[paula_w]

Sangamo Receives Michael J. Fox Foundation Funding to Develop Novel ZFP Therapeutic for the Treatment of Parkinson's Disease


23.01.2007 19:28
http://www.finanznachrichten.de/nach...el-7628476.asp



RICHMOND, Calif., Jan. 23 /PRNewswire/ -- Sangamo BioSciences, (Nachrichten) Inc. announced today that it has been awarded funding by The Michael J. Fox Foundation for Parkinson's Research (MJFF) to support the development of a ZFP Therapeutic(TM) to treat Parkinson's disease (PD). The $950,000 award will be paid over a period of two years.

Sangamo will develop zinc-finger DNA-binding protein transcription factors (ZFP TFs(TM)) to activate the expression of glial cell line-derived neurotrophic factor (GDNF), a potent neurotrophic factor that has shown promise in preclinical testing to slow or stop the progression of Parkinson's disease.

"We are enthusiastic about Sangamo's novel approach to GDNF as it fits squarely within our desire to promote alternative therapeutic strategies for this and other promising molecules," said Brian Fiske, PhD, associate director of research programs for MJFF. "The work is of particular interest given that Sangamo already has similar technology in clinical trials for diabetic neuropathy and peripheral artery disease and, with a positive result, could proceed rapidly into clinical trials for PD."

Sangamo's technology platform permits the development of highly specific ZFP TFs that can be used to regulate or modify genes at the DNA level. Using a ZFP TF, a gene can be activated in a patient's own cells in its natural cellular context. Use of a ZFP TF to activate the GDNF gene in the brain -- as compared to addition of a GDNF cDNA or the recombinant protein -- may allow the delivery of a more physiologically relevant dose of the growth factor. This may be of particular significance when targeting potent natural growth factors such as GDNF, where sufficient but not super-physiological levels of the therapeutic protein are required to achieve potency with safety.

"We are very pleased that The Michael J. Fox Foundation is recognizing and supporting the potential of Sangamo's unique technology with this award," said Edward Lanphier, Sangamo's President and CEO. "We believe that our approach to therapeutic gene regulation, which mimics the natural regulation of the gene in the body, has advantages over other approaches that add GDNF as either the gene or the recombinant protein. Success in this project would establish the validity of the use of ZFP TFs in the brain and set the stage for the application of this powerful technology to other genes that may have a therapeutic benefit in PD."

About Sangamo BioSciences, Inc.

Sangamo BioSciences, Inc. is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development program is currently in a Phase 2 clinical trial for evaluation of safety in patients with diabetic neuropathy. Phase 1 clinical trials are ongoing to evaluate a ZFP Therapeutic for peripheral artery disease. Other therapeutic development programs are focused on ischemic heart disease, neuropathic pain, cancer and infectious and monogenic diseases. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP TF(TM)) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFN(TM)) for therapeutic gene modification as a treatment for a variety of monogenic diseases, such as X-linked SCID and hemophilia, and for infectious diseases, such as HIV. Sangamo has established several Enabling Technology Agreements with companies to apply its ZFP Technology to enhance the production of protein pharmaceuticals. For more information about Sangamo, visit the company's web site at http://www.sangamo.com/ .

This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references to the research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. See the company's SEC filings, and in particular, the risk factors described in the company's Annual Report on Form 10-K and its most recent 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.

Paula

[jeanb]

Paula

Thank you for posting this! Most excellent news indeed!!

[ZucchiniFlower]

Great news, Paula! Thanks for posting. More from their web site:

http://www.sangamo.com/human/human_thera_overview.html

[paula_w]

Does this mean we have a watchdog? Sure hope so.


http://www.prnewswire.com/cgi-bin/st...04511375&EDATE=

Foundation Awards $4.6 Million to 10 Industry Teams for Parkinson's Drug Development Research



Paula

[GregD]

Paula,
Thanks for posting this information. I think it's the first I've heard about it. I'm off to learn more about ZFP TFs.

GregD

[paula_w]

http://www.bctechnology.com/scripts/...s.cfm?id=24001
Michael J. Fox Foundation Funds Allon to Evaluate AL-108 for Parkinson’s Disease

http://www.allontherapeutics.com

Vancouver, BC, January 24, 2007--(T-Net)--Allon Therapeutics (TSX:NPC), The Neuro Protection Company, today announced it has received funding from The Michael J. Fox Foundation for Parkinson’s Research to evaluate the effectiveness of Allon’s lead drug AL-108 in pre-clinical models of Parkinson’s disease.

Gordon McCauley, President and CEO of Allon, said the pre-clinical studies will be started immediately and, if successful, Allon would be in a position to begin a Phase II clinical trial to evaluate the drug’s effectiveness in Parkinson’s patients as early as 2008. The Company has specifically designed their Phase I human clinical trials to provide safety coverage that can be applied across a broad range of neurodegenerative diseases.

“The Michael J. Fox Foundation has driven groundbreaking work in Parkinson’s and we look forward to using MJFF funding to extend development of our drug platforms,” said McCauley. “This award provides Allon with an opportunity to expand its work into another major market where there is a large unmet medical need.”

“The Foundation is pleased to provide funding to help determine whether AL-108’s neuroprotection is applicable to Parkinson’s. The work is of particular interest given that, with a positive result, Allon could proceed directly into a Phase II efficacy trial,” said Brian Fiske, PhD, associate director of research programs for MJFF.

Parkinson's disease is the direct result of the deterioration of cells, called dopaminergic neurons, which produce dopamine, a chemical messenger responsible for transmitting signals within the brain. Loss of dopamine causes critical nerve neurons to fire out of control, leaving patients unable to direct or control their movement in a normal manner. The studies funded by the Michael J. Fox Foundation will allow Allon to test whether AL-108 can protect against and/or repair the deterioration of dopaminergic neurons in two animal models of Parkinson’s disease.

An estimated 1.5 million people in North America suffer from Parkinson’s disease, a progressive neurodegenerative disease, and its incidence is expected to increase significantly over the next 25 years as the population ages. There is currently no cure for Parkinson's disease, and although drugs have been developed that can help patients manage many of the symptoms, these drugs do not stop the disease from progressing. Current therapies also have significant long-term side effects and often stop working. According to Datamonitor, sales of Parkinson’s disease drugs in the seven major pharmaceutical markets was approximately $1.8 billion in 2005.

Allon’s drugs are unique in that in preclinical animal studies they prevent and treat the causes of neurodegeneration, including Alzheimer’s disease and stroke. AL-108 has been shown in numerous preclinical animal studies to prevent deterioration of neurons through interaction with tubulin, the protein that forms microtubules. Microtubules are key parts of the communication pathways inside neurons. Assembly of microtubules is essential for the ongoing regeneration of the nervous system and for combating neurodegenerative diseases.

The Company has two products in Phase II human clinical trials. AL-108 is being evaluated in a Phase II trial as a treatment for Mild Cognitive Impairment-Alzheimer’s disease and Allon recently announced a third Phase II study looking at cognitive impairment in schizophrenia in collaboration with TURNS (Treatment Units for Research on Neurocognition and Schizophrenia) funded by the United States National Institute of Mental Health. The intravenous product, AL-208 is being evaluated in a Phase II trial to prevent and treat the mild cognitive impairment (MCI) that commonly occurs after coronary artery bypass graft (CABG) surgery.

About Allon

Allon Therapeutics Inc. is a Canadian biotechnology company developing drugs that protect against neurodegenerative conditions such as Alzheimer’s disease, mild cognitive impairment, stroke, traumatic brain injury, multiple sclerosis and neuropathy. The Company is listed on the Toronto Stock Exchange under the trading symbol "NPC" (Neuro Protection Company) and based in Vancouver. For additional information visit www.allontherapeutics.com.

About the Michael J. Fox Foundation for Parkinson’s Research

Founded in 2000, The Michael J. Fox Foundation for Parkinson’s Research is dedicated to ensuring the development of a cure for Parkinson’s disease within this decade through an aggressively funded research agenda. The Foundation has funded over $90 million in research to date, either directly or through partnerships.

Forward Looking Statements

There are forward-looking statements contained herein that are not based on historical fact, including without limitation statements containing the words “believes,” “may,” “plans,” “will,” “estimate,” “continue,” “anticipates,” “intends,” “expects,” and similar expressions. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause the actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include, among others, Allon’s stage of development, lack of product revenues, additional capital requirements, risks associated with the completion of clinical trials and obtaining regulatory approval to market Allon’s products, the ability to protect its intellectual property and dependence on collaborative partners. These factors should be considered carefully and readers are cautioned not to place undue reliance on such forward-looking statements. The Company disclaims any obligation to update any such factors or to publicly announce the result of any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments.

For further information:

Investor and Media contact:

Aaron Keay
Manager, Investor Relations
Allon Therapeutics Inc.
P: (604) 742 2540
C: (604) 323 6911
E: akeay@allontherapeutics.com

[aftermathman]

for stem cell work. What makes them interesting is that "ReNeuron has recently filed for approval to commence initial clinical studies in the US with its lead ReN001 stem cell therapy for stroke", so a reasonably progressed approach (he says hopefully).

http://www.oxfordshirebioscience.com...QHEQ5O5HAA4972

Guildford, UK, 24 January 2007: ReNeuron Group plc (LSE: RENE) today announces that it has been awarded a grant from the US-based Michael J. Fox Foundation for Parkinson's Research (MJFF) to develop its ReN004 stem cell therapy for Parkinson's disease. The award was made under MJFF's Therapeutics Development Initiative, designed to catalyse and expand industry investment in pre-clinical Parkinson's drug development.

