As of now, only 5% of so-called rare diseases (orphan diseases) have an FDA-approved treatment available to patients. Rare diseases are defined as those affecting fewer than 1 in 200.000 people with almost 80% of all rare diseases being of genetic nature.
With recent progress in gene-editing technologies, including breakthroughs like CRISPR/Cas9 and new therapy delivery technologies, there is a wave of innovative biotech companies entering the field of rare diseases equipped with new ways to address the problem.
In this article, we would like to outline a list of notable companies targeting rare diseases with cutting-edge gene therapies.
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Topics: Biotech Companies