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AJ201_Oral Granules
SBMA, or Kennedy’s disease, is a rare X-linked neuromuscular disorder caused by CAG repeat expansion in the androgen receptor (AR) gene. The mutant AR protein drives muscle and neuron degeneration through cellular toxicity, oxidative stress, and neuroinflammation. SBMA affects ~1 in 40,000 males worldwide and currently has no approved treatment. AJ201 (JM17) is a novel investigational compound, currently is ready for Phase 3 pivotal study. shown to reduce mutant AR toxicity and improve motor function in preclinical SBMA models. It promotes degradation of pathogenic AR protein and induces antioxidant enzymes, proteasome subunits, and heat shock proteins, potentially slowing disease progression.
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- #polyQ diseases
- #Kennedy's Disease
- #Histone Deacetylase 6
- #Fibrotic diseases
- #Peripheral Neuropathy
- #Keloid
- #Rare Diseases
- #Neurology
- #Dermatology
- #Small molecules
- Established 2014
- Company AnnJi Pharmaceutical Co., Ltd.
- Telephone 02-23655677
- Email info@ajpharm.com
- Address 16F.-6, No. 508, Sec. 7, Zhongxiao E. Rd., Nangang Dist., Taipei City 115011 , Taiwan
AnnJi is a clinical-stage drug company dedicated to the development of innovative small molecules. Our pipeline targets therapeutic areas with robust market growth potential such as neurology and dermatology diseases. We are committed to advancing innovative therapies that address significant unmet medical needs, including treatment for rare diseases, like Kennedy’s Disease (SBMA).