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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 Huntington’s disease. In return, uniQure granted Benitec non-exclusive access to the Company’s AAV5 delivery technology for the development of a ddRNAi therapy for Hepatitis B. “The cross-licensing agreement with Benitec fully capitalizes on the strength of our advanced AAV platform and our proven ability to deliver therapeutic genes to target cells with high accuracy and efficacy,” says Jörn Aldag, CEO of uniQure. “The agreement with Benitec opens up promising new avenues to develop therapies for high unmet medical needs such as Huntington’s disease. While our current programs focus on delivering fully functioning therapeutic genes to remedy faulty or malfunctioning genes, Benitec’s ddRNAi technology will allow us to do the opposite – to ‘silence’ the gene responsible for producing the mutant protein that lies at the basis of Huntington’s disease, and to develop a therapy for this devastating disease.” Dr Peter French, CEO of Benitec Biopharma, commented, “Benitec Biopharma is very pleased to have executed this licensing agreement with uniQure, the first company to achieve market approval for a gene therapy product, Glybera, in the West. uniQure have demonstrated their unique ability to take gene therapy-based programs from pre-clinical stages to commercialization, and we are confident that they will be able to achieve a similar outcome in this program. Importantly this agreement also provides Benitec access to uniQure’s AAV delivery technology enabling further development of our ddRNAi treatment for Hepatitis B.” About Huntington’s disease Huntington's disease (HD) is a rare, chronic, incurable, progressive and disabling neurological condition, which continues to challenge the medical community. About 30,000 Americans (or 1 in 10,000 people) have Huntington's disease and at least 150,000 individuals have a 50% chance of inheriting the disease. The symptoms of HD become most evident in adulthood, typically 30 to 55 years, characterized by sudden, abnormal, and uncontrolled jerky movements called chorea. Currently, therapeutics for HD are limited only to symptomatic treatments and there are no treatment options with proven safety and efficacy to slow down disease progression or enhance survival rate. ------------ uniQure and consortium to receive EUR 2.5 million Eurostars grant to develop RNAi gene therapy for Huntington’s disease Amsterdam, The Netherlands – May 21, 2013 – uniQure B.V., a leader in the field of human gene therapy, today announced that with its consortium partners it is to receive a EUR 2.5 million Eurostars grant to develop an RNA interference (RNAi) gene therapy for Huntington’s disease. The consortium is a pan-European collaboration consisting of uniQure as the coordinator, Lausanne University Hospital, Switzerland, University Medical Center Göttingen, Germany, and Maria Curie-Sklodowska University, Poland. The program’s aim is to develop a gene therapy for the treatment of Huntington’s disease (HD), a rare and devastating neurodegenerative disease caused by mutations in the Huntingtin (Htt) gene. As a result of the defective gene, mutated proteins accumulate in the brain and destroy neurons, leading at first to involuntary, random body movements, but eventually to progressive cognitive decline and finally dementia. Onset of the disease usually manifests itself around 35 to 44 years of age, while life expectancy after diagnosis is on average 20 years. The program will start on June 1, 2013 and run for three years. “Our Huntington’s disease program is part of our strategic effort to demonstrate the potential of our AAV-delivery platform in RNAi,” says Jörn Aldag, CEO of uniQure. “The RNAi field has great promise to become a new and important treatment modality. However, the field has been held back by the lack of effective delivery mechanisms. We believe that our AAV technology is ideally suited to deliver RNAi compounds with high accuracy and efficacy. In addition, the development with the consortium of a gene expression system for GDNF will not only benefit the HD program, but also holds great promise for our Parkinson’s disease program, and potentially other CNS disorders.” About the program The consortium’s goals are to develop a regulated gene expression system for glial cell derived neurotrophic factor (GDNF) to improve the maintenance and survival of neurons as a HD gene therapy, and to develop regulated expression of artificial miRNA to conditionally silence the Htt gene. The main outcome of the program is a robust pre-clinical assessment of the first regulated gene therapy vector suitable for optimized treatment of HD patients. The ability to regulate gene expression would additionally represent an exciting innovation in the field of gene therapy, creating new opportunities to tackle challenging diseases where gene expression is only required at certain times. The consortium expects that the program’s results should lead to clinical safety trials within two years after the conclusion of the project. HD-gene therapy www.eurostars-eureka.eu/project/id/7900
