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Intellia lines up deal with Regeneron and a stock offering

Developer of CRISPR/Cas9 therapies finds a partner and looks to future investors to support its pipeline

by Ann M. Thayer
April 15, 2016 | APPEARED IN VOLUME 94, ISSUE 16

Pipeline
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Intellia is pursuing multiple targets with CRISPR/Cas9 edits
Source: Intellia
Intellia is pursuing multiple targets with CRISPR/Cas9 edits
Source: Intellia

Capitalizing on continued drug industry interest in CRISPR/Cas9 technology, the gene-editing therapy start-up Intellia Therapeutics has formed a new partnership and is preparing for an initial public offering (IPO) of stock.

Last week, Cambridge, Mass.-based Intellia signed a licensing and collaboration deal with Regeneron Pharmaceuticals. The partners will develop both the underlying CRISPR/Cas9 technology and in vivo therapies against as many as 10 targets. About half the targets will be diseases—such as the protein-accumulation disorder transthyretin amyloidosis—that may be treated by editing genes in the liver.

Regeneron will pay $75 million up front as well as potential milestone payments. In addition, Regeneron has agreed to invest up to $50 million in Intellia’s next equity financing. Intellia’s other drug industry investor is Novartis, which collaborates with the biotech firm in the areas of chimeric antigen receptor T cells (CARTs) and hematopoietic stem cells.

Meanwhile, Intellia, which is a 2014 spin-off of the CRISPR/Cas9 technology firm Caribou Biosciences, has filed to sell up to $120 million worth of stock. If it goes ahead with an IPO, it will be the second CRISPR start-up to do so. In February, competitor Editas Medicine raised about $100 million through its IPO. Its shares are now trading at more than twice their offering price.

Whether Editas’s good fortune continues and Intellia’s stock warrants a similar value is uncertain. The earliest clinical trials by either company are at least a year away. Editas is collaborating on CART therapies with Juno Therapeutics while a third CRISPR/Cas9 competitor, Crispr Therapeutics, works with Bayer and Vertex Pharmaceuticals.

At the same time, the companies’ leading scientists and founders are embroiled in a patent interference proceeding over rights to the basic CRISPR technology. Cowen & Co. stock analyst Phil Nadeau expects eventually to see multiple cross-licenses along with additional patents on individual therapies.

“The value in the CRISPR franchises will be determined by their ability to develop successful therapeutic products,” Nadeau told clients in a report.

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