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SG006
- Development stage
- Preclinical
- Lead developer
- SineuGene Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Inhalation
01
Overview
SG006 is an antisense oligonucleotide (ASO) developed by Shenji Changhua (also known as SineuGene Therapeutics) for the treatment of spinocerebellar ataxia type 3 (SCA3), or Machado-Joseph disease. SCA3 is a progressive neurodegenerative disorder caused by a CAG repeat expansion in the ATXN3 gene, resulting in the accumulation of toxic mutant ataxin-3 protein. SG006 is designed to selectively target and reduce the levels of ATXN3 mRNA transcripts, thereby decreasing the production of the disease-causing protein. As of early 2024, the program is in the IND-enabling and Investigator-Initiated Trial (IIT) stages of development.
02
Targets
ATXN3 (Ataxin-3)
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