Penn Medicine, CHOP team awarded Breakthrough Prize for developing gene therapy for inherited blindness | Penn Today
Archived: 2026-04-23 17:11
Penn Medicine, CHOP team awarded Breakthrough Prize for developing gene therapy for inherited blindness | Penn Today
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Their discovery started with a group of blind dogs living at a vet school. Now, the work has been awarded the prestigious Breakthrough Prize at the “Oscars of Science.”
Jean Bennett
, and
Albert Maguire
, both emeritus professors of ophthalmology in the
Perelman School of Medicine
at the University of Pennsylvania, and
Katherine High
, an emeritus professor of pediatrics and the founding director of the
Raymond G. Perelman Center for Cellular and Molecular Therapeutics
at
Children’s Hospital of Philadelphia
(CHOP), have received the
Breakthrough Prize in Life Sciences
for their work in developing the first FDA-approved gene therapy for an inherited condition, which dramatically improves sight in people with a form of blindness called Leber Congenital Amaurosis (LCA).
View large image
Katherine High is a recipient of the 2026 Breakthrough Prize in Life Sciences.
(Image: Courtesy of CHOP)
Their work blazed a trail for the more than 140 gene therapy trials for retinal conditions, including macular degeneration and diabetic retinopathy, diseases that collectively impact about 30 million people in the US. Eighty more trials are currently underway.
“Even 20 years ago, treating people with gene therapy was seen by some as an impossibility,” says
Jonathan Epstein
, dean of the Perelman School of Medicine and executive vice president of the University of Pennsylvania for the Health System. “But this group of incredible physician-scientists persisted and created something that is providing sight to people who would have been completely blind as early as kindergarten. Their belief in the power of life-changing science has led to breathtaking results and richly deserved global recognition.”
The Breakthrough Prizes are called the “Oscars of Science” for their high-profile celebration of research and support from celebrities spanning numerous areas of pop culture. Created in 2012 by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Yuri and Julia Milner, and Anne Wojcicki, the prizes are given out in five categories including Life Sciences, Fundamental Physics, and Math, each with an accompanying $3 million award.
View large image
Mathew Madhavacheril is an assistant professor of physics and astronomy.
(Image: Courtesy of the School of Arts & Sciences)
This year’s accolade now means that nine
Penn-affiliated researchers
have received the Breakthrough Prize, tied for the most with Harvard University. The prior Penn Medicine award winners are
Carl June (2024)
,
Drew Weissman and Katalin Karikó (2022)
, and
Virginia M.Y. Lee (2019)
. Additionally, Penn faculty members
Charles Kane and Eugene Mele
from the
School of Arts & Sciences
(SAS) won the prize for Physics in 2019.
Eight other faculty members from SAS have been part of research groups that were awarded Breakthrough Prizes for Fundamental Physics, as well: A Penn team
won in 2016
as part of the Sudbury Neutrino Observatory Collaboration (
Eugene Beier
,
Joshua Klein
, and
Christopher Mauger
)
and
in 2025
as part of the ATLAS Collaboration at CERN
(
Joseph Kroll
,
Evelyn Thomson
,
Elliot Lipeles
,
Dylan Rankin
, and
Brig Williams
,
part of the Penn ATLAS Group).
Mathew Madhavacheril
, an assistant professor of physics and astronomy in SAS, also received recognition at this year’s Breakthrough Prize ceremony when he was honored with the New Horizons in Physics award, given to researchers early in their careers.
“Science is rarely a straight path, and those who make the most profound discoveries are resilient and persistent, overcoming obstacles along the way,” says Penn President
J. Larry Jameson
. “That is exactly what I see in this year’s awardees, and it has been true of all our remarkable faculty who have been recognized for scientific breakthroughs. Whether they are discovering what lies beneath Alzheimer’s Disease, curing cancer by engineering a patients’ own immune cells, or reversing blindness—they have persisted with imagination and rigor. Their steadfastness has pushed the boundaries of what medicine can achieve.”
“Developing cell and gene therapies has long been a top priority for our organization,” says
Madeline Bell
, CHOP’s CEO. “This breakthrough is the result of decades of investment and collaboration, and reflects our commitment to translating scientific discoveries into therapies that will transform patients’ lives. It has paved the way for many more cell and gene therapy innovations and has given hope to families around the world.”
Read more at
Penn Medicine News
.
View large image
(From left) Jean Bennett, Al Maguire, and Katherine High at the Breakthrough Prize ceremony.
(Image: Courtesy of Penn Medicine)
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Skip to Content
Skip to Content
News from
University of Pennsylvania
Try Advanced Search
Their discovery started with a group of blind dogs living at a vet school. Now, the work has been awarded the prestigious Breakthrough Prize at the “Oscars of Science.”
