EVast Bio Achieves World's First Exosome Therapy Application for Knee Osteoarthritis

/EIN News/ -- MIAMI, March 05, 2025 (GLOBE NEWSWIRE) -- EVast Bio, a U.S.-based clinical-stage biotech company, has achieved an important milestone by conducting the world’s first human application of small extracellular vesicles (sEV) also called exosomes for osteoarthritis (OA) - EVA-100. According to a recent study published in the Journal of Nanobiotechnology, this achievement demonstrates promising results in inflammation control and cartilage protection offering a new therapeutic option for millions of patients suffering from OA. The research showcases a comprehensive development pipeline, from concept to first-in-human validation, marking a significant breakthrough in the treatment of knee osteoarthritis using clinical-grade sEV.

Osteoarthritis, a debilitating joint disease characterized by cartilage degradation and chronic inflammation, affects millions worldwide. Current treatments primarily focus on symptom management. This new study introduces a novel therapeutic approach using small extracellular vesicles (sEV) derived from umbilical cord mesenchymal stromal cells (UC-MSC) offering a potentially longer-lasting disease-modifying benefit.

“Our research marks a significant advancement in anti-inflammatory therapeutics with dual protective and regenerative potentials for osteoarthritis treatment,” the research team explains. “We've developed a standardized, reproducible process for manufacturing clinical-grade extracellular vesicles, demonstrating remarkable potential in both preclinical and initial human studies,” comment Dr. Maroun Khoury, co-founder and Chief Scientific Officer of EVast Bio.

“EVA-100 could revolutionize osteoarthritis treatment, offering a transformative solution for millions if preclinical results are confirmed in both safety and efficacy clinical trials. Our approach aims to go beyond symptom relief by exploring the potential for cartilage regeneration, a key differentiator from existing therapies,” commented Matías Vial, Co-founder & CEO of EVast Bio.

Key Research Findings:

Innovative Manufacturing Process

The team established a standardized manufacturing protocol that meets Good Manufacturing Practice (GMP) standards, ensuring consistent production of clinical-grade sEV. This achievement marks a crucial step in bridging the gap between laboratory research and clinical application.

Highly Reproducible and Standardized sEV Manufacturing at EVast Bio

EVast Bio’s proprietary platform has demonstrated a high level of reproducibility and standardization in the production of small extracellular vesicles (sEVs). Advanced molecular profiling of our sEVs has identified a consistent set of microRNAs (miRNAs) and proteins, reinforcing their stability and reliability for therapeutic applications. Notably, our research shows that a core group of highly specific miRNAs and proteins is consistently present across multiple donor-derived sEV samples.

Mechanism of Action

The study provides insights into how sEV combat osteoarthritis at the cellular level. Our researchers discovered that EVA-100 create an anti-inflammatory environment within affected joints by strategically guiding macrophages toward an anti-inflammatory state. This mechanism is supported by comprehensive miRNA and proteome analysis, highlighting the sophisticated molecular approach of the treatment.

A recently published study by the the Western Ontario Registry for Early OA (WOREO) Knee Study team suggests that while synovitis increases in OA, the exhaustion and dysfunction of immune-regulatory macrophages are linked to greater pain, making them a promising therapeutic target.

Preclinical Success

In preclinical studies using an OA mouse model, intra-articular injection of EVA-100 demonstrated positive therapeutic effects including hyaline cartilage regeneration – restoring joint function and mobility, a prolonged retention of sEV in the knee joint – ensuring sustained therapeutic action.

Pioneering a New Standard for OA Treatment

The therapy, EVA-100, leverages clinical-grade sEV derived from umbilical cord mesenchymal stromal cells (UC-MSC) and has shown transformational potential. Key highlights include:
-World-First Human Application: EVA-100 was administered to a 56-year-old OA patient, achieving a significant WOMAC score reduction within six months and validated cartilage regeneration through third-party evaluation.
-Regenerative Impact: Evidence of increased cartilage volume (WATSc) six months post-treatment underscores the therapy’s capacity to restore joint health.
-Strong Safety Profile: No adverse effects were reported during a 12-month follow-up, further validating the therapy’s suitability for continued clinical development.

Beyond Osteoarthritis: A Next-Generation Drug Delivery Platform for small molecules.

EVast Bio’s proprietary exosome-based platform extends far beyond osteoarthritis, offering a vehicle for advanced drug delivery across multiple therapeutic areas. By leveraging the natural tissue-targeting, immune-modulating, and cargo-loading capabilities of extracellular vesicles (EVs), EVast Bio is developing engineered exosomes designed for targeted delivery of small molecules, RNA therapeutics, and biologics. This customized approach has the potential to enhance bioavailability, reduce systemic toxicity, and enable new treatment strategies in oncology, neurodegenerative diseases, metabolic disorders, and autoimmune conditions.

