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.
A cell-based gene therapy for multiple myeloma has been recommended by the European Medicines Agency for patients with multiple myeloma, an incurable blood cancer that causes plasma cells to grow out of control. Symptoms can include bone fracture or even kidney failure. The drug, Carvykti (ciltacabtagene autoleucel), is a chimeric antigen receptor (CAR) T cell medicine consisting of a patient’s own immune cells which have been engineered to target and kill cancer cells.
Novartis is to explore new ways of delivering gene therapies to the brain under a licence option agreement with Voyager Therapeutics Inc, a US company with adeno-associated virus (AAV) capsid technology. Under the agreement, the Swiss multinational may elect to license novel capsids for use in directing therapies against three undisclosed central nervous system (CNS) targets. Further out, it may exercise options for two additional targets. The goal is to develop gene therapies for neurological diseases that manifest in deep regions of the brain.
An allogeneic cell therapy has been successfully used to treat patients 10 years and older with Duchenne muscular dystrophy, a rare genetic disease that affects both skeletal and cardiac muscle. The therapy, CAP-1002, was tested in a Phase 2, placebo-controlled trial and effectively reduced the deterioration of upper limb function in the patients as well as improved cardiac function. The results of the study, called HOPE-2, were published in The Lancet on 12 March 2022.
BioNTech SE has entered a three-year collaboration with Medigene AG to develop immunotherapies against solid tumours using Medigene’s proprietary T cell receptor (TCR) technology. This technology involves engineering a patient’s own T cells in order to display receptors that can recognise specific cancer antigens. Under the agreement, Medigene’s TCR discovery platform will be used in the development of cancer therapies against tumour targets nominated by BioNTech.