Precision BioSciences Presents New Preclinical Safety Data for PBGENE-HBV Clinical Candidate at the European Association for the Study of the Liver Congress
Precision BioSciences (Nasdaq: DTIL) has announced promising preclinical safety data for its PBGENE-HBV candidate at the European Association for the Study of the Liver Congress. The data demonstrate that PBGENE-HBV can specifically target and cut HBV DNA without affecting the human genome, showing high specificity and no detectable off-target editing. Additionally, non-human primate studies indicated that PBGENE-HBV is well-tolerated, even with multiple administrations. This supports the candidate's potential as a curative treatment for chronic hepatitis B. Precision BioSciences aims to submit an Investigational New Drug (IND) and/or Clinical Trial Application (CTA) for PBGENE-HBV in 2024.
- PBGENE-HBV shows high specificity in targeting HBV DNA without off-target editing in the human genome.
- Preclinical data indicates that multiple administrations of PBGENE-HBV are well-tolerated.
- No detectable off-target editing observed in primary human hepatocytes.
- Preclinical safety data supports the advancement of PBGENE-HBV to clinical trials.
- IND and/or CTA submissions for PBGENE-HBV are on track for 2024, indicating punctual progress.
- Minor and transient elevations in liver transaminases observed, though normalized within 2 weeks.
- Preclinical results are promising but not yet validated in human trials, which poses a validation risk.
Insights
The preclinical data for Precision BioSciences' PBGENE-HBV indicates promising progress for the company. It's important to understand that advancing to clinical trials could significantly enhance the company's valuation. The data showing high specificity and lack of off-target effects aligns with investor expectations for a reliable and safe gene therapy product. This news is particularly important as it de-risks the PBGENE-HBV program and supports the company's roadmap towards potentially lucrative clinical milestones.
Financially, the commitment to submit an Investigational New Drug (IND) application in 2024 represents a key upcoming catalyst. If the subsequent clinical trials show similar positive results, it could lead to substantial partnerships or funding opportunities. However, investors should remain cautious about the typical risks associated with clinical trials, such as unforeseen side effects or regulatory hurdles.
The presented data emphasizes the high specificity of PBGENE-HBV in targeting Hepatitis B virus DNA without off-target genome editing. This specificity is critical because it implies reduced risk of unintended genetic alterations, which is a common concern in gene editing therapies. Additionally, the preclinical trials in non-human primates demonstrating good tolerability across multiple doses is a strong indicator of the potential safety and efficacy in humans.
For chronic Hepatitis B patients, a curative treatment would be a significant breakthrough given the current treatment limitations. The promising preclinical results suggest that PBGENE-HBV could provide a finite, rather than lifelong, treatment solution. Long-term, this has the potential to transform the chronic hepatitis B treatment landscape, providing significant value to patients and the healthcare system.
The news about PBGENE-HBV's preclinical safety data is pivotal for positioning Precision BioSciences in the competitive gene editing market. The ARCUS® platform's ability to deliver targeted and efficient edits differentiates it from other technologies, potentially giving it a competitive edge. Successful advancement to clinical trials could fortify Precision BioSciences' market position and attract strategic partnerships or licensing deals.
From a market perspective, the gene editing field is rapidly advancing with significant interest from both investors and pharmaceutical companies. The favorable preclinical data for PBGENE-HBV enhances its marketability and underscores the growing importance of gene editing solutions in addressing chronic viral infections. However, the competitive landscape is fierce and continued positive results will be necessary to maintain and grow market interest.
- PBGENE-HBV specifically cuts HBV DNA without impacting the human genome
- PBGENE-HBV was well tolerated across multiple administrations with no off-target editing observed
- Preclinical safety data supports advancement of PBGENE-HBV to clinical trials as a potentially curative, finite treatment for chronic hepatitis B with IND and/or CTA on-track for submission in 2024
“We are pleased to present new safety data at EASL for our lead PBGENE-HBV program. These data highlight our ability to specifically target the HBV viral genome, with no detectable off-target editing in the human genome at relevant doses. In addition, the new non-human primate data shows that multiple administrations of PBGENE-HBV are well tolerated, enabling us to multi-dose in clinic,” said Jeff Smith, PhD, Chief Research Officer of Precision BioSciences. “Our work to date continues to highlight the potential of PBGENE-HBV as a curative treatment for chronic hepatitis B and further validates the capabilities of our proprietary ARCUS® platform to deliver safe and efficacious in vivo gene editing therapies. Looking ahead, we are well positioned to progress PBGENE-HBV, our first wholly owned ARCUS program into the clinic with an Investigational New Drug Application (IND) and/or Clinical Trial Application (CTA) in the 2024.”
The data to be presented highlights that PBGENE-HBV specifically cuts HBV DNA leading to elimination of cccDNA and inactivation of integrated HBV DNA without impacting any sites in the human genome, including no editing-associated translocations in HBV infected primary human hepatocytes. In addition, PBGENE-HBV was well-tolerated in non-human primates across multiple dose administrations, with only minor and transient elevations in liver transaminases that are normalized within 2 weeks and non-adverse changes in blood parameters. Preclinical safety data supports the advancement of PBGENE-HBV to clinical trials as a potentially curative treatment for chronic hepatitis B.
Full poster details are included below:
Title: Preclinical safety data for PBGENE-HBV gene editing program supports advancement to clinical trials as a potentially curative treatment for chronic hepatitis B
Abstract Number: LB195
Presenter: Emily Harrison, PhD, Senior Scientist - Hepatitis Research Leader, Precision BioSciences
Date and Time: Wednesday, June 5, 2024, 8:30 AM – Saturday, June 8, 2024, 5:00 PM CEST
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
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’s 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).
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. 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 editing efficiency; the design of PBGENE-HBV to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures; the suitability of ARCUS nucleases for gene elimination, insertion and excision and differentiation from other gene editing approaches due to its small size, simplicity and distinctive cut; the expected timing of regulatory processes (including filings such as IND’s and CTA’s and studies for PBGENE-HBV); expectations about operational initiatives, strategies, and further development of our programs; expectations about achievement of key milestones; and anticipated timing of clinical data. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “strive,” “target,” “will,” “would,” or the negative thereof and similar words and expressions.
Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. These statements are neither promises nor guarantees, but involve number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with raising additional capital and requirements under our current debt instruments and effects of restrictions thereunder; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical studies and clinical trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ ability to identify, develop and commercialize product candidates; potential product liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the
All forward-looking statements speak only as of the date of this presentation 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. Precision consults with various presentation speakers and compensates them for their time and expertise.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240605698770/en/
Investor and Media Contact:
Naresh Tanna
Vice President of Investor Relations
naresh.tanna@precisionbiosciences.com
Source: Precision BioSciences, Inc.
FAQ
What is PBGENE-HBV by Precision BioSciences?
How does PBGENE-HBV affect the human genome?
When will Precision BioSciences submit IND and/or CTA for PBGENE-HBV?
What were the findings from the non-human primate studies for PBGENE-HBV?
What potential does PBGENE-HBV hold for hepatitis B treatment?
