In a summer dominated by news of Vertex Pharmaceuticals’ breakthrough cystic fibrosis combo therapy of Kalydeco with Lumacaftor proving to be effective in treating the underlying cause of the disease in a broader range of CF patients, there have been few other cystic fibrosis drug developers making headlines. Today, however, Dutch-based ProQR Therapeutics, a biotech company currently developing novel mRNA-based treatments for CF, was reported to have filed an IPO with the SEC in a bid to raise up to $75 million in new funding, according to a NASDAQ news release. The launch of an IPO for the company could put their emerging CF therapy into the spotlight.
Founded in 2012 in Leiden, Netherlands, ProQR is a relatively new endeavor. Like Vertex, however, the company is seeking to break free of traditional cystic fibrosis medications that curtail the symptoms of the disease, and instead develop a therapy that, like Kalydeco, treats the underlying genetic underpinnings of CF. In order to accomplish this, their mRNA-based treatment approach repairs the fundamental genetic defect in cystic fibrosis patients: mutations that exist in the CFTR gene in patients’ DNA, which are in turn copied into the RNA. The mutation sets off a genetic chain reaction, leading to the critical breakdown of a key CFTR protein. It is this non-functioning protein that ultimately leads to the severe symptoms that are associated with CF.
ProQR hopes to target the CFTR mutation at the RNA level, thus alleviating the cause of cystic fibrosis symptoms.
While the company is still relatively unknown to the general population, the technology behind its experimental therapy spans both the U.S. and Netherlands — with the RNA modulation technology having been developed at Massachusetts General Hospital in Boston — and the company currently employs 50 people. According to the website, the company is focused specifically on the treatment of cystic fibrosis, with no other diseases indicated in their development pipeline. ProQR also appears to be still in the pre-clinical stage, as there is no current information about upcoming clinical trials on their website or in clinical trials.gov filings.
However, with the start of a new IPO and an audacious goal of treating cystic fibrosis at the genetic level, ProQR offers a promising complement to next-generation CF therapies that could dramatically impact the lives of the 70,000 people worldwide with the disease.