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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020,” Michelle explains.

Disease 80
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New cell therapy model accelerates cancer treatment development

Drug Target Review

Around 2020, Bock assumed the challenge of creating a new division at MD Anderson focused on advancing cell therapies by bridging the gap between research labs and the clinic. While therapies like CAR-T have shown great success in hematologic malignancies, solid tumours remain a challenging area for current treatments.

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Broad Institute and Bayer extend their cardiovascular alliance

Broad Institute

First launched in 2015 and renewed in 2020, the program began as a recognition that while a majority of cardiovascular disease can be associated with lifestyle factors such as tobacco consumption, diet, and level of physical activity, genomics can influence an individual’s predisposition to cardiovascular disease, age of onset, and severity.

Disease 93
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Organoids: the versatile platform for discovery and regeneration

Drug Target Review

8 With the widespread use of induced pluripotent stem cells (iPSCs), organoids can now be generated from the skin or blood cells of both healthy volunteers and disease patients, opening the door to studying various diseases and creating individually customised potential treatments. MedComm (2020). 2009;459(7244):262-5. 2023;4(3):e274.

Treatment 105
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The rising impact of biomarkers in early clinical development

Drug Target Review

Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms. A biomarker is a measurable indicator of a biological process, disease state, or response to a treatment.

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Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Drug Target Review

Dr Aaron Haubner, Senior Manager of North America Medical Affairs and Market Access at Terumo Blood and Cell Technologies , reveals that while promising new treatments emerge, urgent partnerships are needed to ensure this essential blood therapy reaches the patients who need it most.

Disease 52
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David Liu receives Breakthrough Prize in Life Sciences

Broad Institute

Since their initial development, both base editing and prime editing have been used by thousands of laboratories around the world and have enabled the study and potential treatment of many genetic diseases. The Breakthrough Prize is one of the worlds most important science awards.

Science 144