Remove 2021 Remove Disease Remove Treatment
article thumbnail

David Liu receives Breakthrough Prize in Life Sciences

Broad Institute

The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.

Science 139
article thumbnail

A transatlantic collaboration is uncovering the role of key genetic variants in common diseases like type 2 diabetes and obesity

Broad Institute

By Lucy Mutz November 4, 2024 Credit: Juliana Sohn Kasper Lage is managing director of the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease. The overall goal is to translate genetic findings into biological mechanisms underlying disease that could be targeted with new medicines.

Disease 72
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Transforming cancer treatment for greatest impact

Drug Target Review

Cancer is a disease driven by variable genetic mutations. Could you provide insights into the driving factors behind exploring novel treatments for cancer types? Could you provide further details on how this phenomenon could impact broader treatment outcomes? What aspects prompted this research direction?

Treatment 105
article thumbnail

Rare autoantibody diseases: an innovative targeted pathway

Drug Target Review

Approximately three percent of the global population — 240 million people — experience autoantibody diseases, which occur when one’s own body attacks critical organs and tissues. This can create an abnormal immune response that attacks the cells of our bodies and contributes to the development of autoantibody diseases.

Disease 98
article thumbnail

FDA Advisory Committee Review of 2021

Eye on FDA

One meeting of the Oncologic Drugs Advisory Committee is set for February 10 to discuss a new application for the proposed treatment of Non-Small Cell Lung Cancer (NSCLC. But before heading into 2022, it is perhaps time to take stock of AdComms and what they did or did not do during 2021. Fewer Meetings.

FDA 76
article thumbnail

The genetic modifier approach: identifying the right target for rare diseases

Drug Target Review

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

Disease 111
article thumbnail

Antibody Drug Conjugates: windows of opportunity

Drug Target Review

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options.