1. Gene Therapy Products

AAV

Focuses on research and
development of innovative gene
therapy for neuromuscular diseases.​

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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.

Overview​

NM101

Pain relief
Nerve regeneration

  1. 01Administration route and proposed of action​
    • Transfer to the muscles and peripheral nerves via muscular injection​
    • Inhibition of peripheral nerve degeneration and promotion of regeneration ​
    • Allows fundamental treatment by inducing tissue reconstruction​
  2. 02 Substance Characteristics​
    • Plasmid DNA to express IGF-1​
    • Designed to express main isoforms of IGF-1 simultaneously ​
    • Applicable to neurodegenerative disease​
  • Treatable Symptom 01

    Peripheral Neuropathy​

  • Treatable Symptom 02

    Neuropathic Pain

NM301

improvement of muscular function
Neuro-protection

  1. 01Administration route and mechanism of action​
    • Transfer to central nervous system by intrathecal administration ​
    • Inhibit apoptosis and induce tissue regeneration​
    • provide fundamental treatment with tissue reconstruction ​
  2. 02Substance Characteristics​
    • AAV vector expressing HGF isoforms
    • Optimized protein expression and production efficiency
    • Applicable to various neurodegenerative diseases​
  • Treatable Symptom 01

    Amyotrophic Lateral Sclerosis(ALS)

  • Treatable Symptom 02

    Other Degenerative diseases

Status of clinical trials

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Indication Pre-clinical Phase 1 Phase 2 Phase 3
Plasmid
DNA
NM101 Peripheral Neuropathy 2022
AAV NM301 ALS 2023
AAV NM302 Undisclosed