Precision BioSciences Presents Preclinical Safety and Efficacy Data Supporting Repeat Dosing of PBGENE-HBV With a Goal of Curing Chronic Hepatitis B at the Global Hepatitis Summit 2025
Precision BioSciences (NASDAQ: DTIL) presented preclinical data for PBGENE-HBV, the first clinical-stage gene editing therapy for chronic hepatitis B, at the Global Hepatitis Summit 2025. The data supports repeat dosing in the ongoing ELIMINATE-B Phase 1 trial.
Key findings show that in non-human primates, repeated PBGENE-HBV administration was safe and well-tolerated, with no systemic accumulation. Previous data demonstrated up to 99% viral eradication with two doses. Initial results from cohort 1 showed the first 0.2 mg/kg dose was safe, with substantial HBsAg reduction in two of three participants.
The ELIMINATE-B study is actively enrolling HBeAg-negative chronic Hepatitis B patients in Moldova, Hong Kong, and New Zealand, with planned expansion to U.S. sites. The trial will evaluate up to three dose administrations at each dose level, with the company planning to share detailed clinical data throughout 2025.
Precision BioSciences (NASDAQ: DTIL) ha presentato dati preclinici per PBGENE-HBV, la prima terapia di editing genetico in fase clinica per l'epatite B cronica, al Global Hepatitis Summit 2025. I dati supportano la somministrazione ripetuta nel trial in corso ELIMINATE-B di fase 1.
I risultati chiave mostrano che nei primati non umani, la somministrazione ripetuta di PBGENE-HBV è stata sicura e ben tollerata, senza accumulo sistemico. Dati precedenti hanno dimostrato fino al 99% di eradicazione virale con due dosi. I risultati iniziali del coorte 1 hanno mostrato che la prima dose di 0,2 mg/kg era sicura, con una sostanziale riduzione di HBsAg in due dei tre partecipanti.
Lo studio ELIMINATE-B sta attivamente reclutando pazienti con epatite B cronica HBeAg-negativa in Moldova, Hong Kong e Nuova Zelanda, con un'espansione pianificata nei siti degli Stati Uniti. Il trial valuterà fino a tre somministrazioni di dose a ciascun livello di dose, con l'azienda che prevede di condividere dati clinici dettagliati nel corso del 2025.
Precision BioSciences (NASDAQ: DTIL) presentó datos preclínicos para PBGENE-HBV, la primera terapia de edición genética en etapa clínica para la hepatitis B crónica, en la Cumbre Global sobre Hepatitis 2025. Los datos respaldan la administración repetida en el ensayo en curso ELIMINATE-B de fase 1.
Los hallazgos clave muestran que en primates no humanos, la administración repetida de PBGENE-HBV fue segura y bien tolerada, sin acumulación sistémica. Datos previos demostraron hasta un 99% de erradicación viral con dos dosis. Los resultados iniciales del cohorte 1 mostraron que la primera dosis de 0.2 mg/kg fue segura, con una reducción sustancial de HBsAg en dos de tres participantes.
El estudio ELIMINATE-B está reclutando activamente pacientes con hepatitis B crónica negativa para HBeAg en Moldavia, Hong Kong y Nueva Zelanda, con una expansión planificada a sitios en EE. UU. El ensayo evaluará hasta tres administraciones de dosis en cada nivel de dosis, con la empresa planeando compartir datos clínicos detallados a lo largo de 2025.
Precision BioSciences (NASDAQ: DTIL)는 2025 글로벌 간염 정상 회담에서 만성 B형 간염을 위한 첫 번째 임상 단계 유전자 편집 치료제인 PBGENE-HBV에 대한 전임상 데이터를 발표했습니다. 이 데이터는 진행 중인 ELIMINATE-B 1상 시험에서 반복 투여를 지원합니다.
주요 발견은 비인간 영장류에서 반복적인 PBGENE-HBV 투여가 안전하고 잘 견딜 수 있었다는 것이며, 전신 축적이 없었습니다. 이전 데이터는 두 번의 투여로 최대 99%의 바이러스 제거를 보여주었습니다. 코호트 1의 초기 결과는 첫 번째 0.2 mg/kg 투여가 안전했고, 세 명의 참가자 중 두 명에서 HBsAg가 상당히 감소했다는 것을 보여주었습니다.
ELIMINATE-B 연구는 몰도바, 홍콩 및 뉴질랜드에서 HBeAg 음성 만성 B형 간염 환자를 적극적으로 모집하고 있으며, 미국 사이트로의 확장이 계획되어 있습니다. 이 시험은 각 투여 수준에서 최대 세 번의 투여를 평가할 예정이며, 회사는 2025년 동안 자세한 임상 데이터를 공유할 계획입니다.