With a robust GMP-compatible manufacturing process, strong IP portfolio, and growing clinical pipeline, EVast Bio is connecting with pharma and biotech partners to co-develop next-generation EV-based therapeutics that go beyond traditional drug delivery methods.

A Commercial Model Driving Innovation

EVast Bio is leading the exosome revolution in regenerative medicine with a clear strategy for commercialization and expansion:
-Advanced Clinical Trials: EVA-100 is currently in Phase I trials (NCT06431152) with a dose-escalation protocol designed to refine safety and efficacy metrics.
-Promising Results: Combined preclinical and clinical data showcase potential for cartilage repair and long-term joint health.
-Pipeline Expansion: EVA-100+ is under development as an advanced drug-loaded exosome, including RNA loading targeting age-related conditions.
-Strategic Partnerships: Actively pursuing collaborations to scale and license its proprietary sEV technology

Looking Ahead

The successful development and validation of this novel therapeutic approach opens new possibilities for OA treatment but also reinforces EVast Bio’s position as a global leader in regenerative medicine and extracellular vesicle (EV) therapies. The research team has already initiated an early-phase clinical trials initiated this year; as detailed in the recently published, peer reviewed study.

“This comprehensive research pipeline, from development through preclinical testing to initial human validation, provides a strong foundation for advancing extracellular vesicle therapy in clinical practice,” the researchers note. “The potential to offer a safe, effective, and locally-administered treatment for osteoarthritis patients represents a significant step forward in regenerative medicine,” highlights Dr. Maroun Khoury, who is also IMPACT Center director and CSO of Cells for Cells (C4C)—the center of excellence and biotech company sponsoring the study. C4C, the parent company from which EVast Bio was born, has played a crucial role in pioneering this innovation. EVast Bio, its US-based spinoff, presented the groundbreaking results of the first clinical case and trial design evaluating the safety and efficacy of EVA-100. These findings were showcased at prominent conferences, including ISCT and ISEV meetings held in San Francisco, Paris, Lyon, and Vancouver.

To fully leverage its exosome platform, C4C is launching a US-based spinoff focused on osteoarthritis (OA) and longevity. This new venture seeks to raise $18 million to fund the Phase IB studies of EVA-100 and accelerate the development of EVA-100+, an advanced drug-loaded exosome therapy targeting age-related conditions. The company is also pursuing co-development partnerships and expanding its portfolio with customized membrane-engineered and drug-loaded exosomes for additional therapeutic indications based on partners’ interest. This strategic initiative strengthens EVast Bio as a leader in the $2.8 billion US exosomes market, aiming to deliver disease-modifying therapies to an estimated six million eligible patients. In osteoarthritis alone, the Serviceable Addressable Market (SAM) in the US is estimated at $7 billion, highlighting the vast commercial potential of exosome-based therapies in this space.

About the Research

Published in Journal of Nanobiotechnology (volume 23, Article number: 13, 2025), this study represents a collaborative effort to address one of the most common joint diseases affecting global health. The research combines expertise in regenerative medicine, cell biology, and clinical translation to develop an innovative therapeutic approach for osteoarthritis treatment.

For more information, please refer to the full study in the, Journal of Nanobiotechnology,

https://jnanobiotechnology.biomedcentral.com/articles/10.1186/s12951-024-03088-x

About EVast Bio: www.evast.bio

EVast Bio is a US-based clinical-stage biotech spinoff of Cells for Cells and Delaware C Corp pioneering the development of small Extracellular Vesicle (sEV) therapeutics. The proprietary platform harnesses these natural nano-vehicles – microscopic particles released by cells that facilitate intercellular communication – to create next-generation targeted delivery systems. The company innovative approach combines advanced sEV selection technology with precision engineering to develop therapeutics with enhanced safety and efficacy profiles. Through our patented technology, we produce sEVs with unique molecular signatures specifically tailored to target disease pathways. By optimizing drug delivery and reducing adverse events, EVast Bio is transforming the treatment landscape for challenging diseases. Our international clinical-stage pipeline demonstrates the potential of sEV technology to revolutionize therapeutic delivery and advance precision medicine.

About Cells for Cells:

C4C. (www.c4c.cl) is a clinical-stage company developing biotechnology platforms in South-America, including allogeneic cell therapies and extracellular vesicles. The company’s mission is to enhance global healthspan by addressing key hallmarks of high-prevalence chronic diseases.

C4C previously raised $40 million USD, reinforcing its leadership in the biotechnology sector and accelerating the development of innovative therapies to improve patient outcomes worldwide. C4C is committed to time-sensitive and cost-efficient clinical and IP validation, a strategy that enable it to the first-ever clinical proof-of-principle of an exosome-produced therapy targeting joint disease.

Contact:
CEO: mvial@c4c.cl
Scientific related interest: maroun@evast.bio
Business development interest: dsegura@c4c.cl