Novartis-backed, preclinical upstart Intellia Therapeutics ($NTLA) announced May 6th that it’s going for the top range of its IPO, selling 6 million shares at $18 a share to raise a total of $108 million.
Founded in 2014, Intellia quickly caught the attention of both Atlas and Novartis for their use of CRISPR/Cas9 technologies. In fact, Novartis has already agreed to pay $50 million in upfront and research fees over 5 years along with up to $230 million in milestones. And last month, Regeneron ($REGN) paid $75 million upfront to partner with the company.
Intellia’s largest shareholder, however, is Caribou Therapeutics (21.5%), a Californian genome engineering company spun off from Jennifer Doudna’s CRISPR lab at Berkeley. Caribou is responsible for outlicensing the CRISPR tech to Intellia in the first place. Novartis then owns 20.3% while Atlas has a 17% stake.
CRISPR, an acronym that stands for “clustered regularly interspaced short palindromic repeats,” allows scientists to easily and precisely edit the genes of targeted cells. It has the potential to be a powerful tool for treating disease at the genetic level by deleting, repairing or even replacing the genes that cause disease.
Intellia wants to explore the potential applications of CRISPR/Cas9 using proprietary informatics to make the technology more precise, predictable, and efficient.
The Cambridge, MA-based company described their workflow their SEC filing as follows: “We are building a high-throughput, scalable data processing and analysis, or informatics, infrastructure to support various aspects of our platform, including guide RNA selection and analysis of on- and off-target editing in cells. Depending on the desired editing strategy, we use our proprietary bioinformatics methods to design candidate guides and select those that we believe are more likely to be highly specific and have high cutting efficiency. As we grow our experimental data set, we intend to incorporate guide performance into our algorithms to improve their predictive power.”
Their CRISPR informatics-enabled gene editing platform is at the center of their product pipeline. Using this system, Intellia will be able to use the experimental data they collect to improve the predictive power of their algorithms over time. As their repository of data grows, so will the accuracy of their technology.
Because they are still preclinical, we don’t have proven successes to look to, yet. But this is the goal they are setting forth as they get ready for trails. With this new funding, they are poised to invest upwards of $10 million in further developing and testing these technologies as well as hiring new staff to support the process.
CRISPR technology has been considered one of the biggest breakthroughs in biotech in recent memory and could have profound consequences on how we treat and approach certain diseases. This has lead to a number of high IPOs and a lot of pressure on industry leaders to be the first to perfect the technology for public use.
Some analysts are calling gene editing speculation a bubble waiting to pop. And in the past 6 months, we have seen a number of biotechs discount their offerings or pull their IPOs completely.
A few are still making it, however, including fellow Cambridge, MA-based gene editing company Editas ($EDIT), which raised $94.4 million in its IPO back in February. The company priced the shares at $16 apiece – the bottom of their anticipated range of $16 to $18.
Cellectis ($CLLS) of Paris and Sangamo Biosciences ($SMGO) of Richmond, CA, are two more publicly traded biotechs using gene editing to develop products. And Crispr Therapeutics, while still private, has seen around $100 million in venture investment.
As Intellia hands over a chunk of stock to Regeneron and Novartis for $55 million and insiders get set to take their own pile of shares, Intellia’s gross haul has been bumped to around $160 million.
The money raised will be used to push R&D programs, including the submission of at least one IND. They will also begin to acquire useful businesses and technologies to integrate into their research programs, including in vivo and ex vivo product candidates.
One of its in vivo candidates, currently being co-developed with Regeneron, will target transthyretin amyloidosis (ATTR)—a life-threatening disease caused by misfolded transthyretin proteins that accumulate as amyloid fibrils in multiple organs. This candidate could very well be advanced to IND-enabling trials in the next 12 to 24 months, according to the company.
For their ex vivo candidates, Intellia is working with Novartis on a knockout repair insertion technology using hematopoietic stem cells. Their first IND for it isn’t expected to be submitted until 2018, though.
In the world of Biotech, there’s always competition, and multiple companies are rushing to the same ends right now.
Alnylam Pharmaceuticals ($ALNY) is currently running late-stage trials for its RNAi therapeutic patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy.
Editas aims to have its first program for a rare form of blindness called Leber congenital amaurosis in clinical trials by next year.
Intellia’s big backings by Novartis, Atlas, Regeneron, and Caribou may prove to push them to the top. When first choosing to invest in Intellia, Mark Fishman, President of the Novartis Institutes for BioMedical Research (NIBR) said of the technology and the company, “We have glimpsed the power of CRISPR tools in our scientific programs in NIBR, and it is now time to explore how to safely extend this powerful technology to the clinic. CRISPR has the potential to open a new branch of medicine, editing the genome to cure disease. Much remains to be learned, and we are delighted to explore these directions with colleagues from Intellia and Caribou.”