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4w ago

Experimental Fabry Gene Therapy Achieves Near-Break-Even Costs and Advances to Larger Study

A 25-to-30-patient trial will evaluate the therapy’s scalability under a proposed government-owned licensing model for national health systems.

Dr. Michael West, a co-author and Dalhousie University researcher, is shown in this handout image. THE CANADIAN PRESS/Handout

Overview

  • Five men treated between 2016 and 2018 have maintained stabilized organ function and avoided major Fabry-related events over five years.
  • Three participants discontinued biweekly enzyme-replacement therapy, generating $3.7 million in savings against about $4 million in research spending.
  • Only two mild side effects have been recorded in the cohort, and neither was directly attributed to the gene therapy itself.
  • The follow-up study is set to enroll 25 to 30 patients—including women—over a two- to three-year period to confirm long-term efficacy and safety.
  • Investigators are exploring a public-sector development and licensing framework to deliver the one-time treatment through national health systems.