Precision BioSciences Receives U.S. FDA Fast Track Designation for PBGENE-HBV, a First-In-Class Gene Editing Therapy Designed to Eliminate the Root Cause of Chronic Hepatitis B
Precision BioSciences (DTIL) has received Fast Track designation from the FDA for PBGENE-HBV, its lead in vivo gene editing therapy program targeting chronic hepatitis B. The therapy aims to cure the disease by eliminating cccDNA and inactivating integrated HBV DNA in hepatocytes.
The company is currently conducting the global Phase 1 ELIMINATE-B trial across multiple countries including the United States, Moldova, Hong Kong, New Zealand, and the United Kingdom. Initial results have shown encouraging safety and antiviral activity. Updates on the full low-dose cohort and higher dose levels are expected throughout 2025.
The Fast Track designation may enable more frequent FDA communications, rolling review of marketing applications, and potential Priority Review eligibility.
Precision BioSciences (DTIL) ha ottenuto la designazione Fast Track dalla FDA per PBGENE-HBV, il suo principale programma di terapia di editing genico in vivo rivolto all'epatite B cronica. La terapia mira a curare la malattia eliminando il cccDNA e inattivando il DNA HBV integrato negli epatociti.
L'azienda sta attualmente conducendo la sperimentazione globale di Fase 1 ELIMINATE-B in diversi paesi, tra cui Stati Uniti, Moldavia, Hong Kong, Nuova Zelanda e Regno Unito. I risultati iniziali hanno mostrato un profilo di sicurezza incoraggiante e attività antivirale. Aggiornamenti sulla coorte a basso dosaggio completa e sui livelli di dosaggio più elevati sono previsti nel corso del 2025.
La designazione Fast Track potrebbe consentire comunicazioni più frequenti con la FDA, una revisione progressiva delle domande di autorizzazione al commercio e la possibile eleggibilità per la Priority Review.
Precision BioSciences (DTIL) ha recibido la designación Fast Track de la FDA para PBGENE-HBV, su principal programa de terapia de edición genética in vivo dirigido a la hepatitis B crónica. La terapia busca curar la enfermedad eliminando el cccDNA e inactivando el ADN HBV integrado en los hepatocitos.
La compañía está llevando a cabo actualmente el ensayo global de Fase 1 ELIMINATE-B en varios países, incluyendo Estados Unidos, Moldavia, Hong Kong, Nueva Zelanda y Reino Unido. Los resultados iniciales han mostrado seguridad alentadora y actividad antiviral. Se esperan actualizaciones sobre la cohorte de dosis baja completa y niveles de dosis más altos a lo largo de 2025.
La designación Fast Track podría permitir comunicaciones más frecuentes con la FDA, revisión continua de las solicitudes de comercialización y posible elegibilidad para la Revisión Prioritaria.
Precision BioSciences (DTIL)는 만성 B형 간염을 표적으로 하는 주요 생체 내 유전자 편집 치료제 PBGENE-HBV에 대해 FDA로부터 패스트 트랙 지정을 받았습니다. 이 치료제는 간세포 내 cccDNA를 제거하고 통합된 HBV DNA를 비활성화하여 질병을 치료하는 것을 목표로 합니다.
회사는 현재 미국, 몰도바, 홍콩, 뉴질랜드, 영국 등 여러 국가에서 글로벌 1상 ELIMINATE-B 임상시험을 진행 중입니다. 초기 결과는 안전성과 항바이러스 활성이 고무적임을 보여주고 있습니다. 저용량 코호트 전체 및 고용량 수준에 대한 업데이트가 2025년 내내 예정되어 있습니다.
패스트 트랙 지정은 FDA와의 더 빈번한 소통, 마케팅 신청서의 순차적 검토, 우선 심사 자격 가능성을 제공할 수 있습니다.
Precision BioSciences (DTIL) a obtenu la désignation Fast Track de la FDA pour PBGENE-HBV, son principal programme de thérapie d'édition génétique in vivo ciblant l'hépatite B chronique. Cette thérapie vise à guérir la maladie en éliminant l'ADN cccDNA et en inactivant l'ADN HBV intégré dans les hépatocytes.
L'entreprise mène actuellement l'essai mondial de phase 1 ELIMINATE-B dans plusieurs pays, dont les États-Unis, la Moldavie, Hong Kong, la Nouvelle-Zélande et le Royaume-Uni. Les premiers résultats ont montré une sécurité encourageante et une activité antivirale. Des mises à jour concernant la cohorte à faible dose complète et des doses plus élevées sont attendues tout au long de 2025.
La désignation Fast Track pourrait permettre des communications plus fréquentes avec la FDA, un examen continu des demandes de mise sur le marché et une éventuelle éligibilité à l'examen prioritaire.
Precision BioSciences (DTIL) hat von der FDA die Fast Track-Zulassung für PBGENE-HBV erhalten, das führende in vivo Gen-Editing-Therapieprogramm gegen chronische Hepatitis B. Die Therapie zielt darauf ab, die Krankheit zu heilen, indem cccDNA eliminiert und integrierte HBV-DNA in Hepatozyten inaktiviert wird.
