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Aandeel ProQR Therapeutics NV OTC:PRQR.Q, NL0010872495

  • 0,860 12 aug 2022 22:00
  • +0,045 (+5,51%) Dagrange 0,820 - 0,863
  • 1.121.480 Gem. (3M) 452,5K

ProQR Therapeutics 2018

897 Posts
Pagina: «« 1 ... 6 7 8 9 10 ... 45 »» | Laatste | Omlaag ↓
  1. forum rang 4 ivet 1 augustus 2018 13:14
    ProQR Announces Publication on QR-110 for Leber’s Congenital Amaurosis 10 in the Journal Molecular Therapy - Nucleic Acids

    LEIDEN, the Netherlands, Aug. 01, 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, today announced the publication of a peer-reviewed manuscript describing the preclinical validation for the use of QR-110 in the treatment of Leber’s congenital amaurosis 10 (LCA10) in the journal Molecular Therapy - Nucleic Acids.

    LCA10 is a severe inherited retinal dystrophy associated with mutations in the CEP290 gene. QR-110 is an RNA-based drug candidate that has the potential to restore sight or slow down the process of vision loss in patients with LCA10 by correcting the most common mutation causing LCA10, p.Cys998X. This mutation causes incorrect splicing of CEP290 mRNA.

    This publication details the screening process by which QR-110 was identified as a potentially optimal therapeutic candidate using a patient-derived retinal organoid (“eye cup”) model. In additional in vitro testing, QR-110 demonstrated a dose-dependent restoration of mRNA and functional CEP290 protein while in vivo testing showed that QR-110 was well-tolerated following intravitreal injection. The results from this study suggest the pharmacodynamic, pharmacokinetic and safety properties make QR-110 a promising candidate for the treatment of LCA10. On the basis of the work detailed in this publication, QR-110 is currently being evaluated in a Phase 1/2 clinical trial in patients with LCA10.

    “Given that the p.Cys998X mutation is a uniquely human mutation, there is no reliable in vivo model. Therefore, the eye cup model was essential in our preclinical validation process described in the paper,” said Peter Adamson, Ph.D., senior vice president of ophthalmology for ProQR. “The ongoing clinical trial of QR-110 in patients with LCA10 is evaluating the predictive accuracy of the model. If successful, we intend to use the platform to move our additional ophthalmology programs towards clinical development.”

    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 LCA10 is one of the more severe forms. LCA10 is caused by mutations in the CEP290 gene, of which the p.Cys998X mutation is the most common. LCA10 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 LCA10 because of this mutation.

    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 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 and fast track status by the FDA.

    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 Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. 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. 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. These forward-looking statements include, but are not limited to, statements regarding QR-110 and its clinical development and therapeutic potential.  Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, regulatory review or approval process, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and 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.

    ProQR Therapeutics N.V.:
    Smital Shah
    Chief Financial Officer
    T: +1 415 231 6431
    ir@proqr.com

    This message was distributed by GlobeNewswire.
    2321 Rosecrans Ave. Ste 2200, El Segundo, CA, 90245, USA. +1-800-307-6627. www.globenewswire.com
    On behalf of ProQR Therapeutics N.V. Zernikedreef 9, Leiden, 2333CK NL

  2. ousen 4 augustus 2018 11:29
    Op 24-7-18 ook een bescheiden positie ingenomen op € 6,85
    Kijken wat de koers gaat doen. Als deze onder mijn aankoopbedrag komt,
    koop ik wellicht nog wat bij. Zit ook in Gala en verwacht hier ook wel wat van komende weken.
    Heeft iemand overigens kennis van het aandeel Tapimmune?
    Koers gisteren gedaald. Hier ook een kleine positie ingenomen op 7,28.
    Dit is gewoon een gokje.
  3. forum rang 4 Kelewan 6 augustus 2018 22:50
    quote:

    ousen schreef op 4 augustus 2018 11:29:

