Regenerative Medicine

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.

The Belgium-based cell therapy company Promethera Biosciences SA has raised €10 million from the Japanese conglomerate Itochu Corp as part of an ongoing Series D financing round to advance its portfolio of experimental medicines for chronic liver failure, nonalcoholic steatohepatitis (NASH) and fibrosis.

As part of the financing, Tajio Enoki, head of the medical business team in the energy and chemicals unit of Itochu, will join Promethera’s Board of Directors.

Novartis is proposing to acquire the contract manufacturer CellforCure of France in order to boost capacity for the manufacture of cell and gene therapies. Financial terms of the proposed transaction were not disclosed.

Novartis has agreed to offer Kymriah (tisagenlecleucel) the cell-based gene therapy, for an undisclosed discount to patients under the age of 25 years following a negotiation with UK authorities, including the National Institute for Health and Care Excellence (NICE). Supplies of Kymriah will be financed through the Cancer Drugs Fund, a special fund for promising treatments, NICE announced on 16 November.

A Chinese scientist’s reported use of the Crispr gene editing tool to alter a gene in two  embryos which were implanted into a mother’s womb has created an uproar in the scientific community. The edit was performed when the embryos were just a day old and involved alterations to the CCR5 gene in order to prevent the offspring from contracting HIV. They were reported on YouTube videos, rather in a scientific journal, after twin girls bearing the DNA changes were born.

A European research consortium in which Plasticell Ltd is a partner organisation has won a €3.7 million award from the EU to create cellular models of a group of neurological disorders, including Parkinson’s disease, which result from the acute or progressive loss of cells in the brain. The goal is to produce patient-specific human neural models in test tubes, microchips or model organisms that can be used to discover new therapies.

A new gene therapy company that uses chemogenetics-based technology has been launched in the US with plans to develop a product aimed at managing chronic neuropathic pain. CODA Biotherapeutics Inc is based in South San Francisco, California and made its debut on 12 September with a $19 million in Series A financing.

The checkpoint protein on immune cells may have another role to play in treating disease besides fighting cancer. This is the proposition being put forward by Altheia Science Srl, a new Italian biotech which is building a pipeline of agents to modulate programmed cell death-1 (PD-1) and its ligand (PD-L1) for the treatment of Type 1 diabetes and multiple sclerosis.

The European Medicines Agency has delivered a positive opinion recommending use of a new gene therapy, Luxturna (voretigene neparvovec), to treat adults and children suffering from an inherited retinal dystrophy caused by mutations in the RPE65 gene.

The disease causes vision loss that usually leads to blindness. There is currently no treatment for the disorder. Support to patients is limited to measures allowing disease management such as wearing a medical identification bracelet.