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If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients. Novartis’ drug is also playing catch-up with its rivals on data from trials that show the reduction in LDL-C is matched by an improvement in cardiovascular outcomes.
If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients. Novartis’ drug is also playing catch-up with its rivals on data from trials that show the reduction in LDL-C is matched by an improvement in cardiovascular outcomes.
The Danish drugmaker is paying $100 million upfront for rights to antibody drug PRX004, which is heading for phase 2 trials in cardiomyopathy associated with ATTR amyloidosis, a life-threatening disease affecting the heart and nervous system. All told, the deal could be worth up to $1.2
million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. The start of dosing in the phase 1/1b trial of the drug has been delayed by the COVID-19 pandemic, but recruitment of the target 80 participants with advanced solid tumours is reported to be ongoing.
The American Society of Clinical Oncology (ASCO) annual conference was one of the highlights of the year in cancer research and the fact it was held virtually did not distract from some stunning clinical lung cancer trial results presented by AstraZeneca. In the 500 patient trial, patients treated with Trodelvy lived for a median of 12.1
As well as the access agreement for high-risk patients, the Novartis deal also covers a large-scale trial of the PCSK9 inhibitor and the creation of a consortium with academic groups aimed at improving the manufacturing of oligonucleotide drugs in the UK.
Gene-silencing Therapies. Silencing or modifying gene TTR aims to reduce the effect of both variant and wild-type TTR gene, thus reducing hepatic production by targeting its mRNA. The past years have witnessed a shift from off-label and symptomatic therapies to approved TTR stabilizers and gene-silencing therapies.
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