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Amyotrophic Lateral Sclerosis (ALS) met Treeway

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[verwijderd] 14 januari 2015 18:41

we voegen er aldus een nieuw draadje bij voor deze nieuwkomer in de pijplijn van UniQure:

www.cnbc.com/id/102336957

Treeway Announces License and Collaboration Agreement with uniQure to Develop a Gene Therapy for Amyotrophic Lateral Sclerosis (ALS)
4 Hours AgoPR Newswire

COMMENTSStart the Discussion

ROTTERDAM, the Netherlands, January 14, 2015 /PRNewswire/ --

Treeway B.V., founded by entrepreneurs Bernard Muller and Robbert Jan Stuit, both diagnosed with ALS, have announced a collaboration with uniQure N.V., a leading gene therapy company, (NASDAQ:QURE), to develop a gene therapy treatment for ALS.

Under the terms of the licensing and collaboration agreement, uniQure has granted Treeway an exclusive license in this field to uniQure's relevant AAV5 viral vector and GDNF (Glial cell-derived neurotrophic factor) intellectual property. Treeway is responsible for the preclinical and clinical development of the ALS gene therapy treatment. uniQure will provide Treeway with its manufacturing capabilities and will further collaborate with Treeway on ALS gene therapy development. Treeway and uniQure will jointly commercialize any resulting ALS gene therapy with defined geographical rights for commercialization assigned to each company. Financial details of the agreement have not been disclosed.

Inez de Greef, CEO of Treeway, is very pleased about this licensing, collaboration, and co-marketing agreement as it represents a major milestone in executing its mission to develop ALS therapy. "Gene therapy is an innovative and viable technology for developing an ALS therapy and has the potential to change the disease course of ALS. A collaboration between Treeway and uniQure will accelerate this development by bringing two unique companies together," says Ms. de Greef.

Jörn Aldag, CEO of uniQure, adds: "We are impressed by the immense energy and will power of the Treeway founders in seeking a cure for ALS. We believe that by combining their disease know-how and uniQure's gene therapy platform and expertise, we can jointly achieve a step forward in the way ALS is treated in the future."

Note for the editor

about ALS
Amyotrophic Lateral Sclerosis, also known as Lou Gehrig's disease, is a relentless progressive neurodegenerative disease that causes muscle weakness, disability and eventually death, with a median survival of three years. To date, there is no cure for ALS.

uniQure
uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. Our approach is validated by multiple partnerships and the regulatory approval of our lead product Glybera. www.uniQure.com

Treeway B.V.
Treeway is a biotechnology company and has been founded by entrepreneurs Bernard Muller and Robbert Jan Stuit, both diagnosed with ALS. Treeway's strategy is founded on a cohesive combination of approaches that together should provide the highest likelihood of bringing successful treatments for ALS to the patient in the short term.

For questions and more information about Treeway, please contact via Inez de Greef info@treeway.nl or www.treeway.nl / +31-10-298-88-88.

SOURCE Treeway B.V.

zie ook:

finance.yahoo.com/news/treeway-announ...

bilbo3 14 januari 2015 20:38

Wat is nieuw?
Zie www.uniqure.com/partners/

Cedars-Sinai Regenerative Medicine Institute

uniQure is part of a consortium led by the Cedars-Sinai Regenerative Medicine Institute that has initiated animal studies to find a basis for a gene therapy treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. The Cedars-Sinai study involves scientists at the Regenerative Medicine Institute at Cedars-Sinai Medical Center; the University of Wisconsin, Madison; and uniQure. Cedars-Sinai recently received a $2.5 million grant from the US Department of Defense to underwrite the project. Click here for more information.

Prof. Dollar 15 januari 2015 13:43

quote:
invoorentegenspoed schreef op 14 januari 2015 18:41:
we voegen er aldus een nieuw draadje bij voor deze nieuwkomer in de pijplijn van UniQure [..]

Wellicht onder een nieuwe categorie "uniQure Collaboration Programs"... maar of het in de pipeline komt betwijfel ik nog een beetje. uniQure heeft namelijk zelf geen persbericht uitgestuurd. Wel jammer. En Treeway suggereert op haar website van een "eigen" pipeline.

[verwijderd] 15 januari 2015 16:47

ik denk ook niet dat dit de reden van de uptrend is. Op de conference had QURE wel leukere deals kunnen trachten te maken, contacten te leggen etc.

