About Humvira Therapeutics (赫睿源)

Company Overview

Humvira Therapeutics (赫睿源) is a genetic medicine company developing next-generation in vivo gene therapy platforms for severe inherited diseases with high unmet medical need.

The company focuses on platform-driven development of complementary in vivo lentiviral and AAV gene delivery technologies designed to enable durable, single-administration therapeutic effects across multiple organ systems.

Humvira’s strategy centers on selecting the most biologically appropriate in vivo modality for each disease context to achieve long-term systemic correction rather than transient symptom control.

Platform Architecture — HUMx Gateway™

Humvira’s HUMx Gateway™ is a modular in vivo gene therapy framework designed to support scalable expansion across multiple genetically defined rare diseases while preserving a shared biological and delivery backbone.

This platform architecture enables:

· accelerated development across related disease indications

· shared vector and translational infrastructure

· consistent mechanisms of action across programs

· long-term scalability of the therapeutic pipeline

By building around platform architecture rather than isolated programs, Humvira translates advances in gene delivery into a sustainable pipeline of genetic medicines.

Pipeline Focus

Humvira’s development programs are organized around three core therapeutic areas:

· Central Nervous System Lysosomal Storage Disorders (CNS-LSD)

· Severe Combined Immunodeficiency (SCID) and Primary Immunodeficiencies

· Hemophilia A — Plenacogene parvovec (AAV5 / AAV8)

Each program is selected based on clear genetic causation, strong biological rationale for in vivo correction, and the potential for durable clinical impact.

Mission

Humvira’s mission is to develop in vivo genetic medicines capable of delivering durable, system-level correction of rare inherited diseases.

Through platform-driven innovation and rigorous translational science, the company seeks to expand the frontier of what is therapeutically possible in genetic medicine.

Company Information

Humvira Therapeutics (赫睿源)

East Lake High-Tech Campus

Wuhan 430074, China

info@humvira.com