Biotech company Regeneron Pharmaceuticals and gene editing company Intellia Therapeutics, have announced a licensing and collaboration agreement using the CRISPR/Cas9 technique to develop new therapies for a range of diseases. In addition to developing and marketing new therapies, the two companies plan to further advance the CRISPR/Cas9 gene editing tool.
Intellia is set to receive a $75 million upfront payment from Regeneron, with further payments to be dependent upon milestone achievement and successful commercialization of any gene editing-based therapies. The six-year agreement will permit Regeneron to develop CRISPR-based therapies for a maximum of 10 disease targets, the majority of which could be treated by applying the gene editing technique to liver cells.
The first target for the partnership will be transthyretin amyloidosis, a progressive disease in which deposits of amyloid protein accumulate in organs and tissues throughout the body. These deposits most often buildup in the peripheral nervous system, however they can also affect the functioning of vital organs, including the heart, kidneys and gastrointestinal tract.
“Our industry-leading human genetics research with the Regeneron Genetics Center is already identifying important genetic targets, building on our long- standing expertise in genetic engineering,” said Dr. George D. Yancopoulos, Chief Scientific Officer of Regeneron and President of Regeneron Laboratories. “We believe combining these capabilities with Intellia’s technology holds real promise for serious diseases that have been historically difficult to address, and expands our ability to help patients where antibody-based therapies may not be the optimal solution.”
The CRISPR/Cas9 gene editing tool allows researchers to precisely repair specific sequences of DNA, representing a potentially breakthrough therapy for a number of genetic diseases. The technology could be applied to developing new treatments for autoimmune diseases, cancers, and rare inheritable conditions.
“We are excited to be partnering with Regeneron, an industry leader in human genetics research,” said Dr. Nessan Bermingham, Chief Executive Officer and Founder, Intellia Therapeutics. “Regeneron’s focus on advancing science to medicine is an excellent fit with Intellia’s approach, and together, we aim to bring potential cures to patients who are suffering from life-threatening rare diseases and genetic diseases.”