Regenerative Medicine

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.

A new player in the gamma delta T cell therapy arena has brought a cancer therapy to the clinic – this time for acute myeloid leukaemia. Glasgow, UK-based TC BioPharm Ltd has started a Phase 1 study of an allogeneic cell therapy consisting of activated and expanded gamma delta T cells.

Gamma delta T cells are a subset of lymphocytes which have both innate and adaptive immune properties. Besides TC BioPharm, at least two other companies are active in the field: GammaDelta Therapeutics Ltd of the UK and Gadeta BV of the Netherlands.

The Belgian cell therapy company Promethera Biosciences SA has reported a positive safety profile and early efficacy signals from a Phase 2a study of its lead therapy for liver disease, HepaStem. HepaStem is an allogeneic therapy derived from the stem cells of healthy donors.

The stem cells are administered by infusion into the bloodstream of patients where they circulate until reaching the liver, settle and support regeneration.

Catalent Inc, a service provider for the biotech industry, is to pay $1.2 billion to acquire Paragon Bioservices Inc a company with expertise in adeno-associated virus (AAV) vectors for gene therapy. The New Jersey, US-based company said it expects the market for gene therapies to grow by 25% in the coming years.