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Uniqure NV (QURE) Earns “Buy” Rating from Chardan Capital
March 28th, 2016 - By Renee Johnson - 0 comments
Uniqure NV logoChardan Capital reaffirmed their buy rating on shares of Uniqure NV (NASDAQ:QURE) in a research report sent to investors on Wednesday morning, MarketBeat.com reports. They currently have a $40.00 price objective on the stock.
A number of other analysts have also recently commented on the stock. Zacks Investment Research cut shares of Uniqure NV from a buy rating to a hold rating in a research report on Wednesday, November 25th. Cowen and Company restated a buy rating on shares of Uniqure NV in a research report on Monday, November 30th. Leerink Swann restated an outperform rating and issued a $48.00 price objective on shares of Uniqure NV in a research report on Friday, January 8th. Janney Montgomery Scott started coverage on shares of Uniqure NV in a research report on Monday, November 30th. They issued a buy rating and a $40.00 price objective for the company. Finally, Piper Jaffray restated an overweight rating and issued a $35.00 price objective on shares of Uniqure NV in a research report on Wednesday, January 6th. One analyst has rated the stock with a hold rating and eleven have given a buy rating to the company. The stock has a consensus rating of Buy and an average target price of $40.36.
Uniqure NV (NASDAQ:QURE) opened at 11.95 on Wednesday. The firm’s market capitalization is $289.41 million. Uniqure NV has a one year low of $10.61 and a one year high of $36.38. The company has a 50 day moving average price of $13.92 and a 200-day moving average price of $18.07.
Several institutional investors have bought and sold shares of the company. Perceptive Advisors LLC increased its position in Uniqure NV by 40.1% in the fourth quarter. Perceptive Advisors LLC now owns 967,093 shares of the company’s stock worth $15,996,000 after buying an additional 276,824 shares in the last quarter. Eventide Asset Management LLC increased its stake in shares of Uniqure NV by 12.9% in the fourth quarter. Eventide Asset Management LLC now owns 174,900 shares of the company’s stock valued at $2,893,000 after buying an additional 20,000 shares during the period. Columbus Circle Investors increased its stake in shares of Uniqure NV by 493.7% in the fourth quarter. Columbus Circle Investors now owns 113,548 shares of the company’s stock valued at $1,878,000 after buying an additional 94,422 shares during the period. Deutsche Bank AG increased its stake in shares of Uniqure NV by 53.2% in the fourth quarter. Deutsche Bank AG now owns 112,088 shares of the company’s stock valued at $1,853,000 after buying an additional 38,943 shares during the period. Finally, Tocqueville Asset Management L.P. increased its stake in shares of Uniqure NV by 5.6% in the fourth quarter. Tocqueville Asset Management L.P. now owns 79,750 shares of the company’s stock valued at $1,319,000 after buying an additional 4,200 shares during the period.
Uniqure NV is a pharmaceutical company based in the Netherlands. It is primarily engaged in the field of gene therapy for orphan diseases. The Company is focused on patients suffering from genetic or acquired diseases by directing the expression of a therapeutic protein or restoring the expression of a missing protein through a single administration.
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http://www.iramarketreport.com/uniqure-nv-qure-earns-buy-rating-from-chardan-capital/41523/
QURE
New rating:
3/24/2016 HC Wainwright Reiterated Rating Buy $30.00
http://www.marketbeat.com/stocks/NASDAQ/QURE/?RegistrationCode=SocialMedia-StockTwits
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Uniqure NV’s (QURE) “Buy” Rating Reaffirmed at Chardan Capital
Posted by mitch on Mar 24th, 2016 // No Comments
Uniqure NV logoChardan Capital reiterated their buy rating on shares of Uniqure NV (NASDAQ:QURE) in a research report released on Wednesday, Analyst Ratings Network.com reports. Chardan Capital currently has a $40.00 price objective on the stock.
