7 Biotechs Fighting Rare Diseases With Gene-Editing Tech
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.
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..
DiNAQOR, a Swiss biotech startup founded in 2019, develops a novel gene therapy platform consisting of modified adeno-associated vectors (AAV) together with a specific loco-regional delivery system. Their innovation is in utilizing AAV serotypes with increased specificity towards cardiac tissues together with cardiac-specific promoters which improve gene expression in the heart tissues. This could potentially lower the toxicity issues which often accompany the gene-therapy approach. The current lead therapy DINA-001 against monogenic cardiomyopathy is in the preclinical stage.
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