VIDEO: Unmet needs in HAE include faster diagnostics, more effective treatments

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I think there's a huge unmet need diagnostically, particularly for these patients that have HAE with normalcy one inhibitors. So we have good diagnostic tests for type one and type two HAE. We have limited diagnostics for HAE with normal C one inhibitor. And so I think those patients have a difficult journey because the diagnosis is really made over time through mostly clinical treatment and observation. What, you know, what do they respond to, what do they not respond to, and some of the characteristics of their symptoms. So there's a diagnostic unmet need. I think there's also still a therapeutic unmet need.

Again, we're fortunate to have some treatment options, most of which are very effective for most patients. That said, there is still a burden of treatment, meaning these medicines are not always easy to use because of the schedule one has to keep, or the discomfort of doing injections or infusions. Any and all medicines have side effects. So some patients do struggle with side effects from medicines and the medicines aren't perfectly effective for everyone.

So while they're good, we still have gaps either in efficacy or tolerability for some patients. So I think I mentioned all the drugs in development. These are mostly aimed at can we make treatment more effective, particularly when it comes to prevention. Can we have prevent these attacks much more effectively? And secondly, can we make the treatments more tolerable with fewer side effects and safety folds into that. We want to make the treatment as as safe as we can based on the studies that are done. I think those are the trends you're seeing in the treatment is effective, safe, tolerable, easy to use.

And then lastly, like we talked about, accessible, we've gotta try to make these medicines accessible to people because you have the best medicine in the world, but if it just sits on the shelf and no one can get to it, it defeats the purpose entirely folded into that, you know, the research focus for the future. I know our group and others, we really are trying to look at biomarkers. Can we, I diagnose people more accurately, faster, with novel tests, whether those are blood tests, genetic tests, biochemical tests. Can we get to that diagnosis more quickly? There's some interest in artificial intelligence to do that.

You know, can you look at a set of symptoms or a history from a patient and sort of say, you know, there's a high chance this person has HAE and they should be evaluated for that. To pull those people out of the more common types of angioedema, which are allergic in nature, which is where people get stuck a lot of the time and don't respond to those treatments. So I think, I think there's a lot of interest in, in diagnostic algorithms, diagnostic tests. I'd end by saying it would be really nice in the long run to find a, I'll call it a cure, but a long-term treatment for HAE. That's what gene therapy seeks to do.

And I think we have to be cautious about that because we want to be safe, as safe as we can be in developing those treatments. And maybe it's not gene therapy, maybe it's a treatment that you given and it lasts for, you know, a longer period of time as some of these other drugs are seeking to do. But the burden of treatment is a real thing. And so I think if we can find ways to sort of give a treatment, which will give a long, durable response and still be reasonably safe, that's something we're striving for. But we have to go slowly and make sure that we're minimizing the risk of harm in the process of doing these sorts of studies.