The map of cystic fibrosis (CF) research is being redrawn in the U.K. as improvements in treatment, and in particular the introduction of CF modulator drugs, mean people with the rare inherited disease are living much longer.
Whole genome sequencing has substantially accelerated the pace of discovery of genes that cause rare diseases, but while this has brought the diagnostic odyssey of some patients to a conclusion, 50% to 80% remain undiagnosed after initial analysis.
The longstanding ambition of developing an inhaled gene therapy for cystic fibrosis has taken a step forward, with the start of a phase I/II trial of a product using a novel pseudotyped viral vector that it is hoped will circumvent problems encountered in previous studies with other vectors.
EG 427 SAS has closed a €27 million (US$28.3 million) series B round, which will fund it to completion of the first clinical trial of the lead gene therapy program, opening the way for its herpes simplex viral-vectored products to be developed in a range of chronic neuro-urology disorders.
Dealmaking in the Asia Pacific (APAC) region took off this week, with the latest showcasing Genome & Co.’s licensing deal with Ellipses Pharma Ltd. for GENA-104, a phase I-ready immuno-oncology asset, under undisclosed terms Feb. 11.
A large-scale study cross-referencing genomic data from multiple sources with primary care health records has identified genetic overlaps in 72 chronic diseases, opening the way for a more holistic approach to researching, treating and preventing multimorbidity.
For the pharmaceutical industry caught in the crosshairs of a potential trade war, the consequences of U.S. tariffs on China or Europe remain largely speculative, although both would be detrimental, according to a Korea Biotechnology Industry Organization (KoreaBIO) issue briefing Feb. 7.
The European Commission on Feb. 5 cleared Shanghai Henlius Biotech Inc.’s serplulimab (HLX-02) under the brand name of Hetronifly as a first-line combination therapy with carboplatin and etoposide to treat extensive-stage small-cell lung cancer.
The U.K. is continuing to shape up regulation, adding reform of its accelerated drug approval process and its draft guidance on personalized mRNA cancer vaccines to new clinical trial regulations that will come into force early in 2026. The Innovative Licensing and Access Pathway has been relaunched following a review of the industry’s experience of the scheme since its introduction in January 2021, and it will be open for applications from next month.
Europe has more oncology startups than the U.S., but many more U.S. companies scale up to the later growth stage. Given the proven links between patent ownership and access to finance, that critical gap is in part because U.S. companies hold nearly twice as many patents as European counterparts, according to an analysis by the European Patent Office.
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