Precision BioSciences (NASDAQ: DTIL) a présenté des données précliniques sur PBGENE-HBV, la première thérapie d'édition génétique en phase clinique pour l'hépatite B chronique, lors du Global Hepatitis Summit 2025. Les données soutiennent l'administration répétée dans l'essai ELIMINATE-B de phase 1 en cours.
Les résultats clés montrent que chez les primates non humains, l'administration répétée de PBGENE-HBV était sans danger et bien tolérée, sans accumulation systémique. Des données antérieures ont montré jusqu'à 99 % d'éradication virale avec deux doses. Les résultats initiaux du cohorte 1 ont montré que la première dose de 0,2 mg/kg était sûre, avec une réduction substantielle de l'HBsAg chez deux des trois participants.
L'étude ELIMINATE-B recrute activement des patients atteints d'hépatite B chronique négatifs pour HBeAg en Moldavie, à Hong Kong et en Nouvelle-Zélande, avec une expansion prévue sur des sites aux États-Unis. L'essai évaluera jusqu'à trois administrations de dose à chaque niveau de dose, l'entreprise prévoyant de partager des données cliniques détaillées tout au long de 2025.
Precision BioSciences (NASDAQ: DTIL) präsentierte präklinische Daten zu PBGENE-HBV, der ersten klinischen Genbearbeitungstherapie für chronische Hepatitis B, auf dem Global Hepatitis Summit 2025. Die Daten unterstützen die wiederholte Dosierung in der laufenden ELIMINATE-B Phase 1-Studie.
Wichtige Ergebnisse zeigen, dass die wiederholte Verabreichung von PBGENE-HBV bei nichtmenschlichen Primaten sicher und gut verträglich war, ohne systematische Ansammlung. Frühere Daten zeigten eine bis zu 99%ige virale Eliminierung bei zwei Dosen. Erste Ergebnisse aus Kohorte 1 zeigten, dass die erste Dosis von 0,2 mg/kg sicher war, mit einer erheblichen HBsAg-Reduktion bei zwei von drei Teilnehmern.
Die ELIMINATE-B-Studie rekrutiert aktiv HBeAg-negative chronische Hepatitis-B-Patienten in Moldawien, Hongkong und Neuseeland, mit einer geplanten Erweiterung auf Standorte in den USA. Die Studie wird bis zu drei Dosierungen auf jedem Dosierungsniveau bewerten, wobei das Unternehmen plant, im Laufe des Jahres 2025 detaillierte klinische Daten zu teilen.
- First clinical-stage gene editing therapy for chronic hepatitis B
- 99% viral eradication achieved in preclinical studies with two doses
- Initial Phase 1 results show safety and efficacy at lowest dose
- FDA IND clearance received for U.S. trial expansion
- Substantial HBsAg reduction in 2 of 3 participants at lowest dose
- One of three participants did not show substantial HBsAg reduction
- Multiple doses may be required for effective treatment
- Optimal dosing regime not yet determined
Insights
Precision BioSciences' presentation at the Global Hepatitis Summit represents meaningful clinical progress for their lead ARCUS-based gene editing therapy, PBGENE-HBV. The data supporting repeat dosing is particularly significant as it addresses a fundamental requirement for achieving functional cures in chronic HBV.
The preclinical package presented includes several critical findings that de-risk their clinical approach: no systemic accumulation of the drug product in non-human primates, absence of germline distribution (eliminating heritable edit concerns), and no increased off-target risk with additional administrations. These safety parameters are essential hurdles for any gene editing therapy.
The up to 99% viral eradication observed in NHP models with two doses provides strong mechanistic validation. Initial clinical data from the first cohort demonstrates the therapy was well-tolerated at the lowest dose (0.2mg/kg), with HBsAg reductions in two of three patients after just a single administration - a promising early efficacy signal.
The global trial expansion following FDA IND clearance enhances recruitment potential and broadens the genetic diversity of the study population, which is particularly important for a viral disease with genetic variants. The company's execution on their clinical timeline appears on track with planned escalation to higher doses while continuing to evaluate repeat administrations.
For context, chronic HBV affects approximately 296 million people globally with curative options. Current treatments typically suppress viral replication but rarely eliminate the virus completely, leaving patients at risk for the serious complications mentioned in the release.
This clinical development update strengthens Precision BioSciences' competitive positioning in the HBV therapeutic landscape. As the company claims to have "the first and only clinical stage gene editing therapy for chronic hepatitis B," they potentially have a first-mover advantage in applying gene editing technology to this indication.
