Background Information

Pompe disease is a rare, inherited lysosomal storage disorder caused by a deficiency of the enzyme acid alpha-glucosidase, leading to pathological accumulation of glycogen in skeletal muscle, cardiac muscle, and other tissues. Patients experience progressive muscle weakness, respiratory insufficiency, and, in severe infantile forms, life-threatening cardiomyopathy. Despite the availability of approved enzyme replacement therapies, Pompe disease remains a debilitating and ultimately life-limiting condition for many patients.

Current enzyme replacement therapies rely on mannose-6-phosphate-mediated uptake for cellular entry. However, skeletal muscle and diaphragm express relatively low levels of mannose-6-phosphate receptors, resulting in poor enzyme uptake into the tissues that are most critical for long-term functional outcomes. As a result, many patients continue to decline clinically despite years of treatment. In addition, frequent intravenous infusions, long administration times, and high rates of anti-drug antibody formation impose a substantial treatment burden and negatively impact quality of life.

NanoZymeX therapies are designed to overcome the inefficient intracellular delivery of therapeutic enzymes into diseased cells and solve the problem of immunogenicity. After intravenous administration, NanoZymeX lipid nanoparticles circulate systemically and protect the encapsulated enzyme from rapid degradation and immune recognition. The nanoparticle surface is engineered to promote cellular uptake through endocytic pathways that are independent of mannose-6-phosphate receptor binding. The particle itself protects the enzyme from interactions with the antibodies.

Following internalization, the nanoparticles are trafficked through the endosomal–lysosomal pathway. Within the acidic lysosomal environment, the nanoparticle structure destabilizes and releases the active enzyme payload. The liberated enzyme then performs its native catalytic function inside the lysosome, enabling functional substrate clearance. This delivery-driven mechanism allows NanoZymeX therapies to achieve higher effective intracellular enzyme levels in target tissues compared with free enzyme.

Preclinical Validation and Proof of Concept

NanoZymeX has generated a robust preclinical data package demonstrating the feasibility and advantages of enzyme delivery using its proprietary lipid nanoparticle platform. High encapsulation efficiencies were achieved for recombinant human acid alpha-glucosidase while fully preserving enzymatic activity during formulation. In vitro studies in muscle cell models confirmed efficient nanoparticle uptake and intracellular delivery, resulting in significantly higher intracellular enzyme activity compared with free enzyme.

In vivo biodistribution studies in mice demonstrated increased enzyme activity in skeletal muscle and diaphragm following administration of NanoZymeX-GAA compared with free enzyme at equivalent doses. Repeat-dose studies further indicated a favorable tolerability profile and reduced immunogenicity, with lower anti-drug antibody titers relative to conventional enzyme formulations. Together, these data support the translational feasibility of the NanoZymeX platform and its potential to enable next-generation enzyme replacement therapies.

While Pompe disease represents NanoZymeX’s lead indication, the company’s lipid nanoparticle platform is broadly applicable across a wide range of lysosomal storage disorders and other enzyme-deficiency diseases. Many of these conditions share common pathophysiological features, including poor tissue penetration of therapeutic enzymes, dependence on glycosylation-based uptake mechanisms, and high immunogenicity of repeated dosing.

NanoZymeX is actively advancing follow-on programs targeting additional lysosomal enzymes, including alpha-mannosidase, beta-galactosidase, and alpha-galactosidase. By leveraging the same core delivery technology, these programs can be developed more rapidly and efficiently than traditional de novo biologics. This platform strategy positions NanoZymeX to build a scalable pipeline of rare-disease therapies and to address multiple high-unmet-need indications using a single validated delivery engine.

Why NanoZymeX

NanoZymeX is building a first-in-class enzyme delivery platform designed to overcome the core limitation of current enzyme replacement therapies: inefficient intracellular delivery into diseased tissues. Its proprietary lipid nanoparticle technology actively transports therapeutic enzymes into target cells and lysosomes, bypassing glycosylation-dependent uptake mechanisms and enabling higher functional enzyme levels where they matter most, particularly in skeletal muscle and diaphragm.

The platform has demonstrated strong preclinical proof of concept, including high encapsulation efficiency with preserved enzyme activity, superior intracellular delivery in muscle cells, increased enzyme activity in skeletal muscle and diaphragm in vivo, and reduced immunogenicity compared with free enzyme. Combined with a scalable and manufacturing-friendly approach that decouples uptake from glycosylation requirements, NanoZymeX offers a differentiated, capital-efficient path to next-generation enzyme therapies.

Market Opportunity and Value Creation

The global lysosomal storage disorder market exceeds USD 9 billion annually, with Pompe disease representing a multi-billion-dollar segment that continues to show substantial unmet medical need despite multiple approved products. NanoZymeX’s lead Pompe program targets the tissues most poorly corrected by current therapies and aims to deliver clinically meaningful improvements in long-term functional outcomes and treatment burden.

Beyond Pompe disease, the NanoZymeX platform is broadly applicable across multiple lysosomal storage disorders and other enzyme-deficiency diseases, enabling rapid pipeline expansion from a single validated delivery engine. This platform breadth, together with strong intellectual property protection and a regulatory-aligned development strategy leveraging rare-disease incentives, supports venture-scale value creation and multiple partnership or exit opportunities.