FDA has cleared an application for a clinical study of a once-daily powder aimed at treating a rare kidney disease, transitioning six adults from standard cysteamine therapy. Topline data is expected in Q1 2027, pending a timely start.
The pilot will switch approximately six adults from standard cysteamine therapy for seven days; topline data are anticipated in Q1 2027, assuming timely start. Jul 13, 2026 Thiogenesis Receives FDA Rare Pediatric Disease Designation for TTI-0102 for the Treatment of Leigh Syndrome · Jun 17, 2026 Thiogenesis Appoints Dr. Gilad Aharon to Its Board of Directors · Apr 16, 2026 Thiogenesis Therapeutics to Present at 2026 Bloom Burton & Co. Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQB: TTIPF) is a clinical-stage biopharmaceutical company with operations based in San Diego, CA. The Company is publicly traded on the TSX Venture Exchange and in the U.S. on the OTCQB. Thiogenesis is developing sulfur-containing prodrugs that act as precursors to previously approved thiol-active compounds, with the potential to treat serious pediatric diseases with unmet medical needs. Actual results may differ materially due to risks including regulatory actions; the FDA's expectation that, before a registrational trial, the Company establish an optimal dose range, generate sufficient clinical and nonclinical safety data to support chronic administration, and reach agreement with the FDA on an adequate and well-controlled trial design, which may require additional studies, time and expense; delays in study start-up or enrollment; the fact that the study is conducted by an independent investigator and institution; the small size, open-label single-arm design and short treatm The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language. ... Thiogenesis Therapeutics (TTIPF) received FDA clearance on October 16, 2026, to study TTI-0102 in patients with nephropathic cystinosis.
Original title: “FDA clears an application to study a once-daily powder for a rare kidney disease”