The repeat dosing strategy is scientifically and commercially significant. By demonstrating that multiple administrations are both safe and potentially more effective (with up to 99% viral eradication in NHP models), the company has established a rational clinical pathway that could differentiate their approach from competing modalities.
The preliminary human data showing HBsAg reductions after just the first dose at the lowest concentration (0.2mg/kg) suggests dose-response potential as they move to higher doses. This early efficacy signal, while to 2 of 3 patients, is noteworthy for a Phase 1 study primarily designed to evaluate safety.
From a pipeline perspective, PBGENE-HBV represents Precision's lead in vivo candidate, making its progress important for validating their proprietary ARCUS platform technology. Their geographic diversification of clinical sites (Moldova, Hong Kong, New Zealand, and now the U.S.) demonstrates strategic execution to accelerate enrollment - a critical factor for maintaining development timelines and cash runway.
While no specific financial metrics were provided, this progress potentially positions Precision to achieve significant development milestones throughout 2025. The global HBV market represents a substantial opportunity, with chronic infection affecting approximately 296 million people worldwide and current treatments largely to viral suppression rather than cure.
- PBGENE-HBV, the first and only clinical stage gene editing therapy for chronic hepatitis B –
- Repeat dosing of PBGENE-HBV supported by definitive preclinical safety and toxicology studies -
- ELIMINATE-B global Phase 1 study for chronic hepatitis B actively enrolling patients and will evaluate up to three dose administrations of PBGENE-HBV at each dose level –
- Precision remains on track to continue reporting clinical data throughout 2025 –
“We are excited to share new supportive preclinical data for our lead clinical program PBGENE-HBV, which is currently enrolling patients in the ELIMINATE-B Phase 1 study. These new data highlight the potential to safely administer repeated doses of PBGENE-HBV, a LNP delivered gene editor, to achieve durable functional cures in chronic hepatitis B patients,” said Dr. Murray Abramson, MD, MPH, Senior Vice President and Head of Clinical Development at Precision BioSciences. “As we look to translate these results into the clinic, our Phase 1 trial is evaluating up to three dose administrations per patient at each dose level to assess the cumulative effect of editing and eliminating viral DNA with the goal of cure. After demonstrating that the first administration at the lowest dose level was safe and well tolerated, we have commenced subsequent administrations at the lowest dose level.”
Presentation Details:
Title: PBGENE-HBV definitive preclinical toxicokinetic and toxicology data enables advancement to clinical trials for a potentially curative gene editing treatment for chronic hepatitis B
Oral Presentation Number: 30267
Date and Time: March 21, 2025, 9:20-9:30 am PDT
Preclinical safety data to be presented at GHS 2025 support the activity of multiple doses of PBGENE-HBV in order to increase cumulative editing and drive potential cures. In non-human primates (NHPs), repeated administration of PBGENE-HBV was safe and well tolerated, with no systemic accumulation of PBGENE-HBV drug product. Furthermore, PBGENE-HBV was not distributed to germ cells at any of the analyzed timepoints, supporting the conclusion that there is no risk of heritable genome edits. Additionally, the Company conducted extensive nuclease specificity assessments in HBV-infected primary human hepatocytes and showed no increased off-target risk with additional dose administrations. The Company has previously shared nonhuman primate data showing that the administration of two doses of PBGENE-HBV achieved up to
In February, Precision announced initial results from the first administration of PBGENE-HBV in cohort 1, the lowest dose level of the ELIMINATE-B trial. PBGENE-HBV, which comprises an ARCUS-encoding mRNA encapsulated in a lipid nanoparticle (LNP), was safe and well tolerated in all three participants in cohort 1 after the first administration of a 0.2 mg/kg dose. In addition to safety, PBGENE-HBV demonstrated a substantial reduction in Hepatitis B surface antigen (HBsAg) in two of the three participants following the first of three administrations at the lowest dose level. The Company has commenced dosing planned subsequent administrations at the lowest dose level (0.2mg/kg).
The ELIMINATE-B study is currently enrolling HBeAg-negative chronic Hepatitis B patients at leading global infectious disease sites in
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
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 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).
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 PBGENE-HBV); the design of PBGENE-HBV, including expected repeat dose administrations, to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, with the goal of a cure for chronic hepatitis B; 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 and reporting of clinical data; translation of preclinical studies to viral engagement in humans; and risks of heritable genome edits and off-target editing with additional dose administrations. 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.
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Investor and Media Contact:
Naresh Tanna
Vice President of Investor Relations
naresh.tanna@precisionbiosciences.com
Source: Precision BioSciences, Inc.
FAQ
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