Several other analysts have also commented on the stock. Zacks Investment Research lowered shares of Uniqure NV from a buy rating to a hold rating in a research report on Wednesday, November 25th. Cowen and Company reissued a buy rating on shares of Uniqure NV in a research report on Monday, November 30th. Leerink Swann reissued an outperform rating and set a $48.00 price target on shares of Uniqure NV in a research report on Friday, January 8th. Janney Montgomery Scott started coverage on shares of Uniqure NV in a research report on Monday, November 30th. They set a buy rating and a $40.00 price target for the company. Finally, Piper Jaffray reissued an overweight rating and set a $35.00 price target on shares of Uniqure NV in a research report on Wednesday, January 6th. One equities research analyst has rated the stock with a hold rating and eleven have given a buy rating to the company. The stock currently has an average rating of Buy and a consensus price target of $40.36.
A number of institutional investors have modified their holdings of the company. Tocqueville Asset Management L.P. increased its position in shares of Uniqure NV by 5.6% in the fourth quarter. Tocqueville Asset Management L.P. now owns 79,750 shares of the company’s stock valued at $1,319,000 after buying an additional 4,200 shares during the period. Deutsche Bank AG increased its position in shares of Uniqure NV by 53.2% in the fourth quarter. Deutsche Bank AG now owns 112,088 shares of the company’s stock valued at $1,853,000 after buying an additional 38,943 shares during the period. Columbus Circle Investors increased its position in shares of Uniqure NV by 493.7% in the fourth quarter. Columbus Circle Investors now owns 113,548 shares of the company’s stock valued at $1,878,000 after buying an additional 94,422 shares during the period. Eventide Asset Management LLC increased its position in shares of Uniqure NV by 12.9% in the fourth quarter. Eventide Asset Management LLC now owns 174,900 shares of the company’s stock valued at $2,893,000 after buying an additional 20,000 shares during the period. Finally, Perceptive Advisors LLC increased its position in shares of Uniqure NV by 40.1% in the fourth quarter. Perceptive Advisors LLC now owns 967,093 shares of the company’s stock valued at $15,996,000 after buying an additional 276,824 shares during the period.
Shares of Uniqure NV (NASDAQ:QURE) opened at 11.86 on Wednesday. The company’s market capitalization is $287.23 million. Uniqure NV has a 1-year low of $10.61 and a 1-year high of $36.38. The firm’s 50-day moving average is $14.06 and its 200-day moving average is $18.11.
Uniqure NV is a pharmaceutical company based in the Netherlands. It is primarily engaged in the field of gene therapy for orphan diseases. The Company is focused on patients suffering from genetic or acquired diseases by directing the expression of a therapeutic protein or restoring the expression of a missing protein through a single administration.
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http://www.thevistavoice.org/2016/03/24/uniqure-nvs-qure-buy-rating-reaffirmed-at-chardan-capital/983946/
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uniQure Announces Preclinical Proof of Concept for Gene Therapy Approach in Huntington's Disease
Publication in Molecular Therapy-Nucleic Acids Demonstrates Successful Silencing of Mutated Huntingtin Protein Using microRNA Delivered with uniQure's Proprietary AAV5 Vector
AMSTERDAM, the Netherlands, March 22, 2016 (GLOBE NEWSWIRE) --
uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced the publication of preclinical data supporting its proprietary Huntington's disease gene therapy program, AMT-130. Findings published in the current issue of the peer-reviewed journal Molecular Therapy-Nucleic Acids (www.nature.com/mtna/journal/v5/n3/index.html) provide preclinical proof of concept for uniQure's AMT-130 program and demonstrate the potential of a one-time administration of AAV5-delivered gene therapy into the central nervous system (CNS) to silence the Huntingtin gene (HTT). An inherited, mutated form of HTT causes Huntington's disease, a rare, fatal, neurodegenerative disorder that leads to severe physical and cognitive deterioration.
