Carol Miao, PhD, on the Potential of Ultrasound Mediated Gene Delivery
The principal investigator at Seattle Children’s Research Institute discussed her lab’s preclinical research with UMGD for the treatment of hemophilia.
“These are very exciting results to us, indicating that UMGD can be used as a clinically feasible and very promising new gene therapy treatment for hemophilia A...”
Currently, 2 FDA-approved gene therapy products are available for the treatment of hemophilia B and 1 FDA-approved gene therapy product is available for the treatment of hemophilia A. Although these treatments, which were all approved within the past few years, represent a great leap forward for the treatment of bleeding disorders, they are all based on the use of adeno-associated virus (AAV) vectors, which carry several important limitations. For example, some patients have preexisting antibodies to the AAV capsids that are used to deliver these gene therapies, and as such are ineligible for treatment with them because of concerns related to safety and efficacy caused by potential immune reactions to the vector. In addition, these therapies cannot be redosed for a similar reason—patients will have developed antibodies to the vector after their initial treatment. As such, if the efficacy of these therapies wanes over time, nothing can be done to address it. With this in mind, some academic institutions and companies are continuing to work on novel, nonviral delivery methods for genomic medicines targeted at hemophilia in order to address this unmet need. For example, the lab of Carol Miao, PhD, a principal investigator at Seattle Children’s Research Institute, is currently working on ultrasound mediated gene delivery (UMGD) method for treating hemophilia. Notably, preclinical research on the application of UMGD in large animal models was presented by Miao at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD.
At the conference, CGTLive® sat down with Miao to learn more. She explained how the UMGD approach works in detail and its advantages over viral delivery methods. She also went over the results of the preclinical work in hemophilia A canine models, noting that the UMGD approach successfully achieved consistent therapeutic levels of gene expression in the dogs and that it was successfully able to rescue the hemophilia A phenotype.
Click here to view more coverage of the 2024 ASGCT Annual Meeting.
