PlayBall10 17 mei 2017 13:21 auteur info PlayBall10 Lid sinds: 22 okt 2004 Laatste bezoek: 25 apr 2024 Aanbevelingen Ontvangen: 106 Gegeven: 24 Aantal posts: 123 ProQR Announces Results for the First Quarter of 2017www.ir.proqr.com/phoenix.zhtml?c=2537... Aanbevelingen 0 ” Quote Reageren Niet oké
I Love Melbourne 17 mei 2017 14:01 auteur info I Love Melbourne Lid sinds: 21 apr 2016 Laatste bezoek: 24 apr 2024 Aanbevelingen Ontvangen: 251 Gegeven: 983 Aantal posts: 688 Het lijkt me dat we op deze berichten vandaag wel gaan stijgen. Wat vinden jullie van de cijfers? Aanbevelingen 0 ” Quote Reageren Niet oké
PlayBall10 17 mei 2017 15:58 auteur info PlayBall10 Lid sinds: 22 okt 2004 Laatste bezoek: 25 apr 2024 Aanbevelingen Ontvangen: 106 Gegeven: 24 Aantal posts: 123 Leerink met een (herhaald) koersdoel van $10 Aanbevelingen 2 ” Quote Reageren Niet oké
PlayBall10 18 mei 2017 12:14 auteur info PlayBall10 Lid sinds: 22 okt 2004 Laatste bezoek: 25 apr 2024 Aanbevelingen Ontvangen: 106 Gegeven: 24 Aantal posts: 123 JMP Securities: MARKET OUTPERFORM Target Price: $14.00De analisten worden steeds positiever. Nu de beleggers nog. Aanbevelingen 1 ” Quote Reageren Niet oké
[verwijderd] 19 mei 2017 14:31 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 Ik ben bescheiden ingestapt...management toont veel zelfvertrouwen en we gaan het zien.Komende data mbt CF zullen veelbetekenend zijn en hoewel niet zonder risico zie ik de huidige koers meer als calloptie dan aandeel en met beperkt verlies , dan wel onbeperkte winstkansen.Nadat er wat meer duidelijkheid bestaat, dan koop ik liever op wat hogere koers extra in, want meen dat er genoeg fantasie zit in het bedrijf.Ik maak me momenteel meer zorgen om de dollar dan de koers van ProQR, want er zijn nogal wat mondiaal onzekere factoren. Aanbevelingen 0 ” Quote Reageren Niet oké
nb 21 mei 2017 13:27 auteur info nb Lid sinds: 09 sep 2009 Laatste bezoek: 25 apr 2024 Aanbevelingen Ontvangen: 474 Gegeven: 85 Aantal posts: 3.309 ik heb mijn nek pittig uitgestoken, hierin. Maar heb op de een of andere manier wel een goed gevoel. Maar je een loer draaien kan altijd. Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 29 mei 2017 12:18 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 Goed je weer hier te zien IVET en dat je bent ingestapt. Ik dacht alleen dat je net als ik een bodemvisser bent. Nu zijn ze nog goedkoop.Groeten,Frank Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 31 mei 2017 15:36 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 ProQR Receives Fast Track Designation from the FDA for QR-110 for Leber’s Congenital Amaurosis Type 10GlobeNewswire•May 31, 2017Comment Key UpdatesProQR receives Fast Track designation by the U.S. Food and Drug Administration (FDA). Closer interaction with FDA could potentially accelerate the development of QR-110 in patients with Leber’s Congenital Amaurosis Type 10 (LCA 10).LCA 10 is one of the most prevalent forms of gene-related blindness in children worldwide and currently there are no therapies commercially available or in clinical development.QR-110 is currently in clinical development with the planned Phase 1/2 open-label trial (PQ-110-001) that will assess the safety, tolerability, pharmacokinetics and efficacy of multiple administrations of QR-110 in one eye of each patient and will include approximately 6 adults and 6 children with LCA 10.Top-line trial results are expected in 2018.LEIDEN, the Netherlands, May 31, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (PRQR) today announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for QR-110, the lead molecule in its ophthalmology pipeline. QR-110 is being developed for the treatment of patients with Leber’s Congenital Amaurosis Type 10 (LCA 10), a rare genetic disease that causes individuals to lose sight, often in the first years of life. QR-110 is a novel investigational RNA oligonucleotide targeting LCA 10 due to the p.Cys998X mutation, which is one of the most prevalent forms of gene-related blindness in children and currently there are no disease modifying therapies commercially available or in clinical development.Fast Track designation is granted by the FDA to drugs that are under development for serious conditions and have the potential to fulfill an unmet medical need. It was established with the intention to bring promising