Intellia Therapeutics announced yesterday a $15m (€12m) funding round led by Novartis and Atlas Venture. The company was created by Atlas and Caribou Biosciences, together with some of the scientists who helped discover CRISPR biology.
CRISPR-Cas is a tool for gene modification which uses a short sequence of RNA (Cas-9) to guide endonuclease. As well as standard “knocking-out” of genes achieved by zinc-finger nuclease, the method can “knock-in” genes, opening up more possibilities for patient disease models and biologics manufacturing.
Intellia CEO and co-founder Nessan Bermingham said the company will have “one of the most comprehensive intellectual property portfolios” covering therapeutic applications of CRISPR.
“Intellia was created to play a leading role in CRISPR-Cas9 therapeutic development, which will be greatly accelerated by decades of innovation across cell and gene therapy, RNA modification and stabilization and oligonucleotide delivery.”
“Discovery of the CRISPR-Cas9 system has been a significant advance toward the long-elusive therapeutic goal of targeting and repairing specific genetic defects.”
The company said it will use CRISPR for preclinical work but is focusing on clinical drug development and planning its first IND filing.
Intellia’s initial therapeutic focus will be ex vivo applications, removing cells from the body, modifying them to alter disease-causing genes, and returning them to the patient. It plans to begin with blood disorders, therapeutic protein production and cancer, focusing on CAR-T and checkpoint inhibitor regulation.
In the longer term, the company plans to tackle in vivo applications of CRISPR, administered either systemically or locally to correct genes inside specific cells of the body. In vivo applications include ophthalmic, central nervous system, muscle, liver, and anti-infective targets.
At this week’s Financial Times Global Pharmaceutical and Biotechnology Conference in London, Novartis CEO Joe Jiminez commented on the possibilities for emerging techniques like CAR-T.
“It’s amazing technology that allows us to take a blood sample from a leukaemia patient, to isolate and genetically engineer the T-cells in that sample and then reinfuse that sample back into the same patient.
“We see these t cells replicate in vivo and go after and eradicate tumours in the blood. I think we’re at the beginning of a renaissance.”
MIT and the Broad Institute have submitted a patent application as inventors of the CRISPR technique. UK biotech Horizon Discovery is among the companies already using CRISPR-Cas for preclinical modelling.