News

A man living with Fabry disease for more than two decades was found to have a second co-occurring genetic condition called polycystic kidney disease, according to a recent report. “This case demonstrates the possibility of two [genetic kidney diseases] in the same individual and the risk of one diagnosis…

A single dose of 4D-310, an investigational gene therapy from 4D Molecular Therapeutics (4DMT), appears generally safe and restored the levels of alpha-galactosidase A (Gal A), the faulty enzyme in Fabry disease, in the first three men enrolled in a Phase 1/2 clinical trial. Notably, all three patients…

Jasper Therapeutics and Avrobio have entered into a non-exclusive research collaboration to evaluate the use of JSP191 in patients with Fabry disease who are being treated with one of Avrobio’s investigational gene therapies. Under this collaboration, JSP191 — an anti-CD117 monoclonal antibody — also will be…

Sangamo Therapeutics’ gene therapy candidate ST-920 safely and effectively increased and sustained the levels of the alpha-GalA protein, the enzyme that is lacking in people with Fabry disease, according to preliminary Phase 1/2 trial data. Four people have been treated at the first two therapy doses being…

FLT190, Freeline Therapeutics’ experimental gene therapy, led to sustained increases in alpha-galactosidase A (Gal A), the faulty enzyme in Fabry disease, in the first two patients given the one-time therapy in the Phase 1/2 MARVEL-1 clinical trial. Notably, the second patient, dosed early this year, has reached near-normal…

A single dose of Avrobio’s investigational gene therapy AVR-RD-01 continues to show a favorable safety and tolerability profile for up to 4.5 years in adolescents and adults with Fabry disease, according to latest data from Phase 1 and Phase 2 clinical trials. Notably, up to 3.5-year data previously showed the…

Lucerastat, an investigational substrate reduction therapy for Fabry disease, has failed to outperform placebo at reducing neuropathic pain in patients, updated results from the MODIFY Phase 3 trial show. The findings mean that Idorsia‘s MODIFY study (NCT03425539) did not meet its primary goal, despite lucerastat being well…

A Phase 1/2 clinical trial of Sangamo Therapeutics’ experimental gene therapy ST-920 in people with Fabry disease has completed dosing in second patient group, the company announced in a press release. Based on initial safety data, a trial monitoring committee has recommended that a planned third patient…

A recent type A meeting with the U.S. Food and Drug Administration (FDA) is likely to lead Protalix Biotherapeutics to resubmit a request for approval of PRX-102 (pegunigalsidase alfa) to treat Fabry disease, after its initial application was rejected by the agency. “We are pleased with the results of the…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…