– PBGENE-HBV is the only drug modality designed to target the root cause of disease by eliminating cccDNA and inactivating integrated HBV
– Clinical trial applications submitted for the first gene editing approach for chronic hepatitis B
– Final clinical candidate safety data and plans for the Phase 1 trial to be shared in November prior to the American Association for the Study of Liver Diseases meeting
DURHAM, NC, USA I September 30, 2024 I Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, today announced that the Company submitted Clinical Trial Applications (CTA) to initiate a Phase 1study evaluating PBGENE-HBV. PBGENE-HBV is the Company’s wholly owned in vivo gene editing program designed to potentially cure chronic hepatitis B virus (HBV) by eliminating cccDNA, the key source of replicating hepatitis B virus, while also inactivating integrated HBV DNA in hepatocytes.
“The CTA submissions for PBGENE-HBV are important milestones for Precision as we pioneer this potentially curative therapy for chronic hepatitis B. These regulatory submissions are the culmination of our team’s dedication, commitment and highly productive interactions with global regulators as we develop the first clinical stage in vivo gene editing program for chronic hepatitis B virus, recognized as one of the largest global public health problems by the World Health Organization with an estimated 300 million people afflicted globally,” said Michael Amoroso, Chief Executive Officer of Precision BioSciences. “Our comprehensive regulatory package, supported by robust non-human primate (NHP) safety studies, the gold standard model for predicting safety in humans, and efficacy in numerous preclinical models of hepatitis B including in NHPs enables us to proceed with planned regulatory submissions in multiple markets around the world. We recently bolstered our clinical team expertise while building a world class hepatitis scientific advisory board to assist in guiding execution of our global Phase 1 trial. Our goal is clear, to generate robust clinical data for the patients afflicted with hepatitis B who are counting on us to significantly increase their chance of achieving a functional cure.”
Dr. Murray Abramson, Senior Vice President, Head of Clinical Development added: “Current standard of care treatment with nucleos(t)ide analogs only offers 1-3% of patients a chance to achieve a functional cure. With our recent regulatory submissions, we are on the cusp of initiating a global first-in-human study for our wholly owned PBGENE-HBV program. PBGENE-HBV is specifically designed to provide a better chance for a functional cure for chronic hepatitis B by eliminating the root source of viral replication, known as cccDNA. This is the first approach to directly target and eliminate cccDNA. We are excited at the prospect of bringing this potentially curative therapy to patients living with hepatitis B. We look forward to initiating the Phase 1 study soon and expect to report data in 2025. More information about the safety of our clinical candidate and Phase 1 clinical trial will be communicated in November when we plan to share a robust overview of the program and our execution plans.”
Precision is on track to submit additional regulatory applications as part of its global Phase 1 regulatory strategy for PBGENE-HBV. The next update on the PBGENE-HBV program is expected to take place before the American Association for the Study of Liver Diseases (AASLD) Annual Meeting in November. Details about how to participate in the update will be provided in advance.
About Hepatitis B and PBGENE-HBV:
Hepatitis B is a leading cause of morbidity in the US and death globally, with no curative options currently available for patients. In 2019, despite the availability of approved antiviral therapies, an estimated 300 million people globally and more than 1 million people in the US were estimated to have chronic hepatitis B infection. An estimated 15% to 40% of patients with HBV infections may develop complications, such as cirrhosis, liver failure, or liver cancer (hepatocellular carcinoma), which account for the majority of HBV-related deaths.
Chronic hepatitis B infection is primarily driven by persistence of HBV cccDNA and integration of HBV DNA into the human genome in liver cells, the primary source of HBsAg in late-stage disease. Current treatments for patients with HBV infection include agents that result in long-term viral suppression as indicated by reduction of circulating HBV DNA, but these therapies do not eradicate HBV cccDNA, rarely lead to functional cure, and require lifelong administration. PBGENE-HBV is a highly specific, novel therapeutic approach to treating patients with chronic HBV infection. It is designed to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is an advanced gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
The ARCUS® platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV).
