Regenerative Medicine

UniQure NV has concluded an investigation of a patient who had a preliminary diagnosis of hepatocellular carcinoma (HCC) whilst participating in the gene therapy trial HOPE-B for haemophilia. In a statement issued on 29 March, the company said “it is highly unlikely the HCC was caused by etranacogene dezaparvovec.” The company has shared the data with the US Food and Drug Administration, which put a clinical hold on the trial in December 2020.

Medigene AG has reported the discovery of novel immunogenic tumour specific antigens derived from the non-coding regions of the human genome. The German biotech company presented the finding at the virtual annual meeting of the American Association for Cancer Research on 10 April.

Bluebird bio Inc announced on 16 February the temporary suspension of two gene therapy trials following reports of two cases of cancer. The trials were being conducted in support of a prospective gene therapy for sickle cell disease. The company is investigating the cause of the cancers in order to determine if there is any relationship with the lentiviral vector used in the studies.

A spin-out from the Erasmus Medical Center in the Netherlands has raised seed funding to discover and develop new T cell therapies for solid tumours including triple negative breast cancer. The company, Pan Cancer T BV, will be investigating T cell therapies which have been equipped with new receptors enabling them to target specific cancer antigens. The group of therapies is known as T cell receptor (TCR) T cells.

A joint venture cell and gene therapy company set up by UK-based Avacta Group Plc and South Korea’s Daewoong Pharmaceutical Co Ltd has closed a Series A financing of $7.3 million to further develop its pipeline of advanced therapies. The venture, AffyXell Therapeutics, was established in 2020 and is located in South Korea.

The US Food and Drug Administration has placed a clinical hold on uniQure NV’s gene therapy programme in haemophilia B following a possibly related serious adverse event, the company announced on 21 December. The hold affects the company’s pivotal Phase 3 study called HOPE-B which recently reported positive initial data at the annual meeting of the American Society of Hematology (ASH).

Syncona Ltd has provided £26.6 million in Series A funding to Resolution Therapeutics Ltd, a company it founded in August 2020 to exploit the wound healing capabilities of macrophages. Macrophages are cells of the innate immune system involved in the inflammatory response to injury or infection as well as in orchestrating the wound healing process that follows.

The Roche Group is to in-license viral vector technology from the German biotech company CEVEC Pharmaceuticals GmbH in order to increase its manufacturing capacity for gene therapies. This will specifically enable the company’s subsidiary Spark Therapeutics to scale up production of its marketed product Luxturna for retinal diseases which is delivered by an adeno-associated virus (AAV). Luxturna was approved in the US for a rare form of inherited vision loss in 2017. An EU approval followed in 2018.

Bayer AG is to pay up to $4 billion to acquire Asklepios BioPharmaceutical Inc (AskBio), a US gene therapy company with a clinical-stage pipeline and a significant manufacturing capacity. It will pay $2 billion upfront for the company and up to $2 billion in success-based milestones, of which three quarters are expected to be due over the next five years.

The gene and cell therapy industry has significant potential. But this will only be realised if manufacturers share information about their technologies, enabling the advanced therapies to be produced at scale and marketed at affordable prices.