The UK medical research charity, LifeArc, is one of three donors of a £1 million award to researchers at University College London, UK, who are pioneering new therapies for motor neuron disease. This is a neurodegenerative disease that affects the nerve cells in the brain and spinal cord resulting in a person’s loss of voluntary movement. The two other donors are the Motor Neuron Disease Association and the My Name’5 Doddie Foundation.
BioNTech SE, the Germany-based vaccine and immunotherapy company, is to take full control of InstaDeep Ltd having made an equity investment in the company in January 2022. InstaDeep is a UK-based technology company that specialises in artificial intelligence and machine learning. BioNTech has a messenger RNA (mRNA) platform and was responsible, with Pfizer, for commercialising the first Covid-19 vaccine. InstaDeep has its headquarters in London, with offices in Paris, France and four other cities in the Middle East and Africa.
Asklepios BioPharmaceutical Inc, a wholly-owned subsidiary of Bayer AG, has entered into a multi-year research collaboration and option agreement with ReCode Therapeutics Inc. Under the agreement, the companies will work together to discover genetic medicines that precisely target a disease. This will be done by developing technology for the use of single vectors as delivery vehicles for gene editing and DNA cargoes.
The developers of the first chimeric antigen receptor (CAR) T cell therapies are to merge their assets under a deal that they say will speed up manufacturing and create new opportunities for treating solid tumours. The deal involves the acquisition of Tmunity Therapeutics Inc of Philadelphia, US, by Kite Pharma, a business unit of Gilead Sciences Inc. The financial terms of the transaction were not disclosed.
AstraZeneca Plc is to acquire a Dutch company with technology for developing T cell therapies for cancer which target antigens specific to a patient’s tumour. The company, Neogene Therapeutics Inc, was founded in Amsterdam, the Netherlands in 2018 and has a pipeline of candidate therapies that consist of engineered T cells equipped with receptors targeting tumour-specific antigens, also known as neoantigens.
Researchers from nine academic centres, together with PACT Pharma Inc of the US, have reported data from a Phase 1 study of a novel cell therapy which produced initial signs of clinical activity in patients with a variety of solid tumours. The treatment is an allogeneic T cell receptor (TCR) T cell therapy which has been edited to recognise the patient’s specific cancer. This was done by equipping the TCR with a neoantigen specific to the cancer.
A new gene therapy company has been launched in the US and UK by a group of academic-entrepreneurs who aim to treat genetic diseases of the eye with therapies delivered by a herpes simplex virus (HSV) vector. The company, Eudora, plans to employ an HSV-1 vector as a delivery vehicle instead of the commonly used adeno-associated virus (AAV) vector. In an announcement on 31 October, Eudora said its HSV vehicle could deliver more drug than an AAV vector, one of the most widely used virus vectors in the industry.
The European Medicines Agency has launched a new project to help academic scientists and those working for non-profit organisations understand the regulatory requirements for developing and eventually commercialising cell, gene and tissue engineered therapies. The project complements the agency’s priority medicines scheme, PRIME, which was launched in 2016 to provide support to developers of medicines that meet a major public health need.
France-based SparingVision SAS has raised €75 million in a Series B financing round to bring its two lead gene therapy assets into clinical development for the treatment of retinitis pigmentosa, an inherited retinal disorder. The adeno-associated virus vector therapies will deliver mutation agnostic treatments to the eye with a view to correcting the disorder.
The first cell-based gene therapy for the treatment of patients with beta thalassaemia, an inherited blood disorder, was approved by the US Food and Drug Administration on 17 August. Zynteglo (betibeglogene autotemcel) is a one-time treatment administered as an ex vivo lentiviral vector gene therapy. A functional beta globin gene is added into a patient’s cells outside of the body and then infused back into the patient. The treatment process is comprised of several steps and will be administered at qualified treatment centres in the US.