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BA-43407
- Development stage
- Unknown
- Lead developer
- BioAxone BioSciences
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
BA-43407 is a self-delivering RNA interference (sdRNA) therapeutic developed by BioAxone BioSciences. It is designed to inhibit the expression of PTEN, a protein that acts as an intrinsic barrier to axon regeneration in the central nervous system. By knocking down PTEN expression in adult neurons, BA-43407 aims to stimulate robust regenerative axon growth after spinal cord injury. The drug leverages RNA interference technology for targeted gene silencing and represents a novel approach for promoting neural repair following CNS trauma[1][2].
02
Targets
PTEN messenger RNA
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