M01 Rnai-based Gene Therapy Of Huntington’s Disease Abstract Gene therapy is currently one of the most advanced approaches investigated for the treatment of Huntington’s disease (HD). There is a high unmet need for disease-modifying HD therapy since to date, no clinical trials have demonstrated treatment efficacy. The therapeutic target in HD is the mutated huntingtin (Htt) and the goal of the gene therapy is to silence the gene with miRNAs expressed from an AAV5 vector. Adeno-associated viral vector (AAV) expressing artificial miRNAs targeting the Htt gene is being developed by uniQure as an RNA interference (RNAi)-based gene therapy of HD. Three major approaches have been undertaken for the development of RNAi-based HD gene therapy: (1) total Htt knockdown by targeting exon 1, (2) targeting the CAG repeats in Htt exon 1 and (3) allele-specific inhibition of mutant Htt expression by targeting SNP rs362331 in exon 50 and SNP rs362307 in exon 67. Initially 100 artificial miRNAs targeting the wild-type and/or mutant Htt gene were designed and verified for their knock-down efficacy in vitro. In the first approach we identified the highly potent miHtt10 and miHtt12 targeting exon 1 therefore inducing strong total Htt knockdown. In the second approach, we tried to target the CAG repeats and discriminate between wt and mutant Htt based on differences in the secondary structure of the mRNA. In the third approach we identified miSNP50 and miSNP7 that specifically inhibited the mutant Htt allele while the wild-type Htt was mildly affected. Next, murine striata were transduced with AAV5-miHtt12, and AAV5-miSNP67 and a strong knockdown of Htt reporter gene was observed within 8 weeks post injection. Ongoing research aims to assess the neuroprotective effect of the non-selective and allele-specific miRNAs targeting Htt and expressed from AAV5 vectors in rodent HD models. Different viral delivery routes by direct intrastriatal injection or in the cerebrospinal fluid are being evaluated. Furthermore, the Htt RNAi silencing efficacy, safety and off-target potential are determined. AAV5 delivery of miRNAs targeting Htt provides a novel approach for HD therapy as it might allow specific inhibition of the mutant Htt gene expression and hence slow down disease progression. Support provided by uniQure and EU-FP7 E!7900.jnnp.bmj.com/content/85/Suppl_1/A94.2 M02 Therapeutic Benefit Of A Htt-lowering Antisense Oligonucleotide Targeting The Cag-repeat In The R6/2 Huntington’s Disease Mouse Model Abstract Background Different therapeutic approaches using antisense oligonucleotides (AONs) are currently under development for Huntington’s disease (HD). Ideally, an AON-based therapeutic approach for HD would selectively lower levels of the transcript carrying the disease-causing CAG-repeat expansion, but not affect the non-expanded transcript. We have previously demonstrated preferential knockdown of the mutant transcript in vitroin fibroblasts derived from HD patients using a CAG-repeat targeting AON consisting of a (CUG)7 sequence. Aims Aim of this study was to gain in vivoproof-of-concept with our (CUG)7 AON in a relevant HD animal model. Methods/techniques R6/2 mice transgenic for an N-terminal fragment of human HTT containing ˜150 CAG repeats (n = 30) received a total of 6 weekly ICV infusions with the (CUG)7 AON or vehicle. Bodyweight was monitored and a battery of motor tests (grip strength, rotarod, open field, 3D kinematic analysis) were performed. In addition, brain volume and striatal metabolites were quantified using MRI and MRS respectively. Two weeks after the last infusion at 12 weeks of age RNA was isolated from 7 different brain regions, followed by RT-qPCR analysis of HTT transcript levels. Results/outcome A highly signficant and strong (˜90%) reduction of mutant HTT mRNA levels was observed throughout the R6/2 brain. In addition, there was a clear positive treatment effect on motor performance, indicated by an increase in rotarod latency and vertical activity in the open field. 3D kinematic analysis of fine motor skills showed a significant improvement of several parameters impaired in R6/2 mice. A trend towards an increased whole brain and cortical volume was observed using MRI volumetry as well as a significant decrease in striatal levels of the astrocytic marker inositol, known to be elevated in the striatum of R6/2 mice and HD patients. Conclusions These data demonstrate our AON targeting the CAG-repeat has therapeutic potential for HD.jnnp.bmj.com/search?fulltext=uniQure&...
