Research and development pipeline

We are developing a pipeline of novel medicines for patients in need. Our pipeline focusses on diseases that are very severe or life threatening, and have limited treatment options. All of the medicines we are developing are RNA therapies and target the underlying cause in the diseases.
Cystic Fibrosis
DISCOVERY
PRE-CLINICAL DEVELOPMENT
PROOF OF CONCEPT TRIALS
LATE STAGE/ REGISTRATION TRIALS
QR-010 for F508del
QR-010 for cystic fibrosis F508del

Two global clinical trials for QR-010 in people with cystic fibrosis (CF) have been completed. Study 001, was a Phase 1b safety and tolerability clinical trial in 70 people with CF due to two copies of the F508del mutation and Study 002, was a proof of concept clinical trial in 18 people with CF due to one or two copies of the F508del mutation. In both clinical trials, QR-010 was observed to be safe and well-tolerated and both trials showed encouraging signals that QR-010 has the potential to be a meaningful therapy for people with CF.

Read more about QR-010 for cystic fibrosis and clinical trials
QRX-042 for W1282X
QRX-042 for cystic fibrosis W1282X

Cystic fibrosis can be caused by a variety of mutations. The most common mutation is called F508del, for which we are developing QR-010. For several other mutations that cause CF there is currently no treatment options of drugs in clinical trials. These mutations are called “Class 1” mutations. With the positive results from the clinical trials with QR-010 we believe we can use similar RNA therapies for a number of the Class 1 mutations. In our labs we are designing and testing potential medicines for these mutations.

QRX-036 for G542X
QRX-036 for cystic fibrosis G542X

Cystic fibrosis can be caused by a variety of mutations. The most common mutation is called F508del, for which we are developing QR-010. For several other mutations that cause CF there is currently no treatment options of drugs in clinical trials. These mutations are called “Class 1” mutations. With the positive results from the clinical trials with QR-010 we believe we can use similar RNA therapies for a number of the Class 1 mutations. In our labs we are designing and testing potential medicines for these mutations.

QRX-052 for R553X
QRX-052 for cystic fibrosis R553X

Cystic fibrosis can be caused by a variety of mutations. The most common mutation is called F508del, for which we are developing QR-010. For several other mutations that cause CF there is currently no treatment options of drugs in clinical trials. These mutations are called “Class 1” mutations. With the positive results from the clinical trials with QR-010 we believe we can use similar RNA therapies for a number of the Class 1 mutations. In our labs we are designing and testing potential medicines for these mutations.

QRX-065 for 621+1G->T
QRX-062 for cystic fibrosis 621+1G->T

Cystic fibrosis can be caused by a variety of mutations. The most common mutation is called F508del, for which we are developing QR-010. For several other mutations that cause CF there is currently no treatment options of drugs in clinical trials. These mutations are called “Class 1” mutations. With the positive results from the clinical trials with QR-010 we believe we can use similar RNA therapies for a number of the Class 1 mutations. In our labs we are designing and testing potential medicines for these mutations.

QRX-075 for 1717-1G->A
QRX-075 for cystic fibrosis 1717-1G->A

Cystic fibrosis can be caused by a variety of mutations. The most common mutation is called F508del, for which we are developing QR-010. For several other mutations that cause CF there is currently no treatment options of drugs in clinical trials. These mutations are called “Class 1” mutations. With the positive results from the clinical trials with QR-010 we believe we can use similar RNA therapies for a number of the Class 1 mutations. In our labs we are designing and testing potential medicines for these mutations.

Ophthalmology
DISCOVERY
PRE-CLINICAL DEVELOPMENT
PROOF OF CONCEPT TRIALS
LATE STAGE/ REGISTRATION TRIALS
QR-110 for LCA10
QR-110 for LCA10

We are developing QR-110 for Leber's congenital amaurosis 10 (LCA 10), a genetic eye disorder and the leading genetic cause of childhood blindness. We have begun a clinical trial for QR-110, a potentially life changing therapy for people living with LCA 10 due to the p.Cys998X mutation in the CEP290 gene.

Read more about QR-110 for Leber's congenital amaurosis type 10 and clinical trials
QR-421a for Usher Exon 13
QR-421a for Usher Exon 13

We are developing a novel investigational drug called QR-421a to treat patients with Usher syndrome due to a mutation in a specific part of the USH2A gene, called exon 13. QR-421a is designed to exclude exon 13 from the USH2A mRNA, thereby removing the mutation in exon 13. This approach is also known as exon skipping. QR-421a is intended to be administered via intravitreal injections. Beyond QR-421a, we are developing QR-411 for patients have Usher syndrome due to the c.7595-2144G>A mutation in USH2A.

Read more about QR-421a for Usher syndrome type 2
QRX-504 for FECD
QRX-504 for FECD

Fuchs endothelial corneal dystrophy (FECD) is a common inherited condition characterized by the dysfunction and degeneration of the corneal endothelium, a single cell layer on the inside of the cornea in the eye. There are different types of this disease and we focus on age-related FECD (FECD3). Some patients with age-related FECD develop advanced disease with corneal edema and corneal clouding. These symptoms can lead to complete vision loss and the need for surgery and a corneal transplant. Our program, QRX-504, aims to prevent corneal dystrophy in patients with a mutation in the TCF4 gene by using a single stranded RNA oligonucleotide.

