Servier has entered into an agreement with Kaerus Bioscience, a UK-based biotechnology company co-founded by Medicxi, to acquire KER-0193, a potential treatment for Fragile X syndrome (FXS). Financial details include an upfront payment, with development and commercial earn-outs that could bring the total value of the deal to $450 million.
Enlaza Therapeutics Inc and Vertex Pharmaceuticals Inc agreed to collaborate on drug discovery aimed at creating small format drug conjugates and T cell engagers to treat certain autoimmune diseases and to improve conditioning in sickle cell disease and beta-thalassemia. Enlaza will receive $45 million in an upfront payment and equity investment. Vertex will work with Enlaza’s covalent biologics platform, called War-Lock, which uses proprietary non-natural amino acids to precision-engineer biologic medicines, resulting in an expanded therapeutic window.
Replicate Bioscience has entered into a multi-year research collaboration with Novo Nordisk A/S to develop self-replicating RNA (srRNA) therapies for obesity, type 2 diabetes and other cardiometabolic diseases. The deal combines Novo Nordisk’s expertise in metabolic disorders with Replicate’s proprietary srRNA platform, designed to enable scalable and durable protein expression.
A gene therapy being developed by Beacon Therapeutics Holdings Ltd to treat X-linked retinitis pigmentosa has shown progress in two ongoing Phase 2 trials, SKYLINE and DAWN. The product, laru-zova, was generally well tolerated by participants evaluated in the 36-month SKYLINE trial and by participants in the nine months DAWN trial, showing improvements across key measures of visual function, including low luminance visual acuity and microperimetry.
A gene therapy being developed by Beacon Therapeutics Holdings Ltd to treat X-linked retinitis pigmentosa has shown progress in two ongoing Phase 2 trials, SKYLINE and DAWN. The product, laru-zova, was generally well tolerated by participants evaluated in the 36-month SKYLINE trial and by participants in the nine months DAWN trial, showing improvements across key measures of visual function, including low luminance visual acuity and microperimetry.
YolTech Therapeutics Co Ltd, a Shanghai, China-based gene therapy company, raised approximately $45 million in a Series B financing led by the AstraZeneca-CICC healthcare investment fund. The proceeds will support the advancement of YolTech’s clinical programmes and its strategic development plans, the company said on 11 September. Yoltech, a developer of in vivo genome editing therapies, said it is advancing four clinical-stage programmes for:
Britain’s plans for expanding its position as a global centre for biopharmaceutical research and development hit a barrier on 10 September when Merck & Co Inc announced a decision to stop development of a £1 billion research centre in London, and vacate two other laboratories in the city.
Genmab A/S has received a ‘breakthrough therapy’ designation from the US Food and Drug Administration for an antibody-drug conjugate (ADC) which is in development for endometrial cancer and has shown promising activity in heavily pre-treated patients. The designation was announced on 26 August, just weeks after the company presented new data at the 2025 American Society of Clinical Oncology annual meeting in Chicago, US. The drug, rinatabart sesutecan, showed a 50% objective response rate and two complete responses in heavily pre-treated patients with advanced disease.
Food and Drug Administration Commissioner Marty Makary has announced plans to update the regulation of pharmaceutical advertising in the US in order to prevent deceptive messages being sent to patients by developers. On 9 September, the agency issued warning letters to pharmaceutical companies to remove misleading advertisements. Approximately 100 cease-and desist letters were sent to companies judged to have issued misleading ads.
Switzerland-based Aphaia Pharma AG has started a second Phase 2 trial of a new oral candidate drug for obesity which aims to restore endogenous endocrine balance in individuals with the disorder. The drug is a coated formulation of glucose, which is included on the World Health Organization’s list of essential medicines. It is designed to be released in the small intestine to restore endogenous nutrient-sensing signalling pathways and stimulate the release of hormones that control multiple functions including appetite, hunger, satiety and glucose metabolism.