Regenerative Medicine

A US company developing models of cancer tumours derived from the tissues of actual patients has received $70 million in Series A financing in order to advance its diagnostic technology. Xilis Inc of Durham, North Carolina has taken the concept of the organoid, a miniaturised organ, and adapted it to oncology. The financing was led by Mubadala Capital with participation by the new investors GV (formerly Google Ventures) and Life Sciences Partners (LSP) as well as existing investors.

Two European companies have decided to combine their expertise to develop a candidate gene therapy for the treatment of hereditary angioedema (HAE), a rare protein deficiency that can cause swelling in multiple parts of the body. Pharming Group NV, which already has an HAE product on the market, is to pay Orchard Therapeutics Plc for rights to a preclinical gene therapy programme for the same disease. The therapy, OTL-105, is an investigational ex vivo autologous haematopoietic stem cell therapy delivered with a lentiviral vector.

A new regenerative medicines company deploying regulatory T cells as potential therapies was launched in the US on 23 June with a Series A financing of $95 million. Abata Therapeutics plans to treat patients with severe autoimmune and inflammatory diseases, starting with non-relapsing multiple sclerosis, a disease for which there are no approved therapies. The Series A funding was provided by a syndicate led by Third Rock Ventures. Other investors included the biotech company ElevateBio LLC and the charity Juvenile Diabetes Research Foundation.

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.