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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.
Posted In: DTIL
