"Certainly, I've been anxious about data...I did an experiment, I put a lot of time into it, I'm hoping to get good results...we all know that feeling, right? The stakes are just so different here...this kid's life depends on whether this experiment worked."

--Kiran Musunuru, MD, PhD

One of the highlights of the ASGCT 28th Annual Meeting was the announcement on May 15 of a historic medical breakthrough: a child diagnosed with a rare genetic disorder had been successfully treated for the first time with a customized CRISPR gene editing therapy.

The infant, KJ Muldoon, was born with a rare metabolic disease called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. When he was just six months old, KJ received his personalized therapy at the Children's Hospital of Philadelphia (CHOP), where ASGCT Members Kiran Musunuru, MD, PhD; and Rebecca Ahrens-Nicklas, MD, PhD; led the group of researchers who developed KJ's treatment.

KJ is now growing and thriving. He recently left the hospital where he has spent the majority of his life. And the day after presenting the research that led to this first-of-its-kind development, Dr. Musunuru told us that there was so much more to the story.

Listen to our first episode of Soundbites of the Annual Meeting with Dr. Kiran Musunuru.

Did you attend the 28th Annual Meeting?

• Watch all sessions, including Dr. Musunuru's presentation, on demand on the virtual meeting platform through July 18!
• Not a member? Join now and receive 19 months of membership for the price of 12.

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

In honor of Molecular Therapy’s 25th anniversary, this episode is the second in a series hosted by Dr. Joseph Glorioso, Editor-in-Chief of Molecular Therapy, that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past 25 years.

In this episode, Dr. Glorioso will discuss the reviews, AAV vector development, back to the future, with author Sheila Mikhail; M34, Inc.; and The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy; with author Dr. Mark Kay, Stanford University.

If you enjoy this deep dive into research that is critical to the field, check out two more ASGCT events happening this fall: Advancing Cell and Gene Therapies for Cancer and Breakthroughs in Targeted In Vivo Gene Editing. During these events – in October and November, respectively – you’ll be able to hear directly from researchers about their own work related to these topics, or you can submit your own abstract for possible presentation. We hope to see you there!

Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

In honor of Molecular Therapy’s 25th anniversary, this episode is the first in a series hosted by Dr. Joseph Glorioso, Editor-in-Chief of Molecular Therapy, that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past 25 years by expert authors. In this episode, Dr. Glorioso will discuss two of these reviews, “Gene therapy then and now: A look back at changes in the field over the past 25 years,” with author Dr. Terry Flotte, UMass Chan Medical School, and “From concept to cure: The evolution of CAR-T cell therapy,” with author Dr. Carl June, University of Pennsylvania.

Want to learn about more recent developments in the field directly from the people who are doing the research? There's still time to register and join us for ASGCT’s 2025 Annual Meeting, May 13 to 17, in New Orleans or virtually! If you become a member of the Society, you can save $465 on your registration for the premier event in the field. Check out the full program and register for the meeting today!

'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.