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Which of these do you think are farthest along? Gene therapy seems to be working a little bit in ex-vivo approaches (you take cells out, modify them, and put t

• aaavl2821 7y ago
  Re akuous and decibel, I don't know too much about their programs Re eye diseases / blindness, there is at least one approved gene therapy for a form of congenital blindness, luxturna: https://www.fda.gov/news-events/press-announcements/fda-approves-novel-gene-therapy-treat-patients-rare-form-inherited-vision-loss https://www.fda.gov/news-events/press-announcements/fda-appr... the eye is one of the areas where gene therapy delivery is currently feasible. other areas include the CNS using AAV9. Perhaps the most impactful gene tx yet approved is Avexis' Zolgensma (Avexis was acq by Novartis for $8-9B in 2018). Zolgensma is a potential cure for spinal muscular atrophy, the leading genetic cause of infant death. Zolgensma did $160M in its first full quarter on the market Other areas where we will likely see approved gene therapies soon include blood diseases like hemophilia, beta thalessemia. In addition to blood, eye and motor neurons, the liver is a popular target for gene therapy as all of these tissues are easier to deliver gene tx to with current technology Delivering gene tx to other tissues is not really feasible as far as i know at this point