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Find Clinical Drug Pipeline Developments & Deals by GeneTx Biotherapeutics
GTX-102 an antisense oligonucleotide delivered via intrathecal administration and designed to target and inhibit expression of UBE3A-AS. Nonclinical studies show that GTX-102 reduces levels of UBE3A-AS and reactivates expression of paternal UBE3A allele in neurons of CNS.
GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx. GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration.
Phase 1/2 Clinical Study of GTX-102 that inhibit expression of UBE3A-AS, showed no treatment-related serious adverse events to lower extremity weakness observed in the patients, and initial assessments have shown early signs of clinical activity.
GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and designed to target and inhibit expression of UBE3A-AS used in pediatric patients with Angelman syndrome.
The phase 1/2 study evaluates the safety, tolerability, and plasma and cerebrospinal fluid concentrations of GTX-102 in pediatric patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.
At the highest doses, all five patients experienced a serious adverse event (SAE) of lower extremity weakness believed to be related to local inflammation due to GTX-102. Following these events, the companies paused enrollment and dosing.
The Phase 1/2 open-label, multiple-dose, dose-escalating study will evaluate the safety, tolerability, and plasma and cerebrospinal fluid (CSF) concentrations of GTX-102.
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