Unraveling the promise of genetics for treating progressive illness 

Image by Gerd Altmann from Pixabay

For almost every major common disease, researchers have less understanding of the severe forms than milder cases. And as a result, people with severe forms of diseases often have few treatment options available.  

Thus, the significant unmet medical need for many diseases ranging from multiple sclerosis to Alzheimer’s is to halt disease progression and treat severe forms of the disease. “Most patients eventually do progress. We don’t understand what is causing that progression,” said Dr. Jeffrey Gulcher, chief scientific officer of Genuity Science (Boston). 

What causes disease progression? 

This basic concept concerning disease severity is evident in a range of clinical areas. Consider, for instance, non-alcoholic fatty liver disease, where abnormal amounts of fat are stored in the liver. Roughly one in four people in the U.S. have the condition, which can progress …

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Why Genuity and Emory are partnering on neurodegenerative disease research

[Photo by Anna Shvets from Pexels]

Privately-held Genuity Science and Emory University are collaborating with the hopes to accelerate drug research and development for neurodegenerative diseases such as Alzheimer’s and Parkinson’s.

Clinical breakthroughs for neurodegenerative diseases are rare. The most recent drug to win FDA approval for Alzheimer’s, Namenda (memantine) from AbbVie-acquired Allergan, did so in 2003. And Levodopa, one of the most-effective Parkinson’s therapies, won FDA approval in 1975.

The quest for disease-modifying agents

“There are zero disease-modifying agents for Alzheimer’s or Parkinson’s,” said Dr. Allan Levey, professor and chair of the Emory University neurology department.

For Parkinson’s, drugs can “can reverse symptoms pretty well for many people for many years,” Levey said. But the masking of symptoms can hide the gradual progression of the disease.

While t…

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Researchers at Genuity and Yale shed light on aortic aneurysms

In aortic aneurysm, smooth muscles transform, causing lesions. Photo courtesy of YaleNews.

Researchers at privately-held Genuity Science and Yale University Medical School have revealed why aortic aneurysms form. The scientists discovered that an expansion in abnormal cells in smooth muscle tissue of the aorta can contribute to aortic aneurysm.

The researchers came to that conclusion by using a combination of generative artificial intelligence, single-cell biology and RNA sequencing to uncover causal molecular drivers of cell fate transition in aortic aneurysms.

Subsequent investigations have revealed a possible genetic cause of the disease in humans, by specifically linking a genetic variant in a gene of unknown significance to disease pathogenesis.

In 2018, almost 10,000 people in the U.S. died from ruptured aortic aneurysms, according to the CDC. Currently, the first line of defense against the disease is surgical interv…

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