Wednesday, December 31

Big prizes, biotech moves, and global recognition for Baby KJ.


Despite being one of the rockiest years yet for science — marked by millions of dollars in funding cuts and controversial shake-ups to the federal infrastructure — Philadelphia scientists still managed to celebrate many wins in 2025.

Some institutions expanded their research with new centers dedicated to autoimmunity, HIV, Williams syndrome, and drug development. Others won big grants to develop better drugs for asthma and study the causes of autism.

Local scientists published exciting research on treatments for type 1 diabetes and ovarian cancer, designed self-heating concrete, and proposed ways to turn toxic fungi, snake venom, and trees into medicine.

They also won national and international honors for work in physics, cancer research, and drug repurposing. And although no local scientists won a Nobel Prize this year, two at Monell Chemical Senses Center were recognized by its satirical counterpart, the Ig Nobel Prize.

Here are five notable Philly science wins from 2025:

1. Baby KJ is successfully treated with personalized gene editing therapy

Philadelphia-area child KJ Muldoon, now 16 months old, has already been called a “trailblazing baby” by the top scientific journal Nature and recognized by the publication as one of 10 people who helped shape science in 2025.

This international recognition came after his life-threatening genetic condition was successfully treated with a personalized gene editing therapy earlier this year by doctors at Children’s Hospital of Philadelphia and the Hospital of the University of Pennsylvania.

Baby KJ was born in August 2024 with a metabolic disorder that prevented his liver from being able to process protein. Called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, the disorder puts babies at risk of severe brain damage and is fatal more than half the time.

With few options to treat him, the CHOP and Penn team — led by doctors Kiran Musunuru and Rebecca Ahrens-Nicklas — opted for a gene-editing technology known as CRISPR to create a customized drug for KJ that would fix the genetic mutation that was driving his disease.

After receiving three doses, KJ was able to return home in June — ending his 307-day-long stay at the hospital. Though not a cure, the medication has dramatically improved his liver function and made the effects of his disease milder, doctors say.

» READ MORE: CHOP and Penn treated an infant with a rare disease by editing his genes

2. Penn physicists share the Breakthrough Prize in Fundamental Physics

This year, Penn physicists shared one of science’s biggest honors: the Breakthrough Prize.

They were among 13,000 scientists across more than 70 countries to be recognized for their involvement in particle physics experiments at the European Organization for Nuclear Research, known as CERN, in Switzerland.

These decades-long research collaborations have explored the fundamental structure of particles that make up the universe, using CERN’s Large Hadron Collider, a 17-mile-long particle accelerator.

The Penn team — consisting of more than two dozen scientists, including Joseph Kroll, Evelyn Thomson, Elliot Lipeles, Dylan Rankin, and Brig Williams — was specifically part of the ATLAS Experiment, which played a key role in the discovery of the Higgs boson particle, a critical particle in modern particle physics theory. The Higgs discovery helped confirm how fundamental particles acquire mass.

» READ MORE: Penn physicists share Breakthrough Prize with international team of researchers

3. David Fajgenbaum honored for drug repurposing research

Penn immunologist David Fajgenbaum received one of the nation’s oldest science prizes, the John Scott Award, this year for his pioneering work repurposing existing drugs for new uses.

He entered this field 15 years ago after a rare and deadly diagnosis of idiopathic multicentric Castleman disease nearly killed him. The disease had no approved treatment nor any treatment guidelines at the time.

Then a medical student at Penn, Fajgenbaum started collecting samples of his blood to test for abnormalities. The data helped him identify an existing drug called sirolimus — primarily given to organ transplant recipients — which has put him in remission for the last decade.

Now through his nonprofit Every Cure, Fajgenbaum has made it his mission to use AI technology to match available medications with rare, hard-to-treat diseases.

He published a case study in the New England Journal of Medicine in February, where his AI tool helped identify an off-label treatment for another patient with Castleman disease who, at the time, was entering hospice care after all available treatments had failed. As of that study’s publication, the patient has been in a yearslong remission.

» READ MORE: Penn immunologist David Fajgenbaum receives prestigious John Scott Award

4. Lilly Gateway Labs biotech incubator coming to Philly

Pharmaceutical giant Eli Lilly & Co announced in November its plans to open a Lilly Gateway Labs site — an incubator for early-stage biotech companies — in Center City.

It was a positive sign for a biotech scene that otherwise lags behind other cities.

The incubator, which will be Lilly’s fifth in the United States, will span 44,000 square feet on the first two levels of 2300 Market St. Since the program’s launch in 2019, companies at the other locations (in Boston, South San Francisco, and San Diego) have raised more than $3 billion from investors toward more than 50 therapeutic programs, according to Lilly.

Lilly plans to house six to eight companies at the Philadelphia location, with the goal of welcoming the first startups in the first quarter of 2026.

» READ MORE: Eli Lilly & Co. is opening a Lilly Gateway Labs biotech incubator in Philadelphia

5. Carl June wins international honors for CAR-T research

Penn cancer scientist Carl June added two more international prizes to his trophy case in September for his pioneering work engineering the body’s immune system to fight cancer.

June is known for developing the first FDA-approved CAR-T therapy, an immunotherapy in which regular immune cells are genetically modified to become cancer-killing super soldiers. It has revolutionized treatment for blood cancers, saving tens of thousands of lives since its first use in a 2010 clinical trial he co-led at Penn.

Though his past work is what won him the inaugural Broermann Medical Innovation Award and the 2025 Balzan Prize for Gene and Gene-Modified Cell Therapy this year, his lab has remained busy, working on ways to apply CAR-T to solid cancers, enhance the therapy for lymphoma, and even re-engineer cells inside the body.

June has also made moves on the biotech front: A company he co-founded with the purpose of applying CAR-T to autoimmune diseases, Capstan Therapeutics, was bought by AbbVie this summer for $2.1 billion.

» READ MORE: Penn’s CAR-T pioneer Carl June talks about the future of his award-winning work to train the body to fight cancer



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