RESEARCH TRIANGLE PARK – Precision Biosciences’ two-year collaboration with Gilead Sciences to develop new therapies to cure hepatitis B will end in September.
The two firms announced a deal that could have meant as much as $445 million to Precision in September 2018.
An estimated 257 million people around the world live with the hepatitis B virus (HBV). Current treatments suppress HBV replication, but they don’t completely clear up the infection.
Durham-based Precision, which specializes in genome editing, has hoped its proprietary ARCUS platform can produce a permanent solution to the disease.
It began working with Gilead in 2018. Under their agreement, the two companies have used the ARCUS gene editing system to target the covalently closed circular DNA (cccDNA) that enables HBV to reproduce.
As part of the collaboration, Precision has been responsible for development, formulation and preclinical evaluation of investigational ARCUS nucleases. Biopharmaceutical firm Gilead has funded all R&D efforts and also is responsible for clinical development and commercialization of potential new therapies. When the partnership ends on September 4, Precision will regain all rights to the data generated through the program.
“This was a highly productive and well-aligned collaboration, and we deeply value the opportunity to advance our ARCUS genome editing technology and a potential cure for HBV alongside a global leader of infectious disease,” said Derek Jantz, Ph.D., Precision BioSciences chief scientific officer. “While we consider new partnership opportunities for HBV, we are focused on progressing our internal lead proprietary gene correction program for primary hyperoxaluria type 1, for which we expect to nominate a clinical candidate later this year.”
Primary hyperoxaluria is a rare inherited liver disease characterized by overproduction of oxalate, an end product of the body’s metabolism. The only current definitive treatment is a liver transplant.
Precision said it doesn’t expect any changes in its cash runway, the length of time it can remain solvent if it is unable to raise more money. As of March 31, the company had cash and cash equivalents of $154.2 million, which it said should fund its operations into the second half of 2021.
Precision opened a new Manufacturing Center for Advanced Therapeutics a year ago this week. It was “the first in-house current Good Manufacturing Process (cGMP)-compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf chimeric antigen receptor (CAR) T cell therapy products,” according to Precision.
The facility is located in rental space in the Biopoint Innovation Labs in Research Triangle Park.
Precision’s ARCUS genome editing technology makes possible the production of CAR T cells, a type of immune system cell that recognizes and targets invading germs or cancer cells. These “off-the-shelf” allogeneic cells originate from healthy donors, rather than other therapies on the market that use the patient’s own blood.
This approach aims to overcome the manufacturing-related limitations of the traditional autologous CAR T therapies. Precision can make its products in advance – and in large batches — and then cryopreserve them for shipment, storage and off-the-shelf use.
The ARCUS genome-editing process also streamlines the therapeutic process. It removes a T cell receptor to prevent graft versus host disease – a serious complication of cell-transplant therapies for blood cancers – and avoids the need for donor-patient matching.
Precision spun out of Duke University in 2006 and currently has close to 225 employees. Its pipeline includes a number of “off-the-shelf” CAR T immunotherapy clinical and in vivo gene correction therapy candidates. They are targeted at curing genetic and infectious diseases for which no adequate treatments exist.
Gilead Sciences is headquartered in Forest City, California. It’s investigational drug, remdesivir, is considered one of the most promising potential treatments for COVID-19. Ralph Baric and his team at the UNC Gillings School of Public Health were instrumental in developing the therapy.
(C) N.C. Biotech Center