Flagship Ventures, Polaris Partners and Third Rock Ventures have teamed up to fund a biotech start-up based on a genome-editing platform to the tune of $43 million in Series A financing.
The Cambridge, Mass.-based company, Editas Medicine, said its technology has the potential to use RNA molecules to guide a repair protein to “mistakes” in the genome and knock out, knock down or edit those defective genes to treat a broad range of diseases.
Editas’ goal is to use that genome-editing technology to develop a pipeline of human therapeutics for genetic diseases.
Editas Interim President Kevin Bitterman said the investor consortium became aware of the potential of the new technology early in 2013, when it was just hitting the academic journals, and took the unusual step of assembling a “dream team” of five academic founders for the company, including genetics legend George Church, of Harvard University.
“A few foundational papers came out earlier this year, and that opened the floodgate to what’s been an avalanche of work,” Bitterman told BioWorld Today.
Although it’s not unusual for venture firms to recruit academic scientists as company founders, the scope of Editas’ dream team is unprecedented. The scientific consortium includes scientists who might have gone on to form two, three or more separate companies.
In addition to Church, who pioneered the technology of genomic sequencing and helped to start the Human Genome Project, the roster of founders includes the Broad Institute’s Feng Zhang, who developed the TALE and CRISPR technologies for splicing genes into living cells; Harvard geneticist Robert Winthrop; University of California, Berkeley, and Howard Hughes Medical Institute cell biologist Jennifer A. Doudna; Masssachusetts General Hospital and Harvard Medical School pathologist Keith Joung; and Harvard protein evolution and engineering biologist David Liu.
Editas will develop its therapeutics using CRISPR (clustered, regularly interspaced short palindromic repeats)/Cas9 (CRISPR-associated protein 9) and TALENs (transcription activator-like effector nucleases), which may have the potential to address previously intractable therapeutic indications, according to Editas.
TALENs were named as one of the year’s most exciting developments in Science’s 2012 list of top scientific advances, and 2013 began with two more breakthrough publications in the field. The back-to-back reports were published in the Jan. 3, 2013, advance online issue and showed that CRISPR can be used to target “almost any location in the mammalian genome,” according to Feng Zhang. (See BioWorld Today, Jan. 4, 2013.)
Zhang showed that the system could precisely edit both mouse and human cells.
CRISPR uses RNA to target the gene sequences it wants to cut, binding to those sequences through Watson-Crick base pairing. In the other main genome-editing technologies that have been harnessed in the lab so far, TALENs and zinc-finger nucleases, such recognition is done by proteins, and it is much more complex to design a binding protein than to figure out the complementary RNA sequence.
In a separate paper, Church and his colleagues reported developing a CRISPR system that targeted one genomic locus using guide RNA, with bioinformatics methods to determine the sequences of RNAs that could target about 40 percent of the human exome.
Bitterman said the CRISPR/Cas9 machinery is derived from bacteria, where it serves as an adaptive immune system for the organism, a “bacterial cell memory of viral invaders.”
The system is activated when viral pathogens invade, and the bacterium survives the initial insult. The bacteria then incorporate a small portion of the viral DNA into their own genomes and express the viral DNA as RNA, which then associates with Cas9 and through the nuclease activity of the Cas9 enzyme, eliminates the invader.
A number of research groups reached the conclusion nearly simultaneously that CRISPR biology could potentially be used to edit the human genome as well, and therefore had therapeutic potential.
After that, Editas came together in record time.
“The field has been moving very quickly. The last year and a half in particular has seen this really exponential growth in activity in the space of genome editing,” Editas co-founder Keith Joung told BioWorld Today. The rapid formation of Editas, Joung said, “parallels what’s been going on in the scientific literature and in the field.”
Bitterman said Editas will use the funds from the Series A round, expected to be announced Monday, for two primary efforts. The first is to continue to build out the technology platform. The second is to advance into clinical development with lead programs.
Editas is not yet sharing any information about its pipeline. According to Bitterman, the technology has potential for a “large universe” of diseases, including blood diseases, neurodegenerative diseases, cancer, infectious disease and many other applications.
In other financings news:
• Lorus Therapeutics Inc., of Toronto, upsized its C$5 million (US$4.7 million) offering to about 12.7 million shares, issued at a purchase price of C55 cents each, for gross proceeds of about C$7 million. The company agreed to grant underwriters an overallotment option to purchase that number of additional shares equal to 15 percent of the shares sold pursuant to the offering at the offering price for a period ending 30 days following the closing, which is expected to occur on or about Dec. 13.
• Tigenix NV, of Leuven, Belgium, completed a €12 million (US$16.2 million) financing, disclosed last Wednesday. About 34 million ordinary shares were issued to Gri-Cel SA, a fully owned subsidiary of global health care company Grifols SA, of Barcelona. (See BioWorld Today Nov. 21, 2013.)