News

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.

CureVac NV’s candidate messenger RNA (mRNA) vaccine against Covid-19 has shown an overall efficacy rate of 48% in a large Phase 2b/3 trial during which multiple new strains of the SARS-CoV-2 virus were in circulation. The efficacy rate was achieved against Covid-19 disease of any severity, including single non-respiratory mild symptoms, the developer announced on 30 June.

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.

Intellia Therapeutics Inc has announced gross proceeds of about $600 million from a public offering of its shares on the US Nasdaq market. The offering consisted of 4.1 million shares which were priced at $145 per share on 29 June. The underwriters have also been granted a 30-day option to buy additional stock.

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.

A gene therapy for multiple myeloma that was approved in the US in March has now been recommended for a conditional marketing authorisation by the European Medicines Agency. Abecma (idecabtagene vicleucel) is a chimeric antigen receptor (CAR) T cell therapy targeting the B cell maturation antigen (BCMA), a cell membrane receptor expressed exclusively on late stage B cells and plasma cells.

Actemra (tocilizumab), a biologic treatment for rheumatoid arthritis, has been given an emergency use authorisation in the US for the treatment of adults and children two years and older who have been hospitalised with Covid-19. The approval was based on data from four clinical trials, one of which was RECOVERY, a trial led by researchers in the UK.

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.

The demerger of GlaxoSmithKline Plc into separate consumer healthcare and biopharmaceutical companies will result in a reset of R&D priorities, a compound annual growth rate in sales of more than 5% over the next five years and a new dividend policy, Emma Walmsley announced. The widely anticipated demerger will take effect in mid-2022 creating a new publically listed consumer healthcare company and a biopharma group focused on vaccines and speciality medicines. The consumer healthcare business is currently 68% owned by GSK and 32% by Pfizer Inc.

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.