Neu-001 for the treatment of amblyopia

CRMH
Overview

Centre for Regenerative Medicine and Health (CRMH) has developed a first-in-class small molecule drug Neu-001 for the treament of adult and children amblyopia

  • Neu-001 for the treatment of amblyopia
Problem addressed

Amblyopia ("lazy eye") affects 3–6% of the population worldwide. While early intervention through eye patching shows some success in young children, there are currently no effective pharmacological treatments available, particularly for adults. This represents a significant unmet medical need.

Innovation
  • A first-in-class small molecule therapeutic targeting GPCR in the visual cortex, representing a paradigm shift from traditional eye-focused treatments for amblyopia.
  • Novel mechanism of action enables the treatment of adult amblyopia, a condition previously considered untreatable due to limited brain plasticity.
  • This unique therapeutic strategy offers a potential pharmacological solution for both pediatric and adult patients, addressing a significant gap in current treatment options.
Key impact
  • It has a potential to provide the first pharmaceutical cure for amblyopia, dramatically improving treatment efficiency and outcomes compared to current standard of care.
  • Expands treatment opportunity to adult patients previously considered untreatable, addressing an unmet medical need for tens of millions globally.
  • It improves patient compliance and quality of life through convenient pharmaceutical administration, compared to traditional mechanical interventions.
  • It offers significant healthcare economic benefits by reducing long-term complications and improving treatment efficiency.
Application
  • Primary treatment for newly diagnosed amblyopia in children
  • Treatment for adult amblyopia patients previously considered untreatable

As the branch organization of the Chinese Academy of Sciences,  Hong Kong Institute of Science & Innovation (HKISI) is the first national research institution in Hong Kong SAR approved by the central government of China. Centre for Regenerative Medicine and Health (CRMH), the subsidiary of HKISI, is supported by the Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences. CRMH is funded by CAS and the InnoHK program from the Innovation and Technology Commission, Hong Kong SAR government. With its focus on stem cell therapy & genomic editing, developmental biology, and drug discovery, CRMH promotes the globalization of regenerative medicine research and achievements.

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