Innoputix, Alzheimer’s disease gene therapy research published in international journal

Announcement of joint research results with Professor Sang-Hoon Lee’s team at Hanyang University School of Medicine

Innoputix announced on the 2nd that “the research results that revealed the non-clinical efficacy and various mechanisms of action of the new Alzheimer’s disease biologic under development were published in the international scientific journal ‘Molecular Psychiatry’.”

The company is developing new drugs for Alzheimer’s disease gene therapy using Adeno-Associated Virus (AAV). This treatment candidate has been selected as a non-clinical project by the Dementia Overcoming Research and Development Project Group (supported by the Ministry of Science and Technology and the Ministry of Health and Welfare), and is receiving a national research grant of KRW 1.5 billion from May this year to the end of 2024.

According to the published paper, when the AAV-using therapeutic genes Nurr1 and Foxa2 were delivered and expressed to the hippocampus and lateral ventricle of a chronic animal model of Alzheimer’s disease (3XTg mice) and an acute model (5XFAD mice), cognition and memory significantly increased for a long period of time. seemed In addition, it not only removed amyloid beta and tau proteins, which are pathological substances accumulated in the brain, but also confirmed the activation of immune cells and the inhibitory effect of neuroinflammation. It was also shown to promote the secretion of neurotrophic factors and the restoration of neurotransmission synapses.

This research was led by Professor Sang-Hoon Lee (Department of Biochemistry and Molecular Biology) at Hanyang University Medical School, who has been studying neurodegenerative diseases for decades. Professor Lee said, “The developed gene therapy was developed as a modality for direct injection into the patient’s brain, so it can be expected to have therapeutic efficacy without worrying regarding the drug’s brain-vascular barrier (BBB) ​​permeability. long-term effects can be expected.”

Kim Tae-gyun, CEO of Innoputix, predicted, “AAV gene therapy will be developed as a new concept advanced biopharmaceutical that fundamentally improves the treatment environment in the patient’s brain by using the various efficacy of therapeutic genes and the advantages of high-efficiency expression vectors.”

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