ProQR Therapeutics NV NL0010872495

Biotechnologie.

1. [verwijderd] 12 februari 2018 14:15

   auteur info

   • [verwijderd]

     Lid sinds: 01 jan 0001

     Laatste bezoek: 01 jan 0001

   • Aanbevelingen

     Ontvangen: 0

     Gegeven: 0

   •    Aantal posts: 0

   quote:

   jan941 schreef op 12 februari 2018 13:09:

   Persbericht...
   
   ir.proqr.com/news-releases/news-relea...
   

   Foundation Fighting Blindness will provide up to $7.5 million in funding to develop ProQR’s candidate QR-421a for Usher syndrome type 2A
   
   Foundation Fighting Blindness and ProQR enter into a partnership to develop QR-421a for Usher syndrome type 2A, targeting mutations in exon 13 of the causative USH2A gene.
   Foundation Fighting Blindness will provide milestone-based co-funding of up to $7.5 million to ProQR to advance the program into the clinic.
   QR-421a received orphan drug designation from the FDA.
   There are currently no therapies commercially available or in clinical development for the vision loss associated with Usher syndrome type 2A.
   QR-421a is part of ProQR’s growing ophthalmology pipeline which also includes lead candidate, QR-110 for Leber’s congenital amaurosis 10 currently in clinical trials, and three additional pipeline programs, QR-411 that addresses another genetic mutation resulting in Usher syndrome type 2A, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.
   QR-421a is expected to advance towards the clinic in 2018, with clinical data expected in 2019.
   COLUMBIA, Md. and LEIDEN, the Netherlands, Feb. 12, 2018 (GLOBE NEWSWIRE) -- Foundation Fighting Blindness and ProQR Therapeutics N.V. (NASDAQ:PRQR), today announced that they have entered into a partnership to develop QR-421a for Usher syndrome 2A caused by an exon 13 mutation of the causative USH2A gene. Under the agreement, Foundation Fighting Blindness will provide up to $7.5 million in funding to ProQR for the preclinical and clinical development of QR-421a, which is expected to advance towards the clinic in 2018, and safety and efficacy results from the Phase 1/2 trial in Usher syndrome patients are expected in 2019.
   
   Usher syndrome is a devastating genetic disease in which patients first develop hearing loss and then progressive vision loss, thereby threatening their independence and quality of life. Currently there is no treatment for the ophthalmic manifestation of Usher syndrome type 2A. QR-421a is a first-in-class RNA oligonucleotide that is being developed for the treatment of vision loss associated with the disease. QR-421a is designed to modify the RNA such that functional usherin protein is produced in the retina with the goal of stopping the progression of the disease and potentially gaining peripheral vision. ProQR in-licensed the technology underlying QR-421a from Radboud University Medical Center in the Netherlands, where it was invented by lead investigator Dr. Erwin van Wyck.
   
   Foundation Fighting Blindness’ Clinical Research Institute (FFB-CRI) has also launched a natural history study in 120 people with USH2A mutations. The study — known as RUSH2A (“R” stands for “rate of progression”) — was launched in 2017 and is being conducted at about 20 clinical sites around the world. RUSH2A investigators will use a variety of technologies to monitor changes in vision and retinal structure to document and analyze disease progression. Knowledge and data obtained from this trial are intended to provide a better understanding of how USH2A mutations affect the severity and progression of vision loss and help to inform the development of QR-421a.
   
   “Teaming with corporate partners to help promising therapies move through preclinical and clinical development is central to FFB’s strategy so we are very pleased to enter into this partnership with ProQR,” said Benjamin R. Yerxa, PhD, CEO at Foundation Fighting Blindness. “The fact that there are currently no available treatments for Usher syndrome type 2A makes this work that much more exciting and critical.”
   
   QR-421a for Usher syndrome is the second program in ProQR’s growing ophthalmology pipeline scheduled to enter clinical trials. The lead program in the ophthalmology pipeline, QR-110, is currently in a Phase 1/2 safety and efficacy trial in adult and pediatric patients with Leber’s congenital amaurosis 10, due to the p.Cys998X mutation in the CEP290 gene. This pipeline also contains several other molecules for genetic eye diseases, including QR-411 for Usher syndrome type 2A due to the PE-40 mutation, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.
   
