Luke Roberts, MBBS, PhD, on Early Clinical Data on Congestive Heart Failure Gene Therapy


The medical director of clinical development at AskBio discussed safety and efficacy data from a phase 1 trial of AB-1002.

“For peak VO2, we saw improvements and stabilization in the low dose cohort. And in the high dose cohort, we saw some worsening in 3 patients compared to baseline. It wasn’t very clear why that might be the case... But we're going to conduct further studies. And that'll give us a better indication as to what might really be happening. But those patients interestingly, also exhibited improvements in NYHA functional score, ejection fraction, and quality of life. So, it's very particular to peak VO2.”

AB-1002 gene therapy was well-tolerated and showed some improvements in patients with congestive heart failure, including New York Heart Association (NYHA) class III functional score, Left Ventricular Ejection Fraction, and quality of life measures.

Data from the phase 1 trial of AB-1002 were presented at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland, by Luke Roberts, MBBS, PhD, Medical Director, Clinical Development, Asklepios BioPharmaceutical.

CGTLive® spoke with Roberts to learn more about the updated data from the trial. He gave an update on the trial’s enrollment and progress in dosing, as well as an overview of adverse events across the trial’s 2low dose and high dose cohorts. He shared improvements in disease measures that were observed but noted that there were not clear benefits seen with the 6-minute walk test times and peak VO2 measures. Notably, 3 patients in the higher dose cohort had declines in peak VO2 while still improving on other measures. The cause of these declines is currently being further investigated.

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

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
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