Sangamo Therapeutics posts ST-503 nonclinical safety presentation for small fiber neuropathy
Sangamo Therapeutics, Inc.
Sangamo Therapeutics, Inc. SGMO | 0.00 |
- Sangamo published nonclinical safety results for ST-503, an investigational AAV9 gene therapy designed to repress SCN9A (Nav1.7) for treatment of small fiber neuropathy.
- Six-month GLP study in cynomolgus monkeys reported no dose-limiting toxicity following one-time intrathecal dosing at 10,000,000,000,000, 60,000,000,000,000, 90,000,000,000,000 vg/animal.
- Pharmacology readouts at 6 months showed intended SCN9A repression in dorsal root ganglia, with activity also reported in trigeminal ganglia.
- Mouse fertility and embryo-fetal development study using a surrogate AAV9 ZFR at 5,710,000,000,000 vg/kg found no test-article-related adverse effects.
- Integration and germline assessments reported no evidence of AAV germline transmission to offspring, with liver integration profile described as low-level and polyclonal, supporting a low tumor-risk signal.
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