Harald Petry uniQure, Amsterdam, THE NETHERLANDS Establishing Gene Therapy in the clinic: Glybera and beyond” Abstract The presentation will provide a review of uniQure’s approach to develop AAV-vector based gene therapy medicinal products. A state-of-the art summary for Glybera will be provided. The clinical development program started 10 years ago with the dosing of the first patient in 2005 (Stroes et al., Arterioscler Thromb Vasc Biol. 2008). In total, 3 interventional open label, prospective clinical studies and 2 retrospective analyses on the incidence of disease-related clinical events in the same subjects have been completed in The Netherlands and Canada. EMA approval was achieved in 2012 (Glybera®). Based on this experience we have started clinical development for indications like Haemophila B and Sanfilippo B. A second focus will be on New Technologies including the AAV-delivery of miRNA and the therapeutic potential of this approach. The lead program in uniQure is for the neurodegenerative Huntington’s disease (HD) where the disease-causing huntingtin (HTT) gene is silenced with AAV-delivered miHTT. Proof of concept studies in HD rodent models have shown improvement of neuropathology associated with HTT protein decrease in the brain. The AAV-miHTT vector is currently being optimized for future clinical applications. A short summary of this progam will be shown as well as an outline on how we will get from where we are in the pre-clinical program to start a phase I clinical trial. twitter.com/floszcrxl/status/62055590... ESGCT Pre-clinical evaluation of AAV5-miHTT gene therapy of Huntington’s disease Jana Miniarikova, uniQure biopharma B.V.; Leiden University Medical Center tinyurl.com/ojx72tx twitter.com/floszcrxl/status/64250614... Also accepted at 3rd Conference on Large Animal Models for Neurodegenerative Disorders, Nov. 9-10. pigmod.avcr.cz/konference/program/ twitter.com/prof_dollar/status/642608...
Via "onze Prof": New Abstract: Pre-clinical evaluation of AAV5-miHTT gene therapy of Huntington’s disease. #AAV5 #RNAi #ESGCT15 twitter.com/prof_dollar/status/645161...
Vuilnismannen in verwarring. AMC Magazine, art. Ziekte van Huntington. P.10/11 issuu.com/amcamsterdam/docs/nr._9dec-jan
flosz schreef op 26 januari 2016 08:49 :
Vuilnismannen in verwarring.
AMC Magazine, art. Ziekte van Huntington.
P.10/11
issuu.com/amcamsterdam/docs/nr._9dec-jan Interessant verhaal!
CHDI HD Therapeutics Conf.2016 chdifoundation.org SESSION III - TARGETED THERAPIES FOR HUNTINGTIN LOWERING?Refining experimental gene therapies for Huntington’s and other diseases Pavlina Konstantinova, PhD uniQure Gene therapy is one of the most advanced approaches for the treatment of neurodegenerative diseases?like Huntington’s disease (HD) and amyloid lateral sclerosis (ALS). For HD therapy, the mutated huntingtin (HTT) is silenced with therapeutic miRNAs (miHTT) delivered with adeno-associated viral vector (AAV).?Two major approaches have been undertaken for the development of RNAi-based gene therapy of HD: total HTT silencing by targeting exon 1 and allele-speci c inhibition by targeting heterozygous SNPs linked to the mutant HTT. SNP rs362331 in exon 50 and SNP rs362307 in exon 67 were selected as they have the highest prevalence of heterozygosity in HD. The most e cient miHTT candidates were incorporated in AAV5 vectors and produced using the established uniQure baculovirus-based manufacturing platform. Proof of concept studies have shown e cacy of AAV5-miHTT in a HD rat model and the humanized Hu128/21 mouse model. In both models AAV5-miHTT delivery resulted in a lower concentration of the disease-inducing HTT protein associated with a delay of neurodegeneration and in reduction of mutant HTT aggregates. For ALS gene therapy arti cial miALS targeting c9orf72 were generated and evaluated for silencing e cacy. On-going work aims to evaluate the therapeutic e cacy of the miALS candidates in rodents. Direct intrastriatal delivery of AAV5-GFP by convection-enhanced di usion (CED) injection or cerebrospinal uid (CSF) delivery were evaluated in non-human primates (NHP) and minipigs to identify the best biodistribution pro le for HD and ALS