“The question is how fast can we scale up,” Axel Bouchon, Casebia’s interim chief executive, told The Boston Globe. “Hiring in Cambridge is tough. However, it’s a huge opportunity. If you have an interesting technology and funding, you can tap a pool of scientific talent that is the best in the world.”
The advantage of the joint venture is that Casebia’s management can focus exclusively on its own programs. It also opens the possibility of bringing in additional investors or go public if either seems appropriate. The company has indicated that it hopes to bring three to four programs in hematology and ophthalmology to clinical trials in the next five years.
“This gives us the opportunity to extend our portfolio,” Novak told The Boston Globe. “We’re working on programs we otherwise wouldn’t have the capacity to work on. There will be a lot of cross-fertilization.”
CRISPR Therapeutics focuses on translating CRISPR-Cas9, genome-editing technology, into therapeutics for diseases. Cas9 is an enzyme that can be programmed with RNA to cut DNA at specific, targeted locations within the genome, which allows for easier, more specific gene editing.
CRISPR Therapeutics is not the only company to be working with CRISPR-Cas9. In fact, the technology itself has been tangled up in lawsuits over patents. Jennifer Doudna and Emmanuelle Charpentier, who co-founded CRISPR Therapeutics with Rodger Novak, published the first paper describing the technology. However, Feng Zhang, a researcher at the MIT-Harvard Broad Institute, filed a broad U.S. patent claim on the technology.
The technology was first described in the journal Science in 2012 by Doudna, then a biologist at the University of California, and Charpentier, then at the Max F Perutz Laboratories at the University of Vienna in Austria. However, Zhang, at the Broad Institute, first won a patent for the technology after submitting laboratory notes to prove he was the first inventor.
The patent battles appear to be continuing, and are expected to draw out for quite some time.
Other companies developing CRISPR include Editas Medicine and Intellia Therapeutics, both headquartered in Cambridge, Mass. To date, no drugs or therapies have been created using CRISPR, although drug companies believe it has the potential to revolutionize the industry. Doudna originally founded a company, Caribou Biosciences, but later cofounded Editas with Zhang and George Church, a professor at Harvard and MIT.