Myrtelle Inc., (“Myrtelle” or the “Company”), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA), the healthcare regulatory body of the United Kingdom (UK), granted Innovative Licensing and Access Pathway (ILAP) designation to the…
MoreWakefield, 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…
MorePhase 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,…
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…
MoreMyrtelle Inc. has successfully completed dosing 8 patients with Canavan Disease in its Phase 1/2 clinical trial for the investigational gene therapy rAAV-Olig001-ASPA, demonstrating favorable safety and tolerability, along with significant improvements in functional scales and brain white matter content in early assessments.
MoreMyrtelle Inc. and Forge Biologics have announced a manufacturing partnership to advance Myrtelle's novel gene therapy for monogenic hearing loss, Myr-201, into clinical trials for patients with autosomal recessive deafness 8 (DFNB8).
MoreMyrtelle Inc. has received Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA) for its lead gene therapy product candidate, rAAV-Olig001-ASPA, aimed at treating Canavan disease, facilitating its regulatory pathway and development in the EU.
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
MoreMyrtelle Inc. has entered into a worldwide exclusive licensing agreement with Rescue Hearing Inc. to develop a novel gene therapy for DFNB8 genetic hearing loss, aiming to repair and restore damaged cells and promote neuron survival, offering potential hope for patients with this condition.
MoreMyrtelle Inc. has announced the successful administration of gene therapy in its Phase 1/2 clinical trial for Canavan disease, treating three children aged 3 to 5 years, with plans to expand the study to younger age groups based on recommendations from the Data Monitoring Committee.
MoreMyrtelle Inc. has entered into an exclusive worldwide licensing agreement with Pfizer Inc. for an investigational recombinant adenovirus (rAAV) gene therapy aimed at addressing Canavan disease (CD), a devastating childhood genetic neurological disorder.
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