Developers of cell and gene therapies will have more resources to conduct their research following a commitment by two financial groups to expand a campus located in Stevenage, UK. Stevenage is the home of the Cell and Gene Therapy Catapult, an independent incubator supported by the UK government.
The European Medicines Agency has issued a report summarising actions that it has taken over the past four years to improve the availability of medicines for children. This activity is regulated by a Paediatric Regulation which came into force in the EU on 26 January 2007. The regulation establishes a special committee to coordinate the agency’s work in this area. The committee in turn decides which studies companies must carry out on children, alongside studies for the same disease that are being investigated in the adult population.
Trodelvy (sacituzumab), an antibody drug-conjugate first approved for triple-negative breast cancer, has had its label expanded by the US Food and Drug Administration to include the treatment of metastatic hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative breast cancer. The developer, Gilead Sciences Inc, said the approval was based on statistically significant and clinically meaningful progression-free survival and overall survival data from a Phase 3 study.
The US Food and Drug Administration has approved the first oral treatment for anaemia caused by chronic kidney disease in adults. The drug, Jesduvroq (daprodustat), was developed by GSK Plc and works by increasing the level of the hormone erythropoietin which signals the body to make red blood cells. These cells are deficient in anaemia. Jesduvroq has been approved for adults who have been on dialysis for at least four months. It is not authorised for patients who are not on dialysis.
The US Food and Drug Administration has given a full approval to the cancer treatment Jemperli (dostarlimab), having received follow-up data confirming the drug’s effectiveness in treating endometrial cancer. Jemperli was given an accelerated approval in August 2021 for adult patients with mismatch repair-deficient recurrent or advanced solid tumours. Mismatch repair-deficient tumours contain abnormalities that affect the proper repair of DNA when copied in a cell.
John Reed, the global head of research and development at Sanofi SA, has left the France-based multinational and will be replaced on an interim basis by Dietmar Berger, currently chief medical officer and global head of development. Dr Reed led R&D at Sanofi since joining the company in 2018. His departure was announced by Sanofi on 13 February. Shortly thereafter, Johnson & Johnson Inc announced his appointment as executive vice president for pharmaceutical R&D.
The UK medical research charity, LifeArc, is one of three donors of a £1 million award to researchers at University College London, UK, who are pioneering new therapies for motor neuron disease. This is a neurodegenerative disease that affects the nerve cells in the brain and spinal cord resulting in a person’s loss of voluntary movement. The two other donors are the Motor Neuron Disease Association and the My Name’5 Doddie Foundation.
Rewind Therapeutics SA of Belgium has recruited a new investor to support its programme for treating neurological diseases by restoring the remyelination function of the central nervous system. Sunstone Life Science Ventures A/S, an independent European investment firm, has joined the syndicate supporting Rewind which includes Boehringer Ingelheim Venture Fund, M Ventures and others.
Finland-based TILT Biotherapeutics Ltd has raised €22 million to advance its pipeline of oncolytic viruses to treat cancer. The immunotherapies have been equipped with molecules, including cytokines, that can be delivered locally and systemically to modify the tumour microenvironment and prevent the suppression of immune responses to cancer. The funding will enable the company to advance a group of candidate therapies, including the lead asset TILT-123, in clinical trials. This will be done in combination with immune checkpoint inhibitors.
Scenic Biotech BV of the Netherlands is to work with a unit of the US National Institutes of Health to advance its therapeutic programme for the treatment of Niemann-Pick Type C (NPC) disease, a severe, progressive neurological disorder. Scenic is developing a small molecule compound for the disorder by identifying genetic modifiers. These are naturally occurring genes that compensate for mutations that are responsible for severe diseases. The goal of the collaboration is to get clinical insight into genes that might reduce the severity of NPC in patients.