   “We are excited to team up with the Foundation Fighting Blindness to develop QR-421a for patients that suffer from Usher syndrome due to exon 13 mutations," said Daniel A. de Boer, CEO of ProQR. “They are the leading private funder of retinal disease research with a very patient centric approach which is a core pillar of our strategy. Through this partnership with the Foundation we plan to gain access to important know-how to develop programs in retinal diseases. We expect that the additional funding will allow us to rapidly advance this novel therapy for this orphan disease with a severe unmet need.”
   
   About Foundation Fighting Blindness
   
   The Foundation Fighting Blindness was established in 1971 and has raised more than $725 million for research on preventing, treating and curing blindness caused by inherited retinal diseases. In excess of 10 million Americans, and millions more worldwide, experience or are at risk for vision loss due to retinal degenerations. Through its support of focused and innovative science, and by teaming with industry, the Foundation drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases.
   
2. [verwijderd] 26 februari 2018 16:23

   auteur info

   • [verwijderd]

     Lid sinds: 01 jan 0001

     Laatste bezoek: 01 jan 0001

   • Aanbevelingen

     Ontvangen: 0

     Gegeven: 0

   •    Aantal posts: 0

   Nog geen enkele handel in aandeel ProqR?
   
   Apart :-)
3. cjapio1 26 februari 2018 16:34

   auteur info

   • cjapio1

     Lid sinds: 09 jun 2015

     Laatste bezoek: 02 feb 2025

   • Aanbevelingen

     Ontvangen: 22

     Gegeven: 11

   •    Aantal posts: 127

   zeker apart
4. droom en vreesman 26 februari 2018 16:37

   auteur info

   • droom en vreesman

     Lid sinds: 20 apr 2016

     Laatste bezoek: 03 jan 2025

   • Aanbevelingen

     Ontvangen: 21

     Gegeven: 25

   •    Aantal posts: 63

   Heeft iemand een idee wat er aan de hand is ?
5. [verwijderd] 26 februari 2018 16:39

   auteur info

   • [verwijderd]

     Lid sinds: 01 jan 0001

     Laatste bezoek: 01 jan 0001

   • Aanbevelingen

     Ontvangen: 0

     Gegeven: 0

   •    Aantal posts: 0

   quote:

   Pl4 schreef op 26 februari 2018 16:23:

   Nog geen enkele handel in aandeel ProqR?
   
   Apart :-)
   

   Vind de volumes in Proqr überhaupt erg laag. Regelmatig in de orde van 20k over een hele dag, lastig als je een positie wil op- of afbouwen (om van handelen maar niet te spreken). Buy & (heel lang) hold dan maar.
6. [verwijderd] 26 februari 2018 17:08

   auteur info

   • [verwijderd]

     Lid sinds: 01 jan 0001

     Laatste bezoek: 01 jan 0001

   • Aanbevelingen

     Ontvangen: 0

     Gegeven: 0

   •    Aantal posts: 0

   Heb er een aantal gekocht om te kijken of er wel handel is, er is gewoon geen vraag vandaag :-)
7. [verwijderd] 27 februari 2018 13:08

   auteur info

   • [verwijderd]

