ProQR to Present at Three Scientific Conferences in April
April 22 2019 - 7:00AM
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
upcoming presentations at the Retinal Cell & Gene Therapy
Innovation Summit, the annual meeting of the Association for
Research in Vision and Ophthalmology (ARVO) and the annual meeting
of the American Society for Gene and Cell Therapy (ASGCT).
Retinal Cell & Gene Therapy Innovation
Summit The conference is being held on Friday, April 26,
2019 in Vancouver, BC, Canada.
Abstract title: |
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Novel type of antisense oligonucleotide treatment
(QR-421a) for retinitis pigmentosa (RP) due to exon 13 USH2A
mutations |
Presenter: |
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Robert K. Koenekoop, MD, PhD, principle
investigator of the ProQR Phase 1/2 STELLAR clinical trial of
QR-421a and Professor of Pediatric Surgery, Human Genetics and
Ophthalmology at McGill University, Montreal |
Presentation: |
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Friday, April 26 at 5:00pm PT |
Session: |
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Non Viral Gene Editing |
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Abstract title: |
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Results of first-in-human study of an antisense
oligonucleotide in subjects with LCA10 |
Presenter: |
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Bart P. Leroy, MD, PhD, principle investigator of
the ProQR Phase 1/2 clinical trial of sepofarsen and Chairman and
Head of the Department of Ophthalmology at the Center for Medical
Genetics at the Ghent University |
Presentation: |
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Friday, April 26 at 8:15am PT |
Session: |
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Gene Therapy: Clinical Applications and
Outcomes |
Annual meeting of the Association for Research in Vision
and Ophthalmology (ARVO)
The conference is being held from April 28 – May 2, 2019 in
Vancouver, BC, Canada.
Abstract title: |
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CEP290-associated LCA due to a photoreceptor
cilium defect treated with an intravitreal antisense
oligonucleotide results in improved vision |
Presenter: |
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Artur V. Cideciyan, PhD, co-investigator of the
PQ-110-001 clinical trial of sepofarsen and Research Professor of
Ophthalmology at the Scheie Eye Institute of the University of
Pennsylvania |
Poster: |
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1834 - A0101 |
Session: |
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#240 - Visual Impairment - Measures of Visual
Function |
Date & time: |
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April 29, 2019 from 11:15 am to 1:00 pm PT |
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Abstract title: |
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QR-421a, an antisense oligonucleotide, for the
treatment of retinitis pigmentosa due to USH2A exon 13
mutations |
Presenter: |
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Hester van Diepen, Director Ophthalmology at
ProQR |
Presentation: |
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3250 |
Session: |
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#340 - Retina/RPE New drugs, Mechanisms of
action, and Toxicity |
Date &
Time: |
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April 30, 2019 from 1:00 pm to 1:15 pm PT |
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Abstract
title: |
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Allele specific knock-down of human P23H
rhodopsin mRNA and prevention of retinal degeneration in humanized
P23H rhodopsin knock-in mouse, following treatment with an
intravitreal GAPmer antisense oligonucleotide (QR-1123) |
Presenter: |
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Patricia Biasutto, VP, Project Lead for QR-1123
at ProQR |
Poster: |
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5719 - A0114 |
Session: |
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#506 - Retina/RPE new drugs, mechanism of action,
and toxicity |
Date & time: |
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May 2, 2019 from 8:00 am to 9:45 am PT |
Annual meeting of the American Society for Gene and Cell
Therapy (ASGCT)
The conference is being held from April 28 – May 2, 2019 in
Washington, DC.
Presenter: |
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David Rodman, MD, EVP Research & Development
of ProQR |
Presentation: |
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Tuesday, April 30 at 9:00 – 9:30 am ET |
Session: |
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RNA therapeutics education session |
About sepofarsen
Sepofarsen 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 (also known
as the c.2991+1655A>G 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. Sepofarsen is designed to restore
normal (wild-type) CEP290 mRNA leading to the production of normal
CEP290 protein by binding to the mutated location in the pre-mRNA
causing normal splicing of the pre-mRNA. Sepofarsen 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 received fast-track designation from the
FDA.
About QR-421a
QR-421a is a first-in-class investigational RNA-based
oligonucleotide designed to address the underlying cause of vision
loss in Usher syndrome type 2 and non-syndromic retinitis
pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
QR-421a is designed to restore functional Usherin protein by using
an exon skipping approach with the aim to stop or reverse vision
loss in patients. QR-421a 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
received fast-track designation from the FDA.
About QR-1123
QR-1123 is a first-in-class investigational oligonucleotide
(gapmer) that was developed by Ionis Pharmaceuticals using Ionis’
proprietary antisense technology for the treatment of adRP due to
the P23H mutation in the RHO gene. The therapy aims to inhibit the
formation of the mutated toxic version of the rhodopsin protein by
specifically binding the mutated RHO mRNA. Binding of QR-1123
causes allele specific knockdown of the mutated mRNA by a mechanism
called RNase H mediated cleavage without affecting the normal RHO
mRNA. QR-1123 is intended to be administered through intravitreal
injections in the eye.
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 and Usher syndrome type 2. 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 our participation at the
Retinal Cell & Gene Therapy Innovation Summit, the annual
meeting of the Association for Research in Vision and Ophthalmology
(ARVO) and the annual meeting of the American Society for Gene and
Cell Therapy. 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.ProQR Therapeutics N.V.Investor
Contact:Investor Contact:Hans VitzthumLifeSci AdvisorsT: +1
617-535-7743hans@lifescieadvisors.com
Media Contact:Sara ZelkovicLifeSci Public RelationsT: +1 646 876
4933Sara@lifescipublicrelations.com
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