The European Medicines agency is recommending conditional approval of Tecartus (autologous anti-CD19-transduced CD3+ cells) for the treatment of relapsed or refractory mantle cell lymphoma. The chimeric antigen receptor (CAR) T cell therapy is the third medicine of its type to be reviewed by the agency. It follows the 2018 approvals of Kymriah for leukaemia and lymphoma and Yescarta for lymphoma.
Disruptions caused by the coronavirus pandemic depressed sales at Roche in the first nine months of the year but the company responded to the health crisis with the launch of new diagnostics and a partnership with Regeneron Pharmaceuticals Inc to develop an antibody treatment for the disease. In the third quarter, Roche received six medicine approvals in the US, the EU and Japan.
The US Food and Drug Administration has given its first approval to a treatment for Ebola virus, a potentially fatal disease affecting people in the Democratic Republic of the Congo (DRC) and several other African countries. The treatment, Inmazeb, is a mixture of three monoclonal antibodies which bind to the glycoprotein on the surface of the virus, blocking its attachment to cell receptors and preventing its entry into cells.
Evotec has acquired a new long-term strategic investor and received fresh funds from an existing shareholder to finance an expansion of its drug discovery portfolio and its geographical footprint in Europe and the US. The new investor is the Mubadala Investment Company PJSC, a sovereign wealth fund wholly owned by the government of Abu Dhabi. The existing investor is Novo Holdings A/S. Together they have purchased 11.5 million Evotec shares for €250 million, of which €200 represents the Mubadala investment and €50 million, the investment by Novo.
A preclinical study of an experimental cancer therapy has shown single-agent activity in models of glioblastoma, inducing long-term tumour eradications in a proportion of the treated animals. The results were published on 8 October, 2020 in Science Translational Medicine. The developer is Philogen SpA, a clinical-stage biotechnology company with headquarters in Italy.
A Phase 1/2a study of a candidate factor replacement therapy BIVV001 in adults with severe haemophilia A has shown significant levels of factor activity. The results were published on 10 September in The New England Journal of Medicine. The developer is Swedish Orphan Biovitrum AB which is collaborating with Sanofi SA on the programme.
Haemophilia A is an inherited bleeding disorder in which the blood does not clot properly. It is caused by a lack, or a decrease, of clotting Factor 8.
Switzerland-based Santhera Pharmaceuticals AG has discontinued development of Puldysa (idebenone), its candidate therapy for Duchenne muscular dystrophy after an independent review of an ongoing Phase 3 trial concluded that the trial was unlikely to meet its primary endpoint. The Data and Safety Monitoring Board based its conclusion on an interim analysis of the study, SIDEROS, which was being conducted in Duchenne muscular dystrophy patients using concomitant glucocorticoids.
The Nobel Prize in Chemistry has been awarded to Emmanuelle Charpentier and Jennifer A Doudna for their discovery of CRISPR-Cas9 genetic scissors, a method of genome editing. It marks the first time ever that two female scientists have jointly won the award.
The pair sparked a revolution in genome editing with their work, and CRISPR-Cas9 technology is now used widely in basic science, biotechnology and the development of future therapeutics. The tool can be used to change the DNA of animals, plants and microorganisms with extremely high precision.
This year’s Nobel Prize in Medicine or Physiology has been awarded to a team of scientists who discovered the hepatitis C virus. Harvey J Alter, Michael Houghton and Charles M Rice have made a “decisive contribution to the fight against blood-borne hepatitis,” according to the prize committee.
The virus is a major global health problem that causes cirrhosis and liver cancer. An estimated 71 million people have chronic hepatitis C virus infection, with almost 400,000 people dying from the disease in 2016, according to the World Health Organization.
Vaccibody AS, a clinical-stage Norwegian biotech, has entered an exclusive licensing deal with Genentech to develop and commercialise a DNA vaccine equipped with neoepitopes specific to a patient’s tumour. The vaccine, VB10.NEO, is designed to induce an immune response that is tailored to the tumours. It has already generated data in several cancers in a Phase 1/2a study.