    Op 24-7-18 ook een bescheiden positie ingenomen op € 6,85
    Kijken wat de koers gaat doen. Als deze onder mijn aankoopbedrag komt,
    koop ik wellicht nog wat bij. Zit ook in Gala en verwacht hier ook wel wat van komende weken.
    Heeft iemand overigens kennis van het aandeel Tapimmune?
    Koers gisteren gedaald. Hier ook een kleine positie ingenomen op 7,28.
    Dit is gewoon een gokje.
    Komende weken? Ik verwacht over enige jaren een goede kans op iets heel moois.
    Maar net als gocrucellgo en ivet - ook oud Crucell belegger - heb ik er wel het geduld voor. Heb een flink pakket, met al een aardige winst, maar met de bedoeling die lang te houden. IMHO heb je met korte termijn meer kansen met andere aandelen, al was het maar omdat er te weinig handel is om te kunnen traden in proqr.
  4. [verwijderd] 7 augustus 2018 16:17
    Kelewan, op de eerste plaats dank voor al je postings, zeker ook op het Galapagos forum, wordt zeer gewaardeerd.
    Ikzelf zit verreweg voor het grootste gedeelte in Galapagos en daarnaast ook voor 10% in ProQR en en een plukje in Merus.
    Gezien je kennis aangaande biotech aandelen, vraag ik mij af in welke aandelen in deze sector je nog meer actief bent, of welke je volgt.
  5. forum rang 4 Kelewan 7 augustus 2018 17:55
    quote:

    jan941 schreef op 7 augustus 2018 16:17:

    Kelewan, op de eerste plaats dank voor al je postings, zeker ook op het Galapagos forum, wordt zeer gewaardeerd.
    Ikzelf zit verreweg voor het grootste gedeelte in Galapagos en daarnaast ook voor 10% in ProQR en en een plukje in Merus.
    Gezien je kennis aangaande biotech aandelen, vraag ik mij af in welke aandelen in deze sector je nog meer actief bent, of welke je volgt.
    Dank voor je bericht, maar ik vermoed dat anderen (zie met name het draadje op het Galapagos forum, "waar beleggen jullie nog meer in"), een interessanter antwoord hebben.
    Maar om je vraag te beantwoorden:
    Galapagos is veruit het grootste fonds in mijn porto, Proqr echter ook bijna 10%. De rest is grotendeels anders dan biotech (waarvan ASML en DSM de toppers zijn). Qua biotech zit ik ook nog in Argenx en een paar kleinere posities in Mithra, Kiadis en Zealand.
  6. forum rang 4 ivet 8 augustus 2018 13:13
    ProQR Announces Financial Results for the Second Quarter of 2018

    LEIDEN, The Netherlands, Aug. 08, 2018 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced results for the second quarter of 2018.

    “During the second quarter, we built up momentum across our pipeline of development programs, including the initiation of the WINGS study, the first clinical trial to evaluate QR-313 in patients with dystrophic epidermolysis bullosa. QR-313 is now our third program to enter clinical development,“ said Daniel A. de Boer, chief executive officer of ProQR. “Over the next twelve months, we are looking forward to key clinical data read-outs from all three clinical development programs that could potentially provide broader validation of our platform in ophthalmology and dermatology. The clinical trial evaluating QR-110, our lead program in ophthalmology, in patients with Leber’s congenital amaurosis 10 remains on track for an interim data announcement later this year.”

