Regenerative Medicine

A genome editing therapy designed to inactivate a mutated gene in the liver has reported positive Phase 1 data – the first therapy of its kind to achieve an effect inside the human body. The drug, NTLA-2001, is intended as a one-time treatment for people living with transthyretin (ATTR) amyloidosis, a rare disease caused by misfolded proteins. The study results were reported on 26 June by Intellia Therapeutics Inc and its partner Regeneron Pharmaceuticals Inc, and simultaneously published in The New England Journal of Medicine.

Blackstone Life Sciences has put its financial muscle behind a start-up company that plans to develop allogeneic CAR T cell therapies for cancer. The US private equity group is committing $250 million to the venture, which will use T cells that have been engineered to express a universal chimeric antigen receptor (CAR) against tumour antigens. The T cells will be derived from healthy donors rather than from the patients themselves. They can also be silenced in order to avoid cytokine release syndrome.

uniQure NV is to acquire a France-based gene therapy company whose lead programme is a prospective treatment for temporal lobe epilepsy, the most common form of focal epilepsy. Corlieve Therapeutics SAS has been working on prospective therapies for neurological disorders since its founding in late 2019.

A new gene therapy company based in the Netherlands has raised €31 million in seed financing to advance a preclinical portfolio of potential treatments for muscular and neurodegenerative disorders. VectorY Therapeutics BV was launched in October 2020 to develop vectorised antibodies using an adeno-associated virus vector platform and antibody-based targeted degradation technologies.

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.