ProQR Therapeutics N.V.(Nasdaq:PRQR) today announced that the Company will present three abstracts during the ARVO conference, including additional positive pre-clinical PoC data for QR-110 in LCA 10.

“RNA based therapeutics for patients suffering from genetic eye diseases are a real opportunity to address an unmet need and we are now at the forefront of this with QR-110 advancing into the clinic. We’re also proud to share data that supports other promising candidates in our ophthalmology pipeline at ARVO”, said Noreen R. Henig, Chief Medical Officer at ProQR. “These programs have many common elements with QR-110 and we hope to advance these rapidly with the pre-clinical and clinical experience in LCA 10.”

ARVO Conference
During the 2017 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) to be held on May 7 – 11, 2017 in Baltimore, MD, USA, the Company will present three abstracts reporting pre-clinical data for three programs targeting forms of genetic retinopathy resulting in blindness.

For ProQR’s lead ophthalmic program, QR-110 for Leber’s congenital amaurosis Type 10 (LCA 10), a poster (#249 – B0280) with additional in vitro pharmacodynamic data will be presented on Sunday, May 7from 8:30 – 10:15am ET. ProQR has received IND clearance to start a clinical trial for QR-110 in LCA 10 patients. Children and adults in the trial will be treated for a full year with QR-110 to assess safety and efficacy.

Next in ProQR’s ophthalmology pipeline are two programs each targeting specific mutations that result in Usher syndrome. QRX-411 and QRX-421 are candidate molecules that will be presented in two oral presentations. QRX-411 for the c.7595-2144A>G mutation will be presented on Monday, May 8, 2017 from 8:45 – 9:00am ET. QRX-421 for Exon 13 mutations will be presented on Tuesday, May 9, 2017 from 11:45am – 12:00pm.

Abstract titles:

  • QR-110 Treatment for Leber’s Congenital Amaurosis Type 10: Restoration of CEP290 mRNA Levels and Ciliation in LCA 10 iPSC-Derived Optic Cups
  • QRX-411, an RNA Oligonucleotide (AON) Directing a Splice Correction in USH2A mRNA Caused by the Frequent Deep-Intronic c.7595-2144A>G Mutation Associated with Retinitis Pigmentosa in Usher Syndrome Type 2
  • QRX-421, an RNA Oligonucleotide (AON) Targeting Mutations in Exon 13 of USH2A, Associated with Retinitis Pigmentosa in Usher Syndrome Type 2, is Effective in Skipping Exon 13 in the USH2A mRNA of Patients Fibroblasts and Patient-Derived Optic Cups

The abstracts are published on the ARVO 2017 annual meeting website.

About ProQR 
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis Type 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. 
*Since 2012* 

About QR-110
QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis Type 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore wild-type CEP290 mRNA leading to the production of wild-type CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union.

About Leber’s Congenital Amaurosis Type 10
Leber’s congenital amaurosis is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of which LCA Type 10 (LCA 10) is one of the more severe forms. LCA 10 is caused by mutations in the CEP290 gene of which the p.Cys998X mutation is most common. LCA 10 leads to progressive loss of vision causing most patients to lose their sight in the first few years of life. To date, there are no treatments approved or products in clinical development that treat the underlying cause of the disease. Although prevalence rates vary, we believe approximately 2,000 people in the Western world have LCA 10 because of this mutation.

About QRX-411 
QRX-411 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the USH2A gene. The mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional or absence of USH2A protein. QRX-411 is designed to restore wild-type USH2A mRNA leading to the production of wild-type USH2A protein by binding the mutated pre-mRNA causing normal splicing of the pre-mRNA.

About QRX-421
QRX-421 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional USH2A protein that causes the disease. QRX-421 is designed to exclude exon 13 from the mRNA (exon skipping) and produce truncated but functional USH2A protein, thereby modifying the underlying disease.

