GeneTx Biotherapeutics was setup as a subsidiary of the Foundation for Angelman Syndrome Therapeutics for the development and commercialization of an investigational antisense drug, GTX-102, for the treatment of Angelman syndrome (AS). Angelman syndrome (AS) is a rare genetic disorder that affects approximately one in 15,000 live births. AS is caused by a loss of function of the maternally inherited UBE3A gene. Symptoms of AS ...
GeneTx Biotherapeutics was setup as a subsidiary of the Foundation for Angelman Syndrome Therapeutics for the development and commercialization of an investigational antisense drug, GTX-102, for the treatment of Angelman syndrome (AS). Angelman syndrome (AS) is a rare genetic disorder that affects approximately one in 15,000 live births. AS is caused by a loss of function of the maternally inherited UBE3A gene. Symptoms of AS include developmental delay, impaired motor function, loss of speech, and epilepsy. GeneTx Biotherapeutics’ GTX-102 is designed to compensate for the deficiencies that result from the underlying genetic causes.
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