From Breakthrough to a New Frontier

Philanthropy is helping Penn researchers unlock novel treatments for diseases using mRNA technology

In science, the word “breakthrough” is often used to describe the field’s most important advances.

The word itself, however, can be a bit misleading. The breakthrough is almost never the result of a single moment of inspiration; nearly every innovation comes from years—or even decades—of methodical research that painstakingly uncovers new ways to understand the world.

Penn Medicine researchers Katalin Karikó and Drew Weissman did that hard work. Since the late 1990s, Karikó and Weissman had been exploring how to safely use messenger RNA (mRNA) to create vaccines and new treatments for diseases. Their research didn’t gain much traction until the COVID-19 pandemic. Suddenly, the world needed new vaccines—quickly. Karikó and Weissman’s discoveries were the blueprint for creating the mRNA-based COVID-19 vaccines that were credited with saving millions of lives, and, in 2023, they were awarded the Nobel Prize in Physiology or Medicine.

Their decades of research changed the world when it was most needed. And now, from that seed of knowledge, mRNA is poised to continue changing the world.

In 2022, Penn launched the Institute for RNA Innovation and in November 2023 opened its facility at One uCity Square. Directed by Weissman, the Roberts Family Professor in Vaccine Research, and Co-Directed by James Hoxie, Emeritus Professor of Medicine and former Director of the Penn Center for AIDS Research, the Institute is a hub for researchers across different fields and from various institutions who are studying the myriad ways RNA can be used for new vaccines and treatments.

With over 1,000 members, it encourages meaningful interaction between faculty members who otherwise might never connect. While it took a chance encounter at a copy machine for Weissman and Karikó to meet and begin working together, the Institute brings scientists together with intention. And not just at Penn—the Institute has a truly global reach, collaborating with over 250 other labs around the world.

A portrait of Drew WeissmanWe hope the institute will bring together researchers across Penn and the world to collaborate and advance basic science through therapeutic development.”Drew Weissman
Roberts Family Professor in Vaccine Research
Director of the Institute for RNA Innovation

Philanthropy—including the support of Brian and Aileen Roberts—has been a key driver for mRNA research. In addition to endowing Weissman’s professorship, the Aileen K. and Brian L. Roberts Family Foundation created the Roberts Family-Katalin Karikó Fellowship in Vaccine Development to provide support for early-career scientists in the field of immunology.

“The Penn Institute for RNA Innovation enables investigators on the Penn, CHOP, and Wistar campus to benefit from the advances and new tools that are becoming available in RNA science,” says Hoxie. “We are excited by the impact this field will have for basic, clinical, and translational research, and our campus is the ideal place to develop collaborations and resources that will make new discoveries possible.”

The years of research on mRNA have yielded not only vital COVID-19 vaccines, but an entirely new therapeutic platform that is changing the field of medicine. Researchers are working quickly toward treatments for some of today’s most prevalent diseases, such as cancer and heart disease. And scientists are looking for new uses for mRNA technology every day.

An informational graphic on a deep purple background highlighting efforts to develop a new malaria vaccine using mRNA technology. It features an icon of a mosquito on a heart-like shape, and text stating collaboration with global researchers. The card notes that in 2022 there were approximately 249 million malaria cases worldwide, mainly in Africa. It mentions that existing vaccines are only about 40% effective and are given yearly, while the new mRNA vaccines promise greater efficacy and longevity.

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An educational graphic about the use of mRNA technology in treating Sickle Cell Anemia. The background is deep maroon with a white circle containing a stylized icon of a red sickle cell. The text explains the complications faced by patients with sickle cell anemia due to the stiffness and misshapen nature of their blood cells. It highlights that approximately 300,000 people are born with this inherited condition annually, mainly in Africa and India. The text describes ongoing research efforts to develop a therapy using mRNA to instruct bone marrow cells to repair the genetic defect responsible for sickle cell anemia, potentially leading to a permanent cure with a single injection.

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An educational graphic about the use of mRNA technology in treating heart failure. The background is dark green with a stylized icon of a red heart containing a white ECG line in a white circle. The text describes how heart muscles weaken and become fibrous, leading to heart failure. It explains that researchers have developed a technique using mRNA to instruct T cells to attack the fibrous material, thereby reducing the risk of heart failure. This treatment could also potentially be used for treating fibrosis in the kidneys, lungs, liver, skin, and joints.

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An educational graphic on mRNA treatments for rare diseases. The background is purple with a white circular icon containing a stylized drawing of a DNA double helix. The text explains that researchers are developing mRNA treatments to replace genes or deliver therapeutic proteins to treat and potentially cure rare genetic diseases, including those affecting newborns. It also mentions advances in lipid nanoparticle-targeted gene editing as a promising approach for correcting mutations in DNA for the rarest diseases.

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An informative graphic about cancer vaccines using mRNA technology. The background is a deep maroon color with a white circular icon at the top featuring a stylized red virus. The text explains that mRNA technology, which is used in vaccines for COVID-19, is also being explored to fight cancer by activating the immune system to target cancerous cells within a patient. It notes that mRNA-based approaches are being evaluated for cancer prevention in high-risk individuals.

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“RNA therapeutics represent a paradigm shift in disease treatment, providing unprecedented versatility and precision,” says Sara Cherry, John W. Eckman Professor of Medical Science and Therapeutics Group Lead at the Institute. “The collaboration among diverse RNA-focused researchers at Penn fosters innovation, promising new avenues for impactful contributions in the field well into the future. It’s a very exciting place to be.”

“The Institute for RNA Innovation has already led to many impactful collaborations amongst Penn students and faculty and serves as a magnet to recruit new faculty to Penn who bring unique expertise to our campus to enable inventive new directions of research,” adds Kristen Lynch, Benjamin Rush Professor of Biochemistry and Basic Science Group Lead at the Institute.

In addition, the Institute is working with the Annenberg School for Communication and other partners across campus to combat vaccine misinformation in the public and collaborate with governments around the world to help them affordably and sustainably manufacture their own vaccines.

The blossoming of new mRNA treatments came from years of dedicated research by Karikó and Weissman. Their breakthrough became a foundation—one that will support decades of new advances in medical science. And at the same time, in a lab somewhere at Penn, other researchers are quietly working on the next great scientific discovery, one that we won’t know about for years.


The groundbreaking advances at the Institute for RNA Innovation are powered by the support of Penn donors. To help researchers develop the next generation of mRNA therapeutics, contact Andrew Bellet at (215) 573‑0548 or abellet@upenn.edu.

A vibrant and abstract illustration depicting various colorful, layered scenes against a gradient blue and yellow background. Each segment showcases different activities and settings: the top features a serene outdoor scene with a figure meditating, the middle segments depict lively social and technological environments with people interacting, and the bottom layers show educational and scientific setups.

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