The paper, titled "Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease", was authored by a research team led by Pavlina Konstantinova, Ph.D., Director of Emerging Technologies at uniQure under the direction of Chief Scientific Officer Harald Petry, Ph.D. The publication describes multiple approaches to silencing HTT using expression cassette-optimized artificial microRNAs (miHTTs). Several miHTT scaffolds were incorporated in an AAV5 vector using uniQure's established baculovirus-based manufacturing platform and administered to a humanized mouse model. The data demonstrate strong silencing of mutant HTT and total HTT silencing in vitro and in vivo. Furthermore, it was shown that HTT knock-down efficiency could be increased to 80% by using optimized miHTT scaffolds. The data published today were in part presented at the 11th Annual CHDI Huntington's Disease Therapeutics Conference on February 24, 2016 by Dr. Konstantinova.
Based on these results, uniQure has initiated further studies of AMT-130 to support the filing of an investigative new drug application with the FDA.
"Huntington's disease devastates families and there is currently no effective disease-modifying treatment," commented Charles W. Richard, M.D., Ph.D., Senior Vice President, Research and Development, Neuroscience at uniQure. "We are excited by the results of this study, and believe this degree of knock-down of mutant Huntingtin protein, if duplicated in our ongoing non-human primate safety toxicology studies and future human clinical trials, could significantly alter the course of the disease."
"Dr. Konstantinova and her team have made significant progress in the search for an effective treatment for this cruel neurodegnerative disorder," said Dan Soland, Chief Executive Officer of uniQure. "AMT-130 now represents our third gene therapy product candidate in the CNS area, in addition to AMT-110 in Sanfilippo B and the NIH-sponsored program in Parkinson's disease. We will continue to leverage our deep experience in the CNS field, as well as our validated manufacturing capabilities and AAV5 technology, to advance AMT-130 towards the clinic."
About Huntington's Disease
Huntington's disease is a severe genetic neurodegenerative disorder causing loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration over a 12-15 year period of time. The disease is caused by an autosomal dominant mutation, a cytosine-adenine-guanine (CAG) expansion, in the first exon of the Huntingtin gene leading to a non-functional, aggregation prone mutated protein. Despite the clear etiology, there are no therapies available to treat the disease, delay onset or slow progression of a patient's decline.
About uniQure
uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with CNS, liver/metabolic and cardiovascular diseases. www.uniQure.com
uniQure Forward-Looking Statement
This presentation 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 the future development of our programs in Huntington's disease, Parkinson's disease and Sanfilippo B, and the progress of any of our ongoing or planned clinical studies and/or development of our product candidates. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with collaboration arrangements, our and our collaborators' clinical development activities, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's 2014 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 7, 2015. 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: uniQure:
Eva Mulder
Direct: +31 20 240 6103
Main: +31 20 240 6000
e.mulder@uniQure.com
Media inquiries:
Gretchen SchweitzerMacDougall Biomedical Communications
Direct: +49 172 861 8540
Main: +49 89 2424 3494 or +1 781 235 3060
gschweitzer@macbiocom.com
Source: uniQure
Read more: http://www.nasdaq.com/press-release/uniqure-announces-preclinical-proof-of-concept-for-gene-therapy-approach-in-huntingtons-disease-20160322-00648#ixzz43e7FbRQ0
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I took a position yesterday also, took one look at the 1 year chart and I couldn't help myself...
Don't know but got to believe we're seeing more volatility due to shorts. Highest short interest in a year, maybe ever:
http://www.nasdaq.com/symbol/qure/short-interest
http://shortanalytics.com/getshortchart.php?tsymbol=qure
QURE
I do believe it'll bounce back eventually. As you say, just small orders shaking it down.
