Regenerative Medicine

Quell Therapeutics Ltd has secured its first major partnership for the development of its regulatory T cell technology for the treatment of autoimmune diseases. An agreement with AstraZeneca Plc, announced on 9 June, will bring the two parties together to research and develop potential therapies for Type 1 diabetes and inflammatory bowel disease. AstraZeneca is to make an $85 million upfront payment to Quell comprising cash and equity. If the collaboration is successful, with the commercialistion of new products, milestone payments could exceed $2 billion.

DiogenX SAS of France has raised €27.5 million in Series A finance from a venture capital syndicate led by the Boehringer Ingelheim Venture Fund and Roche Venture Fund to advance a candidate drug for type 1 diabetes into clinical development. The drug is a recombinant protein that modulates the Wnt/beta-catenin signalling pathway, a regulator of key cellular functions. In preclinical studies it has shown an ability to regenerate insulin-producing beta cells, according to Johannes Zanzinger, investment director at the Boehringer Ingelheim fund.

Novo Nordisk A/S is to collaborate with the Canadian biotech company Aspect Biosystems Ltd to use bioprinting technology to advance its work in cell therapies for diabetes and obesity. Novo is a leading producer of insulins and more recently glucagon-like peptide 1 medicines for the treatment of diabetes. It has also been building a presence in regenerative medicine.

The economic environment may be difficult, but there’s still plenty of money looking for good science, according to a panel of venture capitalists offering advice to start-ups at the Terrapinn Advanced Therapies Congress in London on 14 and 15 March. The conference came shortly after the collapse of Silicon Valley Bank, and all three VCs called on standard banks to become more start-up friendly, and advised start-ups to diversify their funds across a number of banks, wherever possible.

uniQure NV started 2023 with regulatory approvals in both the US and the EU for its gene therapy for haemophilia B as well as a pipeline of seven clinical and pre-clinical gene therapy projects – all in the field of neurology. Hemgenix (etranacogene dezaparvovec) for haemophilia B treats a genetic bleeding disorder and is the first gene therapy for this disease. Until its approval, the standards for haemophilia care were factor replacement therapies as well as a bispecific antibody treatment from Roche specifically developed for haemophilia A.

Adaptimmune Therapeutics Plc has initiated a merger with a US cell therapy company in order to expand its pipeline and develop therapies for solid tumours. The UK-founded company, which has offices in Oxford and Philadelphia, US, is to combine with TCR2 Therapeutics Inc in an all-share transaction valued at about $103 million. The combined company will be 75% controlled by Adaptimmune shareholders, and 25% by those from TCR2. The company will list on the Nasdaq market under Adaptimmune’s stock symbol and be led by Adrian Rawcliffe, the current Adaptimmune CEO.

The Food and Drug Administration is to allow Intellia Therapeutics Inc to include the US in its global trial of a genome editing therapy for hereditary angioedema (HAE), a rare protein deficiency that can cause swelling in multiple parts of the body. To date, the Phase 1/2 study has been taking place at sites in the Netherlands, New Zealand and the UK. The addition of sites in the US will increase the geographical reach of the programme, as well as the number of patients receiving the experimental therapy. Intellia is based in Cambridge, US.

Developers of cell and gene therapies will have more resources to conduct their research  following a commitment by two financial groups to expand a campus located in Stevenage, UK. Stevenage is the home of the Cell and Gene Therapy Catapult, an independent incubator supported by the UK government.

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.