Protocols

Alnylam makes RNAi history again as patisiran gets approved in Europe; Vertex buys into UK-based machine learning group

→ Just days after Alnylam landed a historic FDA OK on patisiran — and quickly launched it as Onpattro — the European Commission has come through with its own marketing authorization. The drug, which treats hereditary ATTR amyloidosis by silencing messenger RNA and blocking the production of TTR protein, is the first RNAi therapy to be approved in Europe. It’s also a likely blockbuster-to-be, with Clarivate pegging 2022 projected sales at $1.21 billion even though the disease afflicts only about 30,000 people worldwide. Inotersen, a rival product from Akcea and Ionis, was approved in Europe in July.

Vertex is partnering with UK-based Genomics plc on their platform for genetics and machine learning. Vertex $VRTX made a £10.5 million equity investment in Genomics plc as part of its £25 million Series B funding round — also announced today. “Human biology and genetics are a foundation of drug discovery at Vertex and the team at Genomics brings together a unique combination of sophisticated data science and human genetics,” said Vertex CSO David Altshuler. “This partnership will pioneer new uses of genomic tools and technologies to advance Vertex’s ongoing work and investment at our Oxford Research Site and globally to bring new medicines to the patients that need them.”

→ We finally know what to call China’s revamped FDA. The drug regulator will now go by the National Medical Products Administration, or NMPA, to reflect its new mandate after a government reorganization separated medicine regulation from other consumer goods — including everything food-related. NMPA reports to the newly created State Administration for Market Regulation.

Evotec and the CHDI Foundation have extended their neuroscience collaboration for a few more years. In the pact, CHDI will help fund the work of up to 75 scientists focused on Huntington’s disease.

Tempus, a precision medicine company providing data analytic services to physicians and researchers, has raised $110 million in Series E financing. Eric Lefkofsky — best known for co-founding Groupon — started the company to assist real-time decision-making for patients, first in cancer care. Harnessing their pool of patient data, the company also operates an organoid development and testing facility to build biological models that they believe can inform clinical trials.


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