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ProQR Therapeutics 2019

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4finance
0
quote:

Wil Helmus schreef op 11 december 2019 08:24:


[...]
Q1 2020 kunnen we weer nieuws verwachten. Resultaten fase 1/2 Stellar trial QR-421a. En Q1 duurt niet zo lang meer.


Ok, thx.
Yearling
1

ProQR Announces First Patient Dosed in Phase 1/2 Aurora Trial of QR-1123 for Autosomal Dominant Retinitis Pigmentosa
11 December 2019 at 7:00 AM EST
LEIDEN, Netherlands and CAMBRIDGE, Mass., Dec. 11, 2019 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced the first patient dosed in the Phase 1/2 Aurora clinical trial of QR-1123 in patients with autosomal dominant retinitis pigmentosa (adRP). Initial data from the study are expected in 2021.

“From previous clinical trials we have seen that RNA therapies can be a promising approach for patients with inherited retinal diseases and there is a strong need for novel approaches in diseases that currently have no treatments,” said David G. Birch, Ph.D., Principal Investigator of Aurora and Scientific Director of the Retina Foundation of the Southwest in Dallas, Texas. “QR-1123 has shown encouraging results in preclinical models and we hope to see that translated to the clinic in this first-in-human trial in patients with adRP.”

David Rodman, M.D., Executive Vice President of Research & Development of ProQR, said, “QR-1123 aims to block expression of the toxic mutated rhodopsin protein in the retina thereby targeting the underlying cause of the vision loss associated with adRP due to the P23H mutation. We are excited to have started a clinical trial for the third RNA therapy in our inherited retinal disease pipeline.”

About the Phase 1/2 Aurora trial

Aurora, or PQ-1123-001, is a first-in-human study that will initially include up to 35 adults with adRP due to the P23H mutation in the rhodopsin (RHO) gene. The trial will include single-dose escalation (open label) groups and multiple-dose escalation (double-masked) groups in which intravitreal injections of QR-1123 or sham procedures will be given in one eye. The objectives of the trial include evaluation of safety and tolerability. Efficacy as measured by improvement of visual function and retinal structure will be assessed through ophthalmic endpoints such as visual acuity, visual field and optical coherence tomography. The trial will be conducted at expert sites in North America.

About QR-1123

QR-1123 is a first-in-class investigational RNA-based oligonucleotide designed to potentially treat adRP due to the P23H mutation in the RHO gene. QR-1123 was discovered and developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology. 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 mutant 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. QR-1123 was in-licensed from Ionis Pharmaceuticals in 2018. QR-1123 has been granted Orphan Drug designation in the United States and received Fast Track designation from the FDA.

About adRP

Autosomal dominant retinitis pigmentosa, or adRP, is a severe and rare genetic disease that causes progressive problems in night vision during childhood, leading to visual field loss and frequently resulting in blindness in mid adulthood. In the United States, the most prevalent mutation associated with adRP is the P23H point mutation (also known as the c.68C>A mutation) in the rhodopsin (RHO) gene and affects approximately 2,500 people. This mutation causes misfolding of the rhodopsin protein that becomes toxic to the photoreceptor cells and at the same time diminishes the function of the wild type allele. Over time this results in cell death and progressive vision loss. There are currently no therapies approved or in clinical development for P23H adRP. A natural history study in patients with P23H adRP has been conducted.

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, Usher syndrome and autosomal dominant retinitis pigmentosa. 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 forward-looking statements include, but are not limited to, statements regarding QR-1123 and the clinical development and the therapeutic potential thereof, and statements regarding PQ-1123-001, including trial design. 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:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or
Hans Vitzthum
LifeSci Advisors
T: +1 617 535 7743
hans@lifesciadvisors.com

Media Contact:
Sara Zelkovic
LifeSci Public Relations
T: +1 646 876 4933
sara@lifescipublicrelations.com

ProQR logo1200x1200.png

Source: ProQR Therapeutics N.V.
Bioteg71
0
Stijgt nu lekker door. Hoop dat het doorzet.. Hoop dat er meer te melden valt op dit draadje, de meesten zijn bezig met andere aandelen op dit forum :(
Staycalm
0
Mee eens Biotech71. Gelukkig is daar nu een apart draadje voor. Nog even geduld en dan wordt het hier drukker, zo opportunistisch werkt het. Je kan er maar beter voor die tijd inzitten als niemand aandacht heeft voor de fundamentals.
Staycalm
2
www.pharmasalmanac.com/articles/tackl...

