Cell therapy weekly: UK announced as global center for new cardiovascular trial

Also this week: US FDA grants orphan drug status to PR001 for use in Gaucher disease and researchers restore fertility in older mice using a simple metabolic compound.

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Feb 13, 2020
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The news highlights:

UK announced as global center for new cardiovascular trial
US FDA grants orphan drug status to PR001 for Gaucher disease
Researchers restore fertility in older female mice with new compound

UK announced as global center for new cardiovascular trial

Novartis (Basel, Switzerland) have declared the UK their global trial center for investigating the potential of inclisiran. Inclisiran made headlines earlier in the year when the RNA-based method demonstrated promise in reducing low-density lipoprotein. Now, Novartis are seeking to recruit patients for their preventative study to combat heart attack or stroke, following the success of their earlier trial on those who had already been affected by the conditions.

“This new trial offers an important opportunity to test the ability of inclisiran to lower the risk of heart disease and stroke in a broad range of people. At the same time, it will demonstrate how a new generation of streamlined trials can provide reliable information about novel treatments for conditions that affect large numbers of NHS patients,” commented Sir John Bell, Regius Professor of Medicine at Oxford University, collaborators on the study.

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US FDA grants orphan drug status to PR001 for Gaucher disease

The US FDA (MD, USA) bestowed orphan drug status to Prevail Therapeutics’s (NY, USA) AAV-based drug for Gaucher disease. Gaucher disease is a neurodegenerative disease that normally results in death during early childhood or infancy. The new AAV treatment aims to restore GBA1 activity and is currently involved in Phase I/II clinical trials for Type 2 Guacher disease patients, with the first patients planned to be treated in the first half of 2020. Obtaining the orphan drug status allows the Prevail Therapeutics to obtain assistance and funding during their research on treating the rare condition.

“We are pleased to receive these important designations from the FDA, which underscore the critical nature of our work,” commented Asa Abeliovich, Founder and Chief Executive Officer of Prevail Therapeutics. “These designations support our conviction that new gene therapies for Gaucher disease are urgently needed — especially for the severe, neuronopathic form of the disease, for which there are no FDA-approved therapies.”

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Researchers restore fertility in older female mice with new compound

By administering small doses of a metabolic compound to older female mice, researchers from The University of Queensland (Brisbane, Australia) have partially restored their fertility. By restoring NAD+ levels in the mice, the team were able to restore or maintain the number and the quality of eggs available. The new treatment potentially offers a solution to infertility and the growing desire to have children later in life.  

“Quality eggs are essential for pregnancy success because they provide virtually all the building blocks required by an embryo,” explained Professor Homer. “We investigated whether the reproductive ageing process could be reversed by an oral dose of a ‘precursor’ compound – used by cells to create the molecule.”

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For more weekly cell therapy news, read previous editions of the cell therapy weekly.

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