Regenerative Medicine

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.

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.