Precision BioSciences Announces Receipt of FDA Fast Track Designation for ECUR-506, iECURE’s Program for the Treatment of Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency

DURHAM, N.C.–(BUSINESS WIRE)– Precision BioSciences, Inc., (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion, elimination, and excision, today announced that its partner iECURE has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for ECUR-506. ECUR-506 is iECURE’s in vivo gene insertion program designed to treat neonatal onset Ornithine Transcarbamylase (OTC) deficiency utilizing a PCSK9-specific ARCUS nuclease, licensed from Precision, that enables insertion of a functional copy of the OTC gene.

The Fast Track designation is designed to facilitate the development and expedite the review of therapeutics to treat serious conditions that fill an unmet medical need. Therapeutics that receive Fast Track designation may be eligible for more frequent meetings with and written communication from the FDA to discuss the therapeutic’s development plan and ensure collection of appropriate data to support potential approval of the therapeutic. Provided relevant criteria are met, programs with Fast Track designation are eligible for accelerated approval and priority review as well. ECUR-506 previously received Rare Pediatric Disease and Orphan Drug designations from the FDA, and Orphan designation from the European Commission for the treatment of OTC deficiency.

“Congratulations to iECURE for this significant regulatory designation and for what it can potentially mean to patients with neonatal onset OTC deficiency. Receiving Fast Track designation from the FDA underscores the severe unmet need for patients with neonatal onset OTC deficiency and the therapeutic potential of this novel gene insertion approach,” said Michael Amoroso, Chief Executive Officer of Precision BioSciences. “This designation represents yet another positive development for Precision’s ARCUS platform as our partner iECURE strives to revolutionize the standard of care for OTC deficiency with a gene insertion program. It also further validates the ARCUS platform as we continue advancing our wholly owned hepatitis B program toward an expected IND and/or CTA in 2024.”

The OTC-HOPE study is a Phase 1/2 first-in-human study in newborn males with genetically confirmed neonatal onset OTC deficiency. It is designed primarily to assess the safety and tolerability of ECUR-506 following intravenous administration of a single dose. Secondary objectives are to assess the pharmacokinetics and efficacy of ECUR-506. The OTC-HOPE study is open for enrollment in the United Kingdom. In addition, trial sites in the United States and Australia are activating and will be enrolling later this year.

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.

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).

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. ECUR-506 is being studied in the OTC-HOPE study, the first clinical meganuclease-based in vivo gene insertion program.

About the OTC-HOPE Study

The OTC-HOPE study is a Phase 1/2 first-in-human clinical trial of ECUR-506 in newborn males with genetically confirmed OTC deficiency and will test differing dose levels of ECUR-506. The study is enrolling newborn males up to seven months of age 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 OTC Deficiency

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 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. iECURE believes their 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. The iECURE management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. iECURE intends to leverage their team’s core strength in research and development strategy to identify what they believe to be the most suitable target and modality for their product candidates to address particular liver diseases. iECURE is 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 their initial pipeline of potential product candidates.

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