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January 22, 2024 — Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that it is initiating GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase 2 trial of AB-1002 (also known as NAN-101) for the treatment of congestive heart failure (CHF). GenePHIT is an adaptive, double-blind, placebo-controlled, randomized, multicenter trial to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy classified as New York Heart Association (NYHA) Class III Heart Failure who have been medically stable for at least 4 weeks.1 The advancement of AB-1002 into Phase 2 marks a significant milestone for this novel gene therapy for CHF and, if successful, could bring this important investigational therapy one step closer to treating patients with high unmet medical need.
GenePHIT will include between 90 and 150 adults with left ventricle ejection fraction between 15 and 35%, who continue to suffer from heart failure symptoms despite guideline recommended therapy. The primary efficacy endpoint at 52 weeks is a modified win ratio of several clinically meaningful assessments.1
“AskBio is excited to initiate GenePHIT under the leadership of Roger Hajjar, MD, Scientific Chair CHF, and Lothar Roessig, MD, Integrated Product Team Lead CHF,” said Jude Samulski, PhD, Co-Founder and Chief Scientific Officer, AskBio. “We believe this trial will help determine the potential of AB-1002 as a treatment for one of the world’s most devastating diseases, and we look forward to learning more about this important investigational cardiac gene therapy. Our hope is that one day AB-1002 will potentially help patients suffering from congestive heart failure.”
The GenePHIT trial, which includes 52-week safety and primary efficacy and four-year long-term follow-up periods, is currently recruiting. AskBio plans to conduct the trial in the U.S. and multiple countries in Europe.1 For more information, please visit clinicaltrials.gov (NCT#05598333) or visit askbio.com.
AB-1002 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. AB-1002 is manufactured by Viralgen Vector Core, a wholly owned and independently operated subsidiary of AskBio.
About Congestive Heart Failure
Heart failure occurs when the heart cannot pump blood efficiently enough to meet the body’s needs, including providing sufficient oxygen to the organs.2 Congestive heart failure results in the slowing of the blood flow out of the heart, which causes the blood returning to the heart through the veins to back up.3 This causes congestion in the body's tissues. Symptoms include swelling in the legs and ankles. Sometimes, fluid collects in the lungs and interferes with breathing. Approximately 26 million people worldwide are living with congestive heart failure.4
For more information: www.askbio.com
References:
[1] Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure (GenePHIT). Available at: https://classic.clinicaltrials.gov/ct2/show/NCT05598333. Accessed January 2024.
[2] CDC. Heart failure. Centers for Disease Control and Prevention. Published 2022. Available at: https://www.cdc.gov/heartdisease/heart_failure.htm. Accessed January 2024.
[3] American Heart Association: Types of Heart Failure. Available at: https://www.heart.org/en/health-topics/heart-failure/what-is-heart-failure/types-of-heart-failure. January 2024.
[4] Savarese G, Lund LH. Global Public Health Burden of Heart Failure. Card Fail Rev. 2017;3(1):7-11. doi:10.15420/cfr.2016:25:2.
[5] Grieger JC, Soltys SM, Samulski RJ. Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector. Mol Ther. 2016;24(2):287-297.
October 28, 2025 
