Pipeline & Programs
Pipeline
<u>Pipeline</u>

Advancing a portfolio of next-generation pediatric medicines designed to transform the lives of children with rare neurodevelopmental and neurodegenerative disorders.

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Stage Preclinical IND Enabling Safety/ESOE Pivotal
CLN-301: CLN3 BATTEN DISEASE

Strategic Overview: Targeting CLN3 Batten Disease with clinically validated AAV delivery mechanisms focusing on Neuronal Ceroid Lipofuscinosis Type 3.

Status: Advanced Clinical Candidate

Projection: Clinical Efficacy 2027

XGR-101: RETT SYNDROME (miREX)

Strategic Overview: Utilizing our proprietary miREX platform to modulate X-chromosome inactivation patterns. First-in-class micro-RNA regulated expression pipeline in a product.

Status: IND-Stage | Platform Technology

Projection: IND & FIH 2027; Clinical ESOE 2027

EFRALEX: NEURODEVELOPMENT & NEURODEGENERATION

Strategic Overview: Targeting EphA4 Receptor—’The Master-Switch’ in Neurodegeneration; uses in-licensed ThecaFlex-DRx™ CNS delivery.

Status: Preclinical

Platform: Neurodegenerative and Neurodevelopmental Disorders