Regenerative Medicine

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.

Oxford BioMedica Plc has teamed up with two UK partners to produce a gene therapy for cystic fibrosis that could be administered to patients through repeat dosing. Boehringer Ingelheim GmbH has an option to commercialise the product.

Sangamo Therapeutics Inc has announced plans to acquire TxCell SA in a deal that will give it ownership of the French company’s regulatory T cell technology for the development of therapies against autoimmune diseases.

A new company has been launched in the UK to develop a peptide-based nanoparticle for the delivery of DNA and RNA into cells for the treatment of cancer and other diseases. The company, NanoGenics Ltd, aspires to replace the live vectors that are currently used to deliver gene therapies with a peptide-based nanoparticle which mimics natural viruses but with qualities enabling repeat dosing.

Kite Pharma, a unit of Gilead Sciences Inc, has entered into a strategic collaboration with Gadeta BV, a privately-held Dutch company, to discover and develop gamma delta T cell receptor (TRC) therapies for cancer. Kite’s first product, a chimeric antigen receptor T cell therapy, has been approved by the US Food and Drug Administration for lymphoma. The company’s collaboration with Gadeta is focused on the treatment of solid tumours.

With three gene therapies on the US market, the Food and Drug Administration has provided six new guidance documents for developers to clarify the regulatory standards for these new medicines.

Three of the new guidance documents relate to regulatory standards for manufacturing and three are product specific. The product-specific guidelines are intended to assist developers of prospective gene therapies for haemophilia, retinal disorders and rare diseases.

Syncona Investment Management Ltd of the UK has launched two new gene therapy companies to complement its existing portfolio which includes Nightstar Therapeutics Plc and Freeline Therapeutics Ltd.