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Pipeline

We’re committed to delivering novel treatments to rare disease patients

Disease Area
Indication
AI Discovery
Preclinical
Clinical planning
Phase 1/2a
Oncology
NF1: cutaneous neurofibroma
NF1: plexiform neurofibroma
Neuro-developmental
Fragile X Syndrome
Angelman Syndrome
Renal/Liver
Autosomal Dominant Polycystic Kidney Disease
Autosomal Recessive Polycystic Kidney Disease
Autosomal Dominant Polycystic Liver Disease
Neuromuscular
Myotonic Dystrophy type-1
Spinocerebellar Ataxia
Pseudoachondroplasia
Ocular
Leber Hereditary Optic Neuropathy
Autosomal Dominant Optic Atrophy

Meet some of our team