Precision BioSciences Submits First Clinical Trial Applications to Initiate Phase 1 Trial for PBGENE-HBV for the Treatment of Chronic Hepatitis B
Precision BioSciences (Nasdaq: DTIL) has submitted Clinical Trial Applications (CTA) to initiate a Phase 1 study for PBGENE-HBV, its in vivo gene editing program designed to potentially cure chronic hepatitis B virus (HBV). The therapy aims to eliminate cccDNA, the key source of replicating HBV, while also inactivating integrated HBV DNA in hepatocytes.
This marks the first gene editing approach for chronic hepatitis B, targeting an estimated 300 million people globally affected by the disease. The company's regulatory package is supported by robust non-human primate safety studies and efficacy in numerous preclinical models. Precision BioSciences plans to initiate the global Phase 1 trial soon and expects to report data in 2025.
Precision BioSciences (Nasdaq: DTIL) ha presentato Domande di Sperimentazione Clinica (CTA) per avviare uno studio di Fase 1 per PBGENE-HBV, il suo programma di editing genetico in vivo progettato per potenzialmente curare l'epatite B cronica. La terapia mira ad eliminare il cccDNA, la principale fonte di replicazione dell'HBV, mentre inattiva anche il DNA HBV integrato negli epatociti.
Questo segna il primo approccio di editing genetico per l'epatite B cronica, mirando a un numero stimato di 300 milioni di persone in tutto il mondo colpite dalla malattia. Il pacchetto normativo dell'azienda è supportato da studi di sicurezza robusti su primati non umani e da efficacia in numerosi modelli preclinici. Precision BioSciences prevede di avviare presto la sperimentazione globale di Fase 1 e si aspetta di riportare dati nel 2025.
Precision BioSciences (Nasdaq: DTIL) ha presentado Solicitudes de Ensayos Clínicos (CTA) para iniciar un estudio de Fase 1 para PBGENE-HBV, su programa de edición genética in vivo diseñado para potencialmente curar la hepatitis B crónica. La terapia tiene como objetivo eliminar el cccDNA, la fuente clave de replicación del HBV, mientras también inactiva el ADN del HBV integrado en los hepatocitos.
Este marca el primer enfoque de edición genética para la hepatitis B crónica, apuntando a unas 300 millones de personas en todo el mundo afectadas por la enfermedad. El paquete regulatorio de la compañía está respaldado por robustos estudios de seguridad en primates no humanos y por eficacia en numerosos modelos preclínicos. Precision BioSciences planea iniciar pronto el ensayo global de Fase 1 y espera informar datos en 2025.
Precision BioSciences (Nasdaq: DTIL)는 PBGENE-HBV는 만성 B형 간염 바이러스(HBV)를 치료하기 위해 설계된 생체 내 유전자 편집 프로그램을 시작하기 위해 1상 연구를 위한 임상시험 신청서(CTA)를 제출했습니다. 이 요법은 HBV의 복제 주요 원인인 cccDNA를 제거하고 간세포 내 통합된 HBV DNA를 비활성화하는 것을 목표로 합니다.
이는 만성 B형 간염을 위한 첫 번째 유전자 편집 접근 방식을 구현하는 것으로, 이 질병에 영향을 받는 전 세계 약 3억 명의 사람들을 대상으로 하고 있습니다. 회사의 규제 패키지는 비인간 영장류에 대한 안전성 연구 및 여러 전임상 모델에서의 효능을 통해 뒷받침되고 있습니다. Precision BioSciences는 곧 글로벌 1상 시험을 시작할 계획이며 2025년 데이터 보고를 기대하고 있습니다.
Precision BioSciences (Nasdaq: DTIL) a soumis des Demandes d'Essai Clinique (CTA) pour initier une étude de Phase 1 pour PBGENE-HBV, son programme d'édition génique in vivo conçu pour potentiellement guérir l'hépatite B chronique. La thérapie vise à éliminer le cccDNA, la principale source de réplication de l'HBV, tout en inactivant également l'ADN de l'HBV intégré dans les hépatocytes.
Ceci marque la première approche d'édition génétique pour l'hépatite B chronique, visant environ 300 millions de personnes dans le monde affectées par la maladie. Le paquet réglementaire de l'entreprise est soutenu par des études de sécurité robustes sur des primates non humains et l'efficacité dans de nombreux modèles précliniques. Precision BioSciences prévoit de commencer prochainement l'essai mondial de Phase 1 et s'attend à communiquer des données en 2025.
Precision BioSciences (Nasdaq: DTIL) hat Anträge für Klinische Studien (CTA) eingereicht, um eine Phase 1-Studie für PBGENE-HBV, sein in vivo-Gentechnologieprogramm, zu starten, das potenziell eine Heilung für das chronische Hepatitis-B-Virus (HBV) anzielt. Die Therapie zielt darauf ab, das cccDNA, die Hauptquelle der HBV-Replikation, zu eliminieren und gleichzeitig integriertes HBV-DNA in Hepatozyten inaktiv zu machen.
