Gene therapy for hereditary diseases
Genotarget develops gene therapeutic drugs for orphan neuromuscular diseases. The pipeline is based on the gene transfer technology using AAV vectors
Pipeline
research program
GT DF 100
LGMD R2 (dysferlinopahty, Miyoshi myopathy)
DYSF gene
DYSF gene
GT CN 100
LDMD R1 (calpainopathy)
CAPN3 gene
CAPN3 gene
GT FP 100
LGMD R9
FKRP gene
FKRP gene
Development
Preclinical
studies
studies
Clinical
trials
trials
Approved
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Contacts
mail@genotarget.com
