular degeneration (AMD) has been successfully administered to a patient in the UK, marking the start of one of the first clinical trials of its kind. The therapy consists of a recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor.

A wave of clinical trial applications for new cell and gene therapies has reached the US Food and Drug Administration triggering a review of clinical guidance documents for developers. In an announcement on 15 January, Scott Gottlieb, the FDA commissioner, said new guidances are being drafted for gene replacement therapies as well as for cell-based gene therapies such as the chimeric antigen receptor (CAR) T cell drugs.

A partnership between German and US companies aims to develop gene therapies for neurodegenerative diseases that would cross the blood-brain barrier and deliver therapeutic agents to the brain.

The collaboration is between Sirion Biotech GmbH, which specialises in viral vector technologies for gene and cell therapies, and Denali Therapeutics Inc, a developer of treatments for diseases of the central nervous system.