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What is Gene Therapy?
Gene Therapy is a revolutionary technique that has the potential to treat or cure diseases by altering a person's genes. It works by replacing a disease-causing gene with a healthy copy, deactivating a faulty gene, or introducing a new or modified gene into the body.
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Our Gene Therapy Platform
Our Next Generation Gene Therapy Platform uses AAV (Adeno-Associated Virus) as a vector to introduce healthy genes to a person's cells. Our patent pending multi-gene therapy sets us apart from any other gene therapy company. Our unique technology introduces multiple genes to stop progression and reverse disease. This ground-breaking technology has the potential to dramatically change the lives of those affected by genetic diseases.
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This next gen vector backbone requires less DNA for therapeutic efficacy — thus lowering dosing and reducing viral burden associated with adverse events. Proof of concept pre-clinical studies for disease applications have been completed for: GNE Myopathy, Limb Girdle Muscular Dystrophy and Lysosomal Acid Lipase Deficiency.
AAV Vector
Multi-Gene Technology
Healthy Cells & Function
Multiple genes are introduced to stop progression and reverse disease.
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Problem
GENE REPLACEMENT THERAPY MAY STOP DISEASE PROGRESSION, BUT IS NOT CURATIVE FOR AFFECTED INDIVIDUALS.
The Genosera Solution
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GENOSERA seeks to change the paradigm:​​
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Intervene in early disease
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Reverse progressive disease in more advanced patients​
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​​Once disease causes debilitating symptoms, especially muscle diseases — difficult to reverse effects​
Our approach: Express TWO Different Genes To correct function and reverse symptoms.
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Need for Next-Generation Dual Gene AAV Therapy in Muscle Diseases
Capability of both stopping progression of disease and reversing the disease, e.g. building back lost muscle mass and strength.
Solution:
Multi-gene
Vector
Wider applications for at least 27 known neuromuscular diseases, and more common conditions, such as sarcopenia.
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Need for Next-Generation Dual Gene AAV Therapy in Muscle Diseases
1. Our main gene expression
package is a Platform Technology.
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4. Potential to increase muscle strength while simultaneously preventing disease progression — halting or correcting disease from progressing
2. Patent-pending multi-gene AAV therapy
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5. Dual gene therapy is a model for improving other Muscular Dystrophy therapies moving forward, including several dozen other forms of disease
3. Effective at doses order of magnitude lower than other AAV therapies in pre-clinical tests
6. Potential to treat millions worldwide with sarcopenia due to aging (GNE) and others requiring increased muscle mass and strength (FST).
4
Need for Next-Generation Dual Gene AAV Therapy in Muscle Diseases
Capability of both stopping progression of disease and reversing the disease, e.g. building back lost muscle mass and strength.
Solution:
Multi-gene
Vector
Wider applications for at least 27 known neuromuscular diseases, and more common conditions, such as sarcopenia.