     Lid sinds: 01 jan 0001

     Laatste bezoek: 01 jan 0001

   • Aanbevelingen

     Ontvangen: 0

     Gegeven: 0

   •    Aantal posts: 0

   ProQR Supports Rare Disease Day 2018 and Honors Henri A. Termeer
   
   LEIDEN, the Netherlands, Feb. 27, 2018 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases including cystic fibrosis, Leber's congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced its support of Rare Disease Day 2018, to be held on February 28, 2018. On this day, patients and their families, patient organizations and healthcare providers come together to raise awareness of rare diseases. ProQR is also co-organizing a commemorative event celebrating the legacy of the late Henri A. Termeer, co-founder of ProQR, being held on the same day in Boston, MA.
   Henri Termeer, former CEO of Genzyme and a visionary and pioneer for rare diseases, was ProQR’s co-founder in 2012 and vice chairman of the supervisory board from January 2014 until he unexpectedly passed away on May 12th, 2017. On Rare Disease Day, which is also Henri’s birthday, family, friends and associates will come together to celebrate the renaming of the “North” plaza to “Henri A. Termeer Square”, across from the Genzyme Center in Cambridge, Massachusetts, which was built by him. In 2019, the Square will become home to a life-size sculpture of Henri Termeer. To learn more, please visit termeertribute.org.
   In recognition of Rare Disease Day, ProQR will hold a luncheon at the Company’s headquarters in Leiden, the Netherlands, to host a special guest family in which all three young children are affected by Leber’s congenital amaurosis 10 (LCA 10). The family will share their experiences of living with LCA and how this rare genetic disease has had an impact on their lives. LCA 10 is a genetic eye disorder and leading genetic cause of childhood blindness. ProQR is currently conducting a Phase 1/2 safety and efficacy trial in children and adults that suffer from LCA 10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene.
   “We fully support Rare Disease Day 2018, which will raise public awareness about rare diseases and bring attention to the importance of research to develop therapies for these patients who have no available treatment options,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “With an estimated 7,000 rare diseases known today and with less than 400 available therapies available, there is a dire need to quickly develop and bring to patients the medicines they need. On this day, it is a privilege to pay tribute to Henri, a good friend and mentor and pioneer in rare diseases. We plan to honor his legacy through completing the mission that we set out on together.”
   About Rare Disease Day
   Rare Disease Day is a global campaign to raise awareness of rare diseases. Since its inception in 2008, led by EURORDIS-Rare Diseases Europe and its Council of National Alliances, thousands of events have taken place worldwide on the last day of February to bring attention to rare diseases and how they impact the lives of patients and loved ones. The theme for 2018 is “Research, continuing on from Rare Disease Day 2017.” To learn more, please visit the Rare Disease Day website.
   About the late Henri A. Termer
   Henri was ProQR’s co-founder, vice chairman and served on the Company’s supervisory board since January 2014. From October 1983 to June 2011, Henri served as chairman, president and chief executive officer of Genzyme Corporation. For ten years prior to joining Genzyme, Henri worked for Baxter International Laboratories, Inc., a manufacturer of human health care products. Henri resigned from Genzyme in June 2011 following the acquisition of Genzyme by Sanofi. Widely acknowledged for his contributions to the biotechnology industry and health care field, Henri was active in the areas of humanitarian assistance, policy issues, and innovation in providing access to health care. He was a member of the board of Massachusetts General Hospital and Partners HealthCare, and a member of the board of fellows of Harvard Medical School. Henri was also a member of the board of the Massachusetts Institute of Technology and serves on its Executive Committee and a board member of the Biotechnology Industry Organization (BIO). He was chairman emeritus of the New England Healthcare Institute, a nonprofit, applied research health policy organization he was instrumental in founding. Henri was a board member of ProQR Therapeutics N.V., Abiomed Inc., Aveo Pharmaceuticals, Verastem, Inc., Moderna Therapeutics and Medical Simulation, and was a board member of Allergan, Inc. from 2014 through its acquisition by Actavis in March 2015. In 2008, he was appointed to Massachusetts Governor Deval Patrick’s Council of Economic Advisors. Henri was chairman of the Federal Reserve Bank of Boston’s board of directors from 2010-2011. Henri studied economics at the Economische Hogeschool (Erasmus University, the Netherlands) and earned an MBA from the Darden School at the University of Virginia.
   About Leber’s Congenital Amaurosis 10
   Leber’s congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of which LCA 10 is the more severe forms. LCA 10 is caused by mutations in the CEP290 gene of which the p.Cys998X mutation is the most common. LCA 10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved or other products in clinical development that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA 10 because of this mutation.
   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 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*
   FORWARD-LOOKING STATEMENTS
   This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such statements include those relating to Rare Disease Day 2018, and our clinical development and therapeutic potential of our product candidates. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.
   
