YolTech to Present Positive Data from Clinical Trial of YOLT-201 in ATTR-CM Patients at European Society of Cardiology Congress 2026

Initial results support potential of YOLT-201 to be disease-modifying and curative treatment for ATTR-CM

SHANGHAI – August 26, 2026 - YolTech Therapeutics, a late clinical-stage biotechnology company pioneering in vivo gene-editing therapies, announced today that initial clinical data from an investigator-initiated trial (IIT) of YOLT-201 in transthyretin amyloidosis with cardiomyopathy (ATTR-CM) patients will be presented at the European Society of Cardiology (ESC) Congress 2026, taking place August 28–31, 2026, in Munich, Germany.

The ESC Congress is the world's largest and most influential cardiovascular conference, bringing together clinicians, researchers, and industry experts from around the globe to discuss the latest advances in cardiovascular medicine.

"ATTR-CM remains a progressive and life-threatening disease for which patients continue to face significant unmet medical need despite recent therapeutic advances. The preliminary clinical results from this study are compelling, demonstrating substantial and durable transthyretin (TTR) reductions alongside a favorable safety and tolerability profile. These findings support the potential of YOLT-201 as a one-time in vivo gene-editing therapy that may address the underlying cause of disease and provide meaningful long-term benefit for patients with ATTR-CM,” commented Hui Yan, M.D., Chief Physician from the First Affiliated Hospital at Zhejiang University School of Medicine. “We are highly encouraged to see the deep and durable TTR reduction with YOLT-201."

The robust clinical results from the IIT of YOLT-201 demonstrated manageable safety and clinically meaningful transthyretin (TTR) reduction. The study enrolled seven ATTR-CM patients. Patients treated with 1.0 mg/kg of YOLT-201 achieved a median serum TTR reductions of 96% from baseline, which was sustained through 52 weeks; repeat dosing restored pharmacodynamic response in patients who had initial inadequate suppression. No dose-limiting toxicities or treatment discontinuations were reported. Favorable safety and tolerability were observed. The most common treatment emergent adverse events (TEAEs) were infusion-related reactions. These findings support in vivo YOLT-201 as a potential one-time curative therapy for ATTR-CM patients.

Presentation Details

Presentation Title:         In vivo gene editing with YOLT-201 for transthyretin amyloid cardiomyopathy – an open-label, first-in-human clinical trial

Session:                         Improving Care in Cardiac Amyloidosis

Date and Time:              August 31, 2026 at 09:40 CEST

Venue:                           Messe München (International Congress Center Munich)

Location:                       Science Box 4 (Research Gateway – Hall A1)

Presenter:                      Hui Yan, M.D., Chief Physician from the First Affiliated Hospital at Zhejiang University School of Medicine

About Transthyretin Amyloidosis with Cardiomyopathy

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, debilitating and potentially fatal disease characterized by the accumulation of misfolded transthyretin (TTR) protein deposits in the heart, leading to impaired cardiac function, heart failure and reduced survival. ATTR-CM may result from inherited mutations in the TTR gene or occur as an age-related condition. The disease is estimated to affect approximately 300,000 to 500,000 individuals worldwide and remains substantially underrecognized and underdiagnosed. Despite advances in treatment, significant unmet medical need remains for therapies that target the underlying cause of disease. The global ATTR market is expected to grow to approximately $22.3 billion by 2035, underscoring the need for novel therapeutic approaches for patients living with ATTR.[i]

About YolTech Therapeutics

YolTech Therapeutics is a clinical-stage biotechnology company pioneering next-generation in vivo gene editing medicines for patients with serious genetic, cardiometabolic and autoimmune diseases. The company has built a fully integrated platform that combines HEPDONE™, its proprietary gene editing technologies, with AI-enabled discovery capabilities and targeted lipid nanoparticle (LNP) delivery systems designed to enable precise, one-time treatment with the potential for lifelong benefit. YolTech is advancing a broad portfolio of wholly owned and partnered programs spanning genetic diseases, cardiovascular and metabolic disorders, and autoimmune diseases, with multiple assets in clinical development. Founded on the vision of redefining genetic medicine through precision in vivo editing, YolTech is committed to developing transformative therapies that address the underlying cause of disease and improve outcomes for patients worldwide.

[i] J. Clin. Med. 2025, 14, 4785; Ando, et al. Orphanet J Rare Dis, 2013; Ruberg, et al. Circulation, 2012; Gertz, et al. Am J Manag Care. 2017;23:S107-S112; Neurol Ther. 2020;9:135-149.; Int J Cardiol. 2018;270:192-196