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HX101
- Development stage
- Unknown
- Lead developer
- Huaxia Shengsheng Pharmaceutical
- 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
- Intrathecal
01
Overview
HX101 is an antisense oligonucleotide (ASO) developed by Huaxia Shengsheng Pharmaceutical (Beijing) Co., Ltd. for the treatment of amyotrophic lateral sclerosis (ALS), specifically targeting patients with mutations in the superoxide dismutase 1 (SOD1) gene. The drug is designed to bind to SOD1 mRNA, facilitating its degradation and thereby reducing the levels of toxic, misfolded SOD1 protein aggregates that contribute to motor neuron degeneration. HX101 is administered via intrathecal injection and is currently undergoing Phase 1 clinical evaluation to assess its safety, tolerability, and pharmacokinetics in ALS patients.
02
Targets
SOD (Manganese Superoxide Dismutase)
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