Dies markiert den ersten Ansatz zur Genbearbeitung bei chronischer Hepatitis B, der sich an schätzungsweise 300 Millionen Menschen weltweit richtet, die von der Krankheit betroffen sind. Das regulatorische Paket des Unternehmens wird durch robuste Sicherheitsstudien an nicht-humane Primaten und die Wirksamkeit in zahlreichen präklinischen Modellen unterstützt. Precision BioSciences plant, bald die globale Phase-1-Studie zu starten und erwartet, 2025 Daten zu berichten.
- First gene editing approach for chronic hepatitis B submitted for clinical trials
- Potential to cure chronic HBV by eliminating cccDNA and inactivating integrated HBV DNA
- Robust non-human primate safety studies and preclinical efficacy data support the regulatory submissions
- Global Phase 1 trial expected to initiate soon with data reporting anticipated in 2025
- Expansion of clinical team expertise and formation of hepatitis scientific advisory board
- None.
Insights
The submission of Clinical Trial Applications (CTAs) for PBGENE-HBV marks a significant milestone in the development of a potential cure for chronic hepatitis B virus (HBV). This gene editing approach, utilizing Precision BioSciences' ARCUS® platform, is designed to eliminate cccDNA and inactivate integrated HBV DNA in hepatocytes, addressing the root cause of the disease.
Key points to consider:
- PBGENE-HBV is the first clinical stage in vivo gene editing program for chronic HBV
- Current treatments only offer a
1-3% chance of functional cure - HBV affects an estimated 300 million people globally
- The company plans to initiate a global Phase 1 trial soon, with data expected in 2025
This innovative approach could potentially revolutionize HBV treatment, offering a higher chance of functional cure compared to existing therapies. However, as with any novel gene editing therapy, safety and efficacy in humans remain to be proven in clinical trials.
Precision BioSciences' advancement of PBGENE-HBV to clinical trials represents a significant step in the company's pipeline development. This move positions DTIL as a frontrunner in the gene editing space for HBV treatment, potentially opening up a vast market opportunity.
Investor considerations:
- Market potential: With 300 million HBV patients globally, a successful therapy could generate substantial revenue
- First-mover advantage: Being the first in-vivo gene editing approach for HBV could provide a competitive edge
- Regulatory progress: Multiple CTA submissions indicate confidence in preclinical data and regulatory strategy
- Timeline: Phase 1 data expected in 2025, suggesting a long runway before potential commercialization
While this news is promising for DTIL's long-term prospects, investors should be aware that the company's
- PBGENE-HBV is the only drug modality designed to target the root cause of disease by eliminating cccDNA and inactivating integrated HBV
- Clinical trial applications submitted for the first gene editing approach for chronic hepatitis B
- Final clinical candidate safety data and plans for the Phase 1 trial to be shared in November prior to the American Association for the Study of Liver Diseases meeting
“The CTA submissions for PBGENE-HBV are important milestones for Precision as we pioneer this potentially curative therapy for chronic hepatitis B. These regulatory submissions are the culmination of our team’s dedication, commitment and highly productive interactions with global regulators as we develop the first clinical stage in vivo gene editing program for chronic hepatitis B virus, recognized as one of the largest global public health problems by the World Health Organization with an estimated 300 million people afflicted globally,” said Michael Amoroso, Chief Executive Officer of Precision BioSciences. “Our comprehensive regulatory package, supported by robust non-human primate (NHP) safety studies, the gold standard model for predicting safety in humans, and efficacy in numerous preclinical models of hepatitis B including in NHPs enables us to proceed with planned regulatory submissions in multiple markets around the world. We recently bolstered our clinical team expertise while building a world class hepatitis scientific advisory board to assist in guiding execution of our global Phase 1 trial. Our goal is clear, to generate robust clinical data for the patients afflicted with hepatitis B who are counting on us to significantly increase their chance of achieving a functional cure.”
Dr. Murray Abramson, Senior Vice President, Head of Clinical Development added: “Current standard of care treatment with nucleos(t)ide analogs only offers 1
Precision is on track to submit additional regulatory applications as part of its global Phase 1 regulatory strategy for PBGENE-HBV. The next update on the PBGENE-HBV program is expected to take place before the American Association for the Study of Liver Diseases (AASLD) Annual Meeting in November. Details about how to participate in the update will be provided in advance.
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 is 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 (including PBGENE-HBV) 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 or providing a better chance of 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 and the acceptance of these filings by regulatory agencies); the translation of preclinical safety and efficacy studies and models to safety and efficacy in humans, the suitability of PBGENE-HBV for the treatment of hepatitis and the targeting of the root cause of the disease, 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, and involve a 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 our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and 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; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ or other licensees’ ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the
View source version on businesswire.com: https://www.businesswire.com/news/home/20240930788909/en/
Investor Contact:
Naresh Tanna
Vice President, Investor Relations
Naresh.Tanna@precisionbiosciences.com
Source: Precision BioSciences, Inc.
FAQ
What is PBGENE-HBV and how does it aim to treat chronic hepatitis B?
When did Precision BioSciences (DTIL) submit Clinical Trial Applications for PBGENE-HBV?
What is the potential impact of PBGENE-HBV compared to current hepatitis B treatments?
When does Precision BioSciences (DTIL) expect to report data from the PBGENE-HBV Phase 1 trial?
When will Precision BioSciences (DTIL) provide the next update on the PBGENE-HBV program?
