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Company Agilis Biotherapeutics LLC
DescriptionUbiquitin protein ligase E3A (UBE3A) gene therapy
Molecular Target Ubiquitin protein ligase E3A (UBE3A) (E6AP)
Mechanism of Action 
Therapeutic ModalityGene therapy
Latest Stage of DevelopmentPreclinical
Standard Indication Neurology (unspecified)
Indication DetailsTreat Angelman syndrome
Regulatory Designation U.S. - Orphan Drug (Treat Angelman syndrome);
EU - Orphan Drug (Treat Angelman syndrome)

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