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.
A new gene therapy has been approved in Europe, paving the way for its use in patients with beta-thalassaemia, a rare blood disorder. The treatment, Zynteglo, uses a lentiviral vector to deliver functional copies of a beta-globin gene into a patient’s haematopoietic stem cells in order to correct for a shortage of beta-globin. It was given a positive opinion by the European Medicines Agency on 29 March and a conditional approval by the European Commission on 29 May.
Syncona Ltd, an active investor in cell and gene therapy, has launched a new company, Quell Therapeutics Ltd, which will develop cell therapies to treat autoimmune conditions based on engineered T regulatory (Treg) cells. Tregs are a subset of T cells with the potential to down-regulate the immune system.
Rinri Therapeutics Ltd, a spin-out of Sheffield University in the UK, has received £1.4 million in seed funding from a European syndicate in order to advance a prospective stem cell therapy to restore hearing. The company aims to reverse neuropathic sensorineural hearing loss by repairing the damaged cytoarchitecture of the inner ear.
A new French company working in the field of stem cell culture has raised €7.1 million in a Series A financing round to advance its technology for mass producing stem cells. TreeFrog Therapeutics SAS was founded in November 2018 on the basis of years of academic research.