Hemophilia CRISPR/Cas9 Therapy Deal Expanded by Regeneron, Intellia – Hemophilia News Today
The two companies signed a six-year agreement in 2016 to develop, license, and commercialize gene editing-based therapies.
“We’re pleased to expand our work with Intellia, a like-minded group of scientists focused on maximizing the potential of CRISPR/Cas9 in order to help as many patients as possible,” George D. Yancopoulos, MD, PhD, co-founder, president, and chief scientific officer of Regeneron, said in a press release.
Gene editing is a means of making precise, targeted changes in an organism’s genetic code. The changes include “correcting” harmful mutations with healthy DNA sequence, removing disease-causing sequences, and inserting genetic code that has been lost due to DNA copy errors.
CRISPR/Cas9 is one of the newer and more powerful tools for gene editing. Discovered in bacteria, the technique consists of an RNA molecule targeting specific DNA sequences and the Cas9 enzyme. Cas9 cuts the DNA at the targeted location to enable the cell’s own DNA repair machinery to add or delete genetic material, or to alter the DNA by replacing an existing segment with a designed DNA sequence.
RNA and DNA are both nucleic acids, with RNA being produced from DNA. While performing different functions, they have complementary sequences that “recognize” each other, thereby binding together.
Under the original agreement, the two companies jointly research therapeutic targets that can be developed by either company alone, or co-developed under certain conditions.
With the expanded agreement, Regeneron will provide Intellia with an up-front $70-million payment and further equity investment of $30 million. In turn, Regeneron will receive a non-exclusive license from Intellia to develop and market up to 10 ex vivo CRISPR/Cas9 products in defined cell types.
The new deal extends the existing collaboration until April 2024, which can then be renewed by Regeneron for two more years. Also, it gives Regeneron the right to discover and develop CRISPR/Cas9-based therapies for an additional five in vivo liver targets, up to a total of 15 targets.
“We believe the precise in vivo gene insertion capabilities jointly developed with Intellia could be a promising therapeutic platform with significant potential in many diseases, including those that have been historically difficult to treat,” Yancopoulos said.
Overall, the collaboration seeks to leverage Intellia’s CRISPR/Cas9 platform to accelerate both companies’ efforts to develop new therapeutics, including products for hemophilia A and B.
In preclinical studies, they successfully inserted the gene coding for the blood clotting protein factor IX (FIX) into the liver of non-human primates. FIX is missing or defective in hemophilia B patients. The inserted gene produced normal or higher levels of circulating human FIX, according to the companies.
“We’re excited to work with Regeneron on what could potentially be a cure for hemophilia A and B,” said John M. Leonard, MD, Intellia’s CEO and president. “We believe that our CRISPR/Cas9-based technology addresses the limitations of current replacement and gene therapy approaches, and importantly, may provide a durable, potentially life-long solution to these genetic diseases.”
Published at Mon, 08 Jun 2020 13:00:00 +0000