Aggressive Goals
ProQR has plans to become a multiproduct platform company independently developing and commercializing therapies for patients that suffer from IRDs. Our Vision 2023 strategy includes having at least two commercial products and at least three late-stage and seven early-stage programs in development by 2023, while expanding our RNA platform capabilities in other therapeutic areas through our subsidiary Amylon Therapeutics (brain disease) and spin-out Wings Therapeutics (dermatology) and establishing selective partner programs in non-core therapeutic areas and non-rare diseases.
Given the high unmet need across many of the 350+ IRDs known today, we believe this integrated approach will allow us to more efficiently turn our scientific innovation into multiple potentially life-changing medicines for patients. With our experienced team, predictive translational models and capabilities in precision medicine, we are well-positioned to execute on this long-term strategy and advance the field of medicines for inherited blindness
Staycalm
2
Nice:

Axiomer® Technology
One of the technologies applied to RNA therapy development at ProQR is our proprietary, Axiomer RNA editing platform, which involves editing oligonucleotide (EON)-directed therapeutic editing. Endogenous adenosine deaminase acting on RNA (ADAR) is recruited by a single-strand EON and transduces I groups to G groups, thereby allowing targeted G-to-A mutations.
Given that more than 20,000 diseases are caused by G-to-A mutations, we believe this technology has the potential to yield new medicines for currently untreatable diseases. Previously, ProQR established a first partnership around the Axiomer technology with Galapagos N.V. focused on select fibrosis targets.
Wil Helmus
0
Ambitieuze plannen zeg. Laten we hopen dat ze het waar gaan maken. Dan is het kassa voor ons
4finance
0
quote:

Staycalm schreef op 12 december 2019 18:45:


Nice:

Axiomer® Technology
One of the technologies applied to RNA therapy development at ProQR is our proprietary, Axiomer RNA editing platform, which involves editing oligonucleotide (EON)-directed therapeutic editing. Endogenous adenosine deaminase acting on RNA (ADAR) is recruited by a single-strand EON and transduces I groups to G groups, thereby allowing targeted G-to-A mutations.
Given that more than 20,000 diseases are caused by G-to-A mutations, we believe this technology has the potential to yield new medicines for currently untreatable diseases. Previously, ProQR established a first partnership around the Axiomer technology with Galapagos N.V. focused on select fibrosis targets.


Ziet er allemaal goed uit. Ik heb nog 3k stuks aandelen. Misschien ga ik weer wat bijkopen. Vandaag eerst nog wat Arbutus (ABUS) bijgekocht.

BPPP
0
Nou, dat gaat goed. Weet iemand hoe groot de short posities zijn? Dat kon wel eens tot een squeeze leiden; terug naar de $24.- !
Tom3
0
quote:

BPPP schreef op 12 december 2019 20:45:


Nou, dat gaat goed. Weet iemand hoe groot de short posities zijn? Dat kon wel eens tot een squeeze leiden; terug naar de $24.- !


Shorts vindt je op de Nasdaq site van ProQR: die waren altijd erg laag. Voorlopig stijgen we alleen op grond van peptalk van De Boer zo lijkt het. Zeker van Dinko Velerio geleerd. Ik zie liever harde data en een wetenschappelijk artikel over hoe je dat Axiomer heelhuids in een cel kunt inbrengen. Veel RNA medicijnen zijn tot heden gestrand op de "delivery".
Wil Helmus
0
Artikel van 6 december, maar volgens mij nog niet geplaatst hier.

Tackling Genetic Eye Diseases with Novel RNA Therapies

www.pharmasalmanac.com/articles/tackl...
Bioteg71
0
quote:

Wil Helmus schreef op 13 december 2019 09:19:


Artikel van 6 december, maar volgens mij nog niet geplaatst hier.

Tackling Genetic Eye Diseases with Novel RNA Therapies

www.pharmasalmanac.com/articles/tackl...


FF goed kijken :), een paar reacties naar boven kijken....zie bericht van Staycalm.
Tom3
0
quote:

Wil Helmus schreef op 13 december 2019 09:19:


Artikel van 6 december, maar volgens mij nog niet geplaatst hier.

Tackling Genetic Eye Diseases with Novel RNA Therapies

www.pharmasalmanac.com/articles/tackl...


