Omer A. Abdul Hamid, MD, on Improving Gene Therapy’s Effect and Accessibility

"One thing that's advanced our space a lot is the cost of genetic testing has gone down tremendously, which has increased our access to genetic testing and counseling. And this really opened up the doors for a lot of patients that went undiagnosed for years, and now we're finding a diagnosis, finding a community and finding hope that there's going to be a treatment for them. So, I think we need to improve and expand upon our genetic testing and our understanding of genetics, we still have a lot to learn, we still have genes that haven't been discovered yet.”

Omer A. Abdul Hamid, MD, pediatric neurologist, Nemours Children’s Health, gave a talk at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, on gene therapy logistics, specifically on logistics during the day of dosing, a topic that has only become more salient as the prevalence of gene therapies in the clinic grows.

CGTLive® spoke with Hamid to learn more about the logistics of gene therapies and unmet needs that remain in order to make them more accessible to patients and to support their development for more underserved diseases. He touched on a few topics that he thinks will enable gene therapy to have the most benefit to patients, including improved genetic testing and improved genetic understanding of current diseases, as well as improving accessibility for international patients. He also emphasized the importance of newborn screening to treat patients earlier in their disease courses, and beyond that, the potential for treating patients with gene therapies in utero to halt disease progression as early as possible.

REFERENCE

Gene Transfer Therapy: Dosing Day Logistics. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Orlando, FL.