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 200000 people with almost 80% of all rare diseases being of genetic nature. while only
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, I would like to outline a list of notable companies targeting rare diseases with cutting-edge gene therapies.
Generation Bio is known for its platform consisting of proprietary non-viral closed-ended DNA (ceDNA) construct and cell-targeted lipid nanoparticle (ctLNP) delivery system. It provides three times higher capacity of viral capsids and the ability to carry larger and possibly multiple gene material without activation of the immune system. The company’s current pipeline addresses liver and retina-related pathologies with future potential in oncology, skeletal muscles, and CNS diseases. Generation Bio’s lead therapies for Phenylketonuria (PKU) and Hemophilia A are at the pre-clinical development stage.
Founded in 2016, the company raised a $110 million Series C in 2020 led by T. Rowe Price. This transaction brought the company’s total capital up to $235 million and brought together some prominent investors, including Fidelity Management and Research Company, and Atlas Venture. The company went public in Jun 2020 raising $200 million..
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