About Usher syndrome
Usher syndrome is the leading cause of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. To date, there are no treatments approved or products in clinical development that treat the vision loss associated with the disease. Usher syndrome Type 2 is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene.

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    I would list pros. But I think I check out the cons. Because I saw a comedy sketch using this system Asian girl poking fun at Asian men, And I couldn help but think to myself “so now. That pathetic, I think it nice for PoC to see how other PoC view them who are outside the equation. To our site. which neither Asian or white, It looks pretty damn pathetic. appears self hating. I assume that if another member of your race points you out as being self hating, You deny their allegations. But if a person who isn apart of your race, Completely out of the equation points out how you make yourself look and notices a similar thing that the people in your race pointed out to you, Then maybe it open your eye area to how bad you look. appears bad. Of coarse not the amount of couples are like this, keep in mind. Just like not all white women with black men are frugal racist, And not all black men with white women are uncle toms, But I wanted to drop a paragraph here on the negativity I see making use of their pairing.

    It doesn situation. If they love some other, everything. I am an East Asian feminine. rankings marry a white guy, simply because if we had children, They would be treated more advanced than i was. If i were to bring a child into our planet, Mixed with a race besides caucasian, I would really worry for them and their experiences they’ll have in this society. no less if i have kids with a white man, Our children might be more accepted in society and won have to endure as much sht. I have cousins who’ re mixed race, and may even easily pass off as white and i NEVER hear them complain, Or even touch upon injustices towards them.

    I don think much relating to this. People say its so common but I rarely view it. If I ever do notice it, Its only nerdy white guys dating asian girls and I not sure why its exactly the nerds, Maybe theres a connection with shod and non-shod? Also I don think most white men have yellow fever because thats a false label. Most asian for women who live white fever because statistically they prefer white guys more. Also numerous people that practice this are just using each other,The white guy just wants a submissive girl and has a creepy fetish and the asian lady wants the green card or status so I think people need to be a bit careful with regard to this relationship. A lot of asian girls I met told me they think inferior and have self hate issues, They wish they were white this is why they only want white men so a lot of white men are fetishized, Put on a pedestal and used by working on this. Not all are like that but these type of people are out there and people need to be a bit cautious when dating. I don see anything wrong with it as long as people aren using each other. Other then that I don think much tends to make.

    Everyone is going to have a different opinion on interracial marriage and theres really no wrong or right answer. everyone handles hyperhidrosis differently with different views so not everyone will agree. Its just an opinion and, please remember you shouldn take it to heart.

    Most asian women I knew growing up wanted white men. They found them more attractive wanted the beautiful mixed children. They found them more easy to fall in love with and be attracted to as a preference.

    I only ran into a small few that did not feel because of this. So when I see such couple, I can gather there is plausible that the woman feels this way from my own experiences. Some see white as being better or more fine to them. I am being real here.

    I am an Asian female don mind asian or white males but decided that it was my choice to accept who I am and that Asian is beautiful and they are the men.

    I wouldn care if the Asian woman or man dated interracially exceeding just white people or if they didn date white people 99% of the time instead of their own race, But that not how they are.

    99% of the East Asian us citizens I see who are not “Fresh from the boat” Are married or in a special connection with white people. It idiotic. They don date ones own race, They don date double races, it just white people. Both the men and the ladies. and consequently I just see them as thirsty and deliberately looking for a white partner to give them the half white babies they want because they like white physical features more. the kind that East Asians do this IS self hate. They don date whites, people don date Indians, They don date what other race, They don date their selves, They date white others.

    coming from a Black girl. I fine along with it, It none of my opportunity. All relationships are different. Race is not to do with it. Of course like all interracial and family relationships or romance. There are likely to be some problems due to cultural differences and other things, As long as it isn affecting your relationship. I personally have a problem with people, Who assume negative reasons for other people, since they’re from a different ethnic group, It shows how shallow it is as a chinese date person. Love the person while accepting who they really are. You can really tell what a couple will almost certainly act like from an outer appearance. There are good and bad points to every relationship.

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