Regenerative Medicine

Three industry associations have issued an appeal to the European Commission to exempt advanced therapy medicinal products (ATMPs) from the EU’s genetically modified organism (GMO) legislation. This is to make the region more competitive as a site for clinical trials. The appeal was issued in a statement from the Alliance for Regenerative Medicine; the European Federation of Pharmaceutical Industries and Associations; and the European Association of Bioindustries. A paper was also published online on 24 May in the journal Human Gene Therapy.

Dyno Therapeutics Inc, based in Cambridge, Massachusetts, US, has raised $100 million in a Series A financing round to expand its technology platform for designing adeno-associated virus (AAV) vectors for gene therapies. The company is using artificial intelligence to improve the performance of capsids, the cell-targeting protein shells of AAV vectors.

A new US company with technology for developing tissue-targeted gene therapies for multiple diseases, has launched with $140 million in venture capital financing and collaboration income from AbbVie Inc. Capsida Biotherapeutics Inc is based on technology developed by Viviana Gradinaru and colleagues at the California Institute of Technology in the US.

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.