8. [verwijderd] 27 februari 2018 13:09

   auteur info

   • [verwijderd]

     Lid sinds: 01 jan 0001

     Laatste bezoek: 01 jan 0001

   • Aanbevelingen

     Ontvangen: 0

     Gegeven: 0

   •    Aantal posts: 0

   Bovenstaande press release geeft nog weer eens aan dat het om meer gaat dan beurskoersen en geld verdienen. Mooi bedrijf!
9. PlayBall10 5 maart 2018 15:16

   auteur info

   • PlayBall10

     Lid sinds: 22 okt 2004

     Laatste bezoek: 02 feb 2025

   • Aanbevelingen

     Ontvangen: 106

     Gegeven: 24

   •    Aantal posts: 123

   30th Annual ROTH Conference
   On Monday, March 12, 2018 at 12:00 pm PT, Smital Shah, Chief Financial Officer of ProQR, will present at the 30th Annual Roth Conference. The conference is being held at the Ritz-Carlton Laguna Niguel, in Dana Point, CA.
   The live and archived webcast of the presentation will be accessible from the ‘Investor Relations’ section of ProQR’s website (www.proqr.com) under ‘Events and Presentations’. The archived webcast will be available for 30 days following the presentation date.
   ir.proqr.com/news-releases/news-relea...
10. [verwijderd] 5 maart 2018 16:19

    auteur info

    • [verwijderd]

      Lid sinds: 01 jan 0001

      Laatste bezoek: 01 jan 0001

    • Aanbevelingen

      Ontvangen: 0

      Gegeven: 0

    •    Aantal posts: 0

    Dat zal de koers ongetwijfeld een boost gaan geven. Er is veel goed nieuws te melden en goed dat ProQR hier acte de presence geeft.
11. [verwijderd] 6 maart 2018 12:50

    auteur info

    • [verwijderd]

      Lid sinds: 01 jan 0001

      Laatste bezoek: 01 jan 0001

    • Aanbevelingen

      Ontvangen: 0

      Gegeven: 0

    •    Aantal posts: 0

    Overnamekandidaat? beurskrant.com/2018/03/06/stijgingspo...
12. [verwijderd] 8 maart 2018 16:11

    auteur info

    • [verwijderd]

      Lid sinds: 01 jan 0001

      Laatste bezoek: 01 jan 0001

    • Aanbevelingen

      Ontvangen: 0

      Gegeven: 0

    •    Aantal posts: 0

    quote:

    Vgekr schreef op 6 maart 2018 12:50:

    Overnamekandidaat? beurskrant.com/2018/03/06/stijgingspo...
    

    Dank voor de tip, ziet er interessant uit. Zit je er zelf ook in?
13. PlayBall10 12 maart 2018 16:21

    auteur info

    • PlayBall10

      Lid sinds: 22 okt 2004

      Laatste bezoek: 02 feb 2025

    • Aanbevelingen

      Ontvangen: 106

      Gegeven: 24

    •    Aantal posts: 123

    labiotech.eu/proqr-cystic-fibrosis-da...
14. Tom3 14 maart 2018 21:49

    auteur info

    • Tom3

      Lid sinds: 25 jan 2015

      Laatste bezoek: 01 feb 2025

    • Aanbevelingen

      Ontvangen: 2924

      Gegeven: 630

    •    Aantal posts: 11.398

    “To date, we’ve dosed about 85 patients, and found that QR-010 improved their respiratory symptoms,” says de Boer. After these first studies in humans, ProQR is now preparing for larger trials that confirm these results.
    
    Kennelijk gaan ze toch verder (zonder partner?) Het lijkt me dat ze niet genoeg middelen hebben omdat zelf allemaal te doen. Het zou toch een stunt zijn als Galapagos een minderheidsaandeel in het bedrijf zou nemen. Dan zitten we er warmpjes bij.
15. PlayBall10 15 maart 2018 08:58

    auteur info

    • PlayBall10

      Lid sinds: 22 okt 2004

      Laatste bezoek: 02 feb 2025

    • Aanbevelingen

      Ontvangen: 106

      Gegeven: 24

    •    Aantal posts: 123

    Galapagos, Editas, Ionis und ProQR jagen Vertex im Kampf um den Milliardenmarkt Mukoviszidose
    
    www.fool.de/2018/03/14/galapagos-edit...
16. [verwijderd] 15 maart 2018 20:41

    auteur info

    • [verwijderd]

