Dr. Christopher Janson, the Principal Investigator on Myrtelle’s Canavan Disease gene therapy trial, addresses questions frequently asked by the families in relation to the disease and gene therapy In a clinical trial, what types of measurements can be taken to test how a gene therapy is working? Measurements of the safety and effectiveness of a…
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Cheryl Demos Fludas, Head of Clinical Operations at Myrtelle: Q: Choosing to decide to enroll oneself or a loved one (especially a child) in an experimental clinical trial can be overwhelming. Can you provide a brief overview of that process and what happens once a patient is identified, screened and enrolled? A: The process begins…
Read moreEight patients have been treated in the ongoing Phase 1/2 First-in-Human clinical trial with favorable safety and tolerability observed to date Assessments of the initial 3 patients reaching their six-month follow-up showed improvements on validated functional scales and increases in brain white matter and myelin content Encouraging efficacy and safety data support further development of…
Read morePlasmid and AAV Manufacturing Partnership to Advance Myrtelle’s Novel Gene Therapy For Monogenic Hearing Loss for Phase 1/2 Clinical Trials WAKEFIELD, MASSACHUSETTS AND COLUMBUS, OHIO October 3, 2022 – Myrtelle Inc., (Myrtelle), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and Forge Biologics, a gene therapy-focused contract development and…
Read moreWakefield, Mass., September 7th, 2022 – Myrtelle Inc., (“Myrtelle” or the “Company”), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the European Medicines Agency (EMA) has classified the Company’s lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease, as an Advanced Therapy Medicinal…
Read more1: Can you give us an overview of the step-by-step gene therapy manufacturing process? The manufacturing of gene therapy products is complex and time-consuming, entailing many sequential steps from the time a production process is started to the testing and release of the gene therapy material. At Myrtelle, to make our gene therapies we utilize…
Read moreAnalyses 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…
Read more1: 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…
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