Pipeline

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

Exploratory programs:

Alport Syndrome

Alpha-1 Antitrypsin Deficiency

Facioscapulohumeral Muscular Dystrophy

Pitt Hopkins

Meet some of our team

Dr Anthony Hall

Chief Medical Officer

Dr Mark Youssef

Global Head of Business Development and Licensing

(Pharmaceutical & biotech groups)

Dr Bruce Bloom

Chief Collaboration Officer

(Patient & academic groups)