Disease Focus: Epilepsy

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Phase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

Therapeutic Candidate or Device NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells. Indication Focal epilepsy; drug-resistant mesial temporal lobe epilepsy. Therapeutic Mechanism NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells (hESCs) for the prospective treatment of drug-resistant focal epilepsy (MTLE). NRTX-1001 is delivered as a […]

Development of a Gene Therapy for the Treatment of WWOX related epileptic encephalopathy (WOREE)

Therapeutic Candidate or Device MZ-9138 is an AAV9 gene therapy Indication WWOX-related epileptic encephalopathy Therapeutic Mechanism MZ-9138 delivered to the brain may transduce neurons to create functioning WWOX protein and ultimately improve the phenotype of patients with WOREE Unmet Medical Need WOREE is a severe epileptic disorder resulting in dramatically shortened survival of patients. There […]