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Last Posted: Oct 04, 2024
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Crossing the Equity Chasm: Addressing a Second Valley of Death in Biomedical Innovation

From the article: "Recent transformative advances in health and medicine have successfully traversed the valley of death, including progress in genomics, precision medicine, innovative medicines and vaccines, gene therapy, and artificial intelligence. However, it is critical to call attention to a second, important valley of death: the failure to deliver scientific advances to populations most in need. The second valley of death is fueled by socioeconomic, environmental, political, and systemic factors; health system failures; and persistent challenges in closing the last mile in health care, resulting in significant disparities in morbidity and mortality worldwide. "

Advancing ASO therapies from development to implementation

From the abstract: "A novel application of antisense oligonucleotide (ASO) technology, developed to treat a single patient, adds to the growing number of ‘personalized’ therapies for rare diseases; but pathways to implementation and access are urgently needed. "

A roadmap for affordable genetic medicines

From the abstract: "Nineteen genetic therapies have been approved by the U.S. Food and Drug Administration (FDA) to date, a number that now includes the first CRISPR genome editing therapy for sickle cell disease, CASGEVY (exagamglogene autotemcel). This extraordinary milestone is widely celebrated because of the promise for future genome editing treatments of previously intractable genetic disorders and cancers. At the same time, such genetic therapies are the most expensive drugs on the market, with list prices exceeding $4 million per patient. "

Evolution of Gene Therapy for Inborn Errors of Immunity

From the article: "Inborn errors of immunity comprise more than 500 monogenic defects that predispose affected individuals to life-threatening infections, autoimmunity, and lymphoproliferation. The diagnosis and treatment of inborn errors of immunity have significantly evolved in the past few decades. Currently, a hematopoietic stem cell transplant (HSCT) is the definitive treatment of many IEIs. However, an HSCT carries risks, such as graft-vs-host disease and decreased survival rates in the absence of an HLA-matched donor. Gene therapy (GT) offers a promising alternative by targeting and correcting specific genetic mutations. "

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Disclaimer: Articles listed in the Public Health Genomics and Precision Health Knowledge Base are selected by the CDC Office of Public Health Genomics to provide current awareness of the literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the update, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.

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