Alnylam’s patisiran grabs EMA accelerated review; CRISPR Therapeutics, Casebia enter mRNA deal with CureVac

→ Promising Phase III data on patisiran have proven quite productive for Alnylam, which announced today that the EMA has granted an accelerated assessment for the hereditary ATTR amyloidosis treatment. The RNAi therapeutics company $ALNY plans to apply for market authorization in Europe at year-end 2017, roughly the same time frame as its intended NDA filing in the United States, where the IV drug also got a fast track designation. This news comes as Alnylam proposed another public offering of shares, aiming to raise around $675 million.

→ Philadelphia-based Excision BioTherapeutics has licensed the exclusive use of some new gene editing tech from UC Berkeley. The work was done by Jennifer Doudna — one of the original discoverers of the Cas9 editing tech — and Jillian Banfield, who have been working with uncultivated microbes in search of a next-gen approach that could trump what’s being used by the leaders in this field.

Allergan announced that the FDA has approved Vraylar for the maintenance treatment of adults with schizophrenia, less than two months after issuing a refuse-to-file letter on an application. The drug is designed to minimize relapse risk among patients in the long term. “This approval demonstrates our continued investment in VRAYLAR, as well as our commitment to developing treatments that address unmet needs facing people living with mental illness,” said R&D chief David Nicholson in a statement. Vraylar already has approvals for the acute treatments of schizophrenia and manic or mixed episodes of bipolar I disorder.

CRISPR Therapeutics, together with its joint venture Casebia, is teaming up with CureVac to develop improved Cas9 mRNA constructs for in vivo gene editing. CureVac will also take charge of manufacturing all mRNA needed in clinical development and commercialization of three programs in liver diseases. CRISPR Therapeutics and Casebia promised an unspecified upfront payment, research funding and potential milestones/royalties in return. Also today, the Swiss gene editing company $CRSP announced that Michael Tomsicek, formerly of medical implant manufacturer Abiomed and Merck-acquired pharma Cubist, will join as CFO.

→ Now that Ultragenyx $RARE has completed the acquisition of Dimension Therapeutics, its CEO Annalisa Jenkins is going on to run PlaqueTec in the UK. She’ll be leading the 2018 launch of new tech aimed at better management of coronary artery disease.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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