therapy. CED injection resulted in almost complete transduction of the NHP striatum and di erent areas of the cortex. Similarly, intrastriatal transduction of neuronal and glial cells of AAV5-GFP was observed in minipig putamen and caudate nucleus. Intrathecal delivery of the vector in the minipig resulted in predominant transduction of frontal cortex, cerebellum and lumbal spine. Further studies in HD minipig model will aim to determine the level of HTT silencing in large-brained animals, the safety of the AAV5-miHTT approach and the long-term viral persistence. The miHTT processing and o -target potential will be determined to support the clinical development of the therapeutic candidate. AAV5-miHTT provides a huge therapeutic bene t for the HD patients as it will allow for life-long HTT suppression upon single vector administration. Wednesday February 24th, 2016 9:30am – 10:00am
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 — 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.”www.uniqure.com/news/295/182/uniQure-...
Artikel in Nature: Gene Therapy accepted article preview 3 August 2017; doi: 10.1038/gt.2017.71 This is an unedited manuscript that has been accepted for publication. NPG are providing this early version of the manuscript as a service to our customers. The manuscript will undergo copyediting, typesetting and a proof review before it is published in its final form. Please note that during the production process errors may be discovered which could affect the content, and all legal disclaimers apply.AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease J Miniarikova1,2, V Zimmer3,4, R Martier1,2, C C Brouwers1, C Pythoud3,4, K Richetin3,4, M Rey3,4, J Lubelski1, M M Evers1, S J van Deventer2, H Petry1, N Déglon3,4 and P Konstantinova11Department of Research & Development, uniQure, Amsterdam, the Netherlands 2Department of Gastroenterology and Hepatology, Leiden University Medical Center, Leiden, the Netherlands 3Neurosciences Research Center (CRN), LCMN, Lausanne University Hospital, Lausanne, Switzerland 4Department of Clinical Neurosciences, Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne University Hospital, Lausanne, Switzerland Correspondence: Dr P Konstantinova, Department of Research & Development, UniQure, Paasheuvelweg 25A, Amsterdam 1105BP, The Netherlands. E-mail: p.konstantinova@uniQure.com Received 19 January 2017; Revised 16 June 2017; Accepted 25 July 2017 Accepted article preview online 3 August 2017 Abstract Huntington’s disease (HD) is a fatal progressive neurodegenerative disease caused by a mutation in the huntingtin (HTT) gene. To date, there is no treatment to halt or reverse the course of HD. Lowering of either total or only the mutant HTT expression is expected to have therapeutic benefit. This can be achieved by engineered micro (mi)RNAs targeting HTT transcripts and delivered by an adeno-associated viral (AAV) vector. We have previously showed a miHTT construct to induce total HTT knock-down in Hu128/21 HD mice, while miSNP50T and miSNP67T constructs induced allele-selective HTT knock-down in vitro. In the current preclinical study, the mechanistic efficacy and gene specificity of these selected constructs delivered by an AAV serotype 05 (AAV5) vector was addressed using an acute HD rat model. Our data demonstrated suppression of mutant HTT mRNA, which almost completely prevented mutant HTT aggregate formation, and ultimately resulted in suppression of DARPP-32-associated neuronal dysfunction. The AAV5-miHTT-451 construct was found to be the most efficient, although AAV5-miSNP50T demonstrated the anticipated mutant HTT allele selectivity and no passenger strand expression. Ultimately, AAV5-delivered-miRNA-mediated HTT lowering did not cause activation of microglia or astrocytes suggesting no immune response to the AAV5 vector or therapeutic precursor sequences. These preclinical results suggest that using gene therapy to knock-down HTT may provide important therapeutic benefit for HD patients and raised no safety concerns, which supports our ongoing efforts for the development of an RNAi-based gene therapy product for HD.www.nature.com/gt/journal/vaop/naam/a...
uniQure N.V. - 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 announced that AMT-130, its proprietary gene therapy candidate for Huntington's disease, has received Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). There are currently no approved medical treatments aimed at addressing the underlying cause of Huntington's disease, and AMT-130 has the potential to play a role in this area of high unmet medical need. ODD in the United States provides a special status for investigational drugs being developed for rare diseases considered to affect only up to 200,000 people in the U.S. The ODD programs offer product market exclusivity for up to seven years in the U.S. and ten years in the European Union following regulatory approval, along with tax and financial incentives for companies developing medicines for such orphan indications. "Attaining orphan designation recognizes the potential that AMT-130 holds in delivering meaningful therapeutic benefit to patients suffering from this devastating disease," said Matthew Kapusta, chief executive officer of uniQure. "It supports our ongoing development in Huntington's as we seek to bring the first gene therapy approach to this disease into the clinic next year." 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. Using AAV vectors to deliver micro-RNAs directly into the brain represents a highly innovative approach to treating Huntington's disease.
www.telegraaf.nl/financieel/550351/un... Amsterdam (AFN) - Het Nederlandse biotechnologiebedrijf UniQure heeft een stap gezet in de ontwikkeling van zijn middel AMT-130 tegen de ziekte van Huntington. Het aan de Nasdaq genoteerde bedrijf meldde vrijdag dat de Amerikaanse toezichthouder de speciale weesgeneesmiddelenstatus heeft toegekend aan het middel. Het stempel van de FDA betekent dat het middel aanspraak kan maken op een alleenrecht in de Amerikaanse markt voor maximaal zeven jaar en in de EU voor tien jaar. Ook biedt het mogelijk aanvullende financiële voordelen. De ziekte van Huntington is een zeldzame neurologische aandoening, die de motoriek en het denkvermogen aantast. Er is momenteel geen goedgekeurd medicijn tegen de ziekte voorhanden.
european-biotechnology.com/up-to-date... uniQure’s AMT-130 receives FDA Orphan Drug Designation in Huntington’s disease 09.10.2017 by tg As first gene therapy ever, AMT-130 has been granted FDA Orphan Drug status in Huntington’s Disease. For the Amsterdam-based gene therapy specialist Uniqure NV it’s another good news after losing its licence partner Chiesi: its proprietary gene therapy candidate for Huntington’s disease has gotten orphan drug designation in the largest pharma market globally. AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA which silences the defective huntingtin (HTT) gene. The gene therapy is delivered directly into the brain of patients with the uncurable disease for which there is no causative treatment yet. The neurodegenerative disorder ist caused by accumulation of CAG expansions in the huntingtin (HTT) gene. Clinically, Huntigton disease leads to a loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration over a 12- to 15-year period of time. In March 2016, uniQure published preclinical results, indicating that a one-time administration can block the mutant HTT gene that causes Huntington’s disease. Uniqure demonstrated strong allele-selective silencing of the HTT gene by miSNP50 targeting the heterozygous single-nucleotide polymorphism rs362331 and total HTT silencing exon 1 of the HTT gene by miH12 both in vitro and in vivo. According to Uniqure’s CEO Matthew Kapusta, the company plans to start Phase I studies with its experimental gene therapy next year.
