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Genetics – Innovation

February 27, 2021
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"In 10 years, I expect that gene and cellular therapies will have proliferated, hopefully much more than we can currently conceive. I think there'll be dozens of different gene therapies and even more than one competitive gene therapy for a specific condition. And hemophilia in particular already has at least three that should gain FDA clearance and approval by the end of 2021, at least in hemophilia A, and I expect this to be the case in the ensuing years for several other clinical conditions. The great thing about this is that we are on the brink of being able to, if not cure, then greatly dampen the effect of some of the most disabling diseases that we know as a society. And in doing that, if we're able to create the headroom and I mean the headroom, both from a clinical resourcing and attention and from a cost and financial standpoint, then we'll be able to create an environment where we know that developers of these new technologies will be encouraged and incentivized to develop more and more of them. Hopefully what we find is that the vehicles that are used, specifically the viral vectors that are used, can be used over and over again for several different conditions. So in some, I think in 10 years, there will be a series of different platforms that have been proven out that are safe and effective for delivering non-mutated forms of genes into cells, and they don't have a lot of unintended effects, off target effects, and this will allow us to move from treating single mutation genetic disorders to really focusing on how to dampen the effects of the more complex polygenetic disorders. Common conditions, like diabetes and asthma and heart disease and osteoarthritis. All of these diseases have a genetic basis as well. And we need the bandwidth and the headroom to be able to tackle them."

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