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5 questions facing gene therapy in 2021


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Editor’s note: This story is part of a series on the trends that will shape the industry in 2021. You can find all the articles on our trendline.
Three years ago, the Food and Drug Administration granted a landmark approval to the first gene therapy for an inherited disease, clearing a blindness treatment called Luxturna.
Since then, the regulator has approved one more gene therapy, the spinal muscular atrophy treatment Zolgensma, and given a green light for dozens of biotech and pharmaceutical companies to start clinical testing on others. Genetic medicines for a range of diseases, including hemophilia, sickle cell and several muscular dystrophies, appear in reach, and new science is galvanizing research.  ....

John Evans , Geulah Livshits , Jim Wilson , Brad Loncar , Piper Sandler , Eli Lilly , Emmanuelle Charpentier , Gbola Amusa , Jesse Gelsinger , Jennifer Doudna , Iovance Biotherapeutics , Harvard University , Drug Administration , Biomarin Pharmaceutical , Audentes Therapeutics , Spark Therapeutic , Voyager Therapeutics , Nobel Prize , Editas Medicine , Intellia Therapeutics , Loncar Investments , Dyno Therapeutics , Molecular Therapeutics , ஜான் எவன்ஸ் , ஜிம் வில்சன் , பிராட் லாஂகார் ,