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Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit

pharmaphorum

Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. The post Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit appeared first on.

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Laverock Therapeutics raises £13.5m to accelerate gene silencing platform

BioPharma Reporter

Laverock Therapeutics, the gene editing-induced gene silencing platform for human therapeutic applications, has expanded its seed funding round to Â13.5 million.

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uniQure signs license deal for Apic Bio’s gene therapy for SOD1-ALS

Pharmaceutical Technology

Under the terms of the deal, uniQure will obtain the worldwide rights to develop and market the clinical stage gene therapy, APB-102. uniQure stated that the license of APB-102 further strengthens its gene therapies pipeline developed for the treatment of neurological disorders as well as miRNA-based gene silencing programmes.

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Two biotechs target dry eye disease after Novartis’ Xiidra problems

pharmaphorum

The pipeline for dry eye disease drugs is beginning to heat up, with two biotechs announcing key developments, aiming to disrupt a market led by Allergan and Novartis. billion for its FDA-approved dry eye drug Xiidra during the Japanese pharma’s merger with Shire in 2019, outlining the sales potential in this market niche.

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FDA approves Alnylam’s ultra-rare disease drug Oxlumo

pharmaphorum

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The Swiss pharma’s bet looks to have paid off as inclisiran is likely to be approved in Europe in the coming weeks after backing from CHMP regulators last month.

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High hopes as Inventiva takes NASH contender into phase 3

pharmaphorum

The regulator said it wanted more long-term safety and efficacy data from the REGENERATE study before making a final decision on the drug, which had been expected to be the first entrant into a likely multi-billion dollar market niche. The post High hopes as Inventiva takes NASH contender into phase 3 appeared first on.

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MHRA gives Alnylam ‘innovation passport’ for hypertension drug zilebesiran

pharmaphorum

The UK medicines regulator has awarded Alnylam’s RNAi-based therapy zilebesiran for hypertension an ‘innovation passport ‘, a designation designed to speed up NHS access to promising new medicines.