Precision BioSciences Announces MHRA Approval for Partner iECURE to Expand Phase 1/2 Clinical Trial of ARCUS Gene Editing Program in OTC Deficiency
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Insights
The approval of the Clinical Trial Authorization (CTA) by the UK's MHRA for ECUR-506 to proceed with the OTC-HOPE study is a pivotal development in the field of gene therapy. ECUR-506 employs the ARCUS gene editing platform to insert a functional copy of the OTC gene, potentially offering a transformative treatment for infants with Ornithine Transcarbamylase (OTC) deficiency. This genetic disorder leads to an inability to process ammonia, a toxic byproduct of protein metabolism. The current standard of care, which includes dietary restrictions and ammonia scavengers, only manages symptoms without addressing the underlying genetic issue.
From a research perspective, the ARCUS platform's ability to efficiently insert genes is significant. It represents an advancement from earlier gene editing tools like CRISPR-Cas9, which have been more focused on gene knockout or disruption. The ARCUS technology could potentially correct genetic defects by adding functional genes, which is a substantial leap forward in gene therapy. If ECUR-506 proves to be effective, it could set a precedent for treating a variety of genetic disorders with similar underlying mechanisms. The long-term implications for stakeholders include not only the potential for a cure for patients but also the opening of a new therapeutic market segment in gene editing for Precision BioSciences and its development partners.
The CTA approval in the United Kingdom for Precision BioSciences' partner iECURE to expand the Phase 1/2 OTC-HOPE study is a strong indicator of the regulatory confidence in the ARCUS gene editing platform. For investors, regulatory milestones such as these are critical as they de-risk the development process and can lead to significant value inflection points for the company's stock. Precision BioSciences' licensing strategy with iECURE allows the company to capitalize on its proprietary technology while sharing development risks.
Furthermore, the successful application of ARCUS in a clinical setting could enhance Precision BioSciences' valuation and attract potential partnerships or buyout offers, especially as the company advances its wholly owned hepatitis B program. However, it is crucial to monitor the progression of the clinical trials as gene therapy remains a highly complex and unpredictable field. The cost of development, potential commercialization challenges and the competitive landscape are key factors that could affect the financial outlook for Precision BioSciences and its investors.
The biotech sector closely watches the progression of novel therapies through regulatory milestones and the U.K.'s MHRA CTA approval for ECUR-506 is no exception. This event is particularly noteworthy as it validates the ARCUS platform's utility in gene editing, a technology area that has seen significant investor interest. The approval also suggests that the regulatory environment for gene therapies is becoming more navigable, which could encourage investment in similar biotech ventures.
Looking at the broader market, the success of ARCUS in clinical trials could stimulate further innovation and competition in the gene editing space, potentially impacting the strategies of other biotech firms. The ability to insert functional genes could serve as a benchmark for future gene therapy products. For Precision BioSciences, the expansion of the OTC-HOPE study into the U.K. not only diversifies the geographic footprint of their clinical trials but also provides a strategic advantage in accessing a larger patient population for data collection, which is critical for the robustness of clinical trial outcomes.
- Insertion of a functional OTC gene through ARCUS in vivo gene editing may provide lasting clinical benefit for children with OTC deficiency who are in dire need for effective treatments
- CTA approval for ECUR-506 in the
“Congratulations to iECURE for bringing the first gene editing approach into clinical investigation for patients with OTC deficiency. Approval from the MHRA to expand iECURE’s OTC-HOPE Phase 1/2 study represents yet another milestone, which both advances our partner’s work to potentially revolutionize the treatment of OTC deficiency in infants while validating the differentiated ability of ARCUS to efficiently insert genes and restore function,” said Michael Amoroso, Chief Executive Officer of Precision BioSciences. “To that end, we view progression through multiple regulatory agencies around the world as important proof-points for ARCUS that reflects our broader strategy to leverage ARCUS with select development partners as we continue to advance our wholly owned hepatitis B program toward an investigational new drug (IND) application and/or CTA filing in 2024.”
OTC deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for the detoxification of ammonia. Individuals with OTC deficiency can build up excessive levels of ammonia in their blood potentially resulting in devastating consequences, including irreversible neurological damage, coma, and death. The severe form of the condition emerges shortly after birth and is more common in boys than girls. The only corrective treatment for early onset severe OTC deficiency is a liver transplant. Currently available medical therapies do not correct the disease and do not eliminate the risk of life-threatening symptoms or crises.
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, Precision’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.
About ECUR-506
iECURE’s approach to gene editing for its initial programs, including OTC deficiency, relies on the delivery of two adeno-associated virus (AAV) capsids, each carrying different payloads. ECUR-506 comprises two vectors, an ARCUS® nuclease vector targeting gene editing in the well-characterized PCSK9 gene locus and a donor vector that inserts the desired functional OTC gene. iECURE has licensed the ARCUS nuclease for ECUR-506 from Precision BioSciences. The cut in the PCSK9 site serves as the insertion site for the OTC gene, providing a potential path to permanent expression of a healthy gene.
About the OTC-HOPE Study
The OTC-HOPE study is a Phase 1/2 first-in-human clinical trial of ECUR-506 in baby boys with genetically confirmed OTC deficiency and will test up to two dose levels of ECUR-506. The study is enrolling baby boys aged 24 hours to seven months who are diagnosed with severe neonatal onset OTC deficiency and meet certain other criteria. The primary objective is to assess the safety and tolerability of intravenous administration of a single dose of ECUR-506. It will also assess the pharmacokinetics and efficacy of ECUR-506 administration and the potential effects of ECUR-506 on disease-specific biologic markers, developmental milestones and quality of life.
About iECURE
iECURE is a clinical-stage gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need. We believe our approach has the potential to replace and restore the function of a dysfunctional gene, regardless of mutation, by knocking-in a healthy copy of that gene to offer durable gene expression and long-term, potentially curative, therapeutic benefit. Our management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. We intend to leverage our team’s core strength in research and development strategy to identify what we believe to be the most suitable target and modality for our product candidates to address particular liver diseases. We are collaborating with the University of Pennsylvania’s Gene Therapy Program (GTP) led by James M. Wilson, M.D., Ph.D., to utilize GTP’s world-class translational expertise and infrastructure, which has helped generate our initial pipeline of potential product candidates. For more information, visit https://iecure.com and follow on LinkedIn.
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 and our partners’ product candidates and gene editing approaches including editing efficiency; the suitability of ARCUS nucleases for gene insertion and other gene editing approaches; the expected timing of regulatory processes; expectations about our and our partners’ operational initiatives and strategy; 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,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “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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Naresh Tanna
Vice President of Investor Relations
Naresh.Tanna@precisionbiosciences.com
Source: Precision BioSciences, Inc.
FAQ
What is the significance of the recent CTA approval for ECUR-506 by the MHRA?
What is OTC deficiency and why is it a critical condition?
What is the OTC-HOPE study investigating?
How does Precision BioSciences aim to leverage ARCUS technology with development partners?
