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Applied Therapeutics has a business model that defies the conventional wisdom about start-up success: reviving science abandoned by big pharma; financing from a narrow group of investors, dependent on the goodwill of a single academic institution; all in pursuit of a small molecule solution to one of the biggest, diversely complex and cost-defying challenges in chronic disease – the complications of diabetes.
Infographic: In Vivo explores trends in personalized medicine, looking at the 25 new molecular entities approved in the US last year with an individualized aspect.
Genomic medicine is coming of age. For Illumina chief scientific officer David Bentley, that not only means improved technology, accuracy and coverage of gene sequencing and a broadening into cancer, but also a better understanding among the wider population of disease and precisely the benefits that gene sequencing can bring to patients – actual and pre-symptomatic.
Once the stuff of (largely implausible) science fiction, gene therapy is now a clinical reality and one that is taking an increasing share of the pharma R&D limelight. In Vivo takes a look at how these therapies work, how the field has emerged and where it is likely to go next.
The Gilead subsidiary presented updated data at ASH for its first CD19-targeting CAR-T Yescarta and pivotal results for KTE-X19, which it plans to submit for US FDA approval in mantle cell lymphoma this year.
Phase Ib data at ASH look competitive with new pivotal trial results for Bristol/bluebird’s leading BCMA CAR-T bb2121. The Phase II portion of the CARTITUDE-1 study for JNJ-4528 is fully enrolled.
Data presentations at next week’s ASH meeting will be vital for Autolus to restore confidence in pipeline.
The Cancer Drugs Fund has improved access to oncology drugs, but if it is to remain fit for purpose it will need to evolve.
The EMA has declared itself satisfied with ‘refined manufacturing specifications’ for the beta thalassemia treatment
A bill that would promote genetic testing for infants and children, introduced by California Rep. Eric Swallwell, is gaining support in the US House of Representatives.
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