Neuromyon, a subsidiary of Helixmith, conducts research and development of gene/cell therapy drug for neurodegenerative diseases. AAV is a gene carrier (vector) useful for transferring genes into the body and known to be safer among several viral vectors. In addition, AAV has higher gene transfer efficiency and is capable of cell/tissue specific transfer, than other is highlighted as a vector of gene therapy for various diseases.
‘NM301’ is a gene therapy drug based on AAV vector expressing HGF. Through injection into the intrathecal cavity, it has potential of delay occurrence of symptoms of degenerative disease and promote regeneration of damaged tissue and the nerves and muscles and improving pathologic environment through anti-inflammation activity. Its target indications include amyotrophic lateral sclerosis (ALS). Currently its preclinical study is in progress and aims an IND submission is targeted for 2023.
‘NM101’ is a gene therapy drug based on plasmid DNA expressing IGF-1. Its proposed MOA is the degeneration of tissue via regenerative activity by intramuscular injection. Target indications are peripheral neuropathy and pain. Preclinical studies are in program with a target of an IND submission in 2022.