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Prof. Dollar
0
Dat valt uit het jaarverslag op te maken:

We operate a 53,343 square feet GMP qualified manufacturing facility that we lease in Lexington, Massachusetts. In November 2018, we extended and expanded the facility by leasing an additional 30,655 square feet of the same building. The expanded and extended lease for the facility terminates in June 2029, and subject to the provisions of the lease, may be renewed for two subsequent five-year terms.
Prof. Dollar
1
Resultaten uit de Parkinson-studie (GDNF).

Phase 1 Trial of Convection-Enhanced Delivery of Adeno-Associated Virus Encoding Glial Cell Line-Derived Neurotrophic Factor in Patients with Advanced Parkinson’s Disease

Authors: Davis Palmer Argersinger, B.S.; Codrin Lungu, MD; Dima Hammoud, MD; Peter Herscovitch, MD; Debra Ehrlich, MD; Gretchen Scott; Krystof Bankiewicz, MD, PhD; Kareem Zaghloul, MD, PhD; Mark Hallett, MD; Russell Lonser, MD; John Heiss, MD (Washington, DC)

Introduction:
Parkinson’s disease is progressive and presently incurable. GDNF is a neurotrophic factor that prevented the death of dopaminergic neurons in culture and animal models of Parkinson’s disease (PD).

Methods:
In this Phase 1 clinical trial, 13 adult patients with advanced PD underwent convection-enhanced delivery (CED) of an adeno-associated virus, serotype 2 vector containing glial cell line-derived neurotrophic factor (AAV2-GDNF) to investigate the safety, tolerability, and potential clinical effects of CED of AAV2-GDNF and gadoteridol, a surrogate magnetic resonance imaging (MRI) tracer, into the bilateral putamina (450 µl per hemisphere). Three escalating dose levels were evaluated: 1) 9 x 1010vg (6 patients); 2) 3 x 1011vg (6 patients); and 3) 9 x 1011vg (1 patient). Intraoperative MRI was performed during infusions. Pre-operatively, and at 6-12 month intervals post-operatively, Unified Parkinson’s Disease Rating Scale (UPDRS) Part 3 assessed motor function and positron emission tomography (PET) scanning with [18F]DOPA assessed F-DOPA uptake, a sign of presynaptic dopaminergic integrity.

Results:
The patients tolerated AAV2-GDNF infusion without short- or long-term clinical or radiographic toxicity. MRI tracked AAV2-GDNF infusion within the bilateral putamina. Average coverage of the putamina was 22%. UPDRS Part 3 assessment scores remained stable throughout the study. Increased [18F]DOPA uptake in the infused areas was seen bilaterally in 10/13 patients at 6 months (range: 5-274%, median: 36%), and in 12/13 patients at 18 months after infusion (range: 8-130%, median: 54%).

Conclusion:
Patients with advanced PD tolerated bilateral CED of AAV2-GDNF without toxicity. Gadoteridol in the infusion solution tracked putaminal AAV2-GDNF distribution. PET findings of increased putaminal [18F]DOPA uptake suggest a neurotrophic effect on dopaminergic neurons. Based on a satisfactory safety profile, a follow-up clinical trial is planned to increase putaminal coverage and possibly reverse PD progression and signs.

www.aans.org/Annual-Scientific-Meetin...
Beurs2019
0
quote:

Prof. Dollar schreef op 2 apr 2019 om 17:42:


Dat valt uit het jaarverslag op te maken:

We operate a 53,343 square feet GMP qualified manufacturing facility that we lease in Lexington, Massachusetts. In November 2018, we extended and expanded the facility by leasing an additional 30,655 square feet of the same building. The expanded and extended lease for the facility terminates in June 2029, and subject to the provisions of the lease, may be renewed for two subsequent five-year terms.

Dank Prof. Uitbreiding facility zal niet voor de kat zijn viool zijn gedaan :-) Hoopvol.
Izette
0

Want to participate in a research study? Help shape the future of investing tools and earn a $60 gift card!

Small-caps and large-caps are wildly popular among investors; however, mid-cap stocks, such as uniQure N.V. (NASDAQ:QURE) with a market-capitalization of US$2.2b, rarely draw their attention. Despite this, the two other categories have lagged behind the risk-adjusted returns of commonly ignored mid-cap stocks. QURE’s financial liquidity and debt position will be analysed in this article, to get an idea of whether the company can fund opportunities for strategic growth and maintain strength through economic downturns. Note that this information is centred entirely on financial health and is a top-level understanding, so I encourage you to look further into QURE here.

See our latest analysis for uniQure

Does QURE Produce Much Cash Relative To Its Debt?

Over the past year, QURE has ramped up its debt from US$21m to US$35m , which accounts for long term debt. With this increase in debt, the current cash and short-term investment levels stands at US$235m , ready to be used for running the business. Its negative operating cash flow means calculating cash-to-debt wouldn't be useful. For this article’s sake, I won’t be looking at this today, but you can take a look at some of QURE’s operating efficiency ratios such as ROA here.