Das Unternehmen führt derzeit die globale Phase-1-Studie ELIMINATE-B in mehreren Ländern durch, darunter die USA, Moldawien, Hongkong, Neuseeland und das Vereinigte Königreich. Erste Ergebnisse zeigen eine vielversprechende Sicherheit und antivirale Aktivität. Updates zur vollständigen Niedrigdosis-Kohorte und zu höheren Dosierungsstufen werden im Laufe des Jahres 2025 erwartet.
Die Fast Track-Zulassung kann häufigere FDA-Kommunikationen, eine rollierende Überprüfung von Zulassungsanträgen und eine mögliche Prioritätsbewertung ermöglichen.
- Received FDA Fast Track designation, potentially accelerating development and review process
- Initial safety and antiviral activity data from ELIMINATE-B trial showing encouraging results
- Global Phase 1 trial expansion across five countries
- Complete trial results not expected until 2025
- Still in early Phase 1 stage of development
Insights
The FDA's Fast Track designation for PBGENE-HBV represents a significant regulatory milestone for Precision BioSciences. This designation acknowledges both the serious unmet need in chronic hepatitis B treatment and the potential of their novel ARCUS-based gene editing approach.
Fast Track provides tangible benefits that could accelerate PBGENE-HBV's development timeline, including more frequent FDA interactions, potential rolling review of future applications, and possible eligibility for Priority Review. For a clinical-stage biotech with a
The company's mention of "initial safety and antiviral activity" in their ELIMINATE-B trial suggests preliminary positive signals, though detailed efficacy data remains pending. Their approach targeting both cccDNA (the viral reservoir) and integrated HBV DNA addresses the root cause of chronic infection – a mechanistically sound strategy that differentiates it from current treatments that primarily suppress viral replication.
The global scope of their Phase 1 trial across five countries indicates a robust clinical development program. With data updates expected throughout 2025 from both low-dose cohorts with multiple administrations and higher dose levels, we'll soon have more clarity on this therapy's potential to address the estimated 296 million people living with chronic HBV worldwide.
This Fast Track designation materially enhances Precision BioSciences' development proposition in the competitive gene editing landscape. With their proprietary ARCUS platform now receiving regulatory validation for their lead wholly-owned asset, the company gains important differentiation from other gene editing players.
Chronic hepatitis B represents a substantial market opportunity with inadequate current treatment options. Existing therapies typically require lifelong administration without offering cure potential. A gene editing approach targeting viral cccDNA – essentially addressing the root cause rather than symptoms – could command premium pricing if successful.
The company's strategic decision to pursue higher-risk but wholly-owned programs appears to be gaining traction. This regulatory milestone could potentially attract partnership interest from larger pharmaceutical companies seeking novel modalities for viral diseases, similar to deals we've seen for other innovative HBV approaches.
From a capital allocation perspective, the expedited development pathway should reduce overall program costs while accelerating potential revenue timelines. For a company with market capitalization (
“We are pleased to receive Fast Track designation from the FDA for PBGENE-HBV and believe this classification underscores the urgent need for improved treatment options for patients living with chronic hepatitis B,” said Michael Amoroso, President and Chief Executive Officer at Precision BioSciences. “We’ve been encouraged by the initial safety and antiviral activity we have observed in the ELIMINATE-B trial and look forward to continuing to work closely with the FDA as we progress PBGENE-HBV through clinical development.”
Precision is evaluating PBGENE-HBV in the ongoing global Phase 1 ELIMINATE-B trial, with clinical investigation in
Fast Track designation is designed to facilitate development and expedite the review of drugs that are intended to treat serious or life-threatening conditions and address an unmet medical need. A drug that has received Fast Track designation may be eligible for more frequent meetings and communications with the FDA and rolling review of any application for marketing approval. A drug receiving Fast Track designation may also be eligible for Priority Review if relevant criteria are met.
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 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 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 PBGENE-HBV and our gene editing approaches including editing efficiency, and the suitability of ARCUS nucleases for gene insertion, gene elimination and gene excision and differentiation from other gene editing approaches; the expected timing of regulatory processes and clinical operations, including filings, studies, enrollment and clinical data for PBGENE-HBV; the design of PBGENE-HBV to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures; the encouraging initial safety and antiviral activity observed in the ELIMINATE-B clinical trial; plans to provide ongoing updates on the full low-dose cohort for the PBGENE-HBV study, including multiple dose administrations, and data from higher dose levels throughout 2025; and anticipated timing of clinical data . In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “appear,” “approach,” “believe,” “contemplate,” “could,” “designed,” “encouraged”, “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “strive,” “suggest,” “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
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:
Naresh Tanna
Vice President of Investor Relations
naresh.tanna@precisionbiosciences.com
Source: Precision BioSciences, Inc.
FAQ
What is the significance of FDA Fast Track designation for DTIL's PBGENE-HBV therapy?
How does Precision BioSciences' PBGENE-HBV therapy work to treat hepatitis B?
Where is DTIL conducting the ELIMINATE-B clinical trial for PBGENE-HBV?
When will DTIL release the next clinical data for PBGENE-HBV?
