Guess the Therapy Answer April 8, 2026
Which gene therapy is currently under development by AskBio for NYHA class III nonischemic heart failure with reduced ejection fraction?
Answer: AB-1002.
See below for further reading on AB-1002:
October 22, 2025 — AskBio’s AB-1002, an investigational adeno-associated virus (AAV) vector-based gene therapy, has demonstrated the ability to improve New York Heart Association (NYHA) class in patients with NYHA class III nonischemic heart failure with reduced ejection fraction (HFrEF) who were treated in the phase 1 NAN-CS101 clinical trial (NCT04179643). The 12-month data from the trial were published in Nature Medicine.
According to the Nature Medicine paper, NAN-CS101 treated 6 patients with a dose of 3.25 × 1013 viral genomes of AB-1002 (cohort 1) and 5 patients with 1.08 × 1014 viral genomes of AB-1002 (cohort 2). The study population was composed of 9 men and 2 women. It was reported that 2 of 3 patients in cohort 1 with available data achieved NYHA class II as of 3 months posttreatment and 5 of 6 patients had achieved NYHA class I or II as of 6 months posttreatment, with these changes being maintained at 12 months posttreatment. For the 4 patients in cohort 2 who remained alive at 12 months posttreatment, 2 had achieved NYHA class II as of 3 months posttreatment and maintained this change at 12 months; the other 2 patients in the cohort showed no changes in NYHA class at any time point. In addition to the NYHA class improvements, the Nature Medicine paper notes that improvements in left ventricular ejection fraction were seen among patients in both cohorts and that in cohort 1 improvements in peak oxygen consumption and 6-min walk test performance were observed.
July 15, 2024 — AB-1002 gene therapy has demonstrated a manageable safety profile and some improvements in disease measures including 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 AAV vectors for a breadth of conditions.
