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.
UK-based CHARM Therapeutics Ltd has raised $80 million from an international investor syndicate to support a preclinical product, based on a novel algorithm, for patients with acute myeloid leukaemia (AML). AML accounts for about one out of three leukaemias in adults in the US, but for those over the age of 65 years, the incidence is much higher. Announced on 2 September, the financing was co-led by new investors New Enterprise Associates and SR One with participation from existing groups including NVentures, the investment arm of the chipmaker Nvidia Corp.
Google LLC, the US technology conglomerate owned by Alphabet Inc, has been fined €2.95 billion by the European Commission for what it described as abusive practices in its online advertising business. The fine relates to practices dating from 2014 in which Google was found to have favoured its own online display advertising technology services, to the detriment of other providers.
Scientists across 25 countries representing 51 different nationalities have been awarded €761 million from the European Union to support original research in the physical and life sciences, social sciences and the humanities. The awards were announced on 4 September by the European Research Council, a public body established by the European Commission in 2007. The programme falls under Horizon Europe, the European Union’s seven-year programme for research and development.