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Pivotal safety studies for AMT?260, a novel AAV9?dual microRNA?based vector targeting GRIK2 for the treatment of temporal lobe epilepsy () - Oct 15, 2023 - Abstract #ESGCT2023ESGCT_742;
AMT?260, an AAV9 vector coding for 2 microRNAs was designed to knockdown GluK2 expression by specifically targeting GRIK2 mRNA.AMT?260 administered bilaterally into the hippocampus of a mouse model for TLE (pilocarpine) has already demonstrated efficacy in seizure reductions and improvement in general animal health...microRNA expression was strongly correlated with GRIK2 mRNA knockdown, achieving more than 95% reduction at the highest doses. Safety and vector pharmacokinetics data from mouse and NHP studies show a promising safety profile for further clinical development.
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Preclinical Proof-of-concept of a Novel AAV9-microRNA based Targeting of GRIK2 for the Treatment of Temporal Lobe Epilepsy (Both in-person and online) - Mar 12, 2023 - Abstract #AAN2023AAN_4340;
Convection enhanced, MRI guided intrahippocampal delivery in NHP resulted in local high copies of vector and miRNA expression with substantial knockdown of GRIK2, but with no adverse findings and limited peripheral exposure to the vector. Conclusions AMT-260 shows an excellent preclinical and safety profile and will proceed into clinical testing to treat patients with refractory mesial temporal lobe epilepsy.
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