2023 article

Adeno-associated virus-vectored erythropoietin gene therapy for anemia in cats with chronic kidney disease

Vaden, S. L., Kendall, A. R., Foster, J. D., New, H. L., Eagleson, J. S., May, J. L., … Wilson, J. M. (2023, October 17). JOURNAL OF VETERINARY INTERNAL MEDICINE.

By: S. Vaden n, A. Kendall n, J. Foster, H. New*, J. Eagleson, J. May, A. Traas, M. Wilson ...

co-author countries: United States of America 🇺🇸
author keywords: AAV; cats; red blood cells; renal disease
Source: Web Of Science
Added: October 30, 2023

A treatment of chronic kidney disease (CKD)-associated anemia in cats is needed. SB-001 is an adeno-associated virus-vectored (AAV)-based gene therapeutic agent that is administered intramuscularly, causing the expression of feline erythropoietin.We hypothesized that SB-001 injection would lead to a sustained increase in PCV in cats with CKD-associated anemia.Twenty-three cats with International Renal Interest Society (IRIS) Stage 2 to 4 CKD-associated anemia were enrolled at 4 veterinary clinics.In a prospective clinical trial, cats were treated with 1 of 3 regimens of SB-001 (Lo 1.2 × 109 genome copies [GCs] on Day 0; Lo ± Hi [supplemental 2nd dose of 3.65 × 109 GC on Day 42]; Hi 3.65 × 109 GC IM on Day 0) and followed for 70 days.A response to SB-001 at any time between Day 28 and Day 70 was seen in 86% (95% confidence interval 65, 97%) of all cats. There was a significant (P < .003) increase in PCV from Day 0 to Day 28 (mean increase 6 ± 6 percentage points [pp]; n = 21), Day 42 (8 ± 9 pp; n = 21), Day 56 (10 ± 11 pp; n = 17), and Day 70 (13 ± 14 pp, n = 14). Twelve cats were hypertensive at baseline, 4 of which developed encephalopathy during the study. An additional 6 cats became hypertensive during the study.Results of this study suggest that SB-001 therapy represents a suitable single injection treatment that can address nonregenerative anemia in cats with CKD. It was generally well tolerated; however, hypertension and encephalopathy developed in some cats as previously described in association with erythropoiesis-stimulating agent therapy.