Gene therapy is a revolutionary approach to treating genetic diseases, where a one-time treatment can provide a lifetime of benefits. Instead of treating just the symptoms, gene therapy fixes the disease at the source by replacing the defective gene with a healthy copy.
Gene Therapy
1.
A healthy copy of the patient’s defective gene is loaded into a virus that has been stripped of its own DNA.
2.
Trillions of viruses, each containing a healthy copy of the gene, are injected into the patient’s spinal fluid.
3.
The viruses bind to cells in the patient’s spinal cord and brain and deliver healthy gene’s to the cell’s nucleus.
What is Gene Replacement Therapy?
Gene replacement therapy replaces a faulty gene in an attempt to cure disease or improve your body’s ability to fight disease.
What Gene Therapy is on the horizon for SLC13A5 Epilepsy?
Taysha Gene Therapies has received rare pediatric and orphan drug designation from the FDA and orphan drug designation from the European Commission for TSHA-105 to treat SLC13A5 Epilepsy. This drug was initially developed at UTSW by Rachel Bailey, PhD with funding from TESS Research Foundation.
Press Releases about TSHA-105 for SLC13A5 Deficiency:
Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency
January 19, 2021 Press Release
Taysha Gene Therapies Receives Orphan Drug Designation for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency From the European Commission
Taysha Gene Therapies Announces Late-Breaking Abstract and Poster Presentation on Positive Preclinical Data For TSHA-105 Demonstrating Therapeutic Potential for the Treatment of Epilepsy Caused by SLCA13A5 Deficiency
December 6, 2021 Press Release
Taysha Gene Therapies Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Corporate Update