Design Therapeutics Launches with $45M to Advance Therapy Candidates for FA and Other Disorders

Design Therapeutics has launched with $45 million in funding to advance its lead candidate for the treatment of Friedreich’s ataxia into clinical development, and support programs for other nucleotide repeat disorders such as fragile X syndrome, and myotonic dystrophy. These genetic diseases are caused by excessive repeats…

Retrotope Submits Design of Pivotal Trial for Its Friedreich’s Ataxia Therapy to FDA

Retrotope has submitted the design of a pivotal clinical trial for its Friedreich’s ataxia therapy RT001 to the U.S. Food and Drug Administration. The submission was prompted by results of a Phase 1/2 trial showing that the therapy was safe and exhibited early signs of effectiveness — an ability to improve patients’ physical functioning. A…