Jean Bennett
, and
Albert Maguire
, both emeritus professors of ophthalmology in the
Perelman School of Medicine
at the University of Pennsylvania, and
Katherine High
, an emeritus professor of pediatrics and the founding director of the
Raymond G. Perelman Center for Cellular and Molecular Therapeutics
at
Children’s Hospital of Philadelphia
(CHOP), have received the
Breakthrough Prize in Life Sciences
for their work in developing the first FDA-approved gene therapy for an inherited condition, which dramatically improves sight in people with a form of blindness called Leber Congenital Amaurosis (LCA).
View large image
Katherine High is a recipient of the 2026 Breakthrough Prize in Life Sciences.
(Image: Courtesy of CHOP)
Their work blazed a trail for the more than 140 gene therapy trials for retinal conditions, including macular degeneration and diabetic retinopathy, diseases that collectively impact about 30 million people in the US. Eighty more trials are currently underway.
“Even 20 years ago, treating people with gene therapy was seen by some as an impossibility,” says
Jonathan Epstein
, dean of the Perelman School of Medicine and executive vice president of the University of Pennsylvania for the Health System. “But this group of incredible physician-scientists persisted and created something that is providing sight to people who would have been completely blind as early as kindergarten. Their belief in the power of life-changing science has led to breathtaking results and richly deserved global recognition.”
The Breakthrough Prizes are called the “Oscars of Science” for their high-profile celebration of research and support from celebrities spanning numerous areas of pop culture. Created in 2012 by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Yuri and Julia Milner, and Anne Wojcicki, the prizes are given out in five categories including Life Sciences, Fundamental Physics, and Math, each with an accompanying $3 million award.
View large image
Mathew Madhavacheril is an assistant professor of physics and astronomy.
(Image: Courtesy of the School of Arts & Sciences)
This year’s accolade now means that nine
Penn-affiliated researchers
have received the Breakthrough Prize, tied for the most with Harvard University. The prior Penn Medicine award winners are
Carl June (2024)
,
Drew Weissman and Katalin Karikó (2022)
, and
Virginia M.Y. Lee (2019)
. Additionally, Penn faculty members
Charles Kane and Eugene Mele
from the
School of Arts & Sciences
(SAS) won the prize for Physics in 2019.
Eight other faculty members from SAS have been part of research groups that were awarded Breakthrough Prizes for Fundamental Physics, as well: A Penn team
won in 2016
as part of the Sudbury Neutrino Observatory Collaboration (
Eugene Beier
,
Joshua Klein
, and
Christopher Mauger
)
and
in 2025
as part of the ATLAS Collaboration at CERN
(
Joseph Kroll
,
Evelyn Thomson
,
Elliot Lipeles
,
Dylan Rankin
, and
Brig Williams
,
part of the Penn ATLAS Group).
Mathew Madhavacheril
, an assistant professor of physics and astronomy in SAS, also received recognition at this year’s Breakthrough Prize ceremony when he was honored with the New Horizons in Physics award, given to researchers early in their careers.
“Science is rarely a straight path, and those who make the most profound discoveries are resilient and persistent, overcoming obstacles along the way,” says Penn President
J. Larry Jameson
. “That is exactly what I see in this year’s awardees, and it has been true of all our remarkable faculty who have been recognized for scientific breakthroughs. Whether they are discovering what lies beneath Alzheimer’s Disease, curing cancer by engineering a patients’ own immune cells, or reversing blindness—they have persisted with imagination and rigor. Their steadfastness has pushed the boundaries of what medicine can achieve.”
“Developing cell and gene therapies has long been a top priority for our organization,” says
Madeline Bell
, CHOP’s CEO. “This breakthrough is the result of decades of investment and collaboration, and reflects our commitment to translating scientific discoveries into therapies that will transform patients’ lives. It has paved the way for many more cell and gene therapy innovations and has given hope to families around the world.”
Read more at
Penn Medicine News
.
View large image
(From left) Jean Bennett, Al Maguire, and Katherine High at the Breakthrough Prize ceremony.
(Image: Courtesy of Penn Medicine)
Share this article
Threads
Credits
Writer
From Penn Medicine News
Media Contacts
Frank Otto
More from
Perelman School of Medicine
School of Arts & Sciences
Medicine
Astronomy
Physics
Awards
Faculty
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Health & Medicine
Novel plant-based approach to a better, cheaper GLP-1 delivery system
Research led by Penn Dental’s Henry Daniell investigates the use of a lettuce-based, plant-encapsulated delivery platform as a new oral delivery of two GLP-1 drugs previously approved by the FDA in injectable form.
No brain, no gain: Neuronal activity enhances benefits of exercise
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Research led by Penn neuroscientist J. Nicholas Betley and collaborators finds that hypothalamic neurons are essential for translating physical exertion into endurance, potentially opening the door to exercise-mimicking therapies.
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Arts & Humanities
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In Becky Friedman’s English course Shakespeare in Love, undergraduate students analyze language, genre, and adaptation in the Bard’s plays through the lens of love.
Beating the heat: Designing cooling for bodies in motion
nocred
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Dorit Aviv, director of Weitzman’s Thermal Architecture Lab, studies how humans, technology, and design intersect, paving the way for the development of novel approaches to cooling people efficiently.