
269: Biotech layoffs, eye drug drama, & gene therapy milestones
The Readout Loud
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Sereptus: A Gene Therapy for Duchenne
The sereptus story is kind of interesting in the context of gene therapies more broadly because as you mentioned, it has that conditional approval. The condition is that the this gene therapy also meet its goals in a larger randomized phase three study. And if presumably if it is a success, the FDA expands the approval to a traditional one and makes it available to more boys with Duchenne larger age group., et cetera. So it's an interesting story to watch both scientifically, regularly, and even business termsBecause what serept is doing trying to get patients on commercial therapy? I mean, they're spinning multiple plates at the same time because this trial is ongoing.
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