– PBGENE-HBV is the first-ever investigational in vivo gene editing therapy cleared to enter clinical trials for the treatment of chronic hepatitis B in the United States (U.S.) –
– IND clearance represents a significant regulatory milestone for PBGENE-HBV –
– Company to expand its Phase 1 ELIMINATE-B study to the U.S., joining world-class clinical sites in Moldova, Hong Kong, and New Zealand where strong clinical execution is currently underway –
DURHAM, NC, USA I March 17, 2025 I Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for PBGENE-HBV. PBGENE-HBV is Precision’s lead wholly owned in vivo gene editing program designed to cure chronic hepatitis B by eliminating cccDNA, the key source of replicating hepatitis B virus (HBV), and inactivating integrated HBV DNA in hepatocytes. Precision is in final stages of U.S. site activation, enabling the ELIMINATE-B trial to begin enrolling patients in the U.S. where over one million people are infected with chronic hepatitis B. The ELIMINATE-B trial is actively enrolling patients in Moldova, Hong Kong, and New Zealand and will soon initiate Phase 1 clinical activities in the U.S at the Liver Center at Massachusetts General Hospital. The Company recently announced promising safety and efficacy data for PBGENE-HBV following first dose administration at the lowest dose level in the ELIMINATE-B clinical trial.
“IND clearance to expand the ELIMINATE-B trial for chronic hepatitis B is a first for the gene editing space. This marks the advancement of potentially curative gene editing modalities into major markets for diseases where enormous global disease burden and lack of curative options have unfortunately become the norm,” said Michael Amoroso, President and Chief Executive Officer of Precision BioSciences. “With four regulatory approvals in four months, this clearance validates our parallel global development strategy backed by our robust pre-clinical safety and efficacy package. The ELIMINATE-B trial remains on track following the recent completion of initial dose administration in our first cohort of patients and we look forward to continuing to share clinical data throughout 2025.”
“The IND clearance milestone marks a significant step forward in our mission to advance potential curative treatment options for hepatitis B patients,” said Murray A. Abramson, MD, MPH, Head of Clinical Development at Precision BioSciences. “We’re very excited to extend the ELIMINATE-B trial to the U.S. and look forward to working with the investigators in the U.S., alongside those in Moldova, Hong Kong and New Zealand as we build on the momentum of the ELIMINATE-B phase 1 study.”
Investigators in the ELIMINATE-B trial accrued the first cohort of patients within the first month of open enrollment and are currently administering additional doses to patients in Cohort 1 at the same dose level (0.2 mg/kg). In addition, the Company expects to escalate to a higher dose level in Cohort 2 with the goal to define the optimal dose and number of dose administrations for safely eliminating cccDNA and inactivating integrated HBV DNA. Precision plans to further expand the study to the U.K. and continue accelerating recruitment and evaluation of a genetically diverse patient population in the Phase 1 study. Precision plans to share detailed clinical data throughout 2025.
“Hepatitis B has a significant impact on the lives of people living with hepatitis B in the U.S. and many countries throughout the world. Millions of people are eager for new treatment approaches that can help reduce the burden of hepatitis B which includes liver damage, liver cancer, premature death and social stigma. In the U.S. alone there up to 2.4 million people living with chronic hepatitis B, and despite vaccinations and continued medical developments, the numbers haven’t changed appreciably over the past 15 years,” said Chari A. Cohen, DrPH, MPH, Hepatitis B Foundation President.
About Hepatitis B:
Hepatitis B is a leading cause of morbidity in the US and death globally, with no curative options currently available for patients. Despite the availability of approved antiviral therapies, an estimated 300 million people globally and 1-2 million people in the US are 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 hepatitis B surface antigen (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.
