Regenerative Medicine

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.

The new companies, in which Syncona is the sole institutional investor, are Orbit BioMedical Ltd, a surgical platform company and SwanBio Therapeutics Ltd, a gene therapy company focused on neurological disorders. Syncona, formerly part of the Wellcome Trust, has invested $12 million in Orbit for an 80% stake, and $23 million in SwanBio for a 72% stake.

A gene therapy for a rare, genetic eye disorder has been selected by the US Food and Drug Administration for its Regenerative Medicine Advanced Therapy Designation in recognition of the product’s potential to treat an unmet medical need.

Avacta Group Plc of the UK has shown that it is possible to produce a therapeutic protein in mice by delivering DNA directly into the animal’s muscle tissue. Results of the preclinical proof-of-concept study were reported on 22 May.

A 2016 drug discovery alliance between Germany’s Medigene AG and bluebird bio Inc of the US has been expanded to raise the number of candidate compounds to be developed to six from four. The products are T cell receptor-modified T cell immunotherapies for the treatment of cancer.

Novo Nordisk A/S has announced plans to increase its commitment to cell-based therapies following the achievement of preclinical proof-of-concept for a cell therapy to treat Type 1 diabetes. The diabetes therapy, in which pluripotent stem cells are used to generate insulin-producing beta cells, is expected to enter human trials “within the next few years,” the company said on 16 May.

The US Food and Drug Administration has requested permanent injunctions against stem cell clinics in two regions of the country to prevent them from administering unapproved stem cell treatments to patients with cancer and other diseases. The move comes nearly 10 months after it issued warning letters to the clinics.

The Belgium-based cell therapy company Promethera Biosciences SA has branched out into biologics with the acquisition of Baliopharm AG, a Swiss developer of therapies for immune-mediated inflammatory diseases and cancer. Financial details of the transaction were not disclosed.

Orchard Therapeutics Ltd has come closer to its goal of becoming a global supplier of gene therapies following an agreement with GlaxoSmithKline Plc to take over that company’s portfolio of gene therapies for rare diseases.

Novartis appears set to become a first mover in gene therapy again with the agreed acquisition of AveXis Inc, a company with a late-stage gene replacement therapy for treating spinal muscular atrophy (SMA).

France-based Cellectis SA has acquired a new partner for its portfolio of products for cancer that are based on donor T cells that have been engineered to target specific antigen on cancer cells. The allogeneic portfolio had been licensed to Pfizer Inc and Servier.