| Market Capitalization (Millions $) |
220 |
| Shares
Outstanding (Millions) |
105 |
| Employees |
24 |
| Revenues (TTM) (Millions $) |
30 |
| Net Income (TTM) (Millions $) |
-85 |
| Cash Flow (TTM) (Millions $) |
0 |
| Capital Exp. (TTM) (Millions $) |
2 |
Business Description
Proqr Therapeutics N.V. is a Dutch biotechnology company that specializes in the development of RNA therapies for the treatment of genetic disorders. The company was founded in 2012 and is headquartered in Leiden, the Netherlands.
The primary focus of Proqr Therapeutics is on the development of treatments for rare genetic disorders, which affects a relatively small number of people worldwide. Many of these disorders are caused by a single defective gene that leads to the production of a faulty protein, ultimately leading to disease.
Proqr Therapeutics develops RNA therapies that aim to correct or replace faulty genes, by targeting the RNA (ribonucleic acid) molecules that carry genetic information from DNA to the cells' protein-making machinery.
The company's lead candidate drug, sepofarsen (QR-110), is an RNA therapy being developed to treat a rare genetic disorder called Leber's congenital amaurosis type 10 (LCA10). This hereditary disease affects the retina, causing a progressive loss of vision, often leading to permanent blindness. Sepofarsen works by targeting the mRNA (messenger RNA) molecule that codes for the defective protein in LCA10, and thereby repairing the faulty gene.
Another RNA therapy in development by Proqr Therapeutics is called QR-421a, which targets a genetic mutation that causes Usher syndrome type 2a (USH2A), a progressive disease that causes both hearing and vision loss.
Proqr Therapeutics' RNA therapeutics are based on a proprietary platform technology called Axiomer, which enables the company to design and produce RNA molecules with specific properties that can target specific disease-causing genes.
In addition to its lead candidate drug, sepofarsen, and QR-421a, Proqr Therapeutics has several other RNA therapies in preclinical development for rare genetic diseases such as cystic fibrosis, Huntington's disease, and other genetic disorders that affect the central nervous system.
The company has collaborations with several academic institutions and pharmaceutical companies, including Allergan, Ionis Pharmaceuticals, and the Foundation Fighting Blindness.
Overall, Proqr Therapeutics N.V. is a biotech company that is leading the field of RNA therapies for the treatment of rare genetic disorders, with a focus on developing new treatments that can address the underlying genetic cause of disease.
Company Address: Zernikedreef 9 Leiden 2333
Company Phone Number: 88 166 7000 Stock Exchange / Ticker: NASDAQ PRQR
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