The recently established Blavatnik Therapeutics Challenge Awards (BTCAs) at Harvard Medical School are designed to propel scientific work with exceptional promise for therapeutic translation.
The 2021 award-winning projects are:
Identifying small-molecule therapies for telomere diseases (principal investigator Suneet Agarwal, HMS associate professor of pediatrics at Boston Children’s Hospital). Telomere diseases encompass a spectrum of rare and often-fatal syndromes, including dyskeratosis congenita (DC) and pulmonary fibrosis (PF), caused by mutations in genes regulating the biology and function of telomeres, the tiny caplike structures at the ends of chromosomes that shield them from fraying and damage. The cardinal feature of DC is bone marrow failure and deficient blood cell production. PF involves the progressive scarring and damage to lung cells and the ultimate failure of the lungs in patients affected by the disease. Despite genetic discoveries in the past two decades, there has been no translation of this new knowledge and there are no curative therapies for these conditions.
