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Tarix Orphan

Programs

TXA127 is a pharmaceutical grade formulation of the naturally occurring peptide Angiotensin (1-7), (or A (1-7)). This peptide is part of the renin-angiotensin-system (RAS), and was thought for many years to be an inactive by-product of degradation of other molecules in the RAS. In the 1990s, A(1-7) began to be studied for its action on myocardial tissue, and a specific receptor for A(1-7) was identified. This mas receptor is expressed in a variety of tissues, including bone marrow, brain, heart, lung and muscle. It is now clear that A(1-7) is part of a pathway which includes angiotensin converting enzyme 2 (ACE2) and the mas receptor, and that the actions of ACE2-A(1-7)-mas counteract the detrimental effects of the “classical” RAS axis, ACE-Angiotensin II-AT1 receptor:

RAS diagram
 

Duchenne Muscular Dystrophy (DMD)
In the mdx model of DMD, Angiontensin(1-7) significantly improves mdx gastrocnemius architecture compared to untreated mdx as shown by H&E staining and Sirius red staining.


 

Mdx mice treated with Ang(1-7) also perform better in a treadmill test compared to untreated mdx mice.

DMD ambulation
 

MDC1A – Congenital Muscular Dystrophy
Angiotnesin(1-7), given at 0.5 mg/kg/day, results in improved muscle pathology as shown below. The histology shows less infiltrating cells as seen in the H&E staining as well as vastly improved fibrosis as seen in the Picrosirius red staining.

MDC1A histology
Treatment with Angiotensin(1-7) also results in more functional muscle. At 7 weeks of age, DyW mice treated with Ang(1-7), 0.5 mg/kd/day, exhibit significantly more standups/5 minutes than untreated age-matched DyW littermates.

MDC1A ambulation
 

The video below demonstrates the functional effects of Ang(1-7) on DyW mice. Four mice, two of which are wild type and the other two are DyW (MDC1A) mice, now six weeks old, and treated with TXA127 for four weeks. The DyW mice are always smaller than wild type, but they are moving around just as well as the wild type.

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