Kolon Life Science building overview [사진=코오롱생명과학 제공]
Kolon Life Science, renowned for developing ‘Invossa’, the world’s first osteoarthritis gene therapy and Korea’s first gene therapy, has expressed interest in developing gene therapy for degenerative brain diseases.
According to the Korean Intellectual Property Office on the 22nd, Kolon Life Science is in the process of registering a patent for a composition aimed at preventing or treating degenerative brain diseases. While the patent application was filed in 2022, Kolon Life Science recently initiated the registration process by submitting a request for a prior art search to the Korean Intellectual Property Office.
The composition Kolon Life Science is developing involves inserting two or more of the three protein genes – interleukin 10 (IL-10), glial cell-derived trophic factor (GDNF), and glutamate decarboxylase (GAD) – into a viral vector. This patent pertains to a gene therapy that delivers these proteins, including IL-10, GDNF, and GAD, to target cells within the body following injecting the viral vector, ultimately achieving a therapeutic effect.
The three therapeutic genes, IL-10, GDNF, and GAD, are the core components of ‘KLS-2031’, a neuropathic pain gene therapy currently under development by Kolon Life Science. ‘KLS-2031’ is a CNS treatment designed to block pain signals reaching the brain. As it directly targets the central nervous system, Kolon Life Science conducted research to explore the potential of these components in treating degenerative brain diseases.
The application specification submitted by the company reveals that Kolon Life Science conducted both animal experiments and in vitro experiments using two approaches: a combination of human-derived IL-10 and GDNF, and a combined therapy involving IL-10, GDNF, and GAD.
In animal experiments, the company utilized a mouse model exhibiting Parkinson’s disease to assess the protective effect on dopaminergic neurons. This research is based on the understanding that degenerative brain diseases can cause alterations in the signaling substances of dopaminergic neurons, ultimately leading to a decline in motor function.
Experimental results confirmed that the combined therapy using IL-10 and GDNF significantly reduced dopaminergic neuronal cell death and improved motor dysfunction compared to the placebo (saline) and individual therapies using IL-10 or GDNF. The positive effects on motor dysfunction were even maintained 5 weeks following drug administration, suggesting the potential for long-term benefits. These positive outcomes were similarly observed in the three-gene combination therapy involving IL-10, GDNF, and GAD.
Recognizing the potential impact of oxidative stress on nerve cell damage and disease progression in Parkinson’s disease patients, the Kolon Life Science research team conducted an in vitro experiment to investigate the effectiveness of IL-10, GDNF, and GAD combination therapy in inhibiting nerve cell death caused by oxidative stress.
The results revealed that simultaneous administration of IL-10 and GDNF protein cultures, or co-administration of IL-10, GDNF, and GAD65 protein cultures, effectively inhibited neuronal cell death.
The research team explained, “We discovered that not only the genes for (human-derived) IL-10 and GDNF, but also each respective protein, possess the ability to prevent or treat degenerative brain diseases.” They added, “Given their neuroprotective effect, regardless of the cause of neuronal death, the IL-10, GDNF, and GAD65 genes or proteins hold promise for treating various degenerative brain diseases, including Parkinson’s disease.”
This patent application gains further significance considering that all gene therapy drugs for which Kolon Life Science has registered patents thus far are included in the company’s new drug pipeline.
Kolon Life Science holds major gene therapy patents, including a patent for a composition for pain relief or treatment and a patent for a pharmaceutical composition for preventing or treating osteoarthritis. These patents are directly related to ‘KLS-2031’, a neuropathic pain gene therapy candidate substance, and ‘Invossa’, an osteoarthritis gene therapy drug.
Based on this activity, it’s reasonable to anticipate that the company may embark on full-scale development of gene therapy for degenerative brain diseases in the near future.
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Kolon Life Science Explores Gene Therapy for Degenerative Brain Diseases
Kolon Life Science, renowned for developing ‘Invossa,’ the world’s first osteoarthritis gene therapy and Korea’s first gene therapy, has set its sights on developing gene therapy for degenerative brain diseases. This groundbreaking move signifies a potential leap forward in treating debilitating conditions like Parkinson’s disease, Alzheimer’s disease, and other neurodegenerative disorders.
Patent Application and Composition
The Korean Intellectual Property Office revealed that Kolon Life Science is in the process of registering a patent for a ‘composition for preventing or treating degenerative brain diseases.’ The application, filed in 2022, involves a composition containing two or more of the following protein genes:
- Interleukin 10 (IL-10)
- Glial cell-derived trophic factor (GDNF)
- Glutamate decarboxylase (GAD)
These genes are inserted into a viral vector, which, when injected into the body, produces these proteins in target cells, leading to a therapeutic effect.
KLS-2031 and Neuropathic Pain
The three therapeutic genes – IL-10, GDNF, and GAD – are the key components of ‘KLS-2031’, a neuropathic pain gene therapy currently under development by Kolon Life Science. ‘KLS-2031’ targets the central nervous system (CNS) to block pain signals. Leveraging its experience with ‘KLS-2031’, Kolon Life Science is exploring the potential of these genes in treating degenerative brain diseases.
Animal and In Vitro Experiments
Kolon Life Science has conducted extensive research involving animal and in vitro experiments to evaluate the efficacy of these genes in treating degenerative brain diseases:
Animal Experiments
Animal experiments on a mouse model with induced Parkinson’s disease demonstrated the protective effects of these genes on dopaminergic neurons. These neurons play a crucial role in movement and are affected in degenerative brain diseases.
- The combination therapy of IL-10 and GDNF significantly reduced dopaminergic neuronal cell death and improved motor dysfunction symptoms compared to placebo and single gene therapy.
- The positive effects on motor dysfunction were sustained for up to 5 weeks, suggesting the potential for long-term treatment.
- Similar results were observed with the three-gene combination therapy of IL-10, GDNF, and GAD.
In Vitro Experiments
Kolon Life Science also conducted in vitro experiments to assess the ability of these genes to protect once morest oxidative stress-induced neuronal cell death, a critical factor in Parkinson’s disease.
- The co-administration of IL-10 and GDNF proteins, as well as the combined administration of IL-10, GDNF, and GAD65 proteins, demonstrated a significant inhibition of neuronal cell death.
These experiments strongly suggest the potential of these genes in treating degenerative brain diseases.
Research Team Insights
The research team at Kolon Life Science emphasized the significance of their findings:
- “We have found that not only the genes for (human-derived) IL-10 and GDNF but also each protein has the effect of preventing or treating degenerative brain diseases.”
- “These genes and proteins exhibit neuroprotective effects regardless of the cause of neuronal death, making them potentially effective for various degenerative brain diseases, including Parkinson’s disease.”
Patent Portfolio and Future Development
This patent application adds to Kolon Life Science’s growing portfolio of gene therapy patents. The company holds patents for pain relief and osteoarthritis treatment, aligned with their development of ‘KLS-2031’ and ‘Invossa’, respectively.
This recent patent registration for degenerative brain disease treatment indicates Kolon Life Science’s commitment to expanding its gene therapy research and potentially initiating full-scale development of this promising therapeutic modality.