SOURCE: Precision BioSciences
Post Views: 5,439
– PBGENE-HBV is the only drug modality designed to target the root cause of disease by eliminating cccDNA and inactivating integrated HBV
– Clinical trial applications submitted for the first gene editing approach for chronic hepatitis B
– Final clinical candidate safety data and plans for the Phase 1 trial to be shared in November prior to the American Association for the Study of Liver Diseases meeting
DURHAM, NC, USA I September 30, 2024 I Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, today announced that the Company submitted Clinical Trial Applications (CTA) to initiate a Phase 1study evaluating PBGENE-HBV. PBGENE-HBV is the Company’s wholly owned in vivo gene editing program designed to potentially cure chronic hepatitis B virus (HBV) by eliminating cccDNA, the key source of replicating hepatitis B virus, while also inactivating integrated HBV DNA in hepatocytes.
“The CTA submissions for PBGENE-HBV are important milestones for Precision as we pioneer this potentially curative therapy for chronic hepatitis B. These regulatory submissions are the culmination of our team’s dedication, commitment and highly productive interactions with global regulators as we develop the first clinical stage in vivo gene editing program for chronic hepatitis B virus, recognized as one of the largest global public health problems by the World Health Organization with an estimated 300 million people afflicted globally,” said Michael Amoroso, Chief Executive Officer of Precision BioSciences. “Our comprehensive regulatory package, supported by robust non-human primate (NHP) safety studies, the gold standard model for predicting safety in humans, and efficacy in numerous preclinical models of hepatitis B including in NHPs enables us to proceed with planned regulatory submissions in multiple markets around the world. We recently bolstered our clinical team expertise while building a world class hepatitis scientific advisory board to assist in guiding execution of our global Phase 1 trial. Our goal is clear, to generate robust clinical data for the patients afflicted with hepatitis B who are counting on us to significantly increase their chance of achieving a functional cure.”
Dr. Murray Abramson, Senior Vice President, Head of Clinical Development added: “Current standard of care treatment with nucleos(t)ide analogs only offers 1-3% of patients a chance to achieve a functional cure. With our recent regulatory submissions, we are on the cusp of initiating a global first-in-human study for our wholly owned PBGENE-HBV program. PBGENE-HBV is specifically designed to provide a better chance for a functional cure for chronic hepatitis B by eliminating the root source of viral replication, known as cccDNA. This is the first approach to directly target and eliminate cccDNA. We are excited at the prospect of bringing this potentially curative therapy to patients living with hepatitis B. We look forward to initiating the Phase 1 study soon and expect to report data in 2025. More information about the safety of our clinical candidate and Phase 1 clinical trial will be communicated in November when we plan to share a robust overview of the program and our execution plans.”
Precision is on track to submit additional regulatory applications as part of its global Phase 1 regulatory strategy for PBGENE-HBV. The next update on the PBGENE-HBV program is expected to take place before the American Association for the Study of Liver Diseases (AASLD) Annual Meeting in November. Details about how to participate in the update will be provided in advance.
About Hepatitis B and PBGENE-HBV:
Hepatitis B is a leading cause of morbidity in the US and death globally, with no curative options currently available for patients. In 2019, despite the availability of approved antiviral therapies, an estimated 300 million people globally and more than 1 million people in the US were estimated to have chronic hepatitis B infection. An estimated 15% to 40% of patients with HBV infections may develop complications, such as cirrhosis, liver failure, or liver cancer (hepatocellular carcinoma), which account for the majority of HBV-related deaths.
Chronic hepatitis B infection is primarily driven by persistence of HBV cccDNA and integration of HBV DNA into the human genome in liver cells, the primary source of HBsAg in late-stage disease. Current treatments for patients with HBV infection include agents that result in long-term viral suppression as indicated by reduction of circulating HBV DNA, but these therapies do not eradicate HBV cccDNA, rarely lead to functional cure, and require lifelong administration. PBGENE-HBV is a highly specific, novel therapeutic approach to treating patients with chronic HBV infection. It is designed to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is an advanced gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
The ARCUS® platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV).
SOURCE: Precision BioSciences
Post Views: 5,439
