biotech/pharma algemeen: medicijntests
07-11-2013 20:13 | Door Jeannette Kras www.wel.nl
2600 doden door medicijntests in India
Het is een miljardenindustrie in India, maar tegen een hoge prijs. Tussen 2005 en 2013 zijn waarschijnlijk 2600 mensen overleden door deelname aan medicijnentests. Er waren ook nog eens 11.972 ‘ernstige incidenten’. Dat schrijft dagblad Trouw.
In India nemen ze het niet zo nauw met de regels voor het testen van geneesmiddelen. Door tal van gezondheidsorganisaties is daar felle kritiek op gekomen. Om daar wat aan te doen is een commissie samengesteld, die de situatie moet verbeteren. De commissie kwam onder andere met het advies om de tests alleen nog in erkende centra uit te voeren. Ook zouden deelnemers veel beter geïnformeerd moeten worden en verplicht een standaardformulier voor toestemming moeten ondertekenen.
Een ander punt was de schadevergoeding aan slachtoffers. Volgens de gezondheidsorganisaties wordt er misbruik gemaakt van de armoede en ongeletterdheid van de testpersonen. De commissie zegt hierover dat een schadevergoeding altijd moet worden betaald, tenzij kan worden aangetoond dat schade of overlijden niet komt door de medicijnentest.
Nieuwe wetgeving is niet alleen goed voor de deelnemers, maar ook voor de industrie zelf. Verschillende farmaceutische bedrijven zijn al weggetrokken naar omringende landen. De hoop is dat die weer terugkomen als de regels in orde zijn.
dit bovenstaande artikel heeft niet direct te maken met UniQure zelf, maar voor een ieder die biotech volgt, goed om meer te weten te komen over deze bedrijfstak.
Voor de voormalige CRUCELL aandeelhouders: Minstens 8 jaar oud:
Crucell scientists focus on the discovery and development of transformational immunization products to protect people worldwide against major health threats. Here are just some examples of what we are doing to bring meaningful innovation to global health.
Ebola and Marburg
We are collaborating with international health organizations including WHO and NIAID, and other key stakeholders, governments and public health authorities on the clinical testing, development, production and distribution of the vaccine regimen. The vaccine regimen, which was discovered in a collaborative research program with the National Institutes of Health (NIH), combines a Crucell preventive vaccine with that of Bavarian Nordic (OMX: BAVA), a biotechnology company based in Denmark. This combination vaccine regimen has shown promising results in preclinical studies, and is now planned to be tested for safety and immunogenicity in healthy volunteers in Europe, the USA and Africa starting early January. The company is targeting production of more than one million doses of the vaccine regimen in 2015 – 250,000 of which are expected to be released for broad application in clinical trials by May 2015.
rationeel schreef op 2 december 2014 13:37:
Voor de voormalige CRUCELL aandeelhouders: Minstens 8 jaar oud:
Nog geen 8 wkn. imho.
Niels GEIJSEN Eiwitten direct in de cel inbengen. Hubrecht Instituut.
Embryoselectie of genetische manipulatie; Bas Heijne voelt zich ongemakkelijk bij het idee van een designerbaby. Waar komt dat gevoel vandaan? En is het wel terecht?Meer
elders in biotech ondernemend NL:
ProQR Receives Fast Track Designation from the FDA for QR-110 for Leber’s Congenital Amaurosis Type 10
ProQR receives Fast Track designation by the U.S. Food and Drug Administration (FDA). Closer interaction with FDA could potentially accelerate the development of QR-110 in patients with Leber’s Congenital Amaurosis Type 10 (LCA 10).
LCA 10 is one of the most prevalent forms of gene-related blindness in children worldwide and currently there are no therapies commercially available or in clinical development.
QR-110 is currently in clinical development with the planned Phase 1/2 open-label trial (PQ-110-001) that will assess the safety, tolerability, pharmacokinetics and efficacy of multiple administrations of QR-110 in one eye of each patient and will include approximately 6 adults and 6 children with LCA 10.
Top-line trial results are expected in 2018.
LEIDEN, the Netherlands, May 31, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for QR-110, the lead molecule in its ophthalmology pipeline. QR-110 is being developed for the treatment of patients with Leber’s Congenital Amaurosis Type 10 (LCA 10), a rare genetic disease that causes individuals to lose sight, often in the first years of life. QR-110 is a novel investigational RNA oligonucleotide targeting LCA 10 due to the p.Cys998X mutation, which is one of the most prevalent forms of gene-related blindness in children and currently there are no disease modifying therapies commercially available or in clinical development.
Fast Track designation is granted by the FDA to drugs that are under development for serious conditions and have the potential to fulfill an unmet medical need. It was established with the intention to bring promising drugs to patients sooner by facilitating the development with more frequent FDA interactions and expediting the review process.
“QR-110 is a unique and elegant approach to addressing the underlying genetic defect that leads to blindness in individuals with LCA 10 due to the p.Cys998X mutation. We are very pleased with granting of the Fast Track designation by the FDA for this program as it highlights the need for innovative and efficacious medicines for this devastating disease for which there is currently nothing available,” said Noreen R. Henig, Chief Medical Officer of ProQR. “We are also excited to be able to initiate our first trial for QR-110 as a multidose study and for that we will benefit from the Fast Track Designation. We believe development of QR-110 also opens the possibilities for RNA approaches to target other causes of genetic blindness. We are building our pipeline in ophthalmology and will use our rapid development approach to QR-110 as a model for how to bring RNA therapeutics to patients in need.”
