KGI

Keck Graduate Institute (KGI) is exploring an investigational Cell Penetrating Peptide UBE3A Enzyme Replacement Therapy (CPP-ERT) approach to treating Angelman syndrome.

Discovery & DevPre-clinicalPhase 1Phase 2Phase 3To Patients

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About the Program

A therapeutic approach replacing the missing or nonfunctional UBE3A protein in the brain.

FAST Pillar: Pillar 1: Replace Mom's UBE3A
Collaborators: Barbara Bailus

Media

Backstage Passes to the Brain for Therapeutic Delivery

Pillar 1Replace Mom’s UBE3A

Pillar 2Turn on Dad’s UBE3A

Pillar 3Explore Adjacent Genes

Pillar 4Target Downstream Pathways

KGI

About the Program

Media

About Angelman Syndrome

Research

Pillar 1Replace Mom’s UBE3A

Pillar 2Turn on Dad’s UBE3A

Pillar 3Explore Adjacent Genes

Pillar 4Target Downstream Pathways

Trials & Studies

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News & Events

KGI

About the Program

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