Regenerative Medicine

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.

Promethera Biosciences SA has completed a Series D financing round to support the development of its cell therapies for liver disease, raising €39.7 million from global investors. A total of €10 million of the funding was committed by the Japanese conglomerate Itochu Corp in January.

Kite Pharma is to get its own chief executive under a reorganisation initiated by its parent company Gilead Sciences Inc, which has owned Kite since 2017. The move was disclosed on 2 May with Gilead’s first-quarter earnings announcement.

Daniel O’Day, the Gilead CEO, said in a conference call with analysts that Kite will become a separate business unit, according to Reuters.