Can QURE meet its short-term obligations with the cash in hand?

At the current liabilities level of US$20m, it seems that the business has been able to meet these commitments with a current assets level of US$237m, leading to a 11.85x current account ratio. The current ratio is calculated by dividing current assets by current liabilities. However, a ratio greater than 3x may be considered high by some.


NasdaqGS:QURE Historical Debt, April 4th 2019



NasdaqGS:QURE Historical Debt, April 4th 2019
More
Can QURE service its debt comfortably?

QURE’s level of debt is appropriate relative to its total equity, at 20%. This range is considered safe as QURE is not taking on too much debt obligation, which can be restrictive and risky for equity-holders. Risk around debt is very low for QURE, and the company also has the ability and headroom to increase debt if needed going forward.

Next Steps:

QURE’s low debt is also met with low coverage. This indicates room for improvement as its cash flow covers less than a quarter of its borrowings, which means its operating efficiency could be better. However, the company will be able to pay all of its upcoming liabilities from its current short-term assets. I admit this is a fairly basic analysis for QURE's financial health. Other important fundamentals need to be considered alongside. I suggest you continue to research uniQure to get a better picture of the stock by looking at:
1.Future Outlook: What are well-informed industry analysts predicting for QURE’s future growth? Take a look at our free research report of analyst consensus for QURE’s outlook.
2.Valuation: What is QURE worth today? Is the stock undervalued, even when its growth outlook is factored into its intrinsic value? The intrinsic value infographic in our free research report helps visualize whether QURE is currently mispriced by the market.
3.Other High-Performing Stocks: Are there other stocks that provide better prospects with proven track records? Explore our free list of these great stocks here.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned. Thank you for reading.
Prof. Dollar
0
uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease tools.eurolandir.com/tools/Pressrelea...
T. Montana
0
quote:

Prof. Dollar schreef op 8 apr 2019 om 13:20:


uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease tools.eurolandir.com/tools/Pressrelea...


Heel goed. Ik geloof niet dat deze op je "wat kunnen we dit jaar verwachten lijstje" stond?
Prof. Dollar
0
Klopt, deze stond niet op het lijstje maar was natuurlijk wel te verwachten.

Ik ben benieuwd hoe QURE de voordelen van een eventuele versnelde goedkeuring gaat benutten. Ik had de indruk dat zij voor een fase I/II studie tamelijk veel patiënten willen toelaten. Met oog op de variabele tijd (hoe ontwikkelt de ziekte zich), hebben ze daar later mogelijk baat bij; 1) als de vervolgstudie veilig blijkt te zijn hebben ze meteen veel langer termijn data beschikbaar, 2) als risico-reductie van SCA Type 3 programma.
Izette
0
colt
1
Ik zie (lees) cijfers voorbij gaan E90,- ... E180,- ...en mogelijk binnenkort zelfs boven E380,-. Ik blijf rustig zitten, t wordt spannend hier
Prof. Dollar
0
uniQure Announces Presentations at the Upcoming Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) www.iex.nl/Forum/Topic/1286603/68/Wat...
colt
0
"We hebben het afgelopen jaar aanzienlijke vooruitgang geboekt bij het verbeteren van onze gentherapie-expertise door uitbreiding van onze onderzoekspijplijn", aldus Sander van Deventer, MD, Ph.D., chief scientific officer bij uniQure. "We zijn erg blij met deze een sterke vertoning bij ASGCT en om nieuwe preklinische gegevens te hebben over de ziekte van Huntington, hemofilie A en de ziekte van Fabry. We zien uit naar het hebben van deze gegevens op de conferentie."
"We have made significant progress over the past year in advancing our gene therapy expertise through expansion of our research pipeline,” stated Sander van Deventer, M.D., Ph.D., chief scientific officer at uniQure. “We are very pleased to have such a strong showing at ASGCT, and to have new preclinical data featured in Huntington’s disease, hemophilia A and Fabry disease. We look forward to having these data prominently featured at the conference.”
Prof. Dollar
0
quote:

Prof. Dollar schreef op 15 apr 2019 om 18:30:


uniQure Announces Presentations at the Upcoming Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT)

En nu met de juiste verwijzing: tools.eurolandir.com/tools/Pressrelea...
Prof. Dollar
0
Een halfjaar geleden heeft QURE haar hemofilie A benadering gepresenteerd. Daarin zetten ze hun programma ook af tegen de huidige alternatieven, waarbij Sander van Deventer opmerkte dat er tijdens het onderzoek naar Emicizumab patiënten zijn overleden. Zojuist vernam ik: "3 more deaths on #emicizumab (#Hemlibra) have been reported today, bringing the total to 10. No details available as they occurred in post marketing. 2500 patients currently on emicizumab. We need to know if this number of deaths is more than expected." Bron: twitter.com/ProfMakris/status/1117869...
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