- Intellia Therapeutics, one of several companies working to develop CRISPR/Cas 9 technology commercially, on Monday filed to go public in an initially priced $120 million IPO. Concurrently, the biotech announced a collaboration and licensing deal with Regeneron Pharmaceuticals aimed at advancing up to 10 CRISPR-based programs, focusing primarily on liver diseases.
- Regeneron will pay Intellia $75 million upfront, as well as investing $50 million in Intellia's forthcoming IPO. Additionally, the agreement includes as much as $320 million in milestone payments, according to SEC filing documents.
- The first program to be co-developed with Regeneron will target a rare genetic disorder known as ATTR, which can causes severly impaired nerve or cardiac function.
Intellia is one of several companies developing commerical CRISPR treatments, competing with the likes of CRISPR Therapeutics, Editas Medicine, and others. Editas Medicine has already gone public, with $94 million offering in February.
The new collaboration agreement with Regeneron will help Intellia develop new in vivo therapries, where CRISPR products are delivered directly into the body. Under the six-year agreement, Regeneron will have exclusive rights to develop products against up to 10 targets, at least five of which will be focused on diseases treatable by editing genes found in the liver.
For example, Intellia hopes CRISPR gene-editing can cure the genetic disorder ATTR, which is caused by a buildup of the transthyretin (TTR) protein in tissue. By "knocking out" TTR expression in the liver, Intellia could reduce or eliminate the disease-causing buildup.
In addition to ATTR, Intellia is developing three other liver programs and expects one to two of the programs to reach clinical trials in the next 1 to 2 years.
Partnership with Novartis
Prior to this new deal, Intellia and Novartis had entered into a separate partnership focusing on CAR-T cells and hematopoietic stem cells. Novartis gained exclusive rights to Intellia's CAR-T program, and the companies will jointly advance the stem cell therapies. Unlike the deal with Regeneron, the partnership with Novartis focuses on ex vivo therapies, in which cells are taken out of the body, genetically edited, and then re-introduced.
The Novartis partnership also comes with a hefty schedule of milestone payments. According to regulatory filings, Intellia is eligible for $230 million in milestone payments, along with single-digit royalties. However, Novartis now owns 20.3% of the company.
The company sublicensed its technology from CRISPR pioneer Jennifer Doudna's biotech startup Caribou Biosciences. One of the patents Intellia received is now the subject of interference proceedings between the Broad and Berkeley. If the Broad wins, Intellia could face patent litigation on any of its CRISPR products in development or commercialization.
Furthermore, one of Intellia's key competitors - Editas Medicine - received its CRISPR technology IP from a deal with the Broad, raising the stakes of the ongoing patent case even higher for Intellia.