About PBGENE-HBV (Viral Elimination Program):
PBGENE-HBV is Precision’s wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to potentially cure chronic hepatitis B virus (HBV) infection. Currently, it is estimated that 300 million people worldwide are afflicted with chronic hepatitis B. PBGENE-HBV is the first and only potentially curative gene editing program to enter clinical investigation that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA. Lipid nanoparticle technology for PBGENE-HBV has been provided by Acuitas Therapeutics Inc.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage 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: 2,020
– PBGENE-HBV is the first-ever investigational in vivo gene editing therapy cleared to enter clinical trials for the treatment of chronic hepatitis B in the United States (U.S.) –
– IND clearance represents a significant regulatory milestone for PBGENE-HBV –
– Company to expand its Phase 1 ELIMINATE-B study to the U.S., joining world-class clinical sites in Moldova, Hong Kong, and New Zealand where strong clinical execution is currently underway –
DURHAM, NC, USA I March 17, 2025 I Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for PBGENE-HBV. PBGENE-HBV is Precision’s lead wholly owned in vivo gene editing program designed to cure chronic hepatitis B by eliminating cccDNA, the key source of replicating hepatitis B virus (HBV), and inactivating integrated HBV DNA in hepatocytes. Precision is in final stages of U.S. site activation, enabling the ELIMINATE-B trial to begin enrolling patients in the U.S. where over one million people are infected with chronic hepatitis B. The ELIMINATE-B trial is actively enrolling patients in Moldova, Hong Kong, and New Zealand and will soon initiate Phase 1 clinical activities in the U.S at the Liver Center at Massachusetts General Hospital. The Company recently announced promising safety and efficacy data for PBGENE-HBV following first dose administration at the lowest dose level in the ELIMINATE-B clinical trial.
“IND clearance to expand the ELIMINATE-B trial for chronic hepatitis B is a first for the gene editing space. This marks the advancement of potentially curative gene editing modalities into major markets for diseases where enormous global disease burden and lack of curative options have unfortunately become the norm,” said Michael Amoroso, President and Chief Executive Officer of Precision BioSciences. “With four regulatory approvals in four months, this clearance validates our parallel global development strategy backed by our robust pre-clinical safety and efficacy package. The ELIMINATE-B trial remains on track following the recent completion of initial dose administration in our first cohort of patients and we look forward to continuing to share clinical data throughout 2025.”
“The IND clearance milestone marks a significant step forward in our mission to advance potential curative treatment options for hepatitis B patients,” said Murray A. Abramson, MD, MPH, Head of Clinical Development at Precision BioSciences. “We’re very excited to extend the ELIMINATE-B trial to the U.S. and look forward to working with the investigators in the U.S., alongside those in Moldova, Hong Kong and New Zealand as we build on the momentum of the ELIMINATE-B phase 1 study.”
Investigators in the ELIMINATE-B trial accrued the first cohort of patients within the first month of open enrollment and are currently administering additional doses to patients in Cohort 1 at the same dose level (0.2 mg/kg). In addition, the Company expects to escalate to a higher dose level in Cohort 2 with the goal to define the optimal dose and number of dose administrations for safely eliminating cccDNA and inactivating integrated HBV DNA. Precision plans to further expand the study to the U.K. and continue accelerating recruitment and evaluation of a genetically diverse patient population in the Phase 1 study. Precision plans to share detailed clinical data throughout 2025.
“Hepatitis B has a significant impact on the lives of people living with hepatitis B in the U.S. and many countries throughout the world. Millions of people are eager for new treatment approaches that can help reduce the burden of hepatitis B which includes liver damage, liver cancer, premature death and social stigma. In the U.S. alone there up to 2.4 million people living with chronic hepatitis B, and despite vaccinations and continued medical developments, the numbers haven’t changed appreciably over the past 15 years,” said Chari A. Cohen, DrPH, MPH, Hepatitis B Foundation President.
About Hepatitis B:
Hepatitis B is a leading cause of morbidity in the US and death globally, with no curative options currently available for patients. Despite the availability of approved antiviral therapies, an estimated 300 million people globally and 1-2 million people in the US are 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 hepatitis B surface antigen (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.
About PBGENE-HBV (Viral Elimination Program):
PBGENE-HBV is Precision’s wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to potentially cure chronic hepatitis B virus (HBV) infection. Currently, it is estimated that 300 million people worldwide are afflicted with chronic hepatitis B. PBGENE-HBV is the first and only potentially curative gene editing program to enter clinical investigation that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA. Lipid nanoparticle technology for PBGENE-HBV has been provided by Acuitas Therapeutics Inc.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage 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: 2,020
