All posts by: Michael Zand

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FDA Awards Fast-track Status to Sangamo’s Investigational Gene Therapy SB-525 for Hemophila A – Hemophilia News Today

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2017-05-19 NEWS

FDA Awards Fast-track Status to Sangamo’s Investigational Gene Therapy SB-525 for Hemophila A
Hemophilia News Today
The U.S. Food and Drug Administration (FDA) has granted fast-track status to SB-525, a clinical gene therapy for hemophilia A developed by Sangamo Therapeutics in partnership with Pfizer. SB-525 delivers a human factor VIII cDNA construct and synthetic …

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2017-05-19 NEWS

ALS Research Forum

A New Potential ALS Gene Therapy Delivers A Key Milestone
ALS Research Forum
One approach, which involves gene therapy, suppresses expression of the SOD1 gene through RNA silencing-based mechanisms. This strategy, according to a 2016 study led by University of Massachusetts Medical School’s Christian Mueller, can extend the …