drugs to patients sooner by facilitating the development with more frequent FDA interactions and expediting the review process. “QR-110 is a unique and elegant approach to addressing the underlying genetic defect that leads to blindness in individuals with LCA 10 due to the p.Cys998X mutation. We are very pleased with granting of the Fast Track designation by the FDA for this program as it highlights the need for innovative and efficacious medicines for this devastating disease for which there is currently nothing available,” said Noreen R. Henig, Chief Medical Officer of ProQR. “We are also excited to be able to initiate our first trial for QR-110 as a multidose study and for that we will benefit from the Fast Track Designation. We believe development of QR-110 also opens the possibilities for RNA approaches to target other causes of genetic blindness. We are building our pipeline in ophthalmology and will use our rapid development approach to QR-110 as a model for how to bring RNA therapeutics to patients in need.” About ProQRProQR 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-110QR-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 10Leber’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 the PQ-110-001 StudyPQ-110-001 is an open-label trial that will include approximately 6 children (age 6- 17 years) and 6 adults (= 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients will receive four intravitreal injections of QR-110 into one eye; one every three months for one year. The QR-110 trial is expected to be conducted in three centers with significant expertise in genetic retinal disease in the US and Europe. Aanbevelingen 1 ” Quote Reageren Niet oké
PlayBall10 15 juni 2017 06:28 auteur info PlayBall10 Lid sinds: 22 okt 2004 Laatste bezoek: 25 apr 2024 Aanbevelingen Ontvangen: 106 Gegeven: 24 Aantal posts: 123 Vanmiddag vanaf 14.00 uur ProQR R&D dag.Link naar de webcast: www.webcaster4.com/Webcast/Page/359/2...ProQR R&D dayProQR will organize its second R&D Day on June 15, 2017 in New York, NY. The R&D Day will feature presentations by ProQR management on the progress on its pipeline, including a review and introduction of several near- and medium term value drivers. In addition, several key opinion leaders will discuss the state of the art in research and development on critical areas of the company’s pipeline including CF, inherited blindness, debilitating skin diseases and RNA technologies. Presentations from the speakers will start at 8:00 AM and last until 12:00 PM EST. Kindly RSVP to Ronen Abergel at rabergel@troutgroup.com to reserve a seat or for additional information. WebcastProQR R&D day will be live webcasted, please register using the button below. The webcast is archived on our website for 30 days. PROQR R&D DAY WEBCASTRegister and watch hereREGISTERSummary of discussed topics during R&D DayPresentations on development programs: QR-010 for cystic fibrosis, QR-110 for Leber’s congenital amaurosis Type 10 (LCA 10) and QR-313 for dystrophic epidermolysis bullosa (DEB)An update on the Ophthalmology pipeline including programs for Usher syndrome and the Dermatorlogy pipeline including other DEB mutationsAn introduction to ProQR’s novel RNA editing platform technology called AxiomerSpeakersTo be announcedwww.proqr.com/rd-day/ Aanbevelingen 0 ” Quote Reageren Niet oké
PlayBall10 15 juni 2017 13:24 auteur info PlayBall10 Lid sinds: 22 okt 2004 Laatste bezoek: 25 apr 2024 Aanbevelingen Ontvangen: 106 Gegeven: 24 Aantal posts: 123 ProQR R&D day Highlights Progress on Pipeline and Introduces Axiomer®, a novel proprietary RNA TechnologyKey program features and updates:ProQR to host an R&D day in New York today, June 15, from 8:00am to 1:00pm Eastern Standard Time. The live webcast can be accessed at www.proqr.com/rd-day.Cystic Fibrosis (CF): Full data from the nasal potential difference (NPD) study for QR-010, ProQR’s lead molecule for CF that is studied in two clinical trials in patients, will be presented, along with an update on the ongoing Phase 1b study, for which topline data are expected in September 2017. Steven M. Rowe, M.D., MSPH, a renowned expert in cystic fibrosis research, will discuss the importance of NPD as a biomarker and the unmet needs remaining in the CF treatment landscape.Leber’s Congenital Amaurosis (LCA): Details of the clinical trial