Our Pipeline

ExoVectory is committed to treating the diseases that medicine has not yet been able to cure, giving patients a future that their diagnosis once made unthinkable. We are focused on two therapeutic approaches: T-cell therapy for cancer, and gene therapy for inherited diseases of the brain and heart. Both are made possible by our exosome platform — the delivery system that gets treatment to places nothing else can reach. The diseases we have chosen (PCNSL, CNS disorders, and inherited cardiac conditions) all share the same problem: existing treatments cannot get to them. Ours has the potential to, and that is why we chose them.

Ex Vivo

Our ex vivo therapeutic programme uses ExoVec to engineer CAR/TCR T-cells for the treatment of cancer, focusing on primary central nervous system lymphoma (PCNSL) — a rare and aggressive brain cancer with limited treatment options and a 3-year survival rate of just 30% — as our lead indication.

PCNSL

In Vivo

Our in vivo programme is developing ExoVec as a systemic gene therapy delivery platform for cardiac and CNS diseases, where delivering large therapeutic DNA constructs safely to the target tissue remains the central unsolved challenge in the field.

CNSHeart

TARGET INDICATIONS

Primary Central Nervous System Lymphoma (PCNSL)

PCNSL is a rare and highly aggressive brain cancer that develops in the brain, spinal cord or eyes. With limited treatment options beyond high-dose chemotherapy and a poor prognosis, it represents a significant unmet medical need. In partnership with Leiden University, ExoVectory is using ExoVec to deliver a proprietary TCR precisely into T-cells, offering an efficient and targeted treatment approach for PCNSL with a clear path to expand into broader B-cell lymphomas.

Brain visual
Doctor reviewing CT scan

CNS DISEASES

Delivering gene therapy to the brain remains one of the greatest challenges in medicine. Conventional vectors are blocked by the blood-brain barrier, neutralised by the immune system, or simply too small to carry the DNA constructs required. ExoVec's natural ability to cross the blood-brain barrier and carry DNA of up to 36 kb makes it uniquely suited to CNS indications where no effective gene therapy currently exists.

CARDIAC DISEASES

Genetic cardiac diseases such as cardiomyopathy remain difficult to treat with existing gene therapy vectors due to their large DNA requirements and the immune barriers involved in reaching heart tissue. ExoVec demonstrates superior delivery to heart tissue in both 3D models and in vivo studies, outperforming benchmark vectors and opening a new route to effective gene therapy for these currently incurable conditions.

Hospital monitor visual

DEVELOPMENTAL PLAN

202720282029203020312032
EX VIVO CELL THERAPYPCNSLPreclinical trialCMC and INDFIH Phase 1
IN VIVO DELIVERY PLATFORMMultiple (CNS, Heart)Showcase cell modelsOut-licensing opportunities

VISION & IMPACT BROCHURE

Want the full picture? Our Vision & Impact Brochure covers ExoVectory's founding story, the diseases we are targeting and why we chose them, and our vision for what exosome medicine can achieve.

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Vision and Impact brochure preview

Interested in our pipeline?

ExoVectory is actively seeking partners to co-develop and advance ExoVec across additional indications. Get in touch to learn more.

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