uniQure « Terug naar discussie overzicht

quote:

T. Montana schreef op 22 juni 2016 13:17:

Voyager meldt interim PD data. Kan er geen chocola van maken eerlijk gezegd.
finance.yahoo.com/news/voyager-therap...

quote:

Prof. Dollar schreef op 22 juni 2016 14:13:

[...]
Als ik het zo vlot bekijk...

Dan is Voyager (VYGR) hier mee bezig:
"One approach is to increase dopamine production in specific regions of the brain. One study using this approach approaches uses the gene for the enzyme aromatic amino acid decarboxylase (AADC). This enzyme converts levodopa into dopamine, a neurotransmitter that is deficient in Parkinson's disease. Studies have shown that AADC is gradually lost in Parkinson's disease. The progressive loss of this enzyme is thought to contribute to the need to increase levodopa doses as time goes on. The rationale for this approach is that if a greater amount of AADC is present in the location where dopamine should be released, then a more reliable and perhaps a more robust response to levodopa will occur. Moreover, it is possible that a patient who no longer is obtaining a reliable benefit from levodopa therapy might regain responsiveness to this treatment after gene therapy with AADC. Inherent in this approach treatment is that the patient may alter the effect of his gene therapy by adjusting his daily dose of levodopa, since the effect of this therapy depends on continuing treatment with levodopa."

En is QURE hier mee bezig:
"Another approach is using brain proteins, termed growth factors (because of their role in brain development), that might protect against progression of Parkinson's disease or possibly even reverse it by stimulating regrowth of injured nerve cells. A number of growth factors have been identified over the years. These include glial cell line-derived neurotrophic factor (GDNF) and Neurturin which is similar to GDNF and shares the ability to promote the survival of dopaminergic neurons. In models of Parkinson's disease, GDNF and Neurturin have been shown to promote the survival of dopaminergic neurons. Both a phase 1 and phase 2 study using Neurturin gene therapy targeted to the putamen have been performed. In the phase 2 study, 38 patients were randomized to Neurturin gene therapy or to sham surgery. Unfortunately, there was no significant difference in the main outcome measures at 12 months. While the lack of benefit in the main outcome measures was disappointing, a subgroup of patients followed for 18 months was slightly better in the Neurturin patient than the sham treatment group, suggesting that slightly longer period of observations might be necessary to see a benefit with this gene therapy. Because of this interesting result, a second phase 2 study is underway in which Neurturin gene therapy is also targeted to the substantia nigra."

Bij het stuk onder QURE moet opgemerkt worden dat Neurturin verder door anderen is onderzocht, maar GDNF nog niet (dat doet QURE op dit moment). In het verlengde daarvan een hoopvol abstract: link.springer.com/protocol/10.1007/97... (Dank flosz!)

Voor zowel VYGR als QURE geldt dat ze een toedieningsprotocol moeten hebben, dat veilig is en de juiste plek(ken) bereikt. Daarover gaat deze interim PD data van VYGR. In feite zegt het nog niks, maar het belangrijkste is hun mededeling dat ze voor eind dit jaar top-line data willen publiceren over de motorische skills van patiënten (een indicatie van effectiviteit). Goed om in de gaten te houden!

Tegen die tijd ben ik ook benieuwd wat QURE doet. De tweede cohort moet dan afgerond zijn. Gaat QURE het programma meer naar zich toetrekken? In de pipeline wordt het nog weergegeven als 'academic program' en dat wordt idealiter een 'uniQure program'...

quote:

Prof. Dollar schreef op 24 juni 2016 12:32:

Relevant persbericht van QURE partner:

Synpromics Announces Extension of Collaboration

Edinburgh, UK, 22nd June 2016 / Synpromics Ltd, the leading synthetic promoter company, is pleased to announce an extension to the original collaboration with uniQure announced in January 2015, which focused on the development of synthetic promoters with up-regulated liver cell specific activity suitable for gene expression using an AAV vector.

Synpromics has successfully met the project goals and produced synthetic promoters, of fewer than 250 base pairs, that show high levels of activity. Synpromics and uniQure have now agreed to extend the programme under which Synpromics will further apply its technology and know-how to create ultra-small synthetic promoters. Additionally, Synpromics has developed a proprietary vector screening technology which allows the in vivo screening of synthetic promoter candidates for specificity and activity.

Dr David Venables, CEO of Synpromics, commented, “We are delighted that the first collaboration with uniQure has produced such exciting results and has met its goals. With the agreed extension announced today we can further apply our technology to make shorter promoters and so increase the efficacy of gene therapy. Gene therapy is an exciting new therapeutic area and we are pleased to be able to make a significant contribution”

quote:

T. Montana schreef op 24 juni 2016 21:50:

[...]

Begrijp ik het goed en kunnen ze hiermee (up-regulated) een turbo op hun Hem B AAV zetten en ONCE de hielen laten zien? Dat zou wat zijn.