Regenerative Medicine

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.

Abzena Plc is to apply its antibody technology to chimeric antigen receptor (CAR) T cell development under a new agreement with Tmunity Therapeutics Inc, a company co-founded by Carl June, head of the scientific team that discovered Kymriah.

Allergan Pharmaceuticals International Ltd has exercised an option to develop and commercialise a pre-clinical drug candidate that uses the CRISPR gene editing tool to potentially correct mutations in Leber Congenital amaurosis (LCA), a serious eye disorder.

Bluebird bio Inc, which is developing gene therapies for cancer and rare diseases, is partnering with Regeneron Pharmaceuticals Inc to discover, develop and commercialise new therapies for cancer. The deal includes a $100 million investment by Regeneron in bluebird stock.