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Researchers identify leading compounds for treatment of telomere diseases

Posted: Sat Apr 25, 2020 4:42 am
by ofonorow
https://www.drugtargetreview.com/news/60552/researchers-identify-lead-compounds-for-treatment-of-telomere-diseases/

In the future, the researchers hope to validate PAPD5 inhibition for other diseases involving faulty maintenance of telomeres – and perhaps even ageing itself. They are most excited about two compounds, known as BCH001 and RG7834 that are under further development.

“We envision these to be a new class of oral medicines that target stem cells throughout the body,” said Dr Suneet Agarwal, the study’s senior investigator. “We expect restoring telomeres in stem cells will increase tissue regenerative capacity in the blood, lungs and other organs affected in dyskeratosis congenita and other diseases.”




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“This provided the hope that this could become a clinical treatment,” says Nagpal.

In the future, Agarwal, Nagpal, and colleagues hope to validate PAPD5 inhibition for other diseases involving faulty maintenance of telomeres—and perhaps even aging itself. They are most excited about two compounds, known as BCH001 and RG7834 that are under further development.

“We envision these to be a new class of oral medicines that target stem cells throughout the body,” Agarwal says. “We expect restoring telomeres in stem cells will increase tissue regenerative capacity in the blood, lungs, and other organs affected in DC and other diseases.”