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Myrtelle Inc., a clinical stage gene therapy company, has 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 (CD). EMA OD designation's aim is to encourage the development of treatments for life-threatening rare conditions by providing incentives such as protocol assistance, reduced fees for regulatory activities, and 10 years of market exclusivity in the EU upon drug approval. rAAV-Olig001-ASPA is a novel vector from a class of recombinant AAVs (rAAVs) that directly target oligodendrocytes, the brain cells affected in CD which are responsible for producing myelin – the insulating material that enables proper neuronal function. In CD, the production of myelin is impaired due to a mutation in the Aspartoacylase gene (ASPA) that encodes the enzyme Aspartoacylase (ASPA). The deficiency of ASPA enzyme results in multiple biochemical and anatomic changes, including an inability to metabolize N-Acetylaspartate (NAA), a neurochemical that is abundant in the brain and plays an important role in myelin synthesis and brain bioenergetics. The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function in these cells and thereby improve the metabolism of NAA and myelination in patients with CD. Other designations In addition to EMA’s orphan designation, rAAV-Olig001-ASPA also received EMA’s Advanced Therapy Medicinal Product Classification (ATMP) and US Orphan Drug, Rare Pediatric Disease, and Fast Track designations by the FDA. "The designation by the EMA of rAAV-Olig001-ASPA as an Orphan Drug provides important benefits in the development of this innovative therapy for patients with Canavan disease who currently have significant unmet clinical needs," said Nancy Barone Kribbs, senior vice president of global regulatory affairs at Myrtelle. About Myrtelle Myrtelle is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative and genetic hearing loss diseases. About Canavan disease Canavan disease is a fatal childhood genetic brain disease in which mutations in the aspartoacylase gene (ASPA) prevent the normal expression of aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD, and only palliative treatments are available.

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EMA gives Myrtelle’s Canavan disease gene therapy candidate OD designation

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