– Regulatory feedback and alignment from both U.S. and ex-U.S. regulators supports clinical development path for PBGENE-HBV
– IND/CTA enabling studies with final clinical candidate underway for PBGENE-HBV program; expect to file IND and/or CTA in 2024
– Expected to be the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA
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, including gene insertion, excision, and elimination, today announced that the company has received pre-IND regulatory feedback from the U.S. Food and Drug Administration (FDA) and ex-U.S. agencies. Receipt of this regulatory feedback provides alignment and clarity on Precisionâ€
“We believe the regulatory feedback we have received to date informs and strengthens our clinical strategy and provides a clear pathway towards an IND and/or CTA filing for our lead gene editing program PBGENE-HBV,†said Michael Amoroso, President and Chief Executive Officer at Precision BioSciences. “Based on this feedback, we have initiated the final preclinical studies as well as site selection efforts as we move towards clinical readiness. We look forward to filing regulatory applications in 2024 and continue to believe that PBGENE-HBV has the potential to achieve a functional cure for patients suffering with Chronic Hepatitis B.”
The pre-IND meeting with FDA provided feedback on overall design for the proposed first-in-human clinical study as well as feedback on the toxicology and specificity assessments. This interaction builds on the engagement and alignment achieved through early scientific advice during the FDA INTERACT meeting in July 2023. Similar, productive consultations have been held with regulators in ex-U.S. countries.
Collectively, the feedback and overall alignment obtained from multiple regulatory authorities validates Precisionâ€
PBGENE-HBV is expected to be the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA.
“Current treatment options for patients with HBV are unable to both eliminate cccDNA and inactivate HBsAg expression from integrated HBV DNA genomes in the hepatocyte, which is essential to drive a durable loss of viral markers and potentially achieve a functional cure for patients with HBV,†said Dr. Geoffrey Dusheiko, MD, FCP(SA), FRCS. Emeritus Professor of Medicine, Kingâ€
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. Itâ€
About ARCUS
ARCUS is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert, excise, or eliminate DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI, that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA and stimulate gene insertion at the cut site by homologous recombination. Precision’s platform and products are protected by a comprehensive portfolio including more than 130 granted patents to date.
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 Companyâ€
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The Company intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our product candidates and gene editing approaches including ARCUSâ€
Forward-looking statements are based on managementâ€
All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
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Investor and Media Contact:
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Vice President, Investor Relations
naresh.tanna@precisionbiosciences.com
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