Despite significant improvements in gene therapy in recent years, there are still major drawbacks such as the lack of development of more accessible gene therapies for those with inherited genetic diseases due to a missing or defective gene or gene product. For those individuals, there are limited individualized therapies geared toward their genetic makeup.
Weidong Xiao, professor of pediatrics at the Indiana University School of Medicine and associate director of the Gene and Cell Therapy Program at the Herman B Wells Center for Pediatric Research, and Anh Lam, a former postdoctoral scientist at the IU School of Medicine, have developed modified adeno-associated viral (AAV) capsids that helps to provide more treatment options for patients.
“Through our engineered adeno-associated viral capsids, we are combining chemistry and genetics to create more options of treatments based on each person’s genetic makeup,” Lam said. “Each person has their own immune response, so individuals with preexisting antibodies often reject the capsids on the market which ultimately limits their treatment options.”
Xiao and Lam found that targeting the AAV capsids greatly reduces adverse effects and rejection. For example, for individuals with Duchenne muscular dystrophy the AAV capsids are designed to target the muscles, but if a person is experiencing an inherited blindness, a different AAV capsid serotype is used to target the retina.
Because many patients have preexisting antibodies against these AAV capsid serotypes on the market, the researchers were inspired to develop more engineered capsids to mitigate that or to retarget the AAV viruses towards different organs and tissues, thus opening up many potential therapeutic applications.
IU’s Innovation and Commercialization Office (ICO) filed a provisional patent for the innovation, and currently, Xiao and Lam are seeking opportunities for collaboration and funding in hopes of reaching clinical trials with the therapeutics.
“Our goal is to provide more treatments for larger populations and ICO has helped us move one step closer to that goal,” Xiao said.
Watch a video, featuring an interview with Xiao, about the Gene and Cell Therapy Program at the IU School of Medicine.
Bri Heron, technology marketing manager at Indiana University’s Innovation and Commercialization Office, contributed this story.
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