MEDICAL EXPLAINER
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EnglishVoice Age
Young Adult (18-35)Accents
North American (General) North American (US New York, New Jersey, Bronx, Brooklyn) North American (US West Coast - California, Portland)Transcript
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Cln one disease or infantile Batten disease is a progressive, fatal neurodegenerative disease with early childhood onset. Impacting approximately one in 138,000 live births worldwide. It is caused by loss of function mutations in the Cln one gene, which codes for an enzyme known as P P. T. One. There are no approved disease modifying therapies for its treatment. But today we announced the start of clinical development of Tasia 1 18 are a A V nine gene replacement therapy for Cln one. The clinical study will take place at Queen's University in Ontario Canada under the direction of Dr Jagdish Bwalya. We believe that introducing a functional cln one gene with Tasha 1 18 offers a potentially effective and therapeutic approach that addresses the root cause of this devastating disease. By allowing the expression of healthy PPT one enzyme based on natural history studies. We believe that PPT one enzyme activity of 5% or greater will normalize survival and improve symptoms. Preliminary clinical safety and PPT one enzyme activity data is expected in the first half of 2022. We are hopeful that keisha 1 18 will prove helpful to patients with Cln one. And look forward to reading out data next year
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