Wakefield, Mass., January 11th, 2023 – Myrtelle Inc.,(“Myrtelle”) a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and rAAVen Therapeutics, (“rAAVen”) an AAV engineering company focused on generating innovative vectors for the next generation of gene therapies, today announced a partnership to develop novel recombinant adeno-associated virus (“rAAV”) vectors to…
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Phase 1/2 clinical study is evaluating Myrtelle’s first-of-its-kind proprietary adeno-associated virus (rAAV) vector designed to enable targeting of oligodendrocytes Eight patients have been treated in the ongoing Phase 1/2 First-in-Human clinical trial with favorable findings observed to date Assessments of all treated patients through three-months of follow-up show improvements in neuroimaging and functional scales Wakefield,…
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 European Medicines Agency (EMA) has granted orphan drug (OD) designation for rAAV-Olig001-ASPA, the company’s lead gene therapy product candidate for the treatment of Canavan disease. The OD application is…
Read moreDr. 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…
Read moreCheryl 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…
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