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FDA fast-track boost lifts iECURE gene therapy toward hope for newborns

PHILADELPHIA, PA — A rare-disease gene therapy developed in Philadelphia has cleared a major regulatory hurdle, positioning ...
Morningstar

Arcturus Therapeutics Announces Positive Interim Phase 2 Multiple Dose Data for Ornithine Transcarbamylase (OTC) Deficiency Program

Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a commercial messenger RNA medicines company focused on the development of infectious disease vaccines and opportunities ...
Nasdaq

Arcturus Therapeutics Provides Updates for Ornithine Transcarbamylase (OTC) Deficiency and Cystic Fibrosis (CF) Programs

ARCT-810 Phase 2 study completed enrollment at 0.3 mg/kg dose level in Europe with data expected later this year ARCT-810 to expand Phase 2 clinical program in the U.S. to enroll patients with more ...
Business Wire

Preclinical Data from iECURE’s GTP-506 Demonstrates Potential for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency

PHILADELPHIA--(BUSINESS WIRE)--iECURE, a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver ...
Yahoo Finance

iECURE Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ECUR-506 for Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency

PHILADELPHIA, January 07, 2026--(BUSINESS WIRE)--iECURE, Inc., a genome editing company focused on the development of variant-agnostic in vivo targeted gene insertion therapies for the treatment of ...
Morningstar

iECURE to Present Initial OTC-HOPE Clinical Data of ECUR-506 in Ornithine Transcarbamylase Deficiency at Upcoming Medical Meetings

iECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion therapies for the treatment of liver disorders with significant unmet need, today announced ...
Seeking Alpha

Precision BioSciences Announces Complete Clinical Response in First Infant Dosed by Partner iECURE in Ongoing Phase 1/2 Clinical Trial in Ornithine Transcarbamylase (OTC ...

- Treatment with ECUR-506 resulted in a complete clinical response from three months post exposure to the end of study (six months post exposure) - ECUR-506 was generally well tolerated with no ...

Dad who donated liver to son with rare condition launches fundraiser to build him therapy room

Archie was born with a rare condition and was treated in Glasgow Children's Hospital before being transferred to London to ...
Nasdaq

Arcturus Therapeutics Receives U.S. FDA Fast Track Designation for ARCT-810, mRNA Therapeutic Candidate for Ornithine Transcarbamylase Deficiency

SAN DIEGO--(BUSINESS WIRE)-- Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of ...
Business Wire

Arcturus Therapeutics Provides Updates for Ornithine Transcarbamylase (OTC) Deficiency and Cystic Fibrosis (CF) Programs

SAN DIEGO--(BUSINESS WIRE)--Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global messenger RNA medicines company focused on the development of infectious disease ...
Seeking Alpha

Arcturus Therapeutics Announces Positive Interim Phase 2 Multiple Dose Data for Ornithine Transcarbamylase (OTC) Deficiency Program

Virtual KOL Presentation at 12:00 p.m. ET Today SAN DIEGO--(BUSINESS WIRE)-- Arcturus Therapeutics Holdings Inc. (ARCT), a commercial messenger RNA medicines company focused on the development of ...