Regenerative Medicine

It is often said that cell and gene therapies are a ‘process’ rather than typical drugs. This is based on the fact that their success depends on the quality of their starting materials and how they are manufactured. This could be an autologous chimeric antigen receptor (CAR) T cell-based gene therapy, or any one of the many allogeneic products in clinical development. Manufacturing is the key strategic issue for developers of these therapies, according to speakers at the On Helix annual meeting in Cambridge, UK on 7 July.

Spain-based VIVEbiotech SL has received regulatory approval for an upgrade of its lentiviral vector manufacturing capacity that will significantly expand its ability to produce vectors for both in vivo and ex vivo gene therapies. The company currently has 40 programmes ongoing with four dedicated to in vivo administration.

Galapagos NV is to acquire two companies with a presence in cell therapy as it takes steps to broaden its business under new leadership. The Dutch company has reached separate agreements to purchase CellPoint BV, a manufacturing company in the Netherlands, and AboundBio Inc, a US drug discovery company with an antibody library and antibody engineering capabilities.

Two French companies have joined forces to co-develop manufacturing technologies for AAV-based gene therapies. Coave Therapeutics is to contribute its expertise on viral vectors to the collaboration, while ABL, a contract development and manufacturing organisation, will provide production skills and laboratory space in Lyon.

New data for Kymriah (tisagenlecleucel), the first chimeric antigen receptor (CAR) T cell therapy to be approved for a cancer, have shown durable remission and long-term survival for the children and young adults treated with the therapy. The developer, Novartis, presented the data at the 2022 European Hematology Association Hybrid Congress on 12 June.

A syndicate of international investors has raised $75 million for Code Biotherapeutics Inc, a US company developing genetic medicines using a synthetic DNA delivery platform. The delivery technology is different from the conventional viral vector and is described by its developer as versatile and unimpaired by concerns about immunogenicity.

A Japanese company specialising in advanced therapies has received $30.3 million in Series B funding to advance a programme in tissue and organ repair. LUCA Science Inc is working on a mitochondrial replacement therapy that would restore cellular energy flow in damaged tissues and organs.

Germany-based Immatics NV is to make its cell therapy platform available to Bristol Myers Squibb Co in order to develop a further cohort of immunotherapies for cancer. The collaboration, announced on 2 June, is an expansion of an existing partnership aimed at developing off-the-shelf cell therapies for solid tumours.

Evotec SE has expanded its capabilities in the field of cell therapy with the acquisition of the Italian company Rigenerand Srl, a 2009 spin-off of the University of Modena and Reggio Emilia. The acquisition, valued at €23 million, comes within weeks of Evotec’s agreement with Sernova Corp of Canada to develop a beta cell replacement therapy for diabetes. The prospective therapy will use induced pluripotent stem (iPS) cells produced by Evotec, combined with a pouch developed by Sernova.

A new gene therapy project has been launched by ViGeneron GmbH of Germany and Regeneron Pharmaceuticals Inc that would potentially result in the development of a treatment for an inherited retinal disorder. The two companies announced their collaboration on 6 April but did not identify the disease.