Tag Archives: Rare

First US attempt to cure a rare disease with genome editing fails miserably 

First US attempt to cure a rare disease with genome editing fails miserably Scientists at Sangamo attempted the first human trial of a gene-editing method to cure rare, debilitating enzyme deficiency diseases  After showing early promise, the DNA replacement failed to reduce levels of harmful unprocessed sugars  The gene replacement itself worked, but after nearly… Read More »

Rare disease analytics platform Raremark raises $3.9M

This morning London-based data platform Raremark announced that it landed $ 3.9 million (£3 million) in a funding round led by AlbionVC and Ananda Ventures, with participation from Oltre Ventures and Cass Entrepreneurship Fund. The platform, which focuses on rare diseases, plans on using the new money to build a patient engagement and data analysis… Read More »