Drug intelligence / Profile preview

SPI-5557

Development stage
Preclinical
Lead developer
Sound
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Local Injection
01

Overview

SPI-5557 is a preclinical **small interfering RNA** regenerative hearing-loss candidate being developed by Sound for **severe to profoundly impaired hearing loss**. It is described as a **locally injected siRNA** designed to inhibit **cyclin-dependent kinase inhibitor 1B**, also known as **p27** or **Kip1**, with the goal of driving cochlear supporting cells back into the cell cycle and promoting regeneration of supporting cells and sensory hair cells in the cochlea. The program represents a local RNA-based inner-ear regenerative approach intended to restore hearing in patients with severe to profound impairment; based on the provided pipeline information, it remains **preclinical** and **IND not yet filed**.

02

Targets

CDKN1B (Transmembrane emp24 domain-containing protein 7)

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