for QR-110, ProQR’s lead molecule in the ophthalmology pipeline. Additional preclinical data will be presented.Ophthalmology pipeline: An update and data for the next four programs in the ophthalmology pipeline will be provided, including: QRX-411 and QRX-421 for Ushers syndrome, QRX-504 for Fuchs Endothelial Corneal Dystrophy (FECD) and QRX-1011 for Stargardt’s Disease. Stephen M. Rose, Ph.D., Chief Research Officer at Foundation Fighting Blindness will provide a background on inherited retinal diseases.Dystrophic epidermolysis bullosa (DEB): an update on key pre-clinical functional and delivery data, and an update on preparation for clinical development to be provided for QR-313. M. Peter Marinkovich, M.D., a dermatologist and Director of the Stanford EB disease clinic, will discuss the unmet need in DEB and the current treatment landscape.Axiomer®: ProQR will introduce its novel, proprietary RNA editing platform technology. Art Levin, Ph.D., an internationally recognized expert on the development of oligonucleotide-based therapeutics, will discuss the evolution of RNA therapeutics.As of March 31, 2017, the Company’s current cash of €52.1 million provides a runway into Q3 2018. Aanbevelingen 1 ” Quote Reageren Niet oké
PlayBall10 15 juni 2017 13:25 auteur info PlayBall10 Lid sinds: 22 okt 2004 Laatste bezoek: 25 apr 2024 Aanbevelingen Ontvangen: 106 Gegeven: 24 Aantal posts: 123 Deel 2:LEIDEN, the Netherlands, June 15, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a development stage RNA therapeutics company will provide an update on its product candidates today at an investor event, and introduce Axiomer®, a novel RNA platform technology it pioneered. The R&D Day is hosted by the Company’s management team and will include perspectives from several key opinion leaders. ProQR’s pipeline now includes two clinical programs, one preclinical program and two programs ready to enter development.“Since last year we have made good progress on executing on our strategy to develop life-changing therapies for patients in need, through a diversified pipeline with a balanced risk profile,” said Daniel A. de Boer, CEO of ProQR. “We are pleased to show the progress we have made in our RNA therapeutics pipeline at our second R&D day. Following QR-010 for CF, QR-110 is now in clinical trials for LCA 10, and our third molecule, QR-313 for DEB will move to clinical trials early next year. We are focusing on three important genetic diseases, all with high unmet needs, and all which we believe could greatly benefit from our unique RNA oligonucleotide approach. Within the next 18 months we will have generated clinical data in patients in all these programs.”Axiomer® – editing the RNAProQR is pioneering a next-generation RNA technology called Axiomer®, which we believe has the potential to yield a new class of medicines for genetic diseases. Axiomer® can make single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells. The Axiomer® “Editing Oligo Nucleotides”, or EONs, recruit an endogenously expressed RNA editing system called ADAR, which it can direct to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G). A member of the Scientific Advisory Board, Dr. Levin will present the landscape and evolution of RNA therapeutics and provide his perspective on this unique and proprietary platform technology.“While our focus and priority is on clinical development of our most advanced RNA-based therapeutics to help patients with CF, LCA 10 and DEB, we continue to innovate in RNA science. This innovation effort has led to the discovery of a novel RNA editing technology that we believe can address the underlying cause of a broad range of genetic defects at the RNA level,” said Daniel de Boer. “The invention and patenting of Axiomer® can drive drug discovery and development of a new class of therapeutics, independently and through partnerships.”The R&D Day will feature presentations by ProQR senior management including Daniel de Boer (Chief Executive Officer), Noreen Henig (Chief Medical Officer), Peter Adamson (Head of Ophthalmology), Gerard Platenburg (Chief Innovation Officer) and David Rodman (Chief Development Strategy Officer). Discussions will focus on a review and introduction of several near- and medium term value drivers and progress on the Company’s pipeline. In addition, several leading medical