Design Therapeutics Maps H2 Data Readouts for RESTORE-FA

MarketBeat. Sun, March 15, 2026. RESTORE-FA targets a demonstrable increase in endogenous frataxin (measured in mRNA and protein, including muscle biopsy) after 12 weeks of dosing, with topline data expected in the second half of the year. According to Shah, achieving that type of increase would be notable because it has “never been achieved before.” He said the company is measuring both frataxin mRNA and frataxin protein, describing mRNA as “completely endogenous” and specifying that the assay looks at “normal processed mRNA.” Measurements will be taken in whole blood cells and also in affected tissue via muscle biopsy, though he noted biopsy sampling is limited and would generally be conducted before and after dosing, with a possible third sample.

On the question of GeneTAC selectivity, Shah pointed to preclinical work across multiple FA patient-derived cell types. He said GeneTAC molecules increased endogenous frataxin RNA and led to downstream effects including normalization of protein levels and improvements in functions such as cis-aconitate and cellular respiration. He added that exposure of wild-type genotype cells to an FA GeneTAC molecule had no impact because the repeat number in the wild-type allele is short and therefore does not produce a functional consequence when treated.

Patient population and endpoints in the FA study Shah described RESTORE-FA enrollment criteria as “pretty permissive” beyond genetic confirmation of FA, including ambulatory and wheelchair-bound patients. He said the current study explores both IV and subcutaneous (sub-Q) administration to help determine dose route, dosing interval, and the frataxin response to inform future clinical investigation. While functional assessments are customary in FA trials and are being performed, he said the primary goal of the study is to look for a frataxin response.