Analyses of the first three patients at 6 months post-treatment showed increases in white matter and myelin and improvements on validated functional scales To date, 5 patients have been treated in the ongoing Phase 1/2 First-in-Human clinical trial with favorable safety and tolerability Encouraging early data support further development of rAAV-Olig001-ASPA as a potential therapeutic…
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1: Can you describe the central function of oligodendrocytes and myelin generation? Oligodendrocytes and neurons are types of brain cells. Neurons have a cell body that holds the nucleus (where the cell’s genes are located), dendrites that branch off from the cell body, and an axon that extends away from the cell body like a…
Read moreMyrtelle augments pipeline with gene therapy for monogenic hearing loss, designed to repair and restore damaged cells and promote neuron survival Recent groundbreaking proof-of-concept animal studies show recovery of auditory function Technology also presents potential for an exciting platform for future hearing restoration therapies WAKEFIELD, Mass.–(BUSINESS WIRE)–Myrtelle Inc. (“Myrtelle” or the “Company”), a clinical stage…
Read moreProgram further expands Myrtelle’s pipeline in white matter diseases leveraging proprietary vectors, constructs, capabilities and expertise to target oligodendrocytes and address the underlying cause of disorders involving myelin production. The gene therapy strategy uses a nonpathogenic recombinant adeno-associated virus (rAAV) as a vector to deliver a gene silencing microRNA (miRNA) directly to the central nervous…
Read moreNancy is grateful for her opportunity at Myrtelle, to help bring innovative therapies to patients with serious, incapacitating, and life-threatening genetic disorders. Nancy joined Myrtelle at its inception in 2021 and brings more than 25 years of experience in drug development. Nancy most recently served as Vice President, Head of Regulatory Affairs at Aruvant Sciences where she led the…
Read moreWAKEFIELD, Mass.–(BUSINESS WIRE)–Myrtelle Inc. (“Myrtelle” or the “Company”), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations for its lead clinical-stage gene therapy, rAAV-Olig001-ASPA, for the treatment of…
Read moreArmen Asatryan joined Myrtelle in 2021, shortly after its inception, and has over 20 years of combined experience in pharma/biotech, public health, and clini- cal medicine. Prior to joining the com- pany, Armen held positions of increasing responsibility at Abbott/AbbVie, AveXis/ Novartis, and most recently at AVROBIO, where he was Vice President of Clinical Development…
Read moreMyrtelle’s Phase 1/2 clinical trial introduces first-of-its-kind proprietary recombinant adeno-associated virus (rAAV) vector designed to enable targeting of oligodendrocytes
Read moreMyrtelle’s gene therapy represents a first-of-its kind breakthrough treatment that utilizes a proprietary recombinant adeno-associated virus (rAAV) vector enabling robust targeting of oligodendrocytes, critical for myelination and brain development
Read moreMyrtelle Inc. targets Canavan disease with an exclusive worldwide licensing agreement with Pfizer Inc. for an investigational rAAV gene therapy.
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