De inkopers zijn al een poos bezig en hoewel deze gepubliceerde deal welkom is, niet baanbrekend genoeg om de uptrend mee te verklaren.
Ik blijf van mening dat één (of meer) partijen bezig zijn om hun belang in QURE op te bouwen. De prijs tot boven de 23$ bleek geen enkele belemmering te zijn...dus much more to come (IMHO).

Nu ook weer goed pump en dump zichtbaar: 300 stukjes waren voldoende om de koers ruim 70 cent lager te zetten! (van 22,80 naar 22,08)

flosz 4 april 2015 00:20

Via Bernardus Muller
twitter.com/bernardusmuller

ALS patients press FDA for quick access to controversial biotech drug
www.washingtonpost.com/national/healt...

flosz 13 juni 2015 10:06

The BioReport

Bernard Muller was a successful businessman in the maritime and oil industry, but when he was diagnosed with the neurodegenerative disease ALS in 2010, he turned his entrepreneurial skills toward developing new therapies to treat the disease. Muller co-founded the world’s largest genetic research project for ALS, project MinE, and launched Treeway, a biotech company developing new therapies for ALS. As the Biotechnology Industry Organization kicks off its BIO 2015 International Convention in Philadelphia June 15, Muller is a finalist as one of the organization’s Everyday Superheroes in the pharma/biotech category. We spoke to Muller about his decision to launch Treeway, the active role he sees for patients, and why he thinks traditional approaches to drug development and clinical trial design have not served ALS patients well.
t.co/lQgOjL60wF

flosz 16 juni 2015 21:18

#BIO2015 Everyday Superhero Honorees Announced.Top Honoree: Bernard Muller, Founder & Chief Business Officer, Treeway.

Pharma/Biotech Category
The Pharma/ Biotech Category recognizes employees in the life science industry who have been instrumental in advancing therapies for patients.

Top Honoree: Bernard Muller, Founder & Chief Business Officer, Treeway. Bernard calls himself Patient 3.0. He was diagnosed with ALS and took it as a challenge. In 2012, he co-founded the world’s largest genetic research project for ALS, project MinE. That same year, he also launched a biotech company with the aim of finding a cure or therapy for ALS. His company, Treeway, is now entering Phase 3 clinical trials with its lead product candidate.

twitter.com/treewaytherapy/status/610...

flosz 17 juni 2015 10:41

twitter.com/inezdegreef/status/610953...
Dutch Pride!

Bijlage:

flosz 28 juli 2015 17:43

CEDARS SINAI MEDICAL CENTER
Clive Svendsen, Ph.D., Director & Professor, Board of Governors Regenerative Medicine Institute
Oct. 8 | 4:30pm | La Jolla Ballroom 2
Los Angeles, CA
(Non-Profit Hospital)
Through funding from the California Institute for Regenerative Medicine (CIRM), Cedars Sinai Regenerative Medicine Institute is half-way through the preclinical and manufacturing process to take the world’s first combined stem cell and gene therapy product to treat ALS to the clinic. The product is a human neural progenitor line derived from fetal tissue expanded using a unique “chopping” method that avoids cell dissociation and maintains the cultures in an in vivo environment – closely adapted to that of the living host CNS. They are engineered to produce the powerful growth factor GDNF and then transplanted into the spinal cord of rodents and pigs to test for toxicity, tumorigenicity and migration. Following IND submission, the Institute will plan an 18 patient clinical trial at Cedars-Sinai using a new minimally invasive injection device with a unique unilateral design powered to show changes in progression in the treated limb. Cedars Sinai is partnering with uniQure for the next phase of commercialization of this product. www.cedars-sinai.edu
tinyurl.com/ozga88d
Conf.: 7/8/9 oct. In 2014... & 7/8/9 oct. 2015 Agenda 2015 komt in August..deze info is dus van 2014.

Prof. Dollar 28 juli 2015 18:03

Mooi gevonden, flosz!

[verwijderd] 4 augustus 2015 22:02

ALS dat eens zou werken,....:) :) :)

rationeel 7 augustus 2015 12:35

Waar staat het artikeltje met Jim Rogers?

flosz 4 september 2015 16:40

The ALS Association Announces $11.6 Million in New Research Grants

July 14, 2015
Today, The ALS Association announced its support of 58 new research grants totaling $11,621,638 to find treatments and a cure for the disease. These research awards include investigator-initiated grants, drug development contracts, Milton Safenowitz Postdoctoral Fellowships and support of the NEALS/TREAT ALS™ Clinical Trials Network.
......

