Regenerative Medicine

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.

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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.

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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).

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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.

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UK-based Autolus Ltd has made a submission to the US Securities and Exchange Commission relating to a potential initial public offering of its shares. As of 8 March, the timing and terms of the potential offering had not been determined.

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A zinc finger nuclease gene editing technology is to be used to develop ex vivo cell therapies for cancer, under a new research collaboration between the Gilead Sciences company Kite and Sangamo Therapeutics Inc.

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Synpromics Ltd is bringing its technology for creating synthetic promoters into a partnership with a unit of University College London (UCL) in order to help researchers there develop gene therapies for blood-based disorders. The agreement was announced on 25 January.

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The prospect of being able to re-dose a gene therapy has come into the fore with the launch on 4 January of Generation Bio Inc, a company with technology for delivering DNA directly to the liver using lipid nanoparticles rather than a viral vector.

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Japan’s Takeda Pharmaceutical Company Ltd has announced plans to acquire the Belgium-based cell therapy company TiGenix NV for €520 million, a company with which it already has a licensing deal for the company’s lead product.

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Spark Therapeutics Inc has set the price per patient of its new gene therapy for retinal dystrophy at $850,000 but at the same time opened negotiations with leading US insurers to link payments with the success of the treatment.

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