ORPHAGENEX
The company isdeveloping gene therapy for hereditary diseases..
Our focus is on rare neuromuscular diseases.
The core technology of the pipeline is gene transfer using AAV vectors.
Our focus is on rare neuromuscular diseases.
The core technology of the pipeline is gene transfer using AAV vectors.
OUR MISSION
Development and introduction of drugs for the treatment of neuromuscular diseases
Development
Preclinical
CT
Approved
PC
LGMD R2
GTDF-202
DYSF
Dysferlinopathy
LGMD R1
ORX-1C1
CAPN3
Calpainopathy
LGMD R9
ORX-1F1
FKRP
Dystroglycanopathy
SMA
DMD
ORX-1S1
SMN
DMD
Spinal Muscular
Atrophy
Atrophy
ORX-VEC
Duchenne Muscular
Dystrophy
Dystrophy
co-development
PC – Proof of concept
Preclinical – Preclinical study
CT – Clinical trial
MOSCOW, Prospect Vernatsky 96
MAIL@ORPHAGENEX.RU
© 2025 ORPHAGENEX