A PIPELINE TO TREAT
SEVERE GENETIC DISEASES
Locanabio is developing a portfolio of RNA-targeted gene therapies to severe neurodegenerative, neuromuscular and retinal diseases.
A PIPELINE TO TREAT
SEVERE GENETIC DISEASES
Locanabio is developing a portfolio of RNA-targeted gene therapies to severe neurodegenerative, neuromuscular and retinal diseases.

Indication
Approach
Target
Research
Lead selection
& Optimization
IND Enabling
Clinical
Worldwide Rights
Neuromuscular
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

DUCHENNE
MUSCULAR
DYSTROPHY

Splicing
Single and Multi-
Exon Skipping

NeuroDegeneration
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

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

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
Multi-target
repeat expansion

Ophthalmology
Retinal Disease
Multiple
Undisclosed