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Our approach
B.I.R.C. technology
Pipeline
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Our approach
B.I.R.C. technology
Pipeline
About
Pipeline
We’re committed to delivering novel treatments to rare disease patients
Meet some of our team
Dr Anthony Hall
Chief Medical Officer
Dr Anthony Hall
Chief Medical Officer
Dr Anthony Hall
Chief Medical Officer
Speak to us
Oncology
NF1: cutaneous neurofibroma
NF1: cutaneous neurofibroma
Neuro-developmental
Fragile X Syndrome
Angelman Syndrome
Oncology
Autosomal Dominant Polycystic Kidney Disease
Autosomal Recessive Polycystic Kidney Disease
Autosomal Dominant Polycystic Liver Disease
Oncology
Myotonic Dystrophy type-1
Spinocerebellar Ataxia
Pseudoachondroplasia
Oncology
Leber Hereditary Optic Neuropathy
Autosomal Dominant Optic Atrophy
Disease Area
Indication
AI Discovery
Preclinical
Clinical planning
Phase 1/2a
Oncology
NF1: cutaneous neurofibroma
NF1: plexiform neurofibroma
NF1: cutaneous neurofibroma
NF1: cutaneous neurofibroma
NF1: cutaneous neurofibroma
NF1: cutaneous neurofibroma
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