Gene correction therapy wins orphan drug designation

[Courtesy of ToolGen]

South Korean biotechnology company ToolGen Inc. announced Monday that it received Orphan Drug Designation (ODD) for its rare disease gene correction treatment, TGT-001, from the U.S. Food and Drug Administration (FDA).

ODD supports the development of and expedites approval for therapies, and this latest approval will help accelerate TGT-001’s commercialization.

According to the company, the FDA approved the ODD for TGT-001 on December 14, 2023. With the designation, ToolGen will gain benefits including tax deductions for clinical development expenses, exemption from new drug application fees, and market exclusivity for seven years after approval.

TGT-001 targets Charcot-Marie-Tooth (CMT), a rare genetic disease related to peripheral nerves whose symptoms include muscle atrophy, muscle weakness, sensory loss, walking difficulties, and lack of reflexes.

TGT-001 corrects genes within the body directly, using the CRISPR gene-editing tool to regulate the expression of PMP22 to normal levels.

The treatment strategy was validated via animal testing, according to ToolGen.