Executive Vice President, Chief Scientific Officer and Head of Research & Development, Iteos Therapeutics
Search every verified Louise Rodino-klapac interview, podcast appearance, and on-the-record quote — each transcript cross-checked by AI and human review to confirm speaker identity. Louise Rodino-Klapac, Executive Vice President, Chief Scientific Officer and Head of Research & Development at Iteos Therapeutics, appeared on the podcast "DNA Today" on September 17, 2023, to discuss limb-girdle muscular dystrophies (LGMD). During the episode, she stated that the subtypes her team at Sarepta is researching cover about 70 percent of the LGMD population, and described their goal as delivering a newly functioning version of the relevant gene to make the missing protein. She explained that gene therapy involves adding a normal functioning copy of a gene rather than altering the genome, and that each LGMD subtype requires a tailored therapy because each is caused by a mutation in a different gene. Rodino-Klapac noted that clinical trials for various LGMD subtypes are mostly in phase one, evaluating safety and early efficacy, with hopes for approval within the next decade. She emphasized that the goal of gene therapy is to stabilize or slow disease progression, and that earlier intervention may lead to better outcomes. She encouraged patients to join registries for their specific subtype, stating that natural history studies are critical for understanding the disease and necessary for clinical trials. She also discussed the availability of industry-supported genetic testing programs at commercial laboratories that offer testing at no cost or reduced cost to patients.
“The subtypes that we are researching at Sarepta cover about 70 percent of the limb girdle population and it's our goal to deliver a newly functioning version of that gene to make the protein that's missing.”
“Gene therapy involves delivering a normal functioning copy of the gene; you're not altering the genome of the body, but adding a new copy that makes the protein that's missing or not functional.”
“Each limb girdle subtype is a mutation in a different gene, so each gene therapy is tailored specifically for that subtype, which is why knowing your genetic diagnosis is so important.”
“The goal of gene therapy is to stabilize or slow the progression of the disease, and the earlier the intervention, the better the potential outcome.”
“Currently, there are clinical trials for different limb girdle subtypes, mostly in phase one evaluating safety and early efficacy, with hopes for approval within the next decade.”
“I always encourage patients to join registries for their specific limb girdle subtype, as these natural history studies are critical for understanding the disease and are necessary for clinical trials.”
“Insurance companies approve genetic testing when it impacts management, and for limb girdle muscular dystrophy, insurance authorization is relatively straightforward with proper medical necessity letters.”
“There are industry-supported programs at commercial laboratories offering genetic testing for limb girdle muscular dystrophy at no cost or reduced cost to patients.”
“Genetic technology has advanced rapidly; the first human genome took 13 years to sequence, but now whole genome sequencing can be done in days or even hours, lowering costs and increasing accessibility.”
“Limb girdle muscular dystrophy is not one condition but a collection of disorders with similar phenotypes, each caused by distinct gene mutations, making precise genetic diagnosis essential for treatment.”
In this episode, we are exploring limb-girdle muscular dystrophy (LGMD). Joining us for this conversation are two experts, Dr.
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