Our Science

Expanding the benefit and reach of genetic medicines to help all patients.

Genetic medicines have resulted in remarkable results for patients. Yet, extreme product complexity and cost has severely limited patient access to existing genetic medicines.

Kelonia’s technology will unlock the full promise of genetic medicine by overcoming the central challenge that is shared across all existing technologies: precise and efficient gene delivery directly to the patient.

This will enable improved patient outcomes and dramatically better reach and access than what is possible with the genetic medicines that exist today. At the same time, it can be applied to genetic medicines for a broad range of diseases.

Our technology is built on a well understood gene delivery system

Kelonia uses an advanced lentiviral vector (LVV) engineered for specific and efficient systemic delivery. This is achieved by introducing a targeting molecule that uniquely defines our LVV’s specificity while an optimized fusogen enables efficient delivery of therapeutic transgenes to the target cell. Only the vector envelope is modified, maintaining safe and durable gene delivery, as demonstrated in thousands of patients treated with LVV-modified cells.

Bringing gene therapies into common medical practice

With simple and elegant advancements to LVV technology, Kelonia’s approach to genetic medicines is “off-the-shelf.” This new wave of genetic medicines will reach patients in their communities when they’re needed.

Enabling cell-specific gene delivery via direct infusion

Cell transduction with Kelonia’s cutting-edge LVV is determined by carefully selected targeting molecule(s). The result is exquisitely specific transduction of cells that express the intended ligand. Our technology enables a vast array of potential targeting specificity defined by a single molecule or multiplexed to expand the LVV tropism.

The patient’s cells are engineered within the body to express the therapeutic protein

After administration, Kelonia’s LVV delivers its therapeutic transgenes to the intended cells. The therapeutic transgene is expressed in the target cell genome for durable cellular engineering after a single dose.

The unique design permits exceptionally efficient gene delivery

Kelonia’s cutting-edge LVV is engineered to achieve efficient transduction after a single dose, without the need for in vivo selection or transduction enhancers. This minimizes the dose required, thus avoiding potential immunotoxicites that are associated with large viral dose levels.