I got in at 11.91 and thought I was in at the bottom. I did notice small buy and sell orders which suggests that this is being manipulated to take out stop loss orders for cheap shares possibly.
uniQure Announces Dosing of First Patient in High-Dose Cohort in Phase I/II Hemophilia B Clinical Trial
AMSTERDAM, the Netherlands, March 14, 2016 (GLOBE NEWSWIRE) --
uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced that the Company has treated the first patient in the second cohort of its ongoing AMT-060-01 Phase I/II trial in hemophilia B patients. The primary objective of the second cohort is to assess the safety of the systemic administration of a higher dose of uniQure's AMT-060 gene therapy in hemophilia B patients that present with a severe or moderately-severe disease phenotype. Secondary objectives of the trial include evaluation of Factor IX (FIX) activity levels as well as evaluation of annualized bleeding rates and recombinant FIX usage.
The Company announced preliminary top-line results of the low-dose cohort on January 7th, 2016, showing that AMT-060 was generally well tolerated. Additionally, the first two patients that had completed at least 12 weeks of follow-up showed promising increases in FIX expression levels of 5.5% and 4.5% of normal as of the December 16, 2015 cutoff date, and in four of the five patients dosed the need for continuous, prophylactic rFIX treatment was eliminated as of January 6th, 2016. Including the patients screened and treated in the low-dose cohort, a total of eight patients screened to date for the trial have tested negative for pre-existing anti-AAV5 antibodies. uniQure intends to present an updated analysis of all patients in the low-dose cohort at a scientific conference in the second quarter of 2016.
"Our goal for the second cohort is to evaluate AMT-060's safety profile at a higher dose and to test our hypothesis that with this dose we could achieve further increases in FIX expression levels," said Deya Corzo, Senior Vice President and Therapeutic Area Head, Liver/Metabolism. "The early data we announced in January indicate that our proprietary AAV5 viral vector can achieve a clinically relevant level of expression of the FIX transgene. Our data thereby reproduced previously published long-term FIX expression levels seen with a dose comparable to that used in our low-dose cohort with the same gene cassette but using a different vector."
"The successful dosing of the first patient in the high-dose cohort marks another significant step towards bringing this gene therapy to patients," said Dan Soland, CEO of uniQure. "With the greenlight from the Data Monitoring Committee after the first cohort, we are excited to be able to move the program forward. We strongly believe that the product profile of AMT-060 has the potential to improve the quality of life of hemophilia B patients."
The AMT-060-01 Phase I/II study is a 5-year, open-label, uncontrolled, dose-ascending trial that includes two cohorts, with the low-dose cohort using a treatment of 5x1012 gc/kg and the high-dose cohort using 2x1013 gc/kg. AMT-060 consists of a codon-optimized wild type FIX gene and the LP1 liver promoter together with the AAV5 viral vector, manufactured using uniQure's proprietary insect cell based manufacturing technology. It is administered, without immunosuppressant therapy, through the peripheral vein in a single treatment session for approximately 30 minutes. All patients are screened for pre-existing AAV5 antibodies before treatment and Data Monitoring Committee (DMC) reviews are conducted after each of the first 2 patients in the second cohort.
About Hemophilia B
Hemophilia B is a serious and rare inherited disease in males characterized by insufficient blood clotting. The condition can lead to repeated and sometimes life-threatening episodes of external and internal bleeding following accidental trauma or medical interventions. The episodes can cause long-term damage, for example to the joints, and can be fatal if they occur in the brain. The deficient blood clotting results from the lack of functional human Factor IX, or hFIX. Treatment of hemophilia B today consists of prophylactic or on-demand protein replacement therapy, in which frequent intravenous administrations of plasma-derived or recombinant hFIX are required to stop or prevent bleeding. Hemophilia B occurs in approximately 1 out of 30,000 live births.
