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.
