Spur Therapeutics Announces Platform and Poster Presentations Highlighting New Clinical Data for FLT201 in Gaucher Disease at Upcoming WORLDSymposium(TM)

Spur Therapeutics today announced that it will share updated clinical data from its Phase 1/2 GALILEO-1 and GALILEO-2 trials of avigbagene parvec (FLT201), its Phase 3 gene therapy candidate for Type ...
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Spur Therapeutics Announces Successful End-of-Phase 2 Meeting with FDA for FLT201, Its Gene Therapy Candidate for Gaucher Disease

FDA aligned on single-arm Phase 3 study to support potential accelerated and full approval On track to dose first patient in Phase 3 trial in second half of 2025 LONDON, Feb. 03, 2025 (GLOBE NEWSWIRE) ...
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CENTOGENE Announces Publication Establishing Lyso-Gb1 as a Predictive Biomarker for Gaucher Disease Patients

Results from pivotal study published in Diagnostics journal Confirmation of utility of lyso-Gb1 (glucosylsphingosine) as a sensitive biomarker for Gaucher disease Could predict clinical course of ...
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Freeline Presents Positive New Data from Phase 1/2 GALILEO-1 Trial of FLT201, Its Novel Gene Therapy Candidate, in Gaucher Disease at ESGCT 30th Annual Congress

Substantial reduction in lyso-Gb1 substrate levels, an established biomarker of clinical response, observed in first patient treated with FLT201 Increases of several hundredfold over baseline in ...
Nature

High-throughput screening for small-molecule stabilizers of misfolded glucocerebrosidase in Gaucher disease and Parkinson's disease.

The Nature Index 2025 Research Leaders — previously known as Annual Tables — reveal the leading institutions and countries/territories in the natural and health sciences, according to their output in ...