Thiogenesis Receives FDA Rare Pediatric Disease Designation for TTI-0102 for the Treatment of Leigh Syndrome
San Diego, California--(Newsfile Corp. - July 13, 2026) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQX: TTIPF) ("Thiogenesis" or the "Company"),a clinical-stage biotechnology company developing novel sulfur-based thiol therapies for rare pediatric, metabolic and mitochondrial disorders, today announced that the U.S. Food and Drug Administration ("FDA") has granted Rare Pediatric Disease ("RPD") designation to its lead candidate, TTI-0102, for the treatment of Leigh syndrome.
The FDA grants RPD designation to therapies intended to treat serious or life-threatening diseases that primarily affect children from birth through 18 years of age. The designation provides the Company with the potential, upon approval of a future New Drug Application ("NDA"), to receive a Priority Review Voucher ("PRV"), which may be redeemed to obtain priority review of another marketing application or sold to another sponsor. Historically, PRV's have represented valuable strategic assets for biotechnology companies.
"Receiving Rare Pediatric Disease designation is an important regulatory milestone for our Leigh syndrome spectrum program and further expands Thiogenesis' regulatory portfolio," said Patrice Rioux, M.D., Ph.D., Chief Executive Officer of Thiogenesis. "The designation recognizes the significant unmet medical need in Leigh syndrome and reinforces our confidence in the potential of TTI-0102 to address that need."
"Since receiving FDA clearance of our IND, we have strengthened our Phase 2 protocol by incorporating important pharmacokinetic learnings from our MELAS clinical program. We are now completing final study start-up activities and look forward to initiating the Phase 2 trial and generating clinical data."
About Thiogenesis
Based in San Diego, California, Thiogenesis Therapeutics Corp. (TSXV: TTI) (OTCQX: TTIPF) is a clinical-stage biotechnology company developing TTI-0102, a patented new chemical entity designed to deliver sustained cysteamine exposure through proprietary prodrug chemistry rather than conventional controlled-release formulation technology. This differentiated approach is intended to improve tolerability, convenience and therapeutic exposure relative to existing cysteamine therapies. The Company's lead program is in nephropathic cystinosis, where TTI-0102 is advancing toward late-stage clinical development.Thiogenesis is also developing TTI-0102 for primary mitochondrial diseases, including Leigh syndrome spectrum, and is preparing to initiate a Phase 2 clinical trial.
Forward-Looking Statements
Some statements contained in this news release are forward-looking information within the meaning of Canadian securities laws. Generally, forward-looking information can be identified by the use of forward-looking terminology such as plans, expects, is expected, budget, scheduled, estimates, forecasts, intends, anticipates, believes or variations of such words and phrases including negative or grammatical variations or statements that certain actions, events or results may, could, would, might or will be taken, occur or be achieved or the negative connotation thereof. Investors are cautioned that forward-looking information is inherently uncertain and involves risks, assumptions and uncertainties that could cause actual facts to differ materially. There can be no assurance that future developments affecting the Company will be those anticipated by management. The forward-looking information contained in this press release constitutes management's current estimates, as of the date of this press release, with respect to the matters covered thereby. We expect that these estimates will change as new information is received. While we may elect to update these estimates at any time, we do not undertake to update any estimate at any particular time or in response to any event.
Neither the TSX Venture Exchange nor its Regulation Services Provider as that term is defined in the policies of the TSX Venture Exchange nor the OTC Markets Group Inc. accepts responsibility for the adequacy or accuracy of this news release.
This news release is not an offer of securities for sale in the United States. The securities may not be offered or sold in the United States absent registration or an exemption from registration under U.S. Securities Act of 1933, as amended - the U.S. Securities Act. The Company has not registered and will not register the securities under the U.S. Securities Act. The Company does not intend to engage in a public offering of their securities in the United States.
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Source: Thiogenesis Therapeutics, Corp.
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