About uniQure
uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with CNS, liver/metabolic and cardiovascular diseases. www.uniQure.com
uniQure Forward-Looking Statement
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. 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 the course of development of our program hemophilia B. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with collaboration arrangements, our and our collaborators' clinical development activities, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's 2014 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 7, 2015. 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: uniQure:
Eva Mulder
Direct: +31 20 240 6103
Main: +31 20 240 6000
e.mulder@uniQure.com
Media inquiries:
Gretchen SchweitzerMacDougall Biomedical Communications
Direct: +49 172 861 8540
Main: +49 89 2424 3494 or +1 781 235 3060
gschweitzer@macbiocom.com
Source: uniQure
Read more: http://www.nasdaq.com/press-release/uniqure-announces-dosing-of-first-patient-in-highdose-cohort-in-phase-iii-hemophilia-b-clinical-20160314-00068#ixzz42t9f81yG
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uniQure to Present at the RBC Capital Markets 2016 Global Healthcare Conference
Join the webcast at February 24, at 2.05 PM ET.
Full details and direct link to webcast.
Was hoping to see a bit of a follow-through here today.
I guess that won't be happening.
Brutal market.
Nice move here today! Given market conditions this seems to be a pretty solid play going forward. I hope QURE keeps its gains!
FWIW, sold pre-market @ $17.30. Bullish on QURE, but was concerned about the overall market and also the big spread this morning between bid-ask. Sold on the uptick just before opening.
Looking to get back in ASAP.
QURE
me too...
uniQure BV (QURE) Data Shows Potential Hemophilia B Cure is Achievable - Janney
Janney Capital analyst Debjit Chattopadhyay weighed in on uniQure BV (NASDAQ: QURE) saying the preliminary read from Hemophilia B points to robust FIX-liver expression, showing a potential cure is achievable.
"The two patients who have completed 12 weeks of follow up have FIX levels between 5.5% and 4.5%, which we consider to be curative based on historic data from the St. Jude hospital," Chattopadhyay commented. "This is best highlighted by the fact that four patients, including the 1st two enrolled in the study, have met a the secondary objective in trial by fully discontinuing prophylactic rFIX. While long term follow up would be required to claim full cures, the early data points in the right direction and should alleviate management transition concerns and recatalyse the stock in our view."
The firm said uniQure is attractive at many levels. "uniQure is the only AAV-gene therapy company with both proprietary, comm.-scale manufacturing capabilities and thus far has compelling data across multiple indications and significant partnership with BristolMyer Squibb (cardiovascular centric), which alone should value the company more than its current depressed valuation."
The firm reiterated a Buy rating and $40 fair value target.
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http://www.streetinsider.com/Analyst+Comments/uniQure+BV+%28QURE%29+Data+Shows+Potential+Hemophilia+B+Cure+is+Achievable+-+Janney/11200895.html
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as i am waiting for cc here, anybody got any idea how high we will go by eod ?
Too bad the market is tanking this morning. Hope it doesn't put too much of a damper on the pps. QURE's results are better than $18.00/share especially when underpinned by their strong fiscal position and the new CEO's buyout record (all doubt about the reason for the former CEO's departure is now gone).
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I'm guessing about 200K - 400K shorts will start covering starting at $18 and up. Will be fun. Sit back and watch IMO. $QURE
Agreed. I'm holding and not selling at these levels. Strong PRs will follow on the heals of today's data IMO.
Looks like a few covered yesterday, but there are still a lot of them:
http://shortanalytics.com/getshortchart.php?tsymbol=qure
You know I thought they were at JPM next week and saw it posted and tweeted a lot, but I checked as of yesterday and they are not.
Last pps is $18.00 aand up ~18%. Glad we held!
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Agreed. Impatient sellers will get out of the way and then she will nicely run IMO
i am quite suprised that this company does not receive more press time for their work... this is the way of the future... not my specialty but damn... wallstreet must be stupid...
Love the name and pic. QURE $25+
nice... the presentation is very reassuring... skip the news blip and go straight to the presentation...
All the sellers ran away. Ask at 22.49. Hold your shares TIGHT! This one is going to explode IMO.
presentation looks solid... damn wish i had bought more yesterday...
factor ix levels more than double with lo dose regimens... wow... hi dose if safe looks like a sure cure... wow...
looks like lo dose is safe and more ingoung studies will validate that, while hi dose is next this quarter ...
