Sangamo Therapeutics Gains FDA Approval for ST-503 IND in Idiopathic Small Fibre Neuropathy

Sangamo Therapeutics Gains FDA Approval for ST-503 IND in Idiopathic Small Fibre Neuropathy

By, Admin 21 November 2024

Overview

Sangamo Therapeutics has received clearance from the U.S. Food and Drug Administration (FDA) for its investigational new drug (IND) application for ST-503.

Neuropathic Pain & Its Symptoms

  • Neuropathic pain arises from various conditions affecting the central or peripheral nervous systems, including surgical trauma, spinal cord injuries, and hereditary syndromes. 
  • iSFN, a debilitating peripheral neuropathy, results in symptoms such as burning, stabbing, and lightning-like pain. 

Idiopathic Small Fibre Neuropathy: In US

Affecting approximately 43,000 individuals in the U.S., this condition highlights a significant unmet medical need, as current treatments, such as antidepressants, anticonvulsants, opioids, and topical therapies, offer limited long-term relief.

This experimental therapy is designed to treat idiopathic small fibre neuropathy (iSFN), a chronic form of neuropathic pain.

Aim of Phase 1/2 Trial

  • The Phase 1/2 clinical trial for ST-503 aims to assess its safety and effectiveness in addressing iSFN.
  • Unlike treatments targeting acute or sporadic pain, ST-503 focuses on chronic, intractable pain, which can significantly impact patients' quality of life. 
  • Sangamo plans to commence patient enrolment for the study in mid-2025.

About the Virus: ST-503

  • ST-503 is an epigenetic regulator employing an adeno-associated virus (AAV) vector to deliver a zinc finger repressor targeting the SCN9A gene, responsible for encoding the Nav1.7 sodium channel. 
  • This channel is critical for pain signalling and has been implicated in neuropathic pain conditions. 
  • Traditional approaches using small molecules to inhibit Nav1.7 have faced challenges due to difficulties in achieving selectivity among similar sodium channels, often resulting in off-target effects.

ST-503 During Preclinical Studies

  • In preclinical studies, ST-503 demonstrated promising results by selectively reducing Nav1.7 expressions in sensory neurons, significantly alleviating pain hypersensitivity in animal models. 
  • Administered via a single intrathecal injection, the therapy showed no off-target effects and was well tolerated in non-human primates.

If successful, Sangamo plans to explore ST-503’s potential application in treating other forms of chronic neuropathic pain, addressing a broader spectrum of unmet needs in pain management.

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