Robust Preclinical Data Support Advancement of our Duchenne Programs
In preclinical studies, our EEV-Oligonucleotide approach has led to:
Our lead program is designed to address the underlying genetic cause of Duchenne with the potential to restore functional dystrophin in muscle cells.
Our lead program is designed to address the underlying genetic cause of Duchenne with the potential to restore functional dystrophin in muscle cells.
~30,000 people in the U.S and Europe have Duchenne.1
Duchenne muscular dystrophy – often referred to as Duchenne or DMD – is a rare disease caused by mutations in the DMD gene, which encodes for the dystrophin protein. These mutations lead to inadequate dystrophin production.
Dystrophin is essential to maintaining the structural integrity and function of muscle cells.
Lack of functional dystrophin leads to progressive loss of muscle strength, impacting mobility and causing heart or respiratory complications that contribute to high mortality rates.
Duchenne causes progressive loss of muscle function throughout the body.
To date, currently approved therapies for Duchenne have not shown considerable increases in dystrophin production and often fail to widely distribute across muscle tissue.2-5
Endosomal Escape Vehicle (EEV™)-Oligonucleotides for Duchenne
Our proprietary EEV-Oligonucleotide is designed to address the underlying genetic cause of Duchenne to allow muscle cells to produce functional dystrophin.
Entrada's lead oligonucleotide programs include ENTR-601-44 and ENTR-601-45 for the potential treatment of people living with Duchenne who are exon 44 and 45 skipping amenable, respectively.
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Near complete correction of dystrophin production in skeletal muscles.
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Significant dystrophin production in the heart muscle.
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Wide distribution of dystrophin production throughout the muscle.
Visit our Manuscripts and Presentations pages to learn more
References
Crispi V, Matsakas A. Duchenne muscular dystrophy: genome editing gives new hope for treatment. Postgraduate Medical Journal.2018;94(1111):296-304. doi:10.1136/postgradmedj-2017-135377.
AMONDYS 45 [package insert]. Cambridge, MA: Sarepta Therapeutics, Inc.; 2021.
VILTEPSO [package insert]. Paramus, NJ: NS Pharma, Inc.; 2020.
VYONDYS 53 [package insert]. Cambridge, MA: Sarepta Therapeutics, Inc.; 2019.
EXONDYS 51 [package insert]. Cambridge, MA: Sarepta Therapeutics, Inc.; 2016.