TESS Research Foundation Receives FDA Clearance to Launch First Human Clinical Trial for TSHA-105, a Gene Therapy for SLC13A5 Epilepsy
Milestone Leads Way to Targeted Life Altering Treatment for Severe Seizure Disorder
Menlo Park, CA (August 15, 2025)--TESS Research Foundation, a nonprofit organization committed to improving the lives of those affected by SLC13A5 Epilepsy, announced today that the U.S. Food and Drug Administration (FDA) has granted clearance for its Investigational New Drug (IND) application for TSHA-105—a gene therapy that uses an AAV9 vector to deliver the human SLC13A5 gene. This milestone allows TESS to move forward with a clinical trial (NCT07102524) to test the first-ever targeted treatment for this rare, severe neurological disorder.
“The acceptance of our IND is an important milestone for our SLC13A5 Epilepsy community,” said Kim Nye, Founder and Executive Director of TESS Research Foundation and mom to two children with the disorder. “We are thrilled that the FDA has given us the green light to move forward with a clinical trial for the first treatment ever created specifically for SLC13A5 Epilepsy.”
SLC13A5 Epilepsy Disorder is a rare, recessive neurological disorder caused by variants in the SLC13A5 gene, which codes for a protein responsible for transporting citrate. This disorder typically manifests in infancy, causing debilitating symptoms such as frequent and prolonged seizures, movement and balance problems, developmental delays, severe speech difficulties, and other cognitive challenges. It significantly affects patients’ quality of life, leading to lifelong disabilities.
TSHA-105 Drug was developed by Rachel Bailey, PhD a leader in gene therapy research at UT Southwestern Medical Center (UTSW). Her team employed AAV vector engineering to create a gene replacement approach, demonstrating its potential through preclinical studies in cell culture and animal models.
“It’s incredible to see science move from the research bench into people,” said Dr. Bailey, Assistant Professor at UTSW. “This is patient-centered research – developing a potential treatment centered around the needs of the affected community. Gene therapy has the potential to address the root cause of the disease.”
The natural history of SLC13A5 Epilepsy Disorder was funded by TESS Research Foundation and led by Brenda Porter, MD PhD (Stanford University), Kimberly Goodspeed, MD, Dallas Armstrong MD (UTSW), and Judy Liu, MD PhD (Brown University). Support from the Chan Zuckerberg Initiative helped TESS build the capacity to sponsor the clinical program. Elpida Therapeutics, a nonprofit biotech, led the IND submission, bringing regulatory expertise, strategic clinical development, and experienced project leadership.
“Progress is built on collaboration—where patient advocacy groups, academia, industry, CROs, and donors unite with one shared goal: putting the patient first,” said Dr. Tanya Brown, TESS Scientific Director. “Together, we’re proving that potentially life-changing precision medicines can be developed, even for the rarest of disorders.”
This milestone reflects TESS Research Foundation’s mission: driving cutting-edge research to diagnose, treat, and ultimately cure SLC13A5 Epilepsy, while providing support for affected children and their families.
Further details about clinical trial enrollment are available on Clinicaltrials.gov/study/NCT07102524
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