Jeffrey Chamberlain, PhD, on Exciting New Research at MDA 2024

“One of the more exciting areas, I think, is that there are new generations of delivery vehicles, not only new types of AAV vectors, but also new lentiviral vectors that may be effective. And we're hoping that these more potent delivery vehicles will allow us to lower the dose. And I think that's going to be important for reducing some of the serious adverse events that have been seen in the muscle gene therapy trials.”

Jeffrey Chamberlain, PhD, Professor, Neurology and Medical Genetics, and Adjunct Professor, Biochemistry, and McCaw Endowed Chair of Muscular Dystrophy at University of Washington, was awarded an MDA Legacy Award by the Muscular Dystrophy Association (MDA) at the 2024 MDA Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, for his decades of work in research helping to establish gene therapy treatments for muscular dystrophy, going all the way back to work he did as a graduate with muscle-specific promoters.

CGTLive® spoke with Chamberlain after receiving the award to hear more about his takeaways from this year’s conference. He shared some highlights from the meeting and overviewed some topics and trends that he found exciting. Among these highlights were developing new animal models of Duchenne muscular dystrophy, finding new biomarkers of disease progression, and a surge of antisense oligonucleotide being investigated for muscular dystrophy treatment. One topic he was most excited by was data on new lentiviral vectors as opposed to adeno-associated virus (AAV) vectors.

Click here to view more coverage of the 2024 MDA Conference.

REFERENCE
Muscular Dystrophy Association Announces Recipient of 2024 MDA Legacy Award for Achievement in Research, is Jeffrey Chamberlain, Ph.D., Leading Professor in Gene Therapy. News release. January 30, 2024. https://finance.yahoo.com/news/muscular-dystrophy-association-announces-recipient-145900986.html