February 15, 2019
Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline
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February 13, 2019
Study Questions the Value of Priority Review Vouchers as Incentives
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February 12, 2019
FDA Grants Clementia Rare Pediatric Disease Designation by FOP Drug
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February 8, 2019
Solid Biosciences Reports Disappointing Preliminary Results of DMD Gene Therapy Study
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February 8, 2019
First Results of In Vivo Gene Editing Trial of MPS II Treatment Show…
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February 7, 2019
FDA Approves Sanofi’s Cablivi for Rare Blood-Clotting Disorder aTTP, the First of a…
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February 1, 2019
Regenerative Medicine Moves into the Spotlight
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January 31, 2019
FDA Grants RegenxBio Rare Pediatric Disease Designation for Gene Therapy for CLN2 Form…
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January 28, 2019
Alexion Reports Positive Results for Ultomiris in aHUS, Moving to File for Approval
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January 25, 2019
Learning New Tricks from Pathogenic Bacteria to Target the Microbiome
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January 24, 2019
BridgeBio Enters Collaboration with Cincinnati Children’s to Accelerate Development of Ge...
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January 24, 2019
CAMP4 Therapeutics Enters Rare Disease Research Collaboration with Alnylam
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