Murdoch Children’s Research Institute Debuts Gene Therapy Breakthrough in NYC
Supporters gathered at Hugh Jackman's Old Mates pub to learn about a novel gene therapy for pediatric heart disease.
Friends of the Murdoch Children’s Research Institute (MCRI) convened in New York City on Thursday night at Old Mates, an Australian pub co-owned by actor Hugh Jackman. The event served as a platform for Professor Enzo Porrello, MCRI’s Theme Director of Stem Cell Medicine and Head of the Heart Regeneration Group, to present a significant breakthrough in gene therapy for pediatric heart disease.
Porrello highlighted that heart disease affects approximately one in 100 children, equating to about 40,000 births in the U.S. annually. He emphasized the urgent need for more effective treatments beyond heart transplants, which he described as a last resort and not a cure.
During a panel discussion that included Old Mates co-owner Nicholas Stone and MCRI Co-Chair Sarah Murdoch, Porrello detailed the development of a "single shot injection" gene therapy. He reported that animal studies have demonstrated the therapy's ability to not only prevent the onset of heart disease but also to completely reverse existing conditions. Porrello expressed excitement about moving this therapy from the laboratory to clinical trials.
Sarah Murdoch, MCRI Co-Chair and Chair of the Global Advisory Board, spoke about the institute's legacy, referencing its founder Dame Elisabeth Murdoch. She noted the significant advancements in genetic understanding since MCRI's inception, with the institute now ranking among the top three globally in child health research.
Murdoch also commended Porrello's work, stressing the importance of researchers like him being situated within hospitals to directly observe affected children and identify solutions.
Goldman Sachs returned as the Presenting Sponsor for the second consecutive year, with Asena Advisors, Bondi Partners, and Fabletics also providing sponsorship. The event featured a raffle and a preview of MCRI’s new animation, "For All. Forever," created as part of the organization's 40th anniversary, which will be narrated by Hugh Jackman.