Design Therapeutics Provides RESTORE-FA Clinical Development Update and Reports Second Quarter 2026 Financial Results

CARLSBAD, Calif., Aug. 03, 2026 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc..Positive 

RESTORE-FA Four-Week Data Support Advancement of DT-216P2. As reported in May 2026, DT-216P2 was generally well-tolerated and demonstrated dose-dependent increases in endogenous frataxin mRNA and protein levels, together with improvements across multiple clinical measures following four weeks of intravenous dosing in patients with Friedreich ataxia. 

Modifications to RESTORE-FA. Based on the four-week data, Design is modifying the ongoing cohorts in the RESTORE-FA trial to support the next stage of clinical development. The study will continue to evaluate 1 mpk as the planned go-forward dose, with the intention of enrolling 10 patients in the 12-week cohort. In addition, modifications include specifying endogenous blood FXN protein percent change from baseline as the primary efficacy endpoint and exploring a dose level above 1 mpk. 

Next Steps and Expected Milestones: Design expects to provide an update on its registrational plans in the fourth quarter of 2026, with data following 12 weeks of treatment expected in the first quarter of 2027.

Towards routine genetic testing of repeat expansions in neurogenetic diseases using multiplex CRISPR-Cas9-targeted long read sequencing

Fergelot, P., Boury, C., Penaud, B. et al. Towards routine genetic testing of repeat expansions in neurogenetic diseases using multiplex CRISPR-Cas9-targeted long read sequencing. Sci Rep (2026). doi:10.1038/s41598-026-64095-6 

 We simultaneously targeted nine loci involved in 10 repeat expansion disorders in a single capture panel, including FMR1, HTT, DMPK, CNBP/ZNF9, ATXN2, JPH3, FXN, C9ORF72 and RFC1, covering a broad range of repeat types, sizes and diagnostic needs. Results were compared with standard routine testing methods.

Clinical Challenges in Managing Diabetes Mellitus in Friedreich’s Ataxia

Aarya Naik, MBBS, Hooman Oktaei, MD, Clinical Challenges in Managing Diabetes Mellitus in Friedreich’s Ataxia, Endocrine Practice , 32, S66-S67. doi:10.1016/j.eprac.2026.01.168 

Friedreich’s ataxia (FRDA) is a multisystem disease, with ataxia being the most overt clinical feature. It can be complicated by diabetes mellitus. FRDA-related diabetes has been reported in 5% to 40% of children and adults. Despite the clinical relevance of FRDA-related diabetes mellitus, no evidence-based clinical practice guidelines for screening or management have been set.