Fabry disease

4D Molecular Therapeutics Inc (NASDAQ: FDMT) has posted updated interim data from the Phase 1/2 trial of 4D-310 in Fabry disease at the 18th Annual WORLDSymposium. Following 4D-310 infusion, mean serum AGA enzyme activity was within, or significantly above, the normal range in all three...

Avrobio Inc's (NASDAQ: AVRO) gene therapy for a rare lysosomal storage disorder showed potential durability in the first three patients more than one year after infusion. The data comes from a Phase 1/2 trial testing the safety and efficacy of AVR-RD-04 for cystinosis who had...

Sangamo Therapeutics Inc (NASDAQ: SGMO) announced updated preliminary results from the Phase 1/2 STAAR study of isaralgagene civaparvovec (ST-920), Fabry disease gene therapy candidate. Fabry disease is an inherited disorder that results from the buildup of a particular type of fat...

Avrobio Inc (NASDAQ: AVRO) has updated its pipeline deprioritizing its Fabry disease clinical program to focus on other clinical-stage studies in the lysosomal disorder pipeline.  The company has decided after new data from the five most recently dosed patients in Phase 2 FAB-...

Freeline Therapeutics Holdings plc (NASDAQ: FRLN) gave an update on its investigational Fabry disease treatment FLT190, currently in a Phase 1/2 trial, saying the candidate proved durable in the second patient tested. Fabry disease is an inherited metabolic disorder resulting from...

Sangamo Therapeutics Inc (NASDAQ: SGMO) announced preliminary results from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a gene therapy candidate for Fabry disease.  As of the September 17, 2021 cutoff date, results from the four patients treated in...

4D Molecular Therapeutics Inc (NASDAQ: FDMT) has announced interim data from the Phase 1/2 trial of intravenous 4D-310 in Fabry disease patients. The Company said that 4D-310 demonstrated a manageable safety profile with the initial evidence of clinical activity. The Company said...

The European Commission has approved Amicus Therapeutics Inc (NASDAQ: FOLD) Galafold (migalastat) for use in adolescents with Fabry disease who have an amenable mutation.  Galafold is already approved in the U.S., EU, and Japan for adults who have an amenable variant or...

Protalix BioTherapeutics Inc (NYSE: PLX) has submitted a Type A Meeting request to the FDA regarding PRX-102 Complete Response Letter it received in April. The FDA issued the response letter for pegunigalsidase alfa (PRX-102) in Fabry disease, though no reasons were cited...

Protalix BioTherapeutics Inc (NYSE: PLX) and Chiesi Farmaceutici S.p.A. have provided an update on the clinical development of pegunigalsidase alfa (PRX–102) for the proposed treatment of Fabry disease. PRX–102 is currently being studied in Phase 3 BALANCE trial...

Avrobio Inc (NASDAQ: AVRO) has provided an update on its regulatory plans for AVR-RD-01, its lentiviral gene therapy for Fabry disease, currently in FAB-GT Phase 2 trial. In March, the FDA granted full approval to Sanofi's SA (NASDAQ: SNY) Fabrazyme (agalsidase...

Protalix BioTherapeutics Inc (NYSE: PLX) and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici, have received a Complete Response Letter (CRL) from the FDA related to the marketing application of pegunigalsidase alfa (PRX‑102) for Fabry disease....