tools.eurolandir.com/tools/Pressrelea... One-time Administration of AMT-130 Demonstrates Survival Benefit and Functional Improvement of Huntington's Disease Symptoms in Preclinical Study IND-enabling Toxicology Study Initiated LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 18, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today presented new preclinical data on AMT-130, its proprietary gene therapy candidate for the treatment of Huntington's disease (HD), at the European Society of Gene and Cell Therapy (ESGCT) 25th Anniversary Congress in Berlin, Germany. Data from the study demonstrate that following administration of AMT-130 in Huntington's disease mouse models, significant improvements in both motor-coordination and survival were observed, as well as a dose-dependent, sustained reduction in huntingtin protein. AMT-130 comprises an AAV5 vector carrying a DNA cassette encoding an engineered micro RNA (miHTT) that silences the human huntingtin protein. The study on functional improvement and sustained huntingtin lowering was performed by members of uniQure's research department in collaboration with Charles River Discovery Research Services, Finland. "We are confident that the combination of suppression of neuronal dysfunction, improvement of Huntington's disease symptoms, extended survival and long-term huntingtin lowering observed in these studies, could translate into patient benefit and improve their quality of life," stated Sander van Deventer, M.D., Ph.D., chief scientific officer at uniQure. "We have now begun our investigational new drug-enabling toxicology studies in rodents and non-human primates that will support an IND application for AMT-130 next year." Preclinical Data Findings This study builds on previous data generated at uniQure, demonstrating a long-term significant suppression of mutant huntingtin protein, the cause of Huntington's disease, after a single administration of AMT-130 in the Q175 mouse model of Huntington's disease. The current study was conducted in the rapidly progressing R6/2 mouse model of Huntington's disease, which is characterized by early onset of motor symptoms and a much reduced life-span. A single administration of AMT-130 into the striatum was followed by a significant improvement of motor symptoms including improved coordination on the rotarod (a rotating cylinder to test coordination, physical condition and motor planning) as well as a significantly increased median survival from 120 to 149 days, compared with the control group (p = 0.0270). The data also demonstrate a significant reduction in expression of mutant huntingtin protein. The functional improvements observed in these preclinical studies helped support the Orphan Drug Designation granted to AMT-130 by the U.S. Food and Drug Administration earlier this month and reinforce that huntingtin-lowering gene therapy for Huntington's disease could modify the course of this devastating disease. About Huntington's Disease Huntington's disease is a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration over a 12- to 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.
AAV5-miHTT GT dem.sustained huntingtin lowering&functional improvement in #HD mm uniqure.com/ESGCT%20Huntington%20mous...
uniQure Receives Orphan Medicinal Product Designation in Europe for AMT-130 in Huntington's disease LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Jan. 22, 2018 (GLOBE NEWSWIRE) -- 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 gene therapy candidate for Huntington's disease, has received an Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency. AMT-130 represents the first investigational AAV-gene therapy in Huntington's disease to receive such designation. In October 2017 the company announced that the U.S. Food and Drug Administration granted orphan drug designation for the same indication. There are currently no approved medical treatments aimed at addressing the underlying cause of Huntington's disease, and AMT-130 has the potential to play a role in this area of high unmet medical need. AMT-130 consists of an AAV5 vector carrying an engineered micro-RNA specifically designed to silence the huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein. Using AAV vectors to deliver micro-RNAs directly into the brain represents a highly innovative approach to treating Huntington's disease. "The granting of orphan drug designation in Europe represents another important milestone for our AMT-130 program," said Matthew Kapusta, chief executive officer of uniQure. "Huntington's disease affects approximately 70,000 people in the U.S. and Europe1, making this one of the largest clinical unmet needs in the rare disease field. We expect to file an Investigational New Drug application later this year and be the first AAV-gene therapy to enter clinical development for Huntington's disease." To qualify for OMPD in Europe a therapy must be intended for the treatment of a disease that is life-threatening or chronically debilitating and have a patient prevalence in the European Union of no more than 5 in 10,000. OMPD offers product market exclusivity for ten years in the European Union following regulatory approval, along with tax and financial incentives for companies developing medicines for such orphan indications. About Huntington's disease Huntington's disease is a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration over a 12- to 15-year period of time. The disease is caused by the expansion of CAG trinucleotide in exon 1 of a multifunctional gene coding for protein called huntingtin. Despite the clear etiology, there are no therapies available to treat the disease, delay onset or slow progression of a patient's decline.
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Coca-Cola European Partners
Cofinimmo
Cognosec
Colruyt
Commerzbank
Compagnie des Alpes
Compagnie du Bois Sauvage
Connect Group
Continental AG
Corbion
Core Labs
Corporate Express
Corus
Crescent (voorheen Option)
Crown van Gelder
Crucell
CTP
Curetis
CV-meter
Cyber Security 1 AB
Cybergun
D'Ieteren
D.E Master Blenders 1753
Deceuninck
Delta Lloyd
DEME
Deutsche Cannabis
DEUTSCHE POST AG
Dexia
DGB Group
DIA
Diegem Kennedy
Distri-Land Certificate
DNC
Dockwise
DPA Flex Group
Draka Holding
DSC2
DSM
Duitse aandelen
Dutch Star Companies ONE
Duurzaam Beleggen
DVRG
Ease2pay
Ebusco
Eckert-Ziegler
Econocom Group
Econosto
Edelmetalen
Ekopak
Elastic N.V.
Elia
Endemol
Energie
Energiekontor
Engie
Envipco
Erasmus Beursspel
Eriks
Esperite (voorheen Cryo Save)
EUR/USD
Eurobio
Eurocastle
Eurocommercial Properties
Euronav
Euronext
Euronext
Euronext.liffe Optiecompetitie
Europcar Mobility Group
Europlasma
EVC
EVS Broadcast Equipment
Exact
Exmar
Exor
Facebook
Fagron
Fastned
Fingerprint Cards AB
First Solar Inc
FlatexDeGiro
Floridienne
Flow Traders
Fluxys Belgium D
FNG (voorheen DICO International)
Fondsmanager Gezocht
ForFarmers
Fountain
Frans Maas
Franse aandelen
FuelCell Energy
Fugro
Futures
FX, Forex, foreign exchange market, valutamarkt
Galapagos
Gamma
Gaussin
GBL
Gemalto
General Electric
Genfit
Genmab
GeoJunxion
Getronics
Gilead Sciences
Gimv
Global Graphics
Goud
GrandVision
Great Panther Mining
Greenyard
Grolsch
Grondstoffen
Grontmij
Guru
Hagemeyer
HAL
Hamon Groep
Hedge funds: Haaien of helden?
Heijmans
Heineken
Hello Fresh
HES Beheer
Hitt
Holland Colours
Homburg Invest
Home Invest Belgium
Hoop Effektenbank, v.d.
Hunter Douglas
Hydratec Industries (v/h Nyloplast)
HyGear (NPEX effectenbeurs)
HYLORIS
Hypotheken
IBA
ICT Automatisering
Iep Invest (voorheen Punch International)
Ierse aandelen
IEX Group
IEX.nl Sparen
IMCD
Immo Moury
Immobel
Imtech
ING Groep
Innoconcepts
InPost
Insmed Incorporated (INSM)
IntegraGen
Intel
Intertrust
Intervest Offices & Warehouses
Intrasense
InVivo Therapeutics Holdings Corp (NVIV)
Isotis
JDE PEET'S
Jensen-Group
Jetix Europe
Johnson & Johnson
Just Eat Takeaway
Kardan
Kas Bank
KBC Ancora
KBC Groep
Kendrion
Keyware Technologies
Kiadis Pharma
Kinepolis Group
KKO International
Klépierre
KPN
KPNQwest
KUKA AG
La Jolla Pharmaceutical
Lavide Holding (voorheen Qurius)
LBC
LBI International
Leasinvest
Logica
Lotus Bakeries
Macintosh Retail Group
Majorel
Marel
Mastrad
Materialise NV
McGregor
MDxHealth
Mediq
Melexis
Merus Labs International
Merus NV
Microsoft
Miko
Mithra Pharmaceuticals
Montea
Moolen, van der
Mopoli
Morefield Group
Mota-Engil Africa
MotorK
Moury Construct
MTY Holdings (voorheen Alanheri)
Nationale Bank van België
Nationale Nederlanden
NBZ
Nedap
Nedfield
Nedschroef
Nedsense Enterpr
Nel ASA
Neoen SA
Neopost
Neovacs
NEPI Rockcastle
Netflix
New Sources Energy
Neways Electronics
NewTree
NexTech AR Solutions
NIBC
Nieuwe Steen Investments
Nintendo
