Lep001 – gene therapy for the treatment of congenital lipodystrophy

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Development of an off-the-shelf iPSC derived CAR T cell therapy for the treatment of solid tumors

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Development of an AAV Epigenetic Gene Therapy for Gain-of-Function SCN9A Disorders and Chronic Pain

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Evaluation of an ex vivo lentiviral gene therapy for the treatment of Angelman syndrome

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Genetic Therapy Targeting mHTT mRNA and Somatic Expansion to Treat Huntington’s Disease

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A Novel Gene Therapy to Target Glioblastoma via Custom-Engineered Adenovirus-Associated Viral Vectors

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Parkin Gene Therapy for Parkinson’s Disease

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A novel non-genotoxic antibody conditioning therapeutic to expand access to and safety of HSCT and CD34+ gene therapies

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Hematopoietic Stem Cell Gene Therapy for Alpha Thalassemia

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Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.

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Translational Candidate Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy. Area of Impact Non-destructive treatment option for drug-resistant focal epilepsy…

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