Charting new pathways for genetic medicine

We are Spot Biosystems, a clinical-stage biopharmaceutical company and innovator in a first-of-its-kind application of extracellular vesicles (EVs) as a novel delivery method for gene therapy. EVs are a non-viral alternative to existing viral gene therapy options that have the potential to achieve greater efficacy at higher doses by delivering genetic cargo without triggering immune system responses.

IMPACT

A safer, more potent gene therapy could open the door to numerous applications spanning heart health to rare disease. It also unlocks the ability to reimagine current, approved therapies such as AAV-based treatment for muscular dystrophies.

Initiated in 2025, our lead clinical program is a first-in-human trial in patients with Duchenne muscular dystrophy (DMD). In 2026, we achieved the world’s first non-viral, full length dystrophin delivery to human skeletal muscle. 

Spot Bio’s EV-based technology has been validated through peer-reviewed publications in European Heart Journal (2025) and Nature Biomedical Engineering (2023 and 2026).

PIPELINE

Clinical

SPOT-03 for DMD, currently in a first-in-human clinical trial

PreClinical

Candidate for treatment of cardiac ischemia pending first-in-human clinical study in 2026, as well as additional programs in preclinical testing for further indications