Drug Development Contracts

Drug development contracts are milestone-driven awards designed to rapidly bring the most promising potential therapies closer to clinical trials. Many of the contracts are in partnership with industry. Academic-industry partnerships are invaluable to drive treatment approaches for ALS more rapidly to the clinic.

Clive Svendsen, Ph.D., Director of the Regenerative Medicine Institute at Cedars-Sinai Medical Center in Los Angeles, Calif. will receive $345,275 to develop AAV5-GDNF (TW002) for the treatment of ALS. This study is in partnership Treeway B.V. LTD, Rotterdam, The Netherlands.

tinyurl.com/nspmhbj

flosz 7 december 2015 18:28

Treeway announces collaboration with Cedars AAV5-GDNF (TW002) gene therapy for amyotrophic lateral sclerosis $QURE #ALS www.treeway.nl/treeway-announces-coll...

Rotterdam, December 7th 2015 – Treeway is pleased to announce a collaboration with the Cedars-Sinai Board of Governors Regenerative Medicine Institute for the development of AAV5-GDNF (TW002) gene therapy for amyotrophic lateral sclerosis.
Treeway, a biotech company founded by two ALS patients, obtained an exclusive license agreement from uniQure for the use of AAV5-GDNF earlier this year. GDNF has a promising clinical potential in ALS, but optimization of delivery to the site of action is required. AAV5-GDNF is a gene therapy with a novel administration modality for clinical use and is Treeway’s second product in development (TW002).
To advance the development of AAV5-GDNF gene therapy, Treeway will work with a research team led by Clive Svendsen, PhD, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute in Los Angeles. Svendsen is an expert in human neural stem cell biology and has specifically focused on using neural stem cells, modified to release GDNF, to treat ALS.
Treeway and Cedars-Sinai applied for a TREAT ALS™Drug Development Contract and received the $500,000 award. The Drug Development Contract, an initiative of The ALS association, was designed to rapidly bring promising potential therapeutics closer to clinical trials. The TREAT ALS™Drug Development grant will be used to start a preclinical study in which the safety and efficacy of TW002 in an ALS rat model will be assessed further. The preclinical studies are an important step in the development of TW002 for ALS.
Inez de Greef, CEO of Treeway, is pleased with the collaboration and says: “consistent with Treeway’s philosophy of ‘innovation by collaboration’ we believe that this research will help us to rapidly advance breakthrough science and further the goal of bringing new treatment approaches to ALS patients. We are grateful for the grant awarded by The ALS Association which enables us to jointly work on finding a cure for ALS.”
“The ALS Association is pleased to support funding for academic-industry partnerships to expedite therapy development for ALS”, commented Lucie Bruijn, Ph.D. MBA Chief Scientist from the ALS association.

Bijlage:

flosz 13 februari 2017 18:53

Art. Foliolum (dec.2016), P.6 mbt ALS Treeway incl.TW002

issuu.com/psgroningen/docs/foliolum_d...

Bijlage:

flosz 11 februari 2019 15:46

Even plakken....

Artificial microRNAs targeting C9ORF72 have the potential to reduce accumulation of the intra-nuclear transcripts in ALS (amyotrophic lateral sclerosis)and FTD (frontotemporal dementia) patients ac.els-cdn.com/S2162253119300149/1-s2...
www.iex.nl/Forum/Topic/1306904/3/PubM...

+

What is Frontotemporal Dementia? Get the Essential Facts
twitter.com/brightstarboise/status/10...

flosz 15 februari 2019 15:58

+

Targeting RNA-mediated toxicity in C9ORF72 ALS/FTD by RNAi-based gene therapy $QURE www.cell.com/molecular-therapy-family...

$QURE Announces Publications on AAV Gene Therapy Approach to Treating Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) in the Journal Molecular Therapy Nucleic Acids tools.eurolandir.com/tools/Pressrelea...

Bijlage:

flosz 28 februari 2019 21:46

Experimental Gene Therapy Successfully Silences Key #ALS Gene C9orf72, Preclinical Studies Show alsnewstoday.com/2019/02/18/gene-ther...

Vitavita 30 september 2019 12:06

Don't forget this !

alsnewstoday.com/2019/02/18/gene-ther...

