Pannier sees 'the future is so bright' for gene therapy

Pannier says the ‘future is so bright’ for gene therapy

Development of vaccines for covid-19 in 2020 was a huge step forward for more than 150 years of genetic research and paved the way for an explosion of medical therapies that could save countless lives.

Angie Pannier, Swarts Family Chair in Biological Systems Engineering, delivered the Nebraska Lecture Nov. 17, outlining the history of her field as well as the challenges and promises ahead.

“It’s an exciting time to be in this business,” Pannier said. “This is an exciting time for a patient who could see their life dramatically changed by these therapies.”

Pannier, also a professor of biomedical engineering, paid tribute to many of the giants of science who have advanced genetic research, from Mendel and Morgan to Beadle, Watson, Crick and Franklin, as understanding shifted from identifying goals genes to learning how to manipulate them. and deliver them to patients’ cells to treat disease. There have been setbacks along the way, sometimes tragic, that have forced scientists and regulators to band together and rethink their approaches.

But progress has continued on gene therapies to fight diseases such as blood cancers, a disease that causes blindness, haemophilia and spinal muscular atrophy in infants.

When reports emerged from China in early 2020 of a new pneumonia-like disease, vaccine and gene delivery scientists “knew that…we were going to need a vaccine approach that would allow rapid manufacturing, very efficient and effective results and the ability to mobilize it quickly and across the world,” she said.

The traditional approach to vaccine development has been to take the targeted virus, inactivate it in some way, and re-administer it to patients.

“It can take a long time to isolate the virus, grow it in massive amounts and purify it,” Pannier said. “That’s why it takes us months to prepare for a flu vaccine, for example.

“But there are other ways to achieve this, including using DNA Where RNA and deliver it with non-viral means or viral vectors,” she said. “And that’s where the field really took hold quite quickly because we knew we had decades of research in those areas, and we knew those were technologies that would get us up to speed quickly. “

double strand DNA and single-stranded RNA are the genetic stores of our cells — the former responsible for storing our genetic information and the latter for transmitting it to molecules that control our body’s functions, Pannier said.

Six of 11 covid-19 World Health Organization emergency approved vaccines use so far either DNA Where RNA to deliver disease-fighting genes.

“It gives me chills to say that this is an area I have been in throughout my college career and more than half of covid-19 vaccines use technology that in the late 1990s we feared might not reappear in a market,” Pannier said.

The vaccinations have been a global success and the future is bright, Pannier said. Effectiveness has been demonstrated for gene replacement and vaccines. There have been hundreds of applications to the Food and Drug Administration for clinical trials of gene therapy products over the past two years; we estimate the FDA will approve 10-20 therapies per year by 2025 after a period of about 30 years where only three were approved. And new advances in gene-editing technologies are also set to revolutionize cell and gene therapy.

But challenges also remain. The cost is important.

“These drugs are very expensive to manufacture,” Pannier said. “They cost a lot of money in R&D. Our insurance and healthcare systems are not well set up to price treatment. We must therefore meet these economic challenges.

Pannier’s lab is researching cheaper ways to deliver these therapies, which could lower their costs. One possible solution: to use extracellular vesicles, which are naturally released by most cell types, acting in cell-to-cell communication and transporting cargo from donor cells to recipient cells.

“We thought if the cells were already using it as a delivery vehicle, can we use it as a delivery vehicle for cargo DNA and RNA what do we want to deliver? »

Ultimately, Pannier said, this research could lead to the oral delivery of vaccines, a huge advance over a needle.

“There are a lot of advantages to oral administration of any medication: You have high patient compliance. Most people would rather take a medicine by mouth than get the shot. We do not need medical staff for administration. We have a large area where we can deliver the drug, and we can have a local or systemic response. »

Early results to date are promising.

“The future is so bright. I like teasing my students, it’s suddenly cool to work in the field of gene therapy,” Pannier said.

Nebraska Lectures: Chancellor’s Distinguished Speaker Series is offered once per semester, sponsored by the Office of Research and Economic Development, Office of the Chancellor, and Research Council, in conjunction with the Osher Lifelong Learning Institute. The Nebraska Lectures bring the University community together with the greater Lincoln community and beyond to celebrate the intellectual life of the University of Nebraska–Lincoln by showcasing faculty excellence in research and creative activity . The topics of these free lectures reflect the diversity of faculty achievement in the arts, humanities, social sciences, and physical sciences.

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