InnovatIONS

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Innovating Medicinal Chemistry to Expand the Range of Tissues that RNA-based Therapies Can Target Jul 27, 2023

Graeme Freestone hails from Tadley, a small town in the English countryside 80 kilometers west of London. His father, a nuclear chemist, worked in the neighboring town of Aldermaston, known as Britain’s Los Alamos because it's where the UK designed and maintained nuclear warheads during the Cold War and, in peacetime, manages nuclear waste. Not only is Graeme from a science town, he has science in his blood; one uncle is a chemical engineer and the another is a professor of environmental chemistry. With that background, it’s not surprising that he pursued a chemistry degree at the University of Manchester, followed by a Ph.D. at the University of Oxford. Across the pond in the lab of Professor K.C. Nicolaou at the Scripps Research Institute in San Diego, Graeme’s post-doctoral work focused on the total synthesis of complex natural products. At Ionis, his research focuses on improving the therapeutic profile of antisense and siRNA oligonucleotides to develop therapies to treat cardiovascular, neurological, and metabolic diseases. But chemistry isn’t the only thing Graeme excels in. When he’s not in the lab, he’s training for and competing in Ironman triathlons and ultramarathons. So, what is “innovation” to Dr. Graeme Freestone? “Creating something new, whether that’s new research, new projects or new ideas and putting them in to action.”

Innovating medicines to treat rare childhood neurodegenerative diseases. Mar 30, 2023

Berit Powers, Ph.D., Director, Neurology, Ionis Ohio native Berit Powers went off to the University of Michigan with a keen interest in how the human brain works. A course on the biological correlates of addiction sparked her fascination with the processes that underly human behavior and disease. Later, as a lab assistant at the Cleveland Clinic, she had the “wild” experience of observing the surgical resection of human brain regions that were causing severe, drug-resistant seizures. After shadowing the epileptologist who performed the surgery, she got advice that would shape her career path. He told her, “My major frustration as a neurologist is I have very few treatments to offer my patients. So you go, make the next new drug, give me something I can give my patients.” Those words helped inspire Berit’s journey into translational medicine. Along the way she earned a Ph.D. at the University of Washington and completed her postdoctoral studies at Harvard University. At Ionis, she leads research on investigational medicines to treat Alexander disease and Pelizaeus-Merzbacher disease, two rare neurodevelopmental disorders affecting young children.

Elaine Pirie, Ph.D., Assistant Director, Metabolic Drug Discovery, Ionis Feb 02, 2023

What do sword fighting, fruit fly experiments and a fascination with protein aggregation in the brain have in common? They’re all activities that have consumed Dr. Elaine Pirie. A fencing enthusiast at young age, the St. Paul, Minnesota native started her scientific journey researching retinal degeneration in drosophila at the University of Wisconsin-Madison. Her desire to discover the cause of protein aggregation in ALS and other neurodegenerative diseases and an interest in genetics inspired her graduate and pos-graduate work, including three years as a research assistant in the lab of Nobel Prize-winner Dr. Bruce Beutler. At Ionis, Elaine’s research is focused on understanding the activity of GLP-1 conjugated antisense oligonucleotides in the beta cells of the pancreas. The goal: identifying and advancing new targets for Type 1 and Type 2 diabetes. So, what is Innovation to Dr. Elaine Pirie? It’s when we turn a problem to the side and explore it from a completely different angle. The solutions that come from looking at it in a different way, that’s innovation.

Ian Huggins, Ph.D., Assistant Director, Medicinal Chemistry, Ionis Dec 21, 2022

Starting with his idyllic “free range” childhood exploring nature in northern Utah, Ian has maintained an enduring curiosity about the world. At one point, he even imagined himself as a wildlife biologist tracking wolves in Yellowstone with a camera and notepad. As an undergrad he was fortunate to have mentors who cultivated his love of scientific discovery, which ultimately led him to a career in molecular biology research. His goal: helping to alleviate the suffering caused by disease. Today, he’s doing just that as a member of Ionis’ Medicinal Chemistry department. As part of the Receptor Biology team, Ian works to discover, design and produce ligands, including antibodies and peptides for Ionis’ advanced ligand conjugated antisense, or LICA, technology. So, what is “innovation” to Dr. Ian Huggins? Knowing where we need to be, knowing that we’re not there yet, and finding a way to build a bridge that gets us there.”

Crystal Zhao, Ph.D., Associate Director, Ion-Edit Core Research, Ionis Nov 16, 2022

From Nanjing, China to Woods Hole, Massachusetts to the sunny shores of La Jolla, California, and many points in between, Dr. Zhao’s journey to innovation has been full of discovery. With initial aspirations of becoming a marine biologist, she was ultimately attracted to Ionis because “I was amazed at how much basic research is done” at the company. Also driving her decision was a desire to apply her research to drug discovery that could eventually benefit patients. Now, she’s spearheading core research for an initiative intended to marry Ionis’ pioneering antisense technology with the emerging field of gene editing. So, what is “innovation” to Dr. Crystal Zhao? “It’s a mentality. It’s about our curiosity…It’s about the courage to test and go beyond what we know today.”

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