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis Type 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis Type 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore wild-type CEP290 mRNA leading to the production of wild-type CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union.
About Leber’s Congenital Amaurosis Type 10
Leber’s congenital amaurosis is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of which LCA Type 10 (LCA 10) is one of the more severe forms. LCA 10 is caused by mutations in the CEP290 gene of which the p.Cys998X mutation is most common. LCA 10 leads to progressive loss of vision causing most patients to lose their sight in the first few years of life. To date, there are no treatments approved or products in clinical development that treat the underlying cause of the disease. Although prevalence rates vary, we believe approximately 2,000 people in the Western world have LCA 10 because of this mutation.
About the PQ-110-001 Study
PQ-110-001 is an open-label trial that will include approximately 6 children (age 6- 17 years) and 6 adults (= 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients will receive four intravitreal injections of QR-110 into one eye; one every three months for one year. The QR-110 trial is expected to be conducted in three centers with significant expertise in genetic retinal disease in the US and Europe.
The primary endpoints will be safety and tolerability. Secondary efficacy endpoints will assess the pharmacokinetics and restoration/improvement of visual function and retinal structure through ophthalmic tests such as visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR), mobility course and fixation stability. Changes in quality of life in LCA subjects will also be evaluated. Top-line results from the trial are expected to be available in 2018.
evt ook: finance.yahoo.com/quote/PRQR/?p=PRQR
Wat betekent dit samengevat?
ProQR Receives Orphan Drug Designation from FDA for Drug Candidate QR-313 for Dystrophic Epidermolysis Bullosa and will Present Data at two Scientific Conferences
GlobeNewswire•September 19, 2017Comment
ProQR’s drug candidate QR-313 for dystrophic epidermolysis bullosa (DEB) receives orphan drug designation from the FDA, representing the fifth program in the Company’s pipeline to receive ODD in the U.S.
The Company will be presenting pre-clinical data for QR-313 at two European scientific conferences in Salzburg, Austria – EB2017 Research Conference and ESDR Meeting.
DEB is a severe genetic skin disease with no disease modifying treatments currently available.
QR-313 targets the most common mutations within DEB, which are mutations in exon 73 of the COL7A1 gene and is designed for topical administration.
A first-in-human clinical trial of QR-313 will be initiated in 2018. Clinical data from the program will also be available in 2018.
LEIDEN, the Netherlands, Sept. 19, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (PRQR) today announced that investigational drug QR-313 for dystrophic epidermolysis bullosa (DEB) has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA). QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause in dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. DEB is a rare genetic disease that can lead to severe blistering of the skin resulting in high treatment burden and poor quality of life for patients.
“We are pleased to have ODD designation in the U.S. for our QR-313 program targeting dystrophic epidermolysis bullosa,” said David M. Rodman, MD, Chief Development Strategy Officer of ProQR, “It highlights the unmet need in this devastating disease, for which we aim to make a meaningful difference. Our goal for this disease is to develop a pipeline of programs that can treat DEB mutations in a targeted manner and to actively advance the pipeline through development.”
Poster Presentations at Upcoming Scientific Conferences
The Company will present two posters (# 50 and 51) during the EB2017 - 5th World Conference of Epidermolysis Bullosa Research Conference from September 24-26, 2017 in Salzburg, Austria.
The same posters (# 181 and 194) will also be presented at the 47th Annual European Society for Dermatological Research (ESDR) Meeting on September 29, 2017 in Salzburg, Austria. Poster #181 is selected for a presentation (walk title: Genetics and Cell Based Therapy 2: Epidermolysis bullosa) on September 29 at 14.35-15.30 CET.
The posters are titled:
Local delivery of an antisense oligonucleotide for recessive dystrophic epidermolysis bullosa.
In vitro evaluation of QR-313; an antisense oligonucleotide designed to skip exon 73 from the COL7A1 mRNA.
About Orphan Drug Designation (ODD)
Orphan drugs are intended for the treatment, diagnosis or prevention of serious diseases that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. FDA evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. FDA provides incentives for sponsors to develop products for rare diseases, including development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the U.S.
QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce a functional C7 protein, thereby restoring functionality of the anchoring fibrils.
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. *Since 2012*
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 QR-313 and the clinical development and the therapeutic potential thereof, and statements regarding our pipeline of programs targeting DEB. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that we may not realize the intended benefits afforded by orphan drug designation for our QR-313 program targeting DEB, positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. 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, except as required by law.
ProQR is goed bezig en de moeite om vinger aan de pols te houden!
'verplicht' leesvoer voor biotech-beleggers die van Hollands Glorie houden ;-)
Vanmiddag data van ProQR's CF trial...duimen dat ze goed zijn, want dan heeft Crucell een bijzondere en mogelijk zelfs waardevollere opvolger gecreeërd.
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