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Indication
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Approach
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Target
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Research
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Lead selection
& Optimization |
IND Enabling
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Clinical
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Worldwide Rights
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Neuromuscular
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DUCHENNE
MUSCULAR DYSTROPHY Duchenne muscular dystrophy (DMD) is an X-linked, progressive muscle wasting disease caused by mutations in the DMD gene that result in either truncated non-functional dystrophin protein, or little to no protein. Our approach leverages exon skipping to restore the reading-frame of the RNA to produce a near-full length copy of the dystrophin protein.
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Exon Skipping
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Single and Multi-
Exon Skipping |
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Myotonic Dystrophy type 1
Myotonic Dystrophy type 1 is a genetic
neuromuscular disorder caused by a mutation in the DMPK gene, resulting in a trinucleotide (CUG) repeat expansion in the expressed RNA. Our DM1 program targets and destroys the toxic CUG repeats. |
Destruction /
Blocking |
CUG repeat
expansion |
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NeuroDegeneration
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Huntington’s Disease
Huntington’s Disease is a genetic
neurodegenerative disorder caused by a mutation in the HTT gene, resulting in a trinucleotide (CAG) repeat expansion in the expressed RNA. Our Huntington’s Disease program targets and destroys the excessive CAG repeats. |
Destruction /
Blocking |
CAG repeat
expansion |
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Spinocerebel-
lar Ataxia 1 Spinocerebellar Ataxia Type 1 (SCA1) is a genetic
neurodegenerative disorder caused by a mutation in the ATXN1 gene, resulting in a trinucleotide (CAG) repeat expansion in the expressed RNA. Our SCA1 program targets and destroys the excessive CAG repeats. |
Destruction /
Blocking |
CAG repeat
expansion |
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Amyotrophic Lateral Sclerosis (C9orf72)
C9orf72-related Amyotrophic Lateral Sclerosis
(ALS) is a genetic motor neuron disorder caused by a mutation in the C9orf72 gene, resulting in hexanucleotide (G4C2 and C4G2) repeat expansions. Our C9orf72-ALS program targets and destroys the hexanucleotide repeats. |
Destruction
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Multi-target
repeat expansion |
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