More studies in mice, pigs, and in primates followed, showing survival to degrees “never seen before,” added Sukumar Nagendran, then the company’s chief medical officer. That is our end goal, and until we get there, we will not rest.”. AveXis- BioLife Licenses Spinal Muscular Atrophy (SMA) Patent Portfolio from Nationwide Childrens Hospital and The Ohio State University. Pensacola, FL 32502 “We saw some of the kids standing…most of the kids sitting unassisted, doing things that the textbooks would say they shouldn’t be able to do,” he says. Be the first to rate this post. Dr. Kaspar is an innovator in the field of gene replacement technology – taking a cloned, intact gene and delivering it to patients who have diseases stemming from a missing or mutated gene. Support the SMA clinic at Nationwide Children’s Hospital and coordinate medical services for SMA patients. The intellectual property rights for Zolgensma were licensed from the University of Pennsylvania, Nationwide Children’s Hospital (NCH) and Genethon, a French charity. More studies followed, this time using a SMA-specific mouse model (the now widely used SMNDelta7 mouse) developed earlier by “a colleague of mine at The Ohio State University, Dr. Arthur Burghes … we had connections right next-door, literally,” Kaspar said. http://investors.avexis.com/phoenix.zhtml?c=254285&p=irol-newsArticle&ID=2337894, Press Release. http://www.curesma.org/news/zolgensma-approved.html, Therapeutic Approaches Since 2004, Dr. Kaspar has been working on gene replacement therapy that can halt the disease, with clinical trials showing children walking, talking and living a full life at five years old. Results, they wrote, “demonstrate the unique capacity of AAV9 to efficiently target cells within the CNS [central nervous system], and in particular, widespread neuronal and motor neuron transduction in the neonate, and extensive astrocyte transduction in the adult following intravenous delivery. Diseases like spinal muscular atrophy (SMA). May 24, 2019. Suite 354 The first human clinical trials are conducted with this therapy in SMA patients. No votes so far! All rights reserved. Dr. Kaspar and his team treat some of the first mice with gene replacement therapy and see extraordinary results. Grace Frank has worked as an editor and reporter for leading American newspapers, including The New York Times, The International Herald Tribune and the Tampa Tribune. The role of charities in funding research related to SMA in general and Zolgensma in particular has been quite important. But several more years of work remained before a specific virus — AAV9 — would do the same in key nerve cells of the brain and spinal cord. The NIH RePORTer database lists nearly a half billion in grants, when the search term “Spinal Muscular Atrophy” is used, and every key U.S based researcher has been the principal investigator for millions dollars in NIH grants. Children's researcher Brian Kaspar developed the therapy, and is chief scientific officer for AveXis. Moving mountains so his patients can too. Dr. Kaspar begins working on gene replacement technology for motor neuron disorders, partnering with Dr. Kevin Foust. “These diseases spread throughout the family emotionally. Dr. Brian Kaspar, Scientific Founder & Chief Scientific Officer, AveXis. How Zolgensma (onasemnogene abeparvovec-xioi), the gene therapy once known as AVXS-101, came into being is a tale fairly common to basic science: An idea that progressed from cell work in the lab to experiments with animals and, ultimately, testing as a possible treatment in patients. Spinal muscular atrophy (SMA) is the number one genetic cause of death for infants. “Don’t just do the simple experiment,” says Dr. Kaspar. 57: 649-55. http://www.sophiascure.org/sma-research/the-path-forward, August 26, 2012.

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