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Trigenesis Gene Transfer Therapy
Gene Transfer Therapy is a class of gene therapies currently being studied to address certain neuromuscular diseases. The goal of Gene Transfer Therapy is to transfer a functioning gene into disease-affected cells.
Designed to generate a continual stream of gene transfer therapy candidates with the goal of ushering in a new era of medicine for patients with rare neuromuscular disease. Based on decades of research, Sarepta's approach utilizes rationally designed combinations of vectors, promoters, and transgenes to address the root cause of disease, while considering risks, protein expression, and function.
Sarepta's Gene Transfer Therapy constructs rely on a profound understanding of disease pathology and human biology. Through a precision focus on the core elements of a construct, Sarepta is developing new therapies for patients and families who need them now.
The core components of a Gene Transfer Therapy are selected and engineered according to the principles of informed rational design with the aim of achieving functional protein expression in cells impacted by disease. These essential components include an efficient vector for delivery, a promoter that turns on protein expression, and the transgene itself.
The goal of Gene Transfer Therapy is to overcome protein deficits by selecting an appropriate combination of a vector, promoter, and transgene that work synergistically to address disease pathology.
In developing a Gene Transfer Therapy, informed rational design includes selecting a:
An efficient vector delivers the functioning therapeutic genetic construct to target cells with minimal immune response.
The promoter is selected with the intention of driving robust protein expression in target cells.
The transgene is engineered with the goal of maintaining the functional domains of the protein that address disease pathology.
Gene transfer therapies in development, aim to overcome protein deficits by selecting, based on nonclinical data, an appropriate combination of a vector, promoter, and transgene so that they work synergistically to address disease pathology.
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