As top scientists head toward a summit later this year to hammer out rules governing the use of gene editing, companies developing one form of the technology, known as CRISPR-Cas9, for human medicine continue to raise cash hand over fist.
The latest is Intellia Therapeutics, of Cambridge, MA, which announced today a $70 million round of funding. Intellia made its debut less than a year ago, but it has already forged an alliance with international drug firm Novartis (NYSE: NVS) to use CRISPR-Cas9 for two kinds of treatments: those that would re-engineer a patient’s own T cells, and those that reprogram bone marrow cells, also known as hematopoietic stem cells.
With this cash infusion, Intellia CEO Nessan Bermingham said his company will also explore a third potential use for these therapies. It will work on treatments that would send CRISPR-Cas9’s gene-editing scissors into patient’s livers, where many kinds of diseases might be addressed.
Derived from a bacterial defense system, CRISPR/Cas9 is a potential Nobel-winning biotech discovery that, in just a few years, has caught on around the world as a research tool to cut out or replace genes in all kinds of organisms. Work in human cells has emerged, too, sparking fears that altered reproductive cells or embryos—the human germline—would be used to make babies engineered for looks or intelligence, for example. The U.S. National Academies are planning an international summit later this year.
But Intellia and its rivals take pains to separate themselves from the germline controversy. They are working on therapies that alter somatic cells—those that cannot pass along genetic information to future generations. A big part of the puzzle they need to solve is getting CRISPR-Cas9’s machinery into various types of cells without getting chewed up inside the body or going astray into the wrong cells. Bermingham said Intellia has already in-licensed from an undisclosed source drug-delivery technology called lipid nanoparticles to help shuttle the gene-editing machinery through the body and to the liver.
It’s “premature to answer” what might reach the clinic, or when, said Bermingham. Genetic modification is building a clinical track record, but developers like Intellia would need an “iterative” dialogue with the FDA and other regulators to provide “peace of mind around safety and efficacy.”
“We have to be thoughtful about balancing potential concerns [about unintended side effects] with the severity and outcome of the disease,” said Bermingham.
(As I wrote about several months ago, the methods to identify when and where CRISPR-Cas9 is making unwanted cuts in a patient’s genome are still in development. How precisely those so-called “off target” cuts need to be measured continues to be a big topic of debate in the field.)
Now with 40 employees, Intellia is racing Editas Medicine and CRISPR Therapeutics to turn the research-lab phenomenon into human medicine. The three lay claim to various pieces of intellectual property underlying CRISPR-Cas9, and the outcome of a big patent fight could ultimately sway their fortunes. But that fight remains mired in byzantine U.S. Patent and Trademark Office proceedings. In the meantime, the companies are pressing ahead.
Editas, also of Cambridge, MA, said a few weeks ago it tapped into a large group of investors that include Bill Gates and one of his top advisors to raise $120 million. CRISPR Therapeutics, based in London but with significant operations in Cambridge, MA, raised $64 million from Celgene (NASDAQ: CELG) and others earlier this year.
With that level of cash going into these companies, we’re likely to see the first CRISPR-related IPO well before we see the first CRISPR-related therapy enter clinical trials.
Another form of gene editing called zinc finger proteins, owned by Sangamo Biosciences (NASDAQ: SGMO), is farther ahead of CRISPR, with a program to treat HIV in Phase 2.
If and when CRISPR gets to the clinic, blood-borne diseases, such as various cancers, sickle cell disease, beta thalassemia, and others, could be the first milestone. Intellia and Novartis are one example of drug makers working to make that happen. . Editas is working with Juno Therapeutics (NASDAQ: JUNO), a rival of Novartis, on T cell therapies.
Additionally, a nonprofit group, the Innovative Genomics Initiative of Berkeley, CA, is working with Children’s Hospital in nearby Oakland on a sickle cell disease treatment. As I wrote about a couple weeks ago, IGI researchers should know soon whether their experiments to modify hematopoietic stem cells in sickle-cell-like mice are giving the mice normal red blood cells.
Like its rival Editas, Intellia has tapped into a syndicate of nontraditional private investors for what looks like a crossover financing round; that is, a final shot of cash to prepare the company for a potential IPO, with investors who generally hold public stocks but look to get a head start by taking stakes in late-stage private biotechs. Intellia’s Series B round was led by OrbiMed, with participation from Fidelity, Janus Capital Management, Foresite Capital, Sectoral Asset Management, EcoR1 Capital, and others. Original venture investor Atlas Venture as well as Intellia’s corporate partner Novartis also participated.