June 22, 2016
By Alex Keown, BioSpace.com Breaking News Staff
BERKELEY, Calif. – At 31-years-old, Rachel Haurwitz helms Caribou Biosciences, one of the companies on the cusp of gene editing breakthroughs in the United States using CRISPR-Cas9 gene editing technology co-developed by Caribou’s co-founder Jennifer Doudna.
Although in a field typically dominated by men, Haurwitz has more than held her own as she boldly guides her company into this cutting-edge field. In 2014, when she was 29, Haurwitz was named by Forbes Magazine to the “30 Under 30" list in Science and Healthcare. Haurwitz is an inventor on several patents and patent applications covering multiple CRISPR-derived technologies.
In an interview with StatNews, Haurwitz said she was exposed to CRISPR technology while in graduate school in the lab of Doudna, a professor at U.C. Berkeley. While working in Doudna’s lab, she began to work with CRISPR and that eventually led to her cofounding Caribou with her mentor and ultimately to helming the company. Under her guiding hand, Caribou netted $30 million in Series B financing last month. Caribou said the funds will be used to enable the company to expand its CRISPR gene editing technology platform and “accelerate our efforts in highly promising application areas in agriculture, therapeutics, biological research, and industrial biotechnology.” Last year Caribou raised $11 million in Series A funding.
Under Haurwitz’s guidance, Caribou Biosciences, has formed a strategic alliance with DuPont , and Novartis . Caribou also formed Intellia Therapeutics, which is active in gene therapy. In her interview with StatNews, Haurwitz said her goal is to strike more deals for Caribou. And her deals are not limited to human gene therapy applications. In May, Caribou struck a multi-year deal with Genus PLC, an animal genetics company, to apply gene-editing technology to improve the health of livestock animals.
“CRISPR” refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Cas9 is a CRISPR-associated endonuclease (an enzyme) known to act as the “molecular scissors” that cut and edit, or correct, disease-associated DNA in a cell. The technology was discovered by UC Berkeley professor Jennifer Doudna and Emmanuelle Charpentier.
Gene therapy essentially transforms cells inside a patient to harness their immune system to fight an invading disease on its own. CRISPR-Cas9 is considered revolutionary technology, and as such is likely, at some level, to be used by many companies and institutions.
Gene therapy and the CRISPR-Cas9 platform got a boost this week with support from the newly-founded Parker Institute for Cancer Immunotherapy on the first human testing of the technology, which passed its first initial safety review by U.S. regulators. The gene editing technology will be used to alter a patient’s blood cells, particularly the T cells, in order to kill the cancer cells. T cells are the soldiers of the body’s immune system. The proposed use of this particular trial would be to combat myeloma, melanoma, and sarcomas.
