News

Nerve and heart muscle cells derived from a man with Friedreich’s ataxia (FA) showed differences in the activity of multiple genes, a study reported. In nerve cells, deficiency in the frataxin protein, the underlying cause of FA, influenced genes related to energy-producing glucose metabolism, or glycolysis. In heart…

Prime Medicine’s gene-editing technology — called Prime Editing — successfully corrected the genetic deficits associated with Friedreich’s ataxia (FA) and restored nerve cell projections in preclinical studies using patient cells, the company has announced. The company also announced positive updates from a number of its 18 other developmental programs…

Researchers have developed statistical models to help predict the progression of Friedreich’s ataxia using data such as age at disease onset and genetic information. Although the models are not yet accurate enough to be employed in clinical use, the researchers said this is a first step toward individualized medicine…

People with Friedreich’s ataxia show marked abnormalities in the structure of the upper spinal cord, some of which are correlated with measures of disease duration and severity, a study reports. “These results provide new insights about [the development of Friedreich’s ataxia] and indicate that spinal cord MRI may be…

The investigational therapy DT-216 safely worked to more than double the activity of the FXN gene — involved in energy production in cells — in people with Friedreich’s ataxia (FA). That’s according to new data from a single-ascending dose (SAD) Phase 1 clinical trial, which showed the treatment candidate was…

A $4.8 million grant from the California Institute for Regenerative Medicine (CIRM) will support a research project hoping to move a one-time gene editing treatment for Friedreich’s ataxia closer to clinical trials. “If successful, this approach could prevent the neurologic and cardiac complications in [Friedreich’s ataxia] and address the pressing…

People with Friedreich’s ataxia (FA) who also have diabetes, a condition that causes their blood glucose (sugar) level to become too high, are more likely than those who don’t have diabetes to have worse outcomes from COVID-19, according to a single-center study. Having diabetes increased both the chance of…

Low levels of the frataxin protein — the underlying cause of Friedreich’s ataxia (FA) — and the ensuing problems in mitochondria, the cells’ powerhouses, may activate certain immune responses in the disease, a study suggests. In lab-grown human heart, muscle precursor, and connective tissue cells, suppressing frataxin production was…

A four-week Phase 2 clinical trial that’ll be testing CTI-1601, Larimar Therapeutics’ experimental therapy for Friedreich’s ataxia (FA), is now enrolling in the U.S., the company announced in a press release. Patient recruitment began shortly after the U.S. Food and Drug Administration (FDA) lifted its full hold…

The Phase 1 clinical program of DT-216, Design Therapeutics’ experimental therapy for Friedreich’s ataxia (FA), remains on track, with initial data expected by year’s end. The program includes a single-ascending dose Phase 1a trial (NCT05285540) and a multiple-ascending dose Phase 1b study (NCT05573698), both testing the…