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unicure NV (QURE)

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Last Post: 10/20/2017 4:56:39 AM - Followers: 17 - Board type: Free - Posts Today: 0

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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QURE News: Current Report Filing (8-k) 12/10/2018 06:08:58 AM
QURE News: Statement of Changes in Beneficial Ownership (4) 12/06/2018 09:39:53 AM
QURE News: Current Report Filing (8-k) 12/03/2018 09:07:26 AM
QURE News: uniQure Announces Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 and Confirms Dose for AMT-061 Pivotal Stud... 12/03/2018 07:00:00 AM
QURE News: uniQure Announces Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 and Confirms Dose for AMT-061 Pivotal Stud... 12/03/2018 07:00:00 AM
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#219   Profit taking time .. Good time to short Sunnycupid 10/20/17 04:56:39 AM
#218   QURE $9.15 high today, nice. Thoro 08/14/17 05:39:17 PM
#217   This thing is pretty volatile lately. Thoro 08/03/17 04:45:50 PM
#216   QURE Looked great hitting $9.00 today Thoro 07/25/17 05:05:15 PM
#215   QURE General Information Thoro 07/24/17 07:16:45 PM
#214   Uniqure NV (QURE) Money Flow Index Nearing Key Level Thoro 07/24/17 07:14:19 PM
#213   Erratic Trading Showing Unusual Volume in Shares of Thoro 07/24/17 07:13:09 PM
#212   uniQure N.V. NASDAQ:QURE Sees High Volatility in Session Thoro 07/24/17 07:12:18 PM
#211   uniQure N.V. (QURE) PT Set at $13.00 by Thoro 07/24/17 07:10:51 PM
#210   This dog has always been a turd. Why Rogerthat1 07/19/17 06:51:24 PM
#209   buying here Dutchdaan 05/26/17 03:08:30 PM
#208   Lost all patience in this pos Panzer 01/14/17 05:55:02 PM
#207   Patient is a virtue. redsox17 12/05/16 12:37:43 PM
#206   That's why it's down another 3% jaytea 12/05/16 10:42:46 AM
#205   Trust me they are. They just don't redsox17 12/05/16 10:24:34 AM
#204   The market does not seem to be overly impressed. jaytea 12/05/16 10:17:58 AM
#203   Great news out redsox17 12/03/16 05:31:33 PM
#202   Great newsGo QURE redsox17 11/17/16 09:56:19 AM
#201   I see over 100 % upside from here. redsox17 11/16/16 06:49:00 PM
#200   Not sure but panzers got blasted. Data in Panzer 10/15/16 11:44:13 AM
#199   WTF happened here today? jaytea 09/29/16 03:57:32 PM
#198   Adding all under 9s Panzer 09/13/16 09:50:28 PM
#197   QURE data will show in PPS hang on tight Panzer 09/02/16 09:05:24 PM
#196   QURE will double here into December Panzer 09/02/16 09:04:36 PM
#195   Laaaaaid back QURE love it Panzer 08/26/16 09:43:34 PM
#194   End of summer QURE prediction will happen Panzer 08/25/16 11:33:47 PM
#193   QURE run to 12+ on it watch Panzer 08/25/16 11:32:44 PM
#192   Panzer nailing biotechs sniper Panzer 08/25/16 11:26:32 PM
#191   Looking fantastic QURE Panzer 08/25/16 11:26:05 PM
#190   This trade turning into pure genius muhahahhaha Panzer 08/07/16 08:01:06 PM
#189   Looking fantastic, nice runner QURE Panzer 08/02/16 10:58:38 PM
#188   Just blasted some QURE ask Panzer 07/27/16 03:53:56 PM
#187   End of summer it hits $12 Ch@rter 07/06/16 10:45:41 AM
#186   So much for that -- now see chmcnfunds 06/28/16 03:01:38 PM
#185   Ended up selling @ $7.37 this afternoon. Still chmcnfunds 06/28/16 02:49:24 PM
#184   no kiddin man... ez $$$ here... oldsport 06/28/16 09:00:39 AM
#183   Back in QURE @ $7.01 after a period chmcnfunds 06/28/16 08:27:26 AM
#182   Joehoe it is time to get inn folks Ch@rter 06/16/16 04:56:46 PM
#181   Making a move today - start of a jaytea 05/31/16 02:03:21 PM
#180   Free Fallin jaytea 04/11/16 03:38:31 PM
#179   This biyitch has been all over the map jaytea 04/07/16 02:34:43 PM
#178   Posted message 177 to myself. Was meant for chmcnfunds 04/07/16 10:10:41 AM
#177   Yep. You never know do you. Yesterday it chmcnfunds 04/07/16 10:08:31 AM
#176   too bad... $qure is at bottom... oldsport 04/07/16 08:50:13 AM
#175   FWIW, sold today at $12.60. Being cautious. Supposedly chmcnfunds 04/01/16 03:51:43 PM
#174   Uniqure NV (QURE) Earns “Buy” Rating from Chardan Capital chmcnfunds 03/29/16 11:45:38 AM
#173   New rating: chmcnfunds 03/24/16 12:01:35 PM
#172   Uniqure NV’s (QURE) “Buy” Rating Reaffirmed at Chardan Capital chmcnfunds 03/24/16 11:24:58 AM
#171   Nice move today on news. I think we jaytea 03/22/16 04:29:29 PM
#170   uniQure Announces Preclinical Proof of Concept for Gene chmcnfunds 03/22/16 11:30:04 AM