ProQR Announces Presentations at the European Oligonucleotide and Peptide Therapeutics Conference
October 31 2018 - 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 European Oligonucleotide and Peptide
Therapeutics (EuroTIDES) Conference to be held from November 6 –
November 9, 2018 in Amsterdam, the Netherlands.
Oral presentation on QR-110 clinical data in
LCA10
Title:
Childhood Blindness due to a
Photoreceptor Cilium Defect Treated with an Intravitreal Antisense
Oligonucleotide (QR-110)Presenter:
Peter Adamson, PhD, SVP Ophthalmology Science
of ProQRPresentation: Thursday,
November 8 at 14:15 - 14:45
CETSession:
TRACK 1: Non-Clinical, Preclinical and Clinical
Development
Oral presentation on sterilization of oligonucleotide
drugs
Title:
Evaluating the impact of
sterilization on oligonucleotide based drug
productsPresenter:
Erik van der Hage, PhD, Director Analytical
Development & Quality
Control
Presentation: Thursday,
November 8 at 16:16 – 16:45
CETSession:
TRACK 2: CMC and Analytical Method Development
Strategies
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
(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. QR-110 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. 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 received fast-track designation by the FDA.
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 the most frequent and 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 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. Such
statements include those relating to our participation at the
EuroTIDES conference. 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:Smital
ShahChief Financial Officer T: +1 415 231 6431 ir@proqr.com
Media Contact:Sara ZelkovicLifeSci Public RelationsT: +1 646 876
4933Sara@lifescipublicrelations.com
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