CMTA Alliance Partner ToolGen announced December 18, 2023 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).

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. This treatment is designed to treat CMT1A, which is caused by a duplication of the PMP22 gene.

The CMTA has been working with ToolGen via the CMTA’s unique pre-clinical testing program. Developing strategic research partnerships with companies like ToolGen is a central part of the CMTA’s strategy to accelerate treatments for the CMT community. We congratulate ToolGen on receiving Orphan Drug Designation for its CMT1A treatment candidate and will continue to support their efforts to advance this treatment towards clinical trials.

In a quote from ToolGen in October 2021, Director of R&D Therapeutics Jae Young Lee said, “We are delighted to have this collaboration to strengthen our drug development program for CMT1A where no approved therapy is available. This alliance with the Charcot-Marie-Tooth Association will enable us thorough testing of our drug candidate in suitable CMT1A models and lead us closer to initiating clinical trials.”

What is Orphan Drug Designation

Orphan drug designation provides a range of incentives and support mechanisms to encourage the development of treatments for rare diseases like CMT. These incentives help overcome the financial and logistical challenges associated with developing therapies for conditions with a small patient population, ultimately increasing the likelihood of successful treatment options for individuals with CMT.

Published: December 20, 2023