Skip directly to search Skip directly to A to Z list Skip directly to navigation Skip directly to page options Skip directly to site content


Main|Search|PHGKB
Search PHGKB:

Last Posted: Jul 18, 2024
spot light Highlights

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. "

Sickle Cell Disease Approvals Include First CRISPR Gene Editing Therapy
E Harris, JAMA, January 3, 2024

From the article: "The US Food and Drug Administration (FDA) recently greenlit 2 cell-based gene treatments for sickle cell anemia, including the first therapy involving the genome editing technology known as CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats–CRISPER-associated protein 9). Both treatments are approved for people aged 12 years or older and work by modifying people’s own blood stem cells and then transplanting them into their bodies via a single-dose infusion. They change blood cells in different ways, though. "

How CRISPR gene editing could help treat Alzheimer’s
T Thompson, Nature, December 11, 2023

From the article: "Last month saw the first-ever approval of a gene therapy that uses the CRISPR–Cas9 gene-editing tool, a treatment for the blood conditions sickle-cell disease and ß-thalassaemia that works by precisely cutting out a faulty gene in people’s stem cells. Now, researchers in search of new treatments for Alzheimer’s disease are hoping to deploy similar strategies against forms of the disease that are caused by genetic mutations."

In historic decision, FDA approves a CRISPR-based medicine for treatment of sickle cell disease
A Feurstein, StatNews, December 8, 2023

From the article: "The Food and Drug Administration on Friday approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder. The new medicine, called Casgevy,'s authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations — ushering in a new era of genetic medicines for inherited diseases. "

Search Result Summary


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.

TOP