researchers will discuss the state of the art in research and development in relation to the company’s pipeline:Steven M. Rowe, M.D., MSPH, Professor, Department of Medicine, Pediatrics, and Cell Developmental & Integrative Biology, and Director Gregory Fleming James Cystic Fibrosis Research Center University of Alabama.Dr. Rowe is a respected academic physician scientist and a pioneer in the field of personalized therapeutics for cystic fibrosis, cutting-edge discovery in airway disease biology, and translational research in COPD. He is an international authority in the design and conduct of clinical trials targeting the basic CF defect, and has made key advances in the measurement and interpretation of CFTR function. He directs the Cystic Fibrosis Research Center at UAB, which involves over 100 faculty members and has been continuously funded for over 25 years. A board-certified physician, Dr. Rowe serves as a Special Consultant for Translational Science for the Cystic Fibrosis Foundation. He presently has a laboratory of over 25 individuals, embracing lung research from basic discovery, to translational science, to clinical application.Stephen M. Rose, Ph.D., Chief Research Officer at Foundation Fighting Blindness.Dr. Rose oversees the day-to-day operations of the Foundation Fighting Blindness’ Science Department. He also works closely with the clinical arm of the Foundation to establish a seamless pipeline of studies to move preventions and treatments into clinical trials, partnering with biotech and pharma to maximize potential commercialization. Prior to joining the Foundation in 2004, Dr. Rose was a Director in the NIH Office of Science Policy, where he provided oversight on issues regarding recombinant DNA, including human gene transfer clinical protocols. Dr. Rose currently sits on the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee and is a Health Research Alliance Board member.M. Peter Marinkovich, M.D., Associate Professor, Blistering Disease Clinic Department of Dermatology, Stanford University School of Medicine.Dr. Marinkovich is an Associate Professor of Dermatology, a faculty member of the Program in Epithelial Biology and the Stanford Cancer Biology Program. He has an interest in inflammatory skin disease and is Director of the Stanford Epidermolysis Bullous Disease and Psoriasis Clinics. He is also an attending dermatologist at the VA Palo Alto Medical Center. Dr. Marinkovich’s research focuses on pathogenesis and therapy of epidermolysis bullosa, psoriasis, hair disorders and skin cancers.Art Levin, Ph.D., international RNA expert and member of the ProQR Scientific Advisory Board.Dr. Levin has three decades of experience in RNA drug development from discovery through drug registration, both in large pharma and biotech companies. He has been key to the development of numerous of oligonucleotides, including the first approved antisense medicines, and the first microRNA-targeted therapeutic in clinical trials. Dr. Levin has published over 60 scientific articles and served as a director of the Oligonucleotide Therapeutics Society. He has served on ProQR’s Scientific Advisory Board since the company’s inception. R&D Day Event detailsToday, ProQR will host an R&D day in New York, NY from 8:00am to 1:00pm ET. Please email Ronen Abergel, rabergel@troutgroup.com to receive more information and to reserve a seat.WebcastThe live webcast can be accessed at www.proqr.com/rd-day. The 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 90 days following the presentation date.ir.proqr-tx.com/phoenix.zhtml?c=25370... Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 16 juni 2017 10:41 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 thanx voor het plaatsen Playball...je zit er sneller/dichter op dan ik. Ik ben nog aandeeltjes aan het accumuleren...als belegger natuurlijk altijd uit op koopjes dus koop (nog) niet uit de laat.Vervelende bij Degiro is dat ik alleen op 0,05$ limieten kan zetten, terwijl ik met bijv. UniQure op de cent kan handelen.Overigens raad ik Degiro niet echt aan bij/voor kleine noteringen als PRQR...hun systemen halen het niet bij derden die Degiro makkelijk passeren. Aanbevelingen 0 ” Quote Reageren Niet oké
