Gilead Sciences: In pursuit of cures for intractable diseases

Gilead Sciences (GILD) has news that we felt good about it for a company that saved millions of lives from the minute, but monster viruses HIV and HCV. Curing millions of patients with HCV, has led Gilead to decide that the time has come for bringing cures to many horrible diseases.

Yesterday, September 12, the news announced that Gilead and Precision BioSciences have entered into a strategic collaboration to develop therapies targeting the in vivo elimination of hepatitis B virus (HBV) with Precision’s proprietary genome editing platform, ARCUS.

Gilead, indeed, is after providing a cure for hepatitis B virus (HBV), which is debilitating and life-threatening an estimated 257 million people who are living with this liver viral infection around the world. The current treatments suppress HBV viral replication but do not completely clear the virus. The presence of the covalently closed circular DNA (cccDNA) enables HBV replication if the treatments are stopped. Preliminary studies made by Gilead using ARCUS nucleases that targets HBV cccDNA in vitro have demonstrated significant activity against cccDNA and integrated HBV DNA in human hepatocytes.

ARCUS is a genome editing platform derived from a natural genome editing enzyme called a homing endonuclease. ARCUS is based on the ARC nuclease – a fully synthetic enzyme similar to a homing endonuclease but significantly improved to be the starting point for the first therapeutic-grade genome editing platform. The ARC nuclease can be customized to recognize a DNA sequence within any target gene.

ARC nucleases are created using a set of proprietary in silico and lab-based techniques to ensure maximum gene editing efficiency with minimum off-target activity. Importantly, ARC nucleases can be optimized to control potency and specificity based on the analysis of cutting activity in a relevant model organism.

 Terms of the Collaboration

Under the terms of the collaboration agreement, Precision Biosciences will be primarily responsible for the development, formulation, and preclinical evaluation of the investigational nucleases. Gilead will be responsible for the clinical development and commercialization of potential therapies. Gilead will fully fund the research and development.

Precision is eligible to receive milestone payments of up to an aggregate of $445 million and tiered royalties that go up to the mid-teens for commercial products developed through the collaboration.

Gilead Chief Scientific Officer and Head of Research and Development John McHutchison, MD, said, “Gilead is committed to developing innovative therapies to achieve functional cure for patients with chronic hepatitis B virus infection. We are excited about the potential of genome editing and Precision’s ARCUS technology, which has demonstrated promising in vitro activity. We look forward to exploring this technology as an important component of our HBV cure research efforts.”

Precision Chief Scientific Officer Derek Jantz commented, “Gilead’s cure-based approach to hepatitis B is comprehensive and exciting. Precision is pleased that initial studies with our ARCUS platform have established an important role for genome editing in their HBV program. This is an excellent application for our technology, which has made notable progress toward therapeutic in vivo editing in relevant models over the last year.”

Precision BioSciences mission is to cure genetic disease, overcome cancer, and feed the planet. The firm is striving to achieve this goal with its ARCUS, a therapeutic-grade, naturally-derived genome editing system that combines both specificity and efficacy to help overcome life’s greatest genetic challenges.

For additional information about Precision Biosciences visit

 Prohost Observations

There is no doubt that Gilead is after curing chronic progressive diseases and life-threatening diseases. There is no doubt that the firm is meticulously testing several gene editing technologies in order to realize its dream. We believe Gilead will do whatever it takes to realize its dream, which is helping millions of people be cleared from their debilitating diseases that continue to progress in spite of the  treatments.

Gilead has many good news recently, including the successful outcome of its global collaboration with Galapagos (GLPG) in developing filgotinib for the treatment of inflammatory diseases. The successful Phase 2 TORTUGA trial for ankylosing spondylitis is one of many Phase trials conducted by Galapagos for inflammatory diseases.

The clinical trials include the FINCH Phase 3 trial for rheumatoid arthritis, the DIVERSITY Phase 3 trial for Crohn’s disease (also small bowel and fistulizing Crohn’s disease Phase 2 studies) and the Phase 3 SELECTION trial in ulcerative colitis.

Gilead’s news is promising.

Read the Prohost article about the Gilead/Galapagos collaboration posted September 7, 2018 under News & Comments titled

“Gilead and Galapagos have good news for patients with severely active Ankylosing Spondylitis”

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