Experimental Gene Therapy Successfully Silences Key ALS Gene C9orf72, Preclinical Studies Show
February 18, 2019 by Jose Marques Lopes, PHD

A gene therapy candidate targeting a key amyotrophic lateral sclerosis (ALS) mutation in the C9orf72 gene is able to lower the accumulation of toxic RNA clumps and reduce the activity of this mutated gene, in cells collected from a patient with frontotemporal dementia (FTD) and in a mouse model of ALS, according to two preclinical studies.

uniQure’s miQURE is designed to silence disease-causing genes while not causing off-target damage. It is intended to induce silencing of the entire target organ by using tiny, cell-derived vesicles called exosomes. The gene therapy candidates using miQURE incorporate micro-RNAs (miRNAs) — RNA molecules that regulate gene expression — that can be delivered with a type of harmless viral vector called an adeno-associated virus (AAV) to provide sustained activity.

The most common genetic cause of familial and sporadic ALS and FTD — found in a substantial number of ALS patients and caused by progressive degeneration of the brain’s frontal and temporal lobes — is an expanded GGGGCC repeat, a sequence of four guanine (G) nucleotides followed by two cytosine (C) nucleotides in the first intron of C9orf72. While nucleotides are the building blocks of DNA, introns are DNA bits normally spliced out in protein production.

In humans, loss-of-function mutations in C9orf72 — leading to reduced or complete lack of protein function — have not been associated with disease, suggesting that a reduction in this gene’s expression (protein production) is likely tolerable.

Most studies linking C9orf72 to disease showed an accumulation of so-called RNA foci in the cell nucleus and deposition of specific proteins in the cytoplasm (within the cell but excluding the nucleus). RNA foci are toxic clumps of mutated C9orf72 RNA that sequester RNA-binding proteins, leading to cellular dysfunction and death.

The two studies were conducted by scientists at uniQure and used the company’s miQURE technology, a next-generation gene-silencing platform. Both were published in the journal Molecular Therapy – Nucleic Acids.

In the first, “Artificial microRNAs targeting C9orf72 can reduce accumulation of the intra-nuclear transcripts in ALS and FTD patients,” the researchers showed that miQURE’s miRNAs lead to lower levels of the C9orf72 messenger RNA — generated from DNA to produce the protein — in both the cell nucleus and the cytoplasm.

Findings also revealed an approximately 50% reduction in RNA foci in cells expressing the mutant form of C9orf72.

Then, two different miRNA candidates — miC-101 and miC-451 — were incorporated into the AAV5 subtype of the viral vector. This enabled silencing of C9orf72 in vitro, including in neurons derived from induced pluripotent stem cells (iPSCs) from a patient with FTD. iPSCs are developed by reprogramming cells so that they revert to an embryonic-like state to grow into all cell types.

The second study, “Targeting RNA-mediated toxicity in C9orf72 ALS/FTD by RNAi based gene therapy,” further showed that AAV5 carrying miRNAs that target C9orf72 are able to reduce the accumulation of C9orf72 RNA, as well as induce silencing of the target gene in both the nucleus and cytoplasm.

The previously shown reductions in C9orf72 RNA and RNA foci were then validated in a mouse model of ALS that carries the human form of the mutant gene. Reduction in C9orf72 RNA were found in the brain’s striatum, a key brain area in motor control, upon delivery of the potential gene therapy specifically into this region.

“Our data provide evidence on the efficacy of artificial miRNAs against C9orf72 as a promising AAV-based gene therapy for ALS and FTD,” the scientists wrote.

Sander van Deventer, MD, PhD, uniQure’s chief scientific officer, said in a press release that the ability to silence C9orf72 in the nucleus “may prove to be critical for therapeutic efficacy of gene therapies for these diseases.”

These results support the continuation of uniQure’s gene therapy program in ALS and FTD, according to van Deventer, who also said, “We are very pleased to have these data published in a highly relevant journal for the field and look forward to further exploring this opportunity.”

Prof. Dollar 30 september 2019 12:21

Zeker niet vergeten, maar wel kritisch blijven.

Prof. dr. Jeroen Pasterkamp: "We hebben wel gevonden dat het uitschakelen van het C9orf72-eiwit tot ziekte van het immuunsysteem en kanker kan leiden. Dit betekent dat we dus voorzichtig moeten zijn met het uitschakelen van het mutante eiwit als therapie voor ALS-patiënten." www.als-centrum.nl/kennisplatform/rol...

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