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Trend Chart on Innovative Biotherapies – February 11th, 2019
Trend Chart On....

Trend Chart on Innovative Biotherapies – February 11th, 2019

 FEATURE STORY
● Luxturna®: FDA documents reveal the value of a costly gene therapy
GENE THERAPY
● The application of Adeno-Associated Viral vector gene therapy to the treatment of Fragile X Syndrome
● Adeno-associated virus vector as a platform for gene therapy delivery
● Integrative analysis of pooled CRISPR genetic screens using MAGeCKFlute

CLINICAL TRIALS – DATA
● GenSight’s LHON gene therapy fails to meet endpoints at week 48
● UniQure announces first patient treated in HOPE-B pivotal trial of AMT-061 in patients with hemophilia B
● Solid Bio is hit by another setback as initial biopsies spotlight a flop for Duchenne muscular dystrophy
● Sangamo Therapeutics: in vivo genome editing in patients with MPS II treated with SB-913

INDUSTRIAL LANDSCAPE & AGREEMENTS
● EdiGene completes $10 million round for gene editing-based discovery

MISCELLANEOUS
● β-thalassemia: cost-efficacy analysis of gene therapy versus allogenic hematopoietic stem cell transplantation

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