Friends of Murdoch Childrens Research Institute gather in NYC, learn about breakthrough gene therapy for pediatric heart disease

Friends of Murdoch Children’s Research Institute (MCRI) gathered at Old Mates — the haute Aussie pub co-owned by Hugh Jackman in NYC — on Thursday night.

Mixing downtown cool and authentic Australian pub fare — including sausage rolls, oysters Kilpatrick and pavlova — the packed event also featured Professor Enzo Porrello, MCRI’s Theme Director of Stem Cell Medicine and Head of the Heart Regeneration Group, revealing his latest inspirational breakthrough discovery in gene therapy.

“Heart disease in children is pretty common,” Porrello explained. “It affects one in 100 children… that’s about 40,000 children born in the US every year.”

In Porrello’s work at MCRI, “We’re interested in really the most severe forms of heart disease in children that can result in heart failure,” he said. “So the only treatment for heart failure is a heart transplant. And transplants are not a cure. It’s really a last resort. And we really need better therapies — more targeted therapies — they get to the root cause of disease.”

Appearing on a panel with Old Mates co-owner Nicholas Stone — a founder of Bluestone Lane, who’s also on the Global Advisory Board for MCRI — and MCRI Co-Chair and Chair of MCRI’s Global Advisory Board Sarah Murdoch, Porrello shared how he and MCRI researchers have created a therapy that’s a “single shot injection” that can cure certain heart disease.

“We’ve shown in animal studies that not only can this gene therapy prevent the development of heart disease,” Porrello told the rapt crowd. “It can actually completely reverse established heart disease… which is incredible. We’ve never seen anything like that in our sort of 15 to 20 years of doing this work.”

“And so we’re super excited now to take that gene therapy out of the lab and get it into clinical trials,” he said. “So our focus now is, ‘How do we move this as fast as possible.” He also outlined the critical process that would make the life-saving gene therapy a reality.

Murdoch paid tribute to the roots of the organization and MCRI co-founder Dame Elisabeth Murdoch in her remarks. “She was an extraordinary woman,” Murdoch said, addingthat when MCRI was founded, “We had not mapped the human genome, we didn’t know about genetics the way we do today. And we know that almost every disease has a genetic course. Fast forward to now, we’re top three in the world. It’s extraordinary. I think that what makes it special is that her determination… her legacy lives on deeply through the institute.”

Murdoch also praised Porrello at the Friends of MCRI New York 2026 gathering by saying, “You need researchers like Enzo… located in the hospital where they see the children unwell” to “find answers.”

“MCRI were grateful to Goldman Sachs, who returned as Presenting Sponsor for the second consecutive year, for their continued support and partnership,” organizers said.

Asena Advisors, Bondi Partners and Fabletics were additional sponsors.

There was also a fun raffle this year, plus a sneak peek of MCRI’s new “For All. Forever” animation — part of the organization’s 40th anniversary — which will be narrated by Hollywood star Jackman.