Staycalm 19 juni 2017 11:01 auteur info Staycalm Lid sinds: 23 mrt 2015 Laatste bezoek: 09 feb 2024 Aanbevelingen Ontvangen: 385 Gegeven: 113 Aantal posts: 1.023 Heb de webcast teruggeluisterd dit weekend. Axiomer platform is parel in de dop. Aanbevelingen 0 ” Quote Reageren Niet oké
PlayBall10 19 juni 2017 15:17 auteur info PlayBall10 Lid sinds: 22 okt 2004 Laatste bezoek: 25 apr 2024 Aanbevelingen Ontvangen: 106 Gegeven: 24 Aantal posts: 123 Leerink n.a.v. R&D dag. Koersdoel blijft $ 10 Aanbevelingen 0 ” Quote Reageren Niet oké
PlayBall10 20 juni 2017 16:40 auteur info PlayBall10 Lid sinds: 22 okt 2004 Laatste bezoek: 25 apr 2024 Aanbevelingen Ontvangen: 106 Gegeven: 24 Aantal posts: 123 PRQR: Price: $4.90; Market Cap (M): $115Rating: Buy; Price Target: $40.00 Aanbevelingen 1 ” Quote Reageren Niet oké
[verwijderd] 26 juni 2017 12:45 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 quote:PlayBall10 schreef op 20 juni 2017 16:40:PRQR: Price: $4.90; Market Cap (M): $115Rating: Buy; Price Target: $40.00Grote verschillen in price targets. Dat geeft aan dat men het gewoon niet weet, maar wel fors hoger dan wat er nu op de borden staat. Zeker dat Axiomer is vol potenties en dan is $40.00 nog een schijntje. Ik heb hier een heel goed gevoel bij. NL gaat weer eens goed scoren in de biotech wereld.Groeten,Frank Aanbevelingen 0 ” Quote Reageren Niet oké
[verwijderd] 26 juni 2017 12:47 auteur info [verwijderd] Lid sinds: 01 jan 0001 Laatste bezoek: 01 jan 0001 Aanbevelingen Ontvangen: 0 Gegeven: 0 Aantal posts: 0 quote:Staycalm schreef op 19 juni 2017 11:01:Heb de webcast teruggeluisterd dit weekend. Axiomer platform is parel in de dop. Parels groeien in oesters en die zitten over het algemeen goed dicht. Dus wat er nu naar buiten komt is slechts het tipje,.....Echt goed nieuws dit.Groeten,Frank Aanbevelingen 0 ” Quote Reageren Niet oké
DeZwarteRidder 26 juni 2017 12:53 auteur info DeZwarteRidder Lid sinds: 29 jun 2012 Laatste bezoek: 26 apr 2024 Aanbevelingen Ontvangen: 20850 Gegeven: 9464 Aantal posts: 96.775 quote:PlayBall10 schreef op 20 juni 2017 16:40:PRQR: Price: $4.90; Market Cap (M): $115Rating: Buy; Price Target: $40.00 Wainwright schrijft alles als je maar betaalt:The Firm or its affiliates did not receive compensation from ProQR Therapeutics N.V. for investment banking serviceswithin twelve months before, but will seek compensation from the companies mentioned in this report for investmentbanking services within three months following publication of the research report. Aanbevelingen 0 ” Quote Reageren Niet oké
NielsjeB 1 juli 2017 15:10 auteur info NielsjeB Lid sinds: 29 aug 2014 Laatste bezoek: 26 apr 2024 Aanbevelingen Ontvangen: 3178 Gegeven: 10791 Aantal posts: 4.497 Prospectus en agreement bij de emissie van 1,2 miljoen aandelen ($5 p/a).Prospectus: services.corporate-ir.net/SEC/Documen...Agreement: services.corporate-ir.net/SEC/Documen... Aanbevelingen 0 ” Quote Reageren Niet oké
NielsjeB 5 juli 2017 17:14 auteur info NielsjeB Lid sinds: 29 aug 2014 Laatste bezoek: 26 apr 2024 Aanbevelingen Ontvangen: 3178 Gegeven: 10791 Aantal posts: 4.497 ProQR’s Drug Candidate QRX-411 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMAKey UpdatesProQR’s QRX-411 receives Orphan Drug Designation by the FDA and EMA for the treatment of retinitis pigmentosa, including Usher syndrome, the subtype targeted by QRX-411. Usher syndrome is an inherited condition that is characterized by combined deafness and blindness.QRX-411 targets the pseudo-exon 40 (PE-40) mutation in the USH2A gene and currently there are no therapies commercially available or in clinical development for the vision loss associated with this disease.QRX-411 has shown promising preclinical data in both patient fibroblasts and the optic cup model for mRNA restoration, which was presented at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in May 2017.A lead candidate has been selected for this program and is currently ready for IND-enabling studies.QRX-411 is part of ProQR’s ophthalmology pipeline that currently also includes one clinical compound, QR-110 for Leber’s Congenital Amaurosis Type 10, and three preclinical programs, QRX-421 for Usher syndrome, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.LEIDEN, the Netherlands, July 05, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that the company’s investigational drug QRX-411 has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of retinitis pigmentosa, including Usher syndrome, the subtype targeted by QRX-411. Usher syndrome is the leading cause of combined deafness and blindness due to genetic defects in the Usher gene.ODD in the U.S. and European Union provides a special status for investigational drugs being developed for rare diseases. The ODD programs offer development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the U.S., and ten years in the European Union following market approval.