Translate Bio Presents Preclinical Data Supporting MRT5201 for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting
08 avr. 2019 16h05 HE
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Translate Bio, Inc.
-- MRT5201 was efficiently delivered to the liver of a mouse model of OTC deficiency -- -- Preclinical data demonstrated a single IV dose of MRT5201 was sufficient to protect against hyperammonemia...
Translate Bio Provides Updates on Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency Programs
27 févr. 2019 16h30 HE
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Translate Bio, Inc.
-- Initiated multiple-ascending dose portion of the Phase 1/2 clinical trial of MRT5005 in patients with cystic fibrosis -- -- Received FDA communication related to previously announced IND clinical...