Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart Failure

“One thing that's really interested me is the different approaches that people are taking to AAV therapy. I think AB-1002 is quite unique because we're looking at a really big indication rather than the sort of ultra rare diseases that many investigators are focused on. So, for me, it's been quite interesting to see the sheer breadth of conditions that are being presented here.”

AB-1002 gene therapy has demonstrated a manageable safety profile and some improvements in disease measures including New York Heart Association (NYHA) class III functional score, Left Ventricular Ejection Fraction, and quality of life measures in patients with congestive heart failure. Updated data from a phase 1 trial (NCT05598333) evaluating AB-1002 were presented by Luke Roberts, MBBS, PhD, the medical director of Clinical Development at Asklepios BioPharmaceutical (also known as AskBio), at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland.

CGTLive^® spoke with Roberts to learn more about the findings from the trial. He overviewed 1 patient death that occurred during the trial after treatment with AB-1002, the circumstances of the death, and why it was classified as unrelated to AB-1002 treatment; after coming to this agreement regarding the death, the study, which had been paused, resumed dosing. Roberts noted that other patients dosed since then have been doing well. Roberts also shared his excitement for new research being presented at the AGSCT meeting, including a focus on new approaches with adeno-associated virus (AAV) vectors for a breadth of conditions.

Click here to read more coverage of the 2024 ASGCT Annual Meeting.

REFERENCE

Roberts L, Henry T, Chung E, et al. Safety and Efficacy of AB-1002 Gene Therapy in Patients with Advanced Heart Failure: Results from an Ongoing Phase 1 Clinical Trial. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #9