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UniQure Huntington

Our mission at uniQure is to deliver curative gene therapies that transform the lives of patients. We have an ongoing clinical program in hemophilia B and preclinical proof-of-concept for a gene therapy in Huntington's disease uniQure is developing AMT-130, a gene therapy for Huntington's disease (HD) that silences the huntingtin gene, with the goal of inhibiting the production of the mutant protein. We are very encouraged by the significant reductions in mutant huntingtin protein that we have seen in our preclinical studies uniQure has initiated the first gene therapy clinical trial in Huntington's disease. uniQure has begun dosing patients in a dose-escalating, randomized and controlled Phase I/II clinical study to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington's disease uniQure Announces Latest Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease 02.11.2021 12:0

uniQure's open-label Phase 1b/2 European study will enroll 15 early Huntington's patients, assigned to a low- or high-dose treatment group. Depending on results of these two studies, which will help in determining the therapy's optimal dose, uniQure plans to move forward with either a Phase 3 trial, or a confirmatory study should an accelerated path to approval be possible Dive Brief: UniQure can begin treating patients with the highest dose of its experimental Huntington's disease gene therapy, announcing Thursday that clinical trial monitors found no safety concerns after reviewing data from the first 10 participants who received lower doses. The safety panel's go-ahead clears the way for the Massachusetts-based company to enroll another 16 patients

uniQur

  1. UniQure hat seinen Hut in den Ring für Gentherapien der Huntington-Krankheit geworfen, indem sie ein AAV-Virus entwickelt haben, dass die Anleitung für ein Mikro-RNA-Werkzeug enthält, welches sich an die Huntingtin-Boten-RNA anheftet. Das Medikament besteht also aus dem Gesamtpaket Virus plus Anleitung und nennt sich AMT-130
  2. Before I do that, I'd like to say thank you to the team at the Huntington's Disease Society of America for helping us get this message out to the community. uniQure was founded as Amsterdam Molecular Therapeutics(AMT)in the Netherlands in 1998. In 2012, the innovative work of our research team there resulted in the first gene therapy ever approved in Europe. This experience laid the foundation for our leadership today in the discovery, clinical development, and manufacturing of gene.
  3. Uniqure haben ihr Konzept mit vielen Experimenten an unterschiedlichen Huntington-Modellen belegt: sie konnten die Wirksamkeit in Zellkulturen, bei Huntington-Mäusen, -Ratten und -Affen zeigen. An Schweinen untersuchten sie die Langzeitwirkungen der Behandlung - diese ist hier besonders wichtig, denn es handelt sich um einen unumkehrbaren Eingriff. Bis jetzt hat sich gezeigt, dass das.
  4. uniQure has built a potential first- and best-in-class hemophilia B gene therapy program. uniQure is advancing a promising clinical program focused on hemophilia B, a severe orphan blood clotting disorder. Our gene therapy product candidate etranacogene dezaparvovec (AMT-061) consists of an AAV5 viral vector carrying a gene cassette with the Padua.
  5. Biotech company UniQure said Friday it has dosed the first patient with a gene therapy for Huntington's disease, a degenerative condition marked by destruction of nerve cells in the brain. Two patients have entered the trial, one who has received an injection of the therapy into the brain and one who has undergone an imitation procedure
  6. uniQure Inc.: uniQure Announces Latest Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Diseas

Gentherapie gegen die Huntington-Krankheit UniQure gibt Eckdaten zur geplanten Studie der Sicherheit und Verträglichkeit einer Gentherapie mit AMT-130 bekannt. Die Menge an Huntingtin-Eiweiß soll mit einer einzigen Behandlung mit einem Virussystem reduziert werden. Von Dr Anna Pfalzer 21. Juli 2019 Bearbeitet von Professor Ed Wild Übersetzt von Rebecca Ursprünglich veröffentlicht am 17. uniQure Inc.: uniQure Announces Enrollment of First Two Patients in Second Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease LEXINGTON, Mass. and AMSTERDAM. This week uniQure shared a press release stating that another two patients have entered their HD clinical trial. The drug, AMT-130, is an experimental gene therapy that aims to lower harmful huntingtin protein in the brains of people with HD. It is delivered via a one-time brain surgery procedure, and because this type of treatment is so new, uniQure is proceeding carefully and slowly to.

The FDA Green-lights Clinical Trials for Huntington's

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 hemophilia, Huntington's disease and cardiovascular diseases. www.uniQure.co

Huntington's Disease - Patients - uniQur

Phase I/II Clinical Trial of AMT-130 - Patients - uniQur

  1. istration in NHPs and Widespread Long-Term HTT-Lowering in the Brai
  2. Looking at the market potential ($19.6 B = $16.5B from Hemophilia and $3.1 B from Huntington's disease), if QURE can capture 5% of the total market, it would bring annual sales to $980 M.
  3. uniQure Announces First Two Patients Treated in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease ~ Milestone Marks the First-in-Human AAV Gene Therapy Trial for Huntington's Disease ~ LEXINGTON, Mass. and AMSTERDAM, June 19, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with.
  4. 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 gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases

uniQure Announces Latest Positive Recommendation from Data

Trial of AMT-130, Huntington Gene Therapy, Enrolling High

  1. uniQure remains a very high-risk, very high-reward investment due to its Huntington's program. It is only appropriate for investors looking for this profile. Despite the well-reasoned approach.
  2. UniQure has started screening patients for its phase 1/2 trial of gene therapy AMT-130 for Huntington's disease, and says it hopes to start treating the first subject in late 2019 or early 2020
  3. On behalf of the uniQure Huntington's disease (HD)team, I'm honored to introduce our company and our gene therapy clinical development program to all of you. uniQure has a long history in developing gene therapies, but is a relative newcomer to the HD community, so I'd like to provide a bit of background to begin. Before I do that, I'd like to say thank you to the team at the.
  4. Huntington's disease goes viral as UniQure inches ahead in gene therapy race FDA grants Investigational New Drug status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington's Disease patients By Dr Jeff Carroll January 30, 2019 Edited by Professor Ed Wild Originally published on January 29, 2019 utch-American company uniQure has received.
  5. uniQure Company Profile. uniQure NV engages in the research, development, and commercialization of gene therapies. Its discoveries intend to treat hemophilia, Huntington's disease, glybera, and cardiovascular problems. The company was founded by Sander J. van Deventer in 1998 and is headquartered in Amsterdam, the Netherlands
  6. Gene therapy pioneer uniQure launched the first-in-human adeno-associated virus-based gene therapy clinical trial for Huntington's disease. In June, it dosed its first two patients using a novel therapeutic, AMT-130, delivered directly to the brain

UniQure moves Huntington's gene therapy to next phase of

  1. uniQure Announces FDA Orphan Drug Designation for AMT-130 in Huntington's disease ~ First Investigational Gene Therapy in Huntington's Disease to Receive Designation ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 06, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today.
  2. uniQure is an excellent acquisition target for Sanofi with potential to be first to market in gene therapy treatments for hemophilia B and Huntington disease. Introduction . Last month.
  3. ary data on efficacy
  4. uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington's Disease ~ On Track to Treat First Patient in Phase I/II Study of AMT-130 in 2H19 ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 08, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe.
  5. uniQure Announces Completion of Enrollment in First Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease globenewswire | Weitere Nachrichten 01.04.2
  6. stocks on our watch list. Register / Login. Top Gapper

uniQure Announces Latest Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease - read this article along with other careers information, tips and advice on BioSpac uniQure NV engages in the research, development, and commercialization of gene therapies. Its discoveries intend to treat hemophilia, Huntington's disease, glybera, and cardiovascular problems. The company was founded by Sander J. van Deventer in 1998 and is headquartered in Amsterdam, the Netherlands. Featured Story: Stock Symbols, CUSIP and Other Stock Identifiers. This instant news alert. uniQure Announces Completion of Enrollment in First Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Diseas uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. UniQure are leveraging modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases uniQure Announces Latest Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Diseas

About uniQure. uniQure NV engages in the research, development, and commercialization of gene therapies. Its discoveries intend to treat hemophilia, Huntington's disease, glybera, and cardiovascular problems. The company was founded by Sander J. van Deventer in 1998 and is headquartered in Amsterdam, the Netherlands. Featured Story: Trade Defici Uniqure Aktie (A1XDTV, NL0010696654) Nasdaq 120.523 Stk. Quotrix Düsseldorf 640 Stk. Xetra 29 Stk. L&S Exchange 10 Stk. Tradegate 10 Stk. Berlin. BX Swiss. Frankfurt Die Chorea Huntington, auch Huntingtonsche Chorea oder Huntington-Krankheit (englisch Huntington's disease, HD; ältere Namen: Veitstanz, großer Veitstanz, Chorea major) genannt, ist eine unheilbare erbliche Erkrankung des Gehirns, die durch unwillkürliche, unkoordinierte Bewegungen bei gleichzeitig schlaffem Muskeltonus gekennzeichnet ist, in Demenz mündet und zum Tod führt No significant safety concerns were identified in the first eight Huntington's disease patients treated with uniQure's potential gene therapy AMT-130 in a U.S. Phase 1/2 clinical trial.. Six of these patients received the low dose of the therapy — which is delivered directly into the brain — and two received the high dose

The stock price of uniQure, a gene therapy company, has seen a rise of 20% in a week, while it is up 10% over the last month. The recent rise can be attributed to its Q3 loss of $0.79 per share. I am pleased with this opportunity to join the uniQure Board of Directors at such an exciting time for the Company, stated Ms. Jacques. I look forward to partnering with Board members and providing consult to management as it prepares to submit a BLA in hemophilia B, advances its Huntington's disease clinical program, and develops its pipeline of promising gene therapy candidates Experienced Huntington's disease clinician, consultant and trialist with several assignments as Global Coordinating Principle Investigator of clinical trials in HD (e.g. UniQure, PROOF-HD, PRIDE-HD, LEGATO-HD, Selisistat, Ipsen, AFQ-056). Active involvement in several global networks in HD and intense experience in interactions with research consortia & biotechnology industry from small. * uniqure announces first two patients treated in phase i/ii clinical trial of amt-130 for the treatment of huntington's disease BRIEF-Uniqure Announces The Appointment Of Leonard E. Post, Ph.D. LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 25, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with.

Community Statement from uniQure Huntington's Disease

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 25, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the third quarter of 2021 and highlighted. uniQure Announces Third Quarter 2021 Financial Results and Highlights Recent Company Progress Provided by GlobeNewswire. Oct 25, 2021 11:05 AM UTC ~ Completed 78-week follow-up for hemophilia B. uniQure Announces Enrollment of First Two Patients in Second Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease Benzinga - Jun 16, 2021, 7:07A Gene therapy pioneer uniQure launched the first-in-human adeno-associated virus-based gene therapy clinical trial for Huntington's disease. In June, it dosed its first two patients using a novel therapeutic, AMT-130, delivered directly to the brain. Initial readouts are expected in a year's time. Huntington's disease is a hereditary disease that affects approximately 70,000 people in the.

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 gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases uniQure Announces Latest Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease. Stockhouse.com use cookies on this site. By continuing to use our service, you agree to our use of cookies. Cookies are used to offer you a better browsing experience and to analyze our traffic. We also use them to share usage. uniQure Contacts: FOR INVESTORS: FOR MEDIA: Maria E. Cantor: Chiara E. Russo: Tom Malone: Direct: 339-970-7536: Direct: 617-306-9137: Direct: 339-970-755 uniQure Announces Completion of Additional Patient Procedures Following Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of.

HDBuzz Deutsche Huntington-Hilfe e

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 gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.co Astrid Valles-Sanches ricercatrice UniQure malattia di huntington - huntington's disease 1. D E L I V E R I N G G E N E T H E R A P Y T O P A T I E N T S A I C H R O M A O N L U S A n n u a l M e e t i n g - M a y 1 8 , 2 0 1 9 A pioneer in gene therapy +20 years of experience in AAV gene therapy Research & Development Amsterdam, The Netherlands Development and GMP Manufacturing Lexington.

22.01.2018 - uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-130, its proprietary. 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 gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com. UniQure has claimed a fast-track review from the FDA for a gene therapy for Huntington's disease due to start a phase 1/2 trial later this year. The Netherlands-headquartered biotech says the first patient should be treated before the end of 2019 with the therapy - called AMT-130 - which is based on an adeno-associated virus (AAV) vector.

uniQure to Participate in Upcoming Industry Conferences in November. LEXINGTON, Mass. and AMSTERDAM, Nov. 01, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company. Oral Presentation to Highlight AMT-191 for Fabry DiseaseLEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 19, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that four data presentations, including one oral presentation, will be delivered at the European Society.

Hemophilia - Gene Therapy - uniQur

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 hemophilia, Huntington's disease, spinocerebellar ataxia, and other severe genetic diseases 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 gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com uniQure. UniQure has strengthened patent protections around its experimental gene therapy for Huntington's disease, as it prepares to start human testing later this year in a Phase 1/2 study. The Dutch biotech said Wednesday it secured two patents, one in the U.S. and one in Europe, that cover its gene therapy AMT-130. The therapy aims to silence the.

Pipeline - Gene Therapy - uniQure

UniQure gets out the gate first in race for Huntington's

uniQure's neuroimaging translational studies in non-human primates and Huntington's disease patients shows the importance of careful, individualized neurosurgical planning and meticulous dosing parameters in delivering gene therapy products to the brain, added Joseph J. Higgins, M.D., F.A.A.N., vice president, clinical development. We are excited about the AMT-130 clinical. He noted Uniqure is soon to present Phase 3 results for its gene therapy and will have data for its Huntington's disease treatment before year's end. IBD Newsletters Get exclusive IBD analysis and.

Eine kurze Zusammenstellung weiterer erfolgversprechender Ansätze für Gentherapien der Huntington-Krankheit: UniQure: In einer laufenden klinischen Studie wurden bis jetzt 12 von geplanten 26 Patienten einer Schädeloperation unterzogen. UniQure untersucht (verblindet und randomisiert) die Wirksamkeit des Medikaments AMT-130, dass den Gehirnzellen einen Bauplan für ein Mittel gegen. First HD Patients Dosed in uniQure Gene Therapy Study. This week marks a major milestone in HD research: the first two patients were dosed in the world's first gene therapy study for HD. On June 19th, uniQure announced in a press release that two brave participants had undergone the brain surgery required to deliver the experimental. uniQure licenses RNA interference technology to advance Huntington's disease program Amsterdam, The Netherlands - December 5, 2012 - uniQure B.V., a leader in the field of human gene therapy, today announced a non-exclusive cross-licensing agreement with Benitec Biopharma Ltd. (ASX: BLT) giving uniQure access to Benitec's proprietary DNA-directed RNA interference (ddRNAi) technology in.

UniQure will sich verstärkt auf andere Projekte konzentrieren. Entsprechend unserer angekündigten Strategie werden wir unsere Ressourcen darauf ausrichten, unser Hämophilie-B-Programm in erste klinische Studienphasen zu bringen, das Programm zur Huntington-Krankheit zu einer Proof of Concept-Studie zu führen und unsere F&E-Zusammenarbeit mit Bristol-Myers Squibb voranzutreiben. FOR INVESTORS: FOR MEDIA: Maria E. Cantor Direct: 339-970-7536 Mobile: 617-680-9452 m.cantor@uniQure.com: Chiara Russo Direct: 617-306-9137 Mobile: 617-306-913 DSMB Recommends UniQure To Continue Huntington's Gene Therapy Trial Unchanged . Vandana Singh, Benzinga Staff Writer {{following ? Following : Follow}} May 27, 2021 11:48am 126 Comments. Share.

uniQure Inc.: uniQure Announces Latest Positive ..

~ AMT-130 Poised to Become First AAV Gene Therapy to Enter the Clinic for Huntington's Disease uniQure's AMT-130 for Treatment of Huntington's Disease Advances in Phase I/II Clinical Trial uniQure announced today that they will move forward with the Phase I/II Clinical Trial of AMT-130, a gene therapy candidate for treatment of HD administered in a single dose to deep brain tissues with MRI-guided neurosurgical delivery A Phase 1/2 clinical trial evaluating AMT-130 as a potential gene therapy for Huntington's disease has treated its first two patients, uniQure announced. One person was given AMT-130 and the other a sham surgery as part of a control group. Both will now be followed for three months, after which a Data Safety Monitoring Board will determine the best steps for a next patient group

ASO gene silencing reaches further, lasts longer

uniQure Announces Publications of Preclinical Data for AMT-130 in Huntington's Disease Showing Safety of Administration in NHPs and Widespread Long-Term HTT-Lowering in the Brai LEXINGTON, Mass. and AMSTERDAM, Nov. 01, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced its participation in the following upcoming investor and scientific conferences: The 28 th Annual Meeting of the Huntington Study Group (Virtual), November 4 - 6, 202

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. The stock price of uniQure (NASDAQ:QURE), a gene therapy company, has seen a rise of 20% in a week, while it is up 10% over the last month. The recent rise can be attributed to its Q3 loss of $0. uniQure N.V. (NASDAQ:QURE) said no significant safety concerns were seen in the first four patients enrolled in higher-dose group of its phase 1/2 trial of AMT-130 to treat Huntington's disease. DSMB Recommends UniQure To Continue Huntington's Gene Therapy Trial Unchanged. Benzinga. 07:05AM : uniQure Announces Positive Recommendation to Advance Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntingtons Disease. GlobeNewswire. May-24-21 07:14AM : uniQure N.V. (NASDAQ:QURE) Shares Could Be 46% Below Their Intrinsic Value Estimate. Simply Wall St. May-13-21 07:00AM : uniQure. FDA clearance of the IND enables uniQure to initiate its planned dose-escalating, randomized and controlled Phase I/II clinical trial to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington's disease. uniQure expects to open several clinical sites in the United States and begin dosing patients in the second half of this year

FDA says Voyager can trek on as it removes trial hold forPotential Therapeutic Target for Huntington’s DiseaseNieuws - Vereniging van Huntington - Page #2

According to UniQure, AMT-130 does exactly that as the therapy consists of an AAV5 vector that carries an artificial micro-RNA, which 'silences' the Huntington gene when it reaches the brain. The designation supports UniQure's ongoing development in Huntington's, which is a therapy area the group has long been interested in, alongside haemophilia and cardiovascular diseases Beurscodes, betekenis en hulp bij zoeken Europa AEX Euronext Amsterdam BRU Euronext Brussels PSE Euronext Paris LIS Euronext Lissabon CHX CBOE Europe, grote(re) EU aandele Huntington's disease (HD) is a fatal progressive neurodegenerative disorder caused by a mutation in the huntingtin (HTT) gene. To date, there is no treatment to halt or reverse the course of HD

Dysregulation of dopamine receptor D2 as a sensitiveManagement Team - About - uniQure

uniQure Inc.: uniQure Announces Enrollment of First Two ..

LEXINGTON, Mass. and AMSTERDAM, Sept. 25, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the independent Data Safety Monitoring Board (DSMB) overseeing the Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease has met and reviewed. QURE 30.53$ (-7%) The program, known as AMT-260, is a collaboration with Regenxbio, which has equity in Corlieve and is eligible to receive milestone payments and royalties. As part of the uniQure deal, Corlieve will also receive up to 43.7 million euros ($52.1 million) for development milestones through phase 1/2 and another 160 million. A uniQure gene therapy for hemophilia B in on track for an FDA submission in the first quarter of 2022. Meanwhile, uniQure added to its pipeline with the acquisition of preclinical epilepsy gene. Publication in Science Translational Medicine shows local striatal delivery of microRNA-gene therapy results in widespread brain huntingtin protei.. uniQure NV engages in the research, development, and commercialization of gene therapies. Its discoveries intend to treat hemophilia, Huntington's disease, glybera, and cardiovascular problems. The company was founded by Sander J. van Deventer in 1998 and is headquartered in Amsterdam, the Netherlands. Recommended Story: Technical Analysis of Stocks and What It Means . This instant news alert.

uniQure Trial Proceeds; Voyager Receives Regulatory

uniQure announced that the independent Data Safety Monitoring Board (DSMB) overseeing its phase 1/2 clinical trial (NCT04120493) of AMT-130 for the treatment of Huntington disease (HD) found no significant safety concerns, clearing the final 6 patients in the first cohort for enrollment. 1 The board reviewed the 6-month safety data from the first 2 enrolled patients and the 90-day safety data. uniQure NV engages in the research, development, and commercialization of gene therapies. Its discoveries intend to treat hemophilia, Huntington's disease, glybera, and cardiovascular problems.

Among such companies, uniQure N.V. (NASDAQ: QURE), a gene therapy company, is using its modular technology platform to advance a pipeline of adeno-associated virus (AAV)-based gene therapies for the treatment of patients with severe genetic diseases of the central nervous system (CNS) and liver, including clinical programs in hemophilia B and Huntington's disease, as well as preclinical. No Safety Concerns Observed In Uniqure's Early-Stage Huntington's Gene Therapy Study, DSMB Says. Vandana Singh, Benzinga Staff Writer {{following ? Following : Follow}} February 08, 2021 9.

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 gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. For more information, visi Publication in Science Translational Medicine shows local striatal delivery of microRNA-gene therapy results in widespread brain huntingtin protein lowering in Huntington's disease minipigsLEXINGTON, Mass. and AMSTERDAM, April 08, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs. Bloomberg the Company & Its Products The Company & its Products Bloomberg Terminal Demo Request Bloomberg Anywhere Remote Login Bloomberg Anywhere Login Bloomberg Customer Support Customer Suppor AMT-130 is an experimental gene therapy being developed by uniQure to treat patients with Huntington's disease.The therapy has shown promising results in preclinical studies and has been granted orphan drug status and fast track designation by the U.S. Food and Drug Administration (FDA). Clinical trials began in 2019. How AMT-130 works. Huntington's disease is caused by a mutation in the. AMT-130 is uniQure's first clinical program incorporating its proprietary miQURE™ platform. miQURE is designed to degrade disease-causing genes without off-target toxicity and induce silencing of the entire target organ through secondary exosome-mediated delivery. About Huntington's Disease Huntington's disease is a rare, inherited neurodegenerative disorder that leads to motor. Feb 27, 2020 Hawellek D et al.: oral presentation, CHDI Foundation's 15th Annual Huntington's Disease Therapeutics Conference. Feb 27, 2020 Feb 27, 2020 Über diesen Artike