Vesigen™ Platform

Revolutionary Breakthrough in Non-viral Gene Delivery

Protheragen provides one-stop R&D services for rare allergic diseases.

Platform Overview

Vesigen^TM Platform:
Naturally Targeted Gene Delivery

Gene therapy has made significant progress in recent years, particularly in the treatment of rare and genetic diseases. However, existing gene therapy delivery systems, such as viral vectors, have numerous limitations, including immunogenicity, toxicity, low delivery efficiency, and high production costs. Protheragen has developed a non-viral delivery platform based on megakaryocyte-derived extracellular vesicles (MkEVs), which naturally target bone marrow hematopoietic stem cells (HSCs), enabling efficient and safe gene therapy delivery.

Disruptive Technology
Excellent Team
Diversified Pipeline
Customized Solutions

Platform Core

Formation of Megakaryocyte-derived
Extracellular Vesicles (MkEVs)

The process begins with the expansion and differentiation of hematopoietic stem and progenitor cells (HSPCs) into megakaryocytes. These mature megakaryocytes then naturally produce and release extracellular vesicles (MkEVs) through specialized membrane budding and shedding mechanisms. This biologically derived method yields vesicles inherently capable of homing to bone marrow and carrying diverse therapeutic cargo.

Platform Advantages

Uniquely Advantages of the Vesigen™ Platform

Natural Targeting

Protheragen's megakaryocyte-derived extracellular vesicle (MkEV) platform is naturally bone marrow-targeted, enabling precise delivery of gene-editing payloads to hematopoietic stem and progenitor cells (HSPCs) through a membrane fusion mechanism. This approach avoids the immunogenicity and liver-targeting issues associated with viral vectors ( e.g., AAV) and lipid nanoparticles (LNPs).

Large Cargo Capacity

MkEV can carry a variety of gene-editing tools ( e.g., plasmid DNA, mRNA, proteins, etc. ) without significant payload size limitations, providing flexibility and versatility for complex gene therapies.

Repeat Dosing Potential

MkEV's immune-silencing properties support repeated dosing, overcoming the single-dose limitations of viral vector

Platform Application

Explore Multiple Therapeutic Applications

Gene Therapy for Blood Diseases The Vesigen™ Platform enables curative therapies for monogenic blood disorders such as sickle cell disease, Fanconi anemia, β-thalassemia, and X-SCID. It delivers gene-editing cargo directly to hematopoietic stem cells (HSCs) using naturally targeted MkEVs, offering the potential for lifelong disease correction.
In Vivo CAR-T Therapy Our platform pioneers a novel in vivo cell engineering approach by efficiently delivering chimeric antigen receptor (CAR) genes directly to T cells within the patient’s own body, bypassing the complex and costly traditional ex vivo manufacturing process and significantly expanding accessibility of CAR-T treatments.
Precision Oncology Leveraging the natural targeting properties and flexible payload capacity of MkEVs, the Vesigen™ platform enables targeted delivery of various therapeutic drugs, such as gene editors, tumor suppressors, or immunomodulators, to tumor sites, enabling highly precise and effective interventions in the tumor microenvironment.

Pipeline

Research & Development Pipeline

Projects	Target	Indication
GBD005	To be disclosed	Sickle Cell Disease
GBD009	To be disclosed	β-Thalassemia
GBD014	To be disclosed	X-SCID
GBD017	To be disclosed	Fanconi Anemia
GBD022	To be disclosed	Wiskott-Aldrich Syndrome

Get in Touch with Us

FAQs

Frequently Asked Questions

Q1: What is the key innovation of the Vesigen™ Platform? A: The Vesigen™ platform leverages megakaryocyte-derived extracellular vesicles (MkEVs) as a novel non-viral delivery system. Its breakthrough lies in combining innate bone marrow targeting, immune evasion, and the ability to carry large and diverse therapeutic payloads—overcoming critical limitations of viral vectors and LNPs.
Q2: Which diseases are you currently targeting? A: Our initial focus is on genetic blood disorders, including sickle cell disease, β-thalassemia, X-SCID, Fanconi anemia, etc. We are also expanding into in vivo CAR-T generation and targeted oncology applications.
Q3: What is the advantage of using MkEVs over viral vectors? A: Unlike viral vectors, MkEVs exhibit low immunogenicity, reducing the risk of immune reactions. They also allow for redosability, possess natural tissue tropism, and avoid genomic integration concerns associated with some viral systems.
Q4: How far advanced is your technology? A: Our platform is currently in the preclinical stage. We have generated compelling proof-of-concept data, including safety and efficacy results in non-human primate models, supporting its therapeutic potential.
Q5: Do you have plans for clinical trials? A: Yes. We are advancing our lead programs toward IND-enabling studies and are preparing to initiate clinical trials in the coming years, subject to regulatory approvals.

Vesigen™ Platform

VesigenTM Platform: Naturally Targeted Gene Delivery

Formation of Megakaryocyte-derived Extracellular Vesicles (MkEVs)