AceLink is advancing a small-molecule treatment option for Fabry disease, which could provide a more convenient alternative ...

Fabry's disease is an inherited condition that develops due to a mutation in the GLA gene. For most patients with this disease, the mutation causes the body to produce insufficient amounts of ...

LEXINGTON, Mass. - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company with a market capitalization of $767 million, ...

4D Molecular Therapeutics (4DMT) has said a trial of its gene therapy for Fabry disease has been placed on a clinical hold by the FDA, a few weeks after it reported three cases of atypical ...

“We remain focused on advancing our wholly owned neurology genomic medicine pipeline and progressing our Fabry gene therapy program towards a potential BLA submission in the second half of 2025,” ...

The company said that recently announced partnerships with Roche's Genentech unit and Astellas, and advanced business development discussions for its Fabry gene therapy programme (isaralgagene ...

Pfizer terminated partnership with Sangamo on hemophilia A gene therapy despite positive Phase 3 data. Find out why SGMO ...

PLX READ THE FULL PLX RESEARCH REPORT Reasons To Own / A Compelling Idea for 2025 Protalix Biotherapeutics, Inc. (NYSE:PLX) ...

In addition to the Fabry disease program, Sangamo's diverse pipeline includes isaralgagene civaparvovec (ST-920 or isa-vec), a gene therapy program, and STAC-BBB, an innovative approach targeting ...

Sangamo (SGMO) announced updated data from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene ...

In addition to the Fabry disease program, Sangamo's diverse pipeline includes isaralgagene civaparvovec (ST-920 or isa-vec), a gene therapy program, and STAC-BBB, an innovative approach targeting the ...