Regenerative Medicine

bluebird bio Inc and Novo Nordisk A/S have entered into a research collaboration to jointly  develop in vivo genome editing treatments for genetic diseases, including haemophilia. During a three year research collaboration, the two companies will focus on identifying a gene therapy candidate for haemophilia A.

The Italian biotech company MolMed SpA is withdrawing its cell therapy for use in transplantation procedures, Zalmoxis, from the market. Announcing the decision on 10 October, the company said the decision had been taken “for commercial reasons.”

Zalmoxis (allogeneic genetically modified T cells) was approved for reimbursement in Italy in 2017. It was not immediately clear whether the product was being financially supported by any other European healthcare authorities.

A new US company, Trucode Gene Repair Inc, has been launched with $34 million in financing from Kleiner Perkins and GV, the venture capital arm of Alphabet Inc. The company is advancing an in vivo gene editing technology designed to correct mutations that cause genetic disorders such as sickle cell disease and cystic fibrosis.

Emily Whitehead celebrated her 14^th birthday in May, which was also the seventh year she has been free of cancer. The complete remission of her cancer - acute lymphoblastic leukaemia – is the first of many successes of the immunotherapy, chimeric antigen receptor (CAR) T cells, which are experiencing an expositive growth around the world.

Semma Therapeutics Inc, a company co-founded by stem cell scientist Douglas Melton, is being sold to Vertex Pharmaceuticals Inc to advance a potentially curative treatment for Type 1 diabetes. Vertex will acquire all of the outstanding shares of Semma for $950 million in cash.

Adaptimmune Therapeutics Plc has entered into a collaboration with the Japanese company Noile-Immune Biotech Inc to develop new T cell therapies for cancer. The partnership will involve co-developing Adaptimmune’s T cell products with technology originated by Noile-Immune enabling the T cells to secrete interleukin 7 and the protein CCL19. This is expected to improve the proliferation and trafficking of T cells to solid tumours. Adaptimmune will make an upfront cash payment and milestone payments to Noile-Immune of up to $312 million across all programmes.

Celgene Corp, which is in the process of being acquired by Bristol-Myers Squibb Co, has reached an option agreement with privately-owned Immatics Biotehnologies GmbH of Germany that would give it access to prospective cell therapies for cancer. Celgene already owns clinical-stage T cell therapies for cancer arising from its 2018 acquisition of Juno Therapeutics Inc.

Pfizer Inc has announced a $500 million investment in a new gene therapy manufacturing facility in Sanford, North Carolina, US, not far from the headquarters of Asklepios BioPharmaceutical Inc (AskBio Inc), a developer of adeno-associated viral vector (AAV) technology. The new Pfizer plant will use AskBio’s technology.

Bayer AG is to take full ownership of BlueRock Therapeutics LP, a regenerative medicines company that is developing engineered cell therapies in the fields of neurology, cardiology and immunology. Bayer, together with Versant Ventures, launched BlueRock in 2016 at a time of heightened interest in cell based therapies.

A group of international scientists has successfully repressed the transcription of the mutant gene causing Huntington’s disease, enabling animal models with the disease to show clear improvements in a range of important functions. Results of the research were published online on 1 July in the journal Nature Medicine. The studies were carried out by Sangamo Therapeutics Inc and the CHDI Foundation, a non-profit organisation devoted to Huntington’s disease research.