    Second Quarter 2018 Corporate Highlights

    Initiated WINGS, the first clinical trial to evaluate the safety and efficacy of QR-313 in patients that have recessive dystrophic epidermolysis bullosa (RDEB) due to mutations in exon 73 of the COL7A1 gene
    Interim results expected in late 2018; full data expected in 2019
    Entered into a partnership with EB Research Partnership (EBRP) and EB Medical Research Foundation (EBMRF)
    ProQR will receive up to $5 million in funding for the clinical development of QR-313
    Appointed Yi-Tao Yu, Ph.D., a professor of biochemistry and biophysics at the University of Rochester Medical Center, to the Company’s scientific advisory board
    Dr. Yu’s research focuses on RNA editing and his expertise will help to develop the Company’s novel RNA editing technologies
    Presented at the European Cystic Fibrosis Society (ECFS) conference on eluforsen, formerly known as QR-010
    Abstract title: Exploratory immune assays distinguish healthy volunteer from CF patient cohorts and were validated in a dose escalation study of QR-010 in subjects with cystic fibrosis homozygous for the F508del CFTR mutation
    With current cash on hand of €33.0 million (at June 30, 2018), ProQR’s operation is funded into late 2019
    Subsequent Events

    A paper with preclinical data for QR-110 targeting Leber’s congenital amaurosis 10 (LCA10) was published in Molecular Therapy – Nucleic Acids, an official journal of the American Society of Gene & Cell Therapy
    The research was done in collaboration with scientists at the UCL Institute of Ophthalmology in London, United Kingdom and the Radboud University Medical Center in Nijmegen, the Netherlands
    Presented preclinical data for QR-421a for Usher syndrome at the International Symposium on Usher Syndrome (USH2018)
    Abstract title: Splice modulation to treat USH2A-associated retinal degeneration
    Financial Highlights

    At June 30, 2018, ProQR held cash and cash equivalents of €33.0 million, compared to €48.1 million at December 31, 2017. Net cash used in operating activities during the three month period ended June 30, 2018 was €5.4 million, compared to €9.8 million for the same period last year.

    Research and development costs totaled €6.0 million for the quarter ended June 30, 2018 compared to €7.6 million for the same period last year and comprised of allocated employee costs including share-based payments, the costs of materials and laboratory consumables, outsourced activities, license and intellectual property costs and other allocated costs.

    General and administrative costs decreased to €2.6 million for the quarter ended June 30, 2018 compared to €2.9 million for the quarter ended June 30, 2017.

    Net loss for the three month period ended June 30, 2018 was €7.4 million or €0.23 per share, compared to a €11.3 million loss or €0.47 per share for the same period last year. For further financial information for the period ended June 30, 2018, please refer to the financial statements appearing at the end of this release.
  7. forum rang 4 Pl4 8 augustus 2018 19:32
    quote:

    nb schreef op 8 augustus 2018 19:16:

    Het leuke van een aandeel waar je laag bent ingestapt, is dat het steeds harder gaat. Een stijgende lijn trekt goudzoekers.
    Gaat lekker
    En nog altijd slechts 220 miljoen market cap.

    Als de ontwikkelingen goed doorgaan en de wind komt er echt onder kan huidige koers verveelvoudigen.
  8. forum rang 6 RW1963 8 augustus 2018 21:26
    quote:

    Pl4 schreef op 8 augustus 2018 19:32:

    [...]

    En nog altijd slechts 220 miljoen market cap.

    Als de ontwikkelingen goed doorgaan en de wind komt er echt onder kan huidige koers verveelvoudigen.
    Gaat lekker nu ;-)
    Zou het dezelfde kant op kunnen gaan als Galapagos. Of is dat niet realistisch? ProQR is bij mij de dood of de gladiolen nl.
  9. forum rang 4 Pl4 8 augustus 2018 21:45
    quote:

    RW1963 schreef op 8 augustus 2018 21:26:

    [...]

    Gaat lekker nu ;-)
    Zou het dezelfde kant op kunnen gaan als Galapagos. Of is dat niet realistisch? ProQR is bij mij de dood of de gladiolen nl.
    Moeilijk te voorspellen.

    Maar de pipeline ontwikkelt en de huidige waarde is erg laag volgens mij kijkend naar vergelijkbare US biotechs.

    Interessante samenwerkingen gesloten (ook met Galapagos) en paar interessante mensen in management.
897 Posts
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Koers 0,860   Verschil +0,04 (+5,51%)
Laag 0,820   Volume 1.121.480
Hoog 0,863   Gem. Volume 452.477
12 aug 2022 22:00
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