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ProQR and Galapagos Collaborating on a New Way of Developing Fibrosis Treatments

ProQR Therapeutics and Galapagos are working together on a new approach to treating the tissue scarring seen in cystic fibrosis.

The collaboration will take advantage of ProQR’s proprietary next-generation RNA technology to develop axiomer editing oligonucleotides that can fight the fibrosis-promoting agents Galapagos has identified.

“In this collaboration we will explore a novel application of our Axiomer technology to Galapagos’ fibrosis targets,” Daniel A. de Boer, the CEO of ProQR, said in a press release. “We look forward to working closely with the team at Galapagos. With their leadership in the discovery of novel drug targets and our platform technology and know-how, we can make a meaningful impact together.”

Oligonucelotide editing involves changing an RNA sequence to overcome a gene mutation so the gene can produce a functional protein. A compound known as adenosine in an RNA strand is involved in many of the mutations that leads to a disease.

ProQR’s approach to the problem is changing adenosine to another compound, inosine, in the RNA sequence. It has the potential to treat a number of genetic diseases, including cystic fibrosis.

“With our vast experience in the discovery of novel drug targets, we believe ProQR’s Axiomer technology can be applied to strengthen the validation of novel targets from our platform,” said Onno van de Stolpe, Galapagos’ CEO. “We look forward to collaborating with ProQR and its exciting RNA editing technologies.”

ProQR is collaborating with the Cystic Fibrosis Foundation Therapeutics to develop QR-010 as a potential treatment for cystic fibrosis patients with a CFTR gene mutation known as F508del. Scientists designed the drug to bind to a messenger RNA sequence so the gene can generate a functional protein. RNA is an intermediate molecule between a gene and protein.

A Phase 1b trial (NCT02564354) showed that inhaled QR-010 can help cystic fibrosis patients achieve a meaningful improvement in respiratory function. It also showed that the treatment was safe and patients tolerated it well.

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