“We are pleased with the progress we have made to date with our novel RNA based therapeutic ophthalmology pipeline for patients suffering from genetic eye diseases. Securing orphan drug designations from the FDA and EMA for QRX-411 is a milestone for the program and highlights the importance of addressing the unmet need of this debilitating disease,” said Daniel A. de Boer, CEO of ProQR, “The severe genetic retinal diseases we are targeting do not have any available therapies, especially disease modifying therapies focused on restoring vision or impeding progression of the disease. We believe our novel RNA oligonucleotide approach has the potential to make a meaningful impact in the lives of Usher syndrome patients and others with rare genetic eye diseases.”Chief Development Strategy Officer, David M. Rodman, MD, notes, “At ProQR we have a unique opportunity to combine the flexibility of our oligonucleotide drug discovery platform with accelerated drug development strategies for rare diseases. Orphan drug designation is an important step in rapidly bringing transformational precision medicines to patients with Usher syndrome and many other genetic causes of blindness in children and adults.”ProQR’s growing ophthalmology portfolio includes:QR-110 for Leber’s congenital amaurosis Type 10 (LCA 10) due to the p.Cys998X mutation, which received IND and CTA clearance and is in clinical development (PQ-110-001 Phase 1/2 safety and efficacy study). QR-110 was also granted Fast Track designation by the FDA and Orphan Drug designation by the FDA and EMA.QRX-411 for Usher syndrome type II due to the PE-40 mutation in the USH2A gene, for which a clinical candidate has been selected and is ready for IND enabling development studies.QRX-421 for Usher syndrome type II due to Exon 13 mutations in the USH2A gene, for which a clinical candidate has been selected and is ready for IND enabling development studies.QRX-1011 for Stargardt’s disease due to c.5461-10T>C mutations in the ABCA4 gene, which is in optimization phase.QRX-504 for Fuchs endothelial corneal dystrophy (FECD), for which a clinical candidate has been selected and is ready for IND enabling development studies.About Usher SyndromeUsher 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 II is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene.About QRX-411QRX-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 ProQRProQR 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*FORWARD-LOOKING STATEMENTSThis 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 QRX-411 and the clinical development and therapeutic potential thereof, statements regarding orphan drug designation, including the intended benefits of such status, statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in our ophthalmology portfolio, and statements regarding our oligonucleotide drug discovery platform. 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, 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.Contact:Bonnie OrtegaDirector, Investor RelationsT: +1 858 245 3983ir@proqr.comir.proqr-tx.com/phoenix.zhtml?c=25370... Aanbevelingen 0 ” Quote Reageren Niet oké
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Beurscodes, betekenis en hulp bij zoeken Europa AEX Euronext Amsterdam BRU Euronext Brussels PSE Euronext Paris LIS Euronext Lissabon CHX CBOE Europe, grote(re) EU aandelen NAV Investment Funds (NAV) Noord-Amerika NYS New York Stock Exchange OTC CBOE BZX Exchange (US) TSE Toronto Stock Exchange Kunt u een instrument niet vinden? Zoek dan via de zogenaamde ISIN code. Elk instrument, aandeel etc. heeft een unieke code. Kies vervolgens - wanneer er meerdere resultaten zijn - de notering op de beurs van uw keuze. Waar vind ik die ISIN code? Google de naam van het instrument, aandeel etc. met de toevoeging 'ISIN'. Als zoeken op ISIN code geen resultaten oplevert hebben wij het instrument of aandeel niet in onze koersendatabase.