Gene Therapy Will Continue to be a Major Topic at MDA’s 2024 Conference

Sharon Hesterlee, PhD

This year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference will be held on March 3-6, 2024, in Orlando, Florida. The annual conference is focused on the treatment of neuromuscular disorders in general, but has recently taken a greater focus on gene therapy and cell therapy as more and more therapeutics of these types enter development for these diseases.

In October 2023, CGTLive™ sat down with Sharon Hesterlee, PhD, the chief research officer of MDA, to discuss the highlights of 2023’s meeting and exciting new things to look forward to at the upcoming 2024 meeting. She noted that this year’s meeting will include a new poster tour and a new system of tagging to make it easier for attendees to follow their disease area of interest.

CGTLive: Can you give an overview of what the MDA conference is for anyone who is not familiar with it?

Sharon Hesterlee, PhD: The MDA annual conference is a professional conference. It's both scientific and care focused, so that we have a clinical component as well as basic research. I think that's what makes it kind of unique is having those 2 groups represented almost equally across the agenda so that we can do a lot of cross cutting things that start maybe with their roots in basic research and you can see how things have progressed all the way to new drugs and changes in the way care is practiced.

What are the main reasons that doctors and healthcare professionals would be interested in attending the meeting?

I think it's a really exciting time right now in the world of neuromuscular disease. We've really entered the treatment era with a vengeance after many, many years of not having effective treatments for these diseases. We've started seeing more and more approved and [2023] is a banner year. We've had 7 new drugs approved for neuromuscular disease this year and the year is not over. We're anticipating more before the end of the year. I think the theme of the conference for 2024 is: How are we set up to be successful in administering these drugs? Some of them are complex biologic drugs. [There will be a focus on] really looking at how practices are set up, how drugs are being administered, how we are communicating to families about the availability of these drugs and side effects and expectations, as well as looking at a follow-on pipeline of things that are in development because, of course, we still don't have treatments for all of the neuromuscular diseases. I think it's a sort of big picture: age of treatment and also looking at the continuous development of new therapies.

Are there any highlights from the 2023 meeting that you would like to share?

For anyone who was there in 2023, we had a real treat: our keynote speaker was Dr. Peter Marks [MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research], and it was very timely as we were anticipating the approval of Elevidys, the first gene therapy for Duchenne muscular dystrophy (DMD), which did occur shortly after the conference. That was really exciting and he had a lot of interesting things to say at that conference about how the FDA is moving to be more flexible around its approvals for rare diseases.

We also announced a new internal program at MDA called Kickstart that is focused on ultrarare disorders, where we're basically running an in-house incubator program. Neuromuscular disorders are a huge number of things, and the majority are genetic. As we learn more and more, and identify new genes, we basically have created new disorders, when you look at treating them individually with something like gene therapy. So there are many ultra rare diseases in our program and that's something that we've also focused on last year and that will continue on into this year.

What are you most looking forward to for the 2024 conference? Especially when it comes to gene/cell therapy, is there anything specific you are looking forward to?

We're going to continue our series that we started last year on practical considerations for gene therapy. Again, we are looking at how practices are organized, lessons learned, and how you can be successful. There's a gene therapy approved now for spinal muscular atrophy and one approved for DMD. I think people are starting to get more experience with both of those therapeutics. We will continue that session—it was really popular last year—and we will keep that going this year and expand on some of the themes that people were most interested in last year. I think that's going to be really exciting.

We have a few other series. We have our Lab to Life series—which is always interesting—where we're looking across the spectrum from the mechanistics of a disease all the way through new therapies. That will continue and I think will also be another highlight.

Are there any other sessions, speakers, or topics you're particularly interested in, or that attendees can look forward to?

Something I'd want to highlight for attendees this year that's new that I think will be interesting is we're actually doing a poster tour. There'll be a live poster tour in the evening during the poster sessions, where we're selecting 2 or 3 of some of the more exciting topics and there will be a live talk at the poster and that will move around the room. I think that will really draw a greater focus on posters and the interesting things that are presented there.

Also new for this year, we're doing something to make it very easy to track topics that you're interested in. We will be coding all of the posters and all of the talks according to major groups of neuromuscular disease. So if you're interested in something like metabolic myopathies in particular, or you're particularly interested in amyotrophic lateral sclerosis, or motor neuron disease, we'll have all of that subject matter tagged so that you can easily follow it throughout the conference.

This transcript has been edited for clarity.

Click here to register for the upcoming 2024 MDA Conference.