Using our proprietary EEV™ technology, we are creating a pipeline of oligonucleotide therapies to treat neuromuscular diseases including Duchenne muscular dystrophy (DMD). Our other intracellular programs span multiple therapeutic areas and focus on biologics including antibodies, peptides, enzymes and proteins.
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| Oligonucleotides | |||
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Neuromuscular
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Duchenne Muscular Dystrophy
Lead Exon Skipping Program |
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Preclinical
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Duchenne muscular dystrophy (DMD) is an X-linked recessive disease caused by mutations in the gene that codes for dystrophin, a protein essential for the normal function of muscle cells. Entrada is developing oligonucleotides conjugated to an EEV from our proprietary platform for the treatment of DMD. Limited distribution in muscles and poor endosomal escape are major barriers to achieving higher dystrophin levels with current oligonucleotide therapies. Our proprietary EEV-oligonucleotide conjugates promote enhanced exon skipping, restoration of a functional mRNA reading frame and dystrophin protein production. Learn more about our novel approach to developing a treatment for patients with DMD. |
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Duchenne Muscular Dystrophy
Additional Exon Skipping Programs |
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Discovery
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Other Neuromuscular Diseases
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Discovery
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Beyond Neuromuscular
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Additional Diseases
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Discovery
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| Antibodies and Peptides | |||
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Multiple Therapeutic Areas
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Oncology
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Discovery
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Additional Diseases
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Discovery
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| Enzymes and Proteins | |||
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Inborn Errors of Metabolism
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MNGIE
ENTR-501: Thymidine Phosphorylase |
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Preclinical
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Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is caused by mutations in the TYMP gene. These mutations result in a loss of activity of an enzyme called thymidine phosphorylase. Without thymidine phosphorylase, MNGIE patients accumulate thymidine and deoxyuridine in their tissues and blood, causing mitochondrial depletion. Over time, mitochondrial depletion results in characteristic MNGIE symptoms, including dramatic weight loss as well as gastrointestinal and neurological symptoms. Entrada is developing ENTR-501 as an intracellular enzyme replacement therapy for patients with MNGIE. ENTR-501 consists of an Entrada EEV linked to thymidine phosphorylase and is designed to address the deficiency of enzyme activity inside the cells of MNGIE patients. In 2020, Entrada received Orphan Drug Designation from the U.S. FDA for ENTR-501 for the treatment of MNGIE. |
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