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Pipeline

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

Indication
AI Discovery
Preclinical
Clinical planning
Phase 1/2a
Fragile X syndrome
Neurodevelopmental
Autosomal dominant polycystic kidney disease (ADPKD)
Renal
Neurofibromatosis type 1 - cutaneous neurofibroma (cNF)
Oncology
Neurofibromatosis type 1 - plexiform neurofibroma (pNF)
Oncology
Chronic pancreatitis
Inflammatory
Undisclosed disease
Renal
Friedreich’s ataxia
Neurodevelopmental
Facioscapulohumeral muscular dystrophy (FSHD)
Neuromuscular
Undisclosed disease
Liver
COVID-19
-
Angelman syndrome
Neurodevelopmental
Undisclosed disease
Bone
Autosomal recessive polycystic kidney disease (ARPKD)
Renal
Undisclosed disease
Renal
Undisclosed disease
Neuromuscular
6 x early undisclosed programmes
Multiple

Meet some of our team