Science and Technology

We are developing novel cell therapies that combine cutting-edge iPSC technology and CRISPR-based genome editing to activate the regenerative potential of our patients’ own body and cure muscular dystrophies at their root.

Cell Therapy

At Vita, we strive to transform the way we treat patients by addressing the root cause of their disease using truly curative cellular medicines, rather than merely treating symptoms by lifelong pharmaceutical drug regimens.

By restoring and improving tissue functions on a cellular level, we aim to refine the quality of life for patients whose medical needs are currently unmet. We are developing approaches to fix genetic defects in patient-derived stem cells and reintroduce healthy tissue generated from these cells back into the patient to restore normal function. We are also building cost-effective universal cell types that can treat all patients and bring the economies of scale to our novel cellular therapeutics.

Correct genetic defects

Develop universal
cells and cellular
Medicine to scale

Cell Therapy

Rebuild healthy
cells and tissues

Unlock regeneration

Develop universal cells

Disease target is confirmed and genetic mutation(s) causing the disease are identified

Correct genetic defects

Cells are isolated from a small blood draw from the patient

Rebuild healthy cells and tissues

Blood cells are reprogrammed into iPSCs using the ‘Yamanaka’ factors

Unlock regeneration

Disease-causing genetic mutations are corrected ex vivo using CRISPR technology

Autologous

In the autologous approach, a sample of patient blood is used to generate stem cells with nearly unlimited potential for expansion and differentiation into specialized cell types in our laboratories.

These “pluripotent” stem cells are securely banked and used to create gene-corrected healthy cells and tissues that can be reintroduced into the patient to recover biological function, support true regeneration, and cure disease at the root.

Patient

Disease target is confirmed and genetic mutation(s) causing the disease are identified

Blood draw

Cells are isolated from a small blood draw from the patient

iPSCs

Blood cells are reprogrammed into iPSCs using the ‘Yamanaka’ factors

Genetic correction

Disease-causing genetic mutations are corrected ex vivo using CRISPR technology

iPSCs

Gene-corrected iPSCs are differentiated into disease-specific cell types and tissues

iPSCs

Healthy cells and tissues are transplanted back into the patient

Allogenic Approach

In the allogeneic approach, a “universal” iPSC line is used to generate cells and tissues needed for restoring normal biological function in patients.

These cells lines are engineered to be protected from being attacked by the patient’s immune system, allowing rapid and cost-effective treatment of currently uncurable diseases while harnessing the revolutionary potential of cellular therapy.

Healthy donor

Genomic and biological studies confirm suitability for developing universal cell banks

Blood draw

Cells are isolated from a small blood draw of the healthy donor

iPSCs

Healthy donor’s blood cells are reprogrammed into iPSCs using the “Yamanaka” factors

Hypoimmunogenic engineering

Healthy iPSCs are engineered to enable immune evasion and universal application in all patients

Differentiated cells

Universal iPSCs are differentiated into disease-specific cell types and tissues

Patients

Healthy universal cells and tissues are transplanted into patients

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