En weer wordt de samenwerking met Galapagos aangehaald. Op de AVA heb ik toch echt gehoord dat dit in de koelkast stond. Het schiet niet echt op met het vinden van andere partners. Axiomer is, als je De Boer mag geloven, een wereldvinding waar ook anderen goed aan kunnen verdienen.
Wil Helmus
0
quote:

Tom3 schreef op 13 december 2019 09:56:


[...]

En weer wordt de samenwerking met Galapagos aangehaald. Op de AVA heb ik toch echt gehoord dat dit in de koelkast stond. Het schiet niet echt op met het vinden van andere partners. Axiomer is, als je De Boer mag geloven, een wereldvinding waar ook anderen goed aan kunnen verdienen.

Vreemd, op de site van Galapagos is ook helemaal niks te vinden over een samenwerkingsverband met ProQR. Maar meneer De Boer zal toch geen onwaarheden verkondigen lijkt me? Misschien is de samenwerking onlangs weer aangehaald en kunnen we binnenkort nieuws daarover verwachten?
Tom3
0
@Wil Helmus, dat laatste zou het aandeel doen verdubbelen in waarde. Er zijn heel weinig biotechs die zonder sponsor kunnen. ProQR moet opboksen tegen een Editas die financieel ondersteund wordt voor haar oog trials. Je kunt er in feite niet zonder. Vd Stolpe te gierig?
BPPP
0
quote:

Tom3 schreef op 12 december 2019 22:24:


[...]

Shorts vindt je op de Nasdaq site van ProQR: die waren altijd erg laag. Voorlopig stijgen we alleen op grond van peptalk van De Boer zo lijkt het. Zeker van Dinko Velerio geleerd. Ik zie liever harde data en een wetenschappelijk artikel over hoe je dat Axiomer heelhuids in een cel kunt inbrengen. Veel RNA medicijnen zijn tot heden gestrand op de "delivery".


Hartelijk dank!
BPPP
0
Jaarverslag ProQR 2018 p. 5: "Our Supervisory Board is currently composed of the following members, all of whom are independent under
applicable NASDAQ standards and all of whom, with the exception of Mr. Dinko Valerio and Mr. Antoine Papiernik are independent under the Dutch Corporate Governance Code (DCGC):"

en "Mr. Valerio is founder and former CEO of Crucell N.V., a Dutch biotech company, and founder and former general partner of Aescap Venture, a life sciences venture capital firm. In 1999, Mr. Valerio was one of the founders of Galapagos Genomics N.V., a spinout from Crucell N.V. which develops novel mode of action medicines. In 2017 Mr Valerio became a boardmember of Amylon Therapeutics B.V., a 80% owned affiliate of ProQR Therapeutics N.V. Adding to his corporate experience, Mr. Valerio is a professor in the field of gene therapy of the hematopoietic system at the University of Leiden."
BPPP
0
quote:

Wil Helmus schreef op 13 december 2019 11:23:


[...]
Vreemd, op de site van Galapagos is ook helemaal niks te vinden over een samenwerkingsverband met ProQR. Maar meneer De Boer zal toch geen onwaarheden verkondigen lijkt me? Misschien is de samenwerking onlangs weer aangehaald en kunnen we binnenkort nieuws daarover verwachten?


Naar ik meen werd alleen dit (vorige bericht) op de jaarvergdering genoemd.
Missolapola
0
quote:

Wil Helmus schreef op 13 december 2019 11:23:


[...]
Vreemd, op de site van Galapagos is ook helemaal niks te vinden over een samenwerkingsverband met ProQR. Maar meneer De Boer zal toch geen onwaarheden verkondigen lijkt me? Misschien is de samenwerking onlangs weer aangehaald en kunnen we binnenkort nieuws daarover verwachten?


Uit de Earnings Conference Call van 26 juli jl. waaruit blijkt dat ze bij GLPG wel oog hebben voor RNA :

“Graig Suvannavejh :

Question about BD. Given the company's traditional focus on small molecules and you talk about platforms that you're looking at, can you just give us additional color? Are you looking at, say, biologic approaches or [indiscernible]. Any color there on the platforms looking at.

Onno van de Stolpe

Well, it's not our of first priority to see if we can get the live molecules into our platform or in our pipeline, but also not excluding it at this point in time. It's still very early days. We have to sit together with the teams and see what makes best sense to move forward. But clearly, we will we looking at new areas for Galapagos, including RNA, which seems to be a very attractive area to expand into. So we will be open for various different technologies to answer the research engine of Galapagos.”
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