I agree. The company would not put the extra effort into the results if they were negative.
If the data is good, shorts will get burned. Will need to cover like crazy. Company doing webcast as well as making slides available to download as PDF. Plus JPM coming up next week. Got to believe data has to be stellar IMO. We will see soon. $QURE
Was down to $14.52.
Nasdaq has it listed:
http://www.nasdaqtrader.com/trader.aspx?id=ShortSaleCircuitBreaker
QURE
at 7% ?
didn t notice any halt today
volume was only 450k
Yep, sure wasn't expecting to be in this position -- crazy day. Will be interesting especially since it tripped the circuit breaker today:
QURE uniQure NV Q 1/6/2016 3:01:58 PM
QURE
Hopefully we get some good news.!
QURE to present data @ 6:00 tomorrow and CC @ 8:30.
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UPDATE:
uniQure Announces Conference Call and Webcast to Discuss Topline Results From Low-Dose Cohort in Hemophilia B Phase I/II Gene Therapy Clinical Trial
By GlobeNewswire, January 06, 2016, 04:15:00 PM EDT
AMSTERDAM, the Netherlands, Jan. 6, 2016 (GLOBE NEWSWIRE) --
uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced that the Company will release topline results from its Hemophilia B Phase I/II gene therapy low-dose cohort clinical trial through a press release at 06:00 EST/12:00 CET on January 7, 2016. The Company's management team will hold a conference call and webcast at 08:30 EST/14:30 CET after the press release has been issued to discuss the update.
The conference call can be accessed by dialing one of the numbers listed below five minutes prior to the start of the call and providing the confirmation code: 1640435. The webcast, including accompanying slides also available as a PDF file, can be accessed through the link displayed in the "Events" section on the "Media" page of the uniQure website at http://www.uniqure.com/news/calendar-of-events/. The webcast replay will be available for at least 72 hours following the call.
Conference Call Numbers
London, United Kingdom: +44 (0) 20 3427 1906
New York, United States of America: +1 212 444 0412
Berlin, Germany: +49 (0) 30 3001 90534
Amsterdam, Netherlands: +31 (0) 20 716 8296
Paris, France: +33 (0) 1 76 77 22 31
Milan, Italy: +39 02 3859 1420
Brussels, Belgium: +32 (0) 2 402 3092
Montreal, Canada: +1 514 841 2154
About uniQure
uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with CNS, liver/metabolic and cardiovascular diseases. www.uniQure.com
CONTACT: uniQure:
Direct: +31 20 240 6110
Main: +31 20 240 6000
e.mulder@uniQure.com
Media inquiries:
Gretchen SchweitzerMacDougall Biomedical Communications
Direct: +49 172 861 8540
Main: +49 89 2424 3494 or
+1 781 235 3060
gschweitzer@macbiocom.com
Source: uniQure
This article appears in: News Headlines
Read more: http://www.nasdaq.com/press-release/uniqure-announces-conference-call-and-webcast-to-discuss-topline-results-from-lowdose-cohort-in-20160106-01031#ixzz3wV9keWJ3
Terrible chart and price action today.
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uniQure N.V. (uniQure) is a gene therapy company. The Company is engaged in developing single treatments with curative results for patients suffering from genetic and other severe diseases. The Company is advancing a focused pipeline of gene therapies that have been developed both internally and through partnerships. The Company’s product candidates include AMT-060 for Hemophilia B, AMT-130 for Huntington's disease, S100A1 for congestive heart failure and Glybera for lipoprotein lipase deficiency (LPLD). uniQure has established clinical proof-of-concept in its lead indication, hemophilia B and has achieved pre-clinical, proof-of-concept in Huntington's disease. The Company, through its collaboration with Bristol Myers-Squibb (BMS), is focused on building a portfolio of gene therapies led by the S100A1 program for congestive heart failure.
