DURHAM, N.C. - Precision BioSciences, Inc. (NASDAQ:DTIL), a gene editing company, has unveiled preclinical data for its gene editing candidate PBGENE-HBV, indicating its specificity in targeting HBV DNA without affecting the human genome. This development, announced at the European Association for the Study of the Liver Congress, supports the candidate's advancement to clinical trials as a potential treatment for chronic hepatitis B.
The data presented shows that PBGENE-HBV can precisely cut HBV DNA, leading to the elimination of cccDNA and inactivation of integrated HBV DNA, which are key drivers in the persistence of chronic hepatitis B infection. The treatment was well-tolerated in non-human primates even after multiple doses, with minor and transient liver enzyme elevations that normalized within two weeks, and no adverse changes in blood parameters were noted.
Jeff Smith, PhD, Chief Research Officer at Precision BioSciences, expressed optimism about the PBGENE-HBV program, highlighting its ability to target the HBV viral genome specifically and its tolerability, which enables multi-dosing in clinical settings. Smith emphasized that this advancement validates the capabilities of the company's proprietary ARCUS platform to deliver safe and efficacious in vivo gene editing therapies.
Chronic hepatitis B remains a significant global health issue, with an estimated 300 million people worldwide, including more than 1 million in the US, living with the infection in 2019. Current treatments focus on long-term viral suppression but do not eradicate the virus, often requiring lifelong administration. PBGENE-HBV represents a novel therapeutic approach aimed at directly eliminating the virus, potentially leading to a functional cure.
Precision BioSciences plans to submit an Investigational New Drug Application (IND) and/or Clinical Trial Application (CTA) for PBGENE-HBV in 2024. The company's ARCUS platform is distinguished by its unique gene editing capabilities, including gene insertion, elimination, and excision, which may offer more defined therapeutic outcomes compared to other technologies.
The advancement of PBGENE-HBV to clinical trials could mark a significant step toward providing a curative treatment option for individuals affected by chronic hepatitis B. This press release statement provides the basis for the reported development.
InvestingPro Insights
Precision BioSciences, Inc. (NASDAQ:DTIL) has shown promising preclinical results for its gene editing candidate, PBGENE-HBV, which is set to advance to clinical trials. As investors consider the potential impact of this development on the company's financial health and stock performance, certain metrics and tips from InvestingPro provide additional context:
InvestingPro Data:
- The company's market capitalization stands at $91.63 million, reflecting its scale in the biotechnology market.
- Revenue growth has been robust over the last twelve months as of Q1 2024, with an impressive increase of 88.25%.
- Despite significant revenue growth, the company's operating income margin was -60.09% in the same period, indicating the costs associated with its research and development efforts.
InvestingPro Tips:
- Precision BioSciences holds more cash than debt on its balance sheet, which could provide financial flexibility as it moves PBGENE-HBV into clinical trials.
- Analysts do not anticipate the company will be profitable this year, a consideration for investors as they weigh the prospects of the company's innovative ARCUS platform against its current financial performance.
Investors interested in a deeper dive into Precision BioSciences' financials and future outlook can find a wealth of additional tips on InvestingPro, including 12 more tips for DTIL. To explore these insights and more, visit https://www.investing.com/pro/DTIL and remember to use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
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