ASCAT 2023 | Gene therapy: the future of SCD treatment?

So I get excited. I got excited when Voxelotor came out. I got excited when [crizanlizumab] came out. Gene therapy is one of the more promising tools that we’ll have, hopefully relatively soon. But, you know, the PK agonists are also very exciting. And remember, not everyone’s going to be eligible for gene therapy and so having alternative medicines that are available to most patients with sickle cell are sometimes going to be the better options, especially when we know that not everybody is going to be eligible for gene therapy or it may be limited to certain specific circumstances in terms of patient’s care, i.e., you know, multiple pain episodes, specific types of sickle cell, you know, and people who have chronic organ damage already may not get better from gene therapy, so we have to be very careful about that too.

So there’s three versions of gene therapy that hopefully will be available relatively soon. One is a gene addition program that adds a harmless but detectable hemoglobin that is shown to improve pain episodes. And there are two that, one is in clinical trials still and one is actually up for FDA approval, that help increase fetal hemoglobin, and that’s the hemoglobin that potentially helps to protect people from chronic organ damage, and at least keeps them from developing worsening chronic organ damage if they already have it. So those two are actually up for FDA approval later this year, and we’re hoping that if that goes through well, that then they’ll be available for patients generally starting the beginning part of next year.