QR-411 for Usher c.7595-2144A>G
QR-411 for Usher c.7595-2144A>G

Our QR-411 program is being developed to treat the c.7595-2144A>G mutation in the USH2A gene that leads to vision loss. Our single stranded RNA oligonucleotide aims to repair the genetic defect in the RNA in the eye, such that it leads to a normal "wild-type" mRNA, which then translates into normal protein, thereby modifying the underlying disease. QR-411 has received orphan drug designation from the U.S. Food and Drug Administration and European Medicines Agency.

QRX-1011 for Stargardt’s
QRX-1011 for Stargardt’s

Stargardt’s disease (fundus flavimaculatus) is a form of juvenile macular degeneration which causes progressive central vision loss, reduced visual acuity, impaired color vision and problems in night vision. The signs and symptoms typically appear in late childhood to early adulthood and worsen over time with loss of peripheral vision. QRX-1011, aims to treat vision loss caused by the specific c.5461-10T>C mutation in ABCA4 gene which leads to an important part of the protein being deleted. Using an antisense oligonucleotide which modulates splicing of the mRNA, QRX-1011-mediated correction in the mRNA level leads to inclusion of the deleted sequence and formation of functional, wild-type ABCA4 protein which will potentially stop and perhaps reverse the progression of the disease.

Dystrophic Epidermolysis Bullosa
DISCOVERY
PRE-CLINICAL DEVELOPMENT
PROOF OF CONCEPT TRIALS
LATE STAGE/ REGISTRATION TRIALS
QR-313 for DEB exon 73
QR-313 for DEB exon 73

Dystrophic epidermolysis bullosa (DEB) is one of the most severe forms of EB, a group of rare genetic skin diseases. We are developing QR-313 for people that have DEB due to a mutation in a specific part of the COL7A1 gene called exon 73. We plan to start a DEB wound observational study to better understand the behavior of DEB wounds over time. In addition, we plan to start a first clinical trial of QR-313 in people with DEB due to mutations in exon 73 of the COL7A1 gene. Beyond QR-313 we are working on QRX-323 and QRX-333 that target mutations in exon 80 and exon 3 of the COL7A1 gene respectively.

Read more about QR-313 for DEB and clinical trials
QRX-323 for DEB exon 80
QRX-323 for DEB exon 80

Dystrophic epidermolysis bullosa (DEB) is one of the most severe forms of EB, a group of rare genetic skin diseases. In addition to the clinical trial that we plan to start for QR-313 in DEB patients with an exon 73 mutation, we are working on QRX-323 and QRX-333 to target other mutations that cause DEB.

QRX-333 for DEB exon 3
QRX-333 for DEB exon 3

Dystrophic epidermolysis bullosa (DEB) is one of the most severe forms of EB, a group of rare genetic skin diseases. In addition to the clinical trial that we plan to start for QR-313 in DEB patients with an exon 73 mutation, we are working on QRX-323 and QRX-333 to target other mutations that cause DEB.

Partially owned programs
DISCOVERY
PRE-CLINICAL DEVELOPMENT
PROOF OF CONCEPT TRIALS
LATE STAGE/ REGISTRATION TRIALS
Fibrosis - Partnered with Galapagos
Fibrosis - Partnered with Galapagos

In collaboration with Galapagos N.V. we are applying our Axiomer RNA editing technology to undisclosed novel fibrosis targets. Galapagos is a target discovery company with a focus on inflammation and fibrosis. In this collaboration we aim to validate their novel fibrosis targets and identify therapeutic compounds to potentially target fibrosis.

Fibrosis - Partnered with Galapagos
Fibrosis - Partnered with Galapagos

In collaboration with Galapagos N.V. we are applying our Axiomer RNA editing technology to undisclosed novel fibrosis targets. Galapagos is a target discovery company with a focus on inflammation and fibrosis. In this collaboration we aim to validate their novel fibrosis targets and identify therapeutic compounds to potentially target fibrosis.

Amylon Therapeutics - AT-010 for HCHWA-D
Amylon Therapeutics

ProQR spun out Amylon Therapeutics in September 2017 as a privately-held company focused on the development of therapies for diseases of the central nervous system including a rare genetic disease which leads to strokes at mid-adulthood, called HCHWA-D. ProQR retains a majority ownership stake in Amylon.

Visit the Amylon website






You are now leaving ProQR Therapeutics

ProQR Therapeutics provides links to web sites of other organizations in order to provide visitors with certain information. A link does not constitute an endorsement of content, viewpoint, policies, products or services of that web site. Once you link to another web site not maintained by ProQR Therapeutics, you are subject to the terms and conditions of that web site, including but not limited to its privacy policy.

You will be redirected to

Click the link above to continue or CANCEL