ReNeuron has previously presented data showing that its ReN004 candidate stem cell lines have the potential to reverse the neurological deficits in pre-clinical models of Parkinson's disease. The data indicated good in vivo survival of the cell lines and expression of the appropriate markers for dopaminergic neurons, the cell type deficient in Parkinson's patients. The MJFF grant will allow ReNeuron to build on this pre-clinical data and also develop a biodegradable delivery matrix which the Company believes will be required to successfully implant the required cell type into Parkinson's patients. The grant will fund the ReN004 programme over the next year, accelerating its progress towards the clinic.

In addition to its Parkinson's disease programme, ReNeuron has recently filed for approval to commence initial clinical studies in the US with its lead ReN001 stem cell therapy for stroke. The Company is also developing stem cell therapies for Huntington's disease, Type 1 diabetes and diseases of the retina.

Michael Hunt, Chief Executive Officer of ReNeuron, said:

"We are delighted to have secured this important first grant for our ReN004 therapy from such a pre-eminent and high-profile US grant-awarding body supporting research into Parkinson's disease. Significantly, we regard this award as an important independent validation of the therapeutic approach we are adopting in seeking to address this debilitating disease. Our ReN004 Parkinson's disease programme applies ReNeuron's platform c-mycERTAM cell expansion technology to a further significant area of unmet medical need."

Aftermathman.
(Car due back Friday, mood improving up till then).

[Thelma]

GDNF applied to the MPTP-lesioned nigrostriatal system requires TGF-β for its neuroprotective action

Andreas Schobera, c, , , Heike Peterziela, Christopher S. von Bartheldb, Horst Simona, Kerstin Krieglsteinc and Klaus Unsickera

aIZN, Department of Neuroanatomy, University of Heidelberg, Im Neuenheimer Feld 307, D-69120 Heidelberg, Germany
bDepartment of Physiology and Cell Biology, University of Nevada School of Medicine, Mailstop 352, Reno, NV 89557, USA
cCMPB, Department of Neuroanatomy, University of Göttingen, Kreuzbergring 36, D-37075 Göttingen, Germany

Received 14 June 2006; revised 22 September 2006; accepted 9 October 2006. Available online 1 December 2006.




Abstract
GDNF is a potent neurotrophic factor for nigrostriatal dopaminergic neurons in vitro and in animal models of Parkinson's disease (PD), but has largely failed when tested in therapeutic applications in human PD. We report here that GDNF requires transforming growth factor-β (TGF-β) to elicit its neurotrophic activity. Lesioning the mouse nigrostriatal system with MPTP significantly upregulates striatal TGF-β2 mRNA levels. As expected, GDNF protects against the destructive effects of MPTP, including losses of TH-ir nigral neurons, striatal dopamine and TH-ir fibers. Application of antibodies neutralizing all three TGF-β isoforms to the MPTP-lesioned striatum abolishes the neurotrophic effect of GDNF. We show that TGF-β antibodies are not toxic and do not interfere with retrograde transport of iodinated GDNF, suggesting that TGF-β antibodies do not impair internalization and retrograde trafficking of GDNF. We conclude that striatal TGF-β may be essential for permitting exogenous GDNF to act as a neuroprotective factor.

Keywords: Parkinson's disease; Neurotrophic factors; Dopaminergic neurons; Cell death; Engrailed-1; Retrograde transport

Abbreviations: CSF, cerebrospinal fluid; DA, dopamine; En, engrailed; GDNF, glial cell line-derived neurotrophic factor; GFRα1, GDNF co-receptor alpha1; HPLC-ED, high performance liquid chromatography with electrochemical detection; MLEC, mink lung epithelial cell; MPTP, 1,2,3,6-tetrahydro-1-methyl-4-phenylpyridine hydrochloride; PD, Parkinson's disease; SN, substantia nigra; SNpc, substantia nigra, pars compacta; SN–VTA, substantia nigra–ventral tegmental area; TGF-β, transforming growth factor-beta; TH, tyrosine hydroxylase



Corresponding author. Department of Neuroanatomy and IZN, University of Heidelberg, Im Neuenheimer Feld 307, D-69120 Heidelberg, Germany. Fax: +49 6221 54 5604.

[paula_w]

That's very interesting! I hadn't heard it before and will look for more on it. Thanks Thelma.

Paula

[burckle]

Yes, I agree with you Paula. This is very hopeful news.

All the best,

Lloyd