DURHAM, N.C. - Precision BioSciences, Inc. (NASDAQ:DTIL), a gene editing company, announced today that its partner iECURE has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for its gene therapy candidate ECUR-506. The therapy is aimed at treating neonatal onset Ornithine Transcarbamylase (OTC) deficiency, a genetic disorder that can lead to high levels of ammonia in the blood and severe neurological damage.
The Fast Track program is intended to speed up the development and review of drugs that treat serious conditions and fill an unmet medical need. Therapies with this designation may benefit from more frequent interactions with the FDA and could be eligible for accelerated approval and priority review if they meet certain criteria. ECUR-506 has also received Rare Pediatric Disease and Orphan Drug designations from the FDA, as well as Orphan designation from the European Commission.
Michael Amoroso, CEO of Precision BioSciences, expressed that the FDA's Fast Track designation highlights the critical need for new treatments for neonatal onset OTC deficiency and the potential of the gene insertion approach used in ECUR-506.
The OTC-HOPE study, a Phase 1/2 clinical trial for ECUR-506, is currently enrolling newborn males with OTC deficiency in the United Kingdom, with additional trial sites in the United States and Australia expected to begin enrollment later this year. The study's primary focus is on the safety and tolerability of a single intravenous dose of ECUR-506, with secondary objectives including the assessment of pharmacokinetics and efficacy.
OTC deficiency is a urea cycle disorder that can cause excessive ammonia accumulation in the blood, leading to life-threatening symptoms. The current standard of care includes liver transplantation and medical therapies that do not correct the underlying genetic defect.
Precision BioSciences' proprietary ARCUS platform is used to develop in vivo gene editing therapies, including ECUR-506, which involves the insertion of a functional OTC gene into a specific genetic locus using an ARCUS nuclease.
iECURE is a clinical-stage gene editing company that has licensed the ARCUS nuclease for ECUR-506 from Precision BioSciences. The company's approach to gene editing focuses on in vivo gene insertion for the treatment of liver disorders with significant unmet needs.
This news is based on a press release statement from Precision BioSciences, Inc.
InvestingPro Insights
Precision BioSciences, Inc. (NASDAQ:DTIL) is at the forefront of gene editing technology with its ARCUS platform, and the recent Fast Track designation for its gene therapy candidate ECUR-506 is a testament to the company's potential to address critical unmet medical needs. As investors digest this news, here are some key insights from InvestingPro to consider:
Precision BioSciences holds a market capitalization of $73.73 million, which reflects investor sentiment towards its growth prospects and current financial health. Despite the company's promising technology, analysts have raised concerns about its financial trajectory. An "InvestingPro Tip" highlights that the company is quickly burning through cash, which can be a red flag for investors looking for sustainable growth. Furthermore, the company's stock price has been quite volatile, with a 1-month price total return of -25.3%, underscoring the risks associated with investing in this biotech firm.
The financial data underscores these concerns, with a negative P/E ratio of -0.66, indicating that the company is not currently profitable. Additionally, the revenue growth appears inconsistent, with a substantial increase of 94.15% in the last twelve months as of Q1 2023, followed by a quarterly decline of -33.59% in Q1 2023. This could suggest that while the company has had periods of rapid revenue expansion, it may be facing challenges in maintaining that growth rate.
Investors interested in a deeper dive into Precision BioSciences' financials and future outlook can find more "InvestingPro Tips" at https://www.investing.com/pro/DTIL. There are 10 additional tips available, providing a comprehensive analysis of the company's financial health and market position. For those considering an InvestingPro subscription, use the coupon code PRONEWS24 to receive an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
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