      Lid sinds: 01 jan 0001

      Laatste bezoek: 01 jan 0001

    • Aanbevelingen

      Ontvangen: 0

      Gegeven: 0

    •    Aantal posts: 0

    quote:

    Tom3 schreef op 14 maart 2018 21:49:

    “To date, we’ve dosed about 85 patients, and found that QR-010 improved their respiratory symptoms,” says de Boer. After these first studies in humans, ProQR is now preparing for larger trials that confirm these results.
    
    Kennelijk gaan ze toch verder (zonder partner?) Het lijkt me dat ze niet genoeg middelen hebben omdat zelf allemaal te doen. Het zou toch een stunt zijn als Galapagos een minderheidsaandeel in het bedrijf zou nemen. Dan zitten we er warmpjes bij.
    

    Dat was ook mijn gedachte, maar ik denk dat dat pas gebeurt als Gala de rechten terugkrijgt van abvie. En dan een combi kan maken met eigen targets. Ze zullen niet snel gaan concurreren met Abvie denk ik.
17. Guy2024 20 maart 2018 17:20

    auteur info

    • Guy2024

      Lid sinds: 15 sep 2015

      Laatste bezoek: 02 feb 2025

    • Aanbevelingen

      Ontvangen: 48

      Gegeven: 2

    •    Aantal posts: 306

    Het blijft toch een vreemd aandeel vind ik..Veel positieve geluiden,maar we raken maar niet uit het slop precies..Hoop dat 3 dollar zowat de bodem blijft..
18. Tom3 21 maart 2018 00:33

    auteur info

    • Tom3

      Lid sinds: 25 jan 2015

      Laatste bezoek: 01 feb 2025

    • Aanbevelingen

      Ontvangen: 2924

      Gegeven: 630

    •    Aantal posts: 11.398

    quote:

    Guy-vdk schreef op 20 maart 2018 17:20:

    Het blijft toch een vreemd aandeel vind ik..Veel positieve geluiden,maar we raken maar niet uit het slop precies..Hoop dat 3 dollar zowat de bodem blijft..
    

    Ondanks alle positieve persberichten is het aandeel inderdaad niet vooruit te branden. Vooral die mensen die ingestapt zijn op het dubbele (of hoger) van de huidige koers zullen met gekromde tenen achter hun PC zitten. Lees net dat Galapagos weer 300k aandelen de markt opgooit. Kunnen ze ProQR royaal van bekostigen lijkt me. Wil ik ook nog wel Galapagos aandeelhouder voor worden als het moet.
19. TD_01 23 maart 2018 18:20

    auteur info

    • TD_01

      Lid sinds: 26 sep 2017

      Laatste bezoek: 02 feb 2025

    • Aanbevelingen

      Ontvangen: 54

      Gegeven: 4

    •    Aantal posts: 827

    Ik begrijp het ook niet meer goed! Ik begin er zelfs aan te denken om eventueel mijn verlies te nemen en in iets anders te investeren...
20. [verwijderd] 23 maart 2018 18:40

    auteur info

    • [verwijderd]

      Lid sinds: 01 jan 0001

      Laatste bezoek: 01 jan 0001

    • Aanbevelingen

      Ontvangen: 0

      Gegeven: 0

    •    Aantal posts: 0

    quote:

    TD_01 schreef op 23 maart 2018 18:20:

    Ik begrijp het ook niet meer goed! Ik begin er zelfs aan te denken om eventueel mijn verlies te nemen en in iets anders te investeren...
    

    Ik heb zelf slechte ervaringen met geduld kwijtraken en uitstappen bij fondsen waar ik ooit instapte voor goede redenen. Ik blijf dus zitten ondanks dat ik absoluut niet blij ben met de huidige koers (ingestapt in 6 usd regione, bijgekocht in de afgelopen jaren en zit er nu gemiddeld in voor ongeveer 4 usd).