Transforming Lives through Gene Therapy
Gene therapy has the potential to change the lives of millions by addressing diseases at their genetic root.
With the right technology, it can move from being a therapy for few selected patients, to a scalable treatment for many.
Our mission is to help make that future a reality.
No safe vector exists.
Around 300 million people live with genetic disorders.
But most gene therapies are held back by severe limitations:
Safety Risks
Genotoxicity & Cytotoxicity.
Some viral vectors can integrate into the genome in unintended ways, causing genomic instability, and in rare cases may replicate uncontrollably, creating serious safety concerns.
Immunogenicity
Anaphylactic shock & Rapid clearance.
Many patients cannot receive these treatments at all because they already have immunity to viral vectors. In others, immune responses can trigger severe adverse effects, posing serious safety risks and further limiting the use of current gene therapies.
Limited Cargo
Gene size restrictions.
Small payload size makes delivery of large genes, such as certain blood-clotting factors, problematic. This severely restricts the treatment of diseases associated with longer genes.
Limited Efficiency
Low transduction rates in key tissues.
Gene therapies using AAV often require high doses to work well. Resulting in higher production costs, patient-to-patient variability, and increased adverse effects like liver problems or immune reactions (sometimes serious ones.) These issues make the treatment less reliable and harder to use widely.
High Costs
Unaffordable treatment.
Gene therapies often represent a last hope for patients with severe genetic disorders worldwide. However, their exorbitant prices are frequently exceeding $2-4 million per one-time treatment, making them inaccessible to most individuals. This financial barrier risks transforming gene therapy into a privatized luxury available primarily to those with comprehensive private insurance or substantial personal wealth, exacerbating health inequities and limiting its broader societal benefit.
A Safer, More Accessible Gene Therapy.
Our vision is to unlock the full potential of gene therapy by rethinking how genes are delivered. Led by Dr. Michael Burger, who has 10+ years of experience in gene delivery research, we are developing a fundamentally new approach.
Our gene delivery system is built exclusively from proteins and membranes naturally found in the human body. By avoiding viral components, we aim to create therapies that work in harmony with the body, do not trigger immune rejection, and cannot replicate or spread.
Together with co-founder Fernando Leyra, we are working toward gene therapies that are safer, more precise, scalable, and affordable. Bringing life-changing treatments within reach for millions, not just a select few.
Human Backed.
Led by science and powered by people.
We leverage rigorous research and a persistent focus
on the demands of the patients to engineer
the solutions of tomorrow.