Nokia
Nokia OYJ
Nokia Oyj
Novacyt
NOVO-NORDISK AS
NPEX
NR21
Numico
Nutreco
Nvidia
NWE Nederlandse AM Hypotheek Bank
NX Filtration
NXP Semiconductors NV
Nyrstar
Nyxoah
Océ
OCI
Octoplus
Oil States International
Onconova Therapeutics
Ontex
Onward Medical
Onxeo SA
OpenTV
OpGen
Opinies - Tilburg Trading Club
Opportunty Investment Management
Orange Belgium
Oranjewoud
Ordina Beheer
Oud ForFarmers
Oxurion (vh ThromboGenics)
P&O Nedlloyd
PAVmed
Payton Planar Magnetics
Perpetuals, Steepeners
Pershing Square Holdings Ltd
Personalized Nursing Services
Pfizer
Pharco
Pharming
Pharnext
Philips
Picanol
Pieris Pharmaceuticals
Plug Power
Politiek
Porceleyne Fles
Portugese aandelen
PostNL
Priority Telecom
Prologis Euro Prop
ProQR Therapeutics
PROSIEBENSAT.1 MEDIA SE
Prosus
Proximus
Qrf
Qualcomm
Quest For Growth
Rabobank Certificaat
Randstad
Range Beleggen
Recticel
Reed Elsevier
Reesink
Refresco Gerber
Reibel
Relief therapeutics
Renewi
Rente en valuta
Resilux
Retail Estates
RoodMicrotec
Roularta Media
Royal Bank Of Scotland
Royal Dutch Shell
RTL Group
RTL Group
S&P 500
Samas Groep
Sapec
SBM Offshore
Scandinavische (Noorse, Zweedse, Deense, Finse) aandelen
Schuitema
Seagull
Sequana Medical
Shurgard
Siemens Gamesa
Sif Holding
Signify
Simac
Sioen Industries
Sipef
Sligro Food Group
SMA Solar technology
Smartphoto Group
Smit Internationale
Snowworld
SNS Fundcoach Beleggingsfondsen Competitie
SNS Reaal
SNS Small & Midcap Competitie
Sofina
Softimat
Solocal Group
Solvac
Solvay
Sopheon
Spadel
Sparen voor later
Spectra7 Microsystems
Spotify
Spyker N.V.
Stellantis
Stellantis
Stern
Stork
Sucraf A en B
Sunrun
Super de Boer
SVK (Scheerders van Kerchove)
Syensqo
Systeem Trading
Taiwan Semiconductor Manufacturing Company (TSMC)
Technicolor
Tele Atlas
Telegraaf Media
Telenet Groep Holding
Tencent Holdings Ltd
Tesla Motors Inc.
Tessenderlo Group
Tetragon Financial Group
Teva Pharmaceutical Industries
Texaf
Theon International
TherapeuticsMD
Thunderbird Resorts
TIE
Tigenix
Tikkurila
TINC
TITAN CEMENT INTERNATIONAL
TKH Group
TMC
TNT Express
TomTom
Transocean
Trigano
Tubize
Turbo's
Twilio
UCB
Umicore
Unibail-Rodamco
Unifiedpost
Unilever
Unilever
uniQure
Unit 4 Agresso
Univar
Universal Music Group
USG People
Vallourec
Value8
Value8 Cum Pref
Van de Velde
Van Lanschot
Vastned
Vastned Retail Belgium
Vedior
VendexKBB
VEON
Vermogensbeheer
Versatel
VESTAS WIND SYSTEMS
VGP
Via Net.Works
Viohalco
Vivendi
Vivoryon Therapeutics
VNU
VolkerWessels
Volkswagen
Volta Finance
Vonovia
Vopak
Warehouses
Wave Life Sciences Ltd
Wavin
WDP
Wegener
Weibo Corp
Wereldhave
Wereldhave Belgium
Wessanen
What's Cooking
Wolters Kluwer
X-FAB
Xebec
Xeikon
Xior
Yatra Capital Limited
Zalando
Zenitel
Zénobe Gramme
Ziggo
Zilver - Silver World Spot (USD)
Indices
AEX
865,35
0,00%
EUR/USD
1,0690
+0,17%
FTSE 100
7.847,99
0,00%
Germany40^
17.838,10
+0,38%
Gold spot
2.378,45
+0,74%
NY-Nasdaq Composite
15.683,37
-1,15%
Stijgers
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