Delivering Parkin therapeutics to significant patient populations in need of transformative outcomes.

GENE THERAPY PIPELINE

 

INITIAL INDICATION: PARKIN-PD

  • Most prevalent autosomal recessive PD mutation with 100% penetrance.

  • Leading cause of Young Onset PD.

  • Mutation results in loss-of-function.

 

NOVEL PROACTIVE MECHANISM

  • Protects and restores dopamine neuron health.

  • One-and-done treatment to reverse the course of PD.

 

EFFICIENT DRUG DEVELOPMENT PATHWAY

  • IND-enabling stage.

  • Manufacturing process established, Tox Lot generated.

  • Clinically validated AAV vector with a well-established safety profile.

  • Proven safe and efficient delivery to directly target multiple cell types.

  • Innovative clinical biomarkers.

 

THE RIGHT DRUG FOR THE RIGHT PATIENT

Data validation by PARKIN program paves way for future Idiopathic PD programs.