AMT-060 for Hemophilia B
AMT-060 is a gene therapy for the treatment of Hemophilia B. Hemophilia B is an inherited disease in males characterized by insufficient blood clotting. AMT-060 consists of the adeno-associated virus (AAV) vector carrying a human Factor IX (FIX) gene cassette that the Company has exclusively licensed from St. Jude Children’s Research Hospital in Memphis, Tennessee. The Company produces this vector with its insect cell-based manufacturing process. It has an ongoing Phase I/II clinical trials.
AMT-130 for Huntington's Disease
AMT-130 is developed for the treatment of Huntington's disease (HD). HD is caused by an inherited defect in a single gene that codes for protein called Huntingtin. The Company’s product candidate AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA, which silences the Huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein.
S100A1 for Congestive Heart Faliure
The Company has entered into an agreement with BMS that provides exclusive access to the Company’s gene therapy technology platform for multiple targets in cardiovascular (and other) diseases (Collaboration and License Agreement). The collaboration includes its proprietary gene therapy program for congestive heart failure, which aims to restore the heart's ability to synthesize S100A1. . S100A1 is a calcium sensor and master regulator of heart function, and thereby improve clinical outcomes for patients with reduced ejection fraction. S100A1 deficient hearts show accelerated progression to severe heart failure and increase mortality after cardiac damage. The Company is leading the discovery, non-clinical, analytical and process development effort and is responsible for manufacturing of clinical and commercial supplies using our vector technologies and industrial, proprietary insect-cell based manufacturing platform, while BMS leads development and regulatory activities across all programs and is responsible for all research and development costs. BMS will be solely responsible for commercialization of all products from the collaboration.
Glybera for Lipoprotein Lipase Deficiency
Glybera is a gene therapy that is designed to restore the LPL enzyme activity required to enable the processing, or clearance, of fat-carrying chylomicron particles formed in the intestine after a fat-containing meal. The product consists of an engineered copy of the human LPL gene packaged with a tissue-specific promoter in a non-replicating AAV1 vector, which has a particular affinity for muscle cells. In order to improve activity, the Company uses a naturally occurring variant of the LPL gene that has higher enzyme activity than the normal version of the gene that encodes the protein. It produces Glybera using its insect cell-based manufacturing process. In the European Union, the Company has been granted orphan drug exclusivity for Glybera for treatment of LPLD until October 2022. The first commercial patient in Europe was treated with Glybera in September 2015.
Other Early-Stage Research
The Company is also engaged in the research of several other gene therapy candidates targeting rare and orphan diseases. It focuses on genetic diseases affecting the liver, including hemophilia A, as well as various central nervous system (CNS) disorders.
Vector Development
uniQure seeks to develop next-generation vectors with increased potency to target liver indications in which high relative percentage increases in the secretion of a protein above the disease state would be required for therapeutic benefit. These next-generation vectors may be used in the development of a gene therapy for hemophilia A as well as other therapeutic indications. In January 2014, the Company has entered into a collaboration and license agreement with 4D Molecular Therapeutics, LLC for the discovery and optimization of next-generation AAV vectors targeting the liver and the brain.
The Company competes with AGTC, Abeona Therapeutics, Adverum Biotechnologies, Asklepios BioPharmaceutical, Audentes Therapeutics, Avalanche Biotech, AveXis, Bayer, BioMarin, Bioveratiy, bluebird bio, Dimension Therapeutics, Errant Gene Therapeutics, Expression Therapeutics, Freeline Therapeutics, Genethon, Genzyme, GlaxoSmithKline, Homology Medicines, Lysogene, Megenics, Milo Therapeutics, Nightstarx, Pfizer, REGENXBIO, Renova Therapeutics, Retrosense Therapeutics, Sangamo BioSciences, Shire, Solid Biosciences, Spark Therapeutics, Takara, Voyager, Amgen, Bayer, Biogen, BioMarin, Genzyme, Novartis, Novo Nordisk, Pfizer, and Shire.
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