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January 31, 2005
Promising Drug Treatment
This sounds interesting...
Drug treatment promising for halting Huntington's-related nerve deathDALLAS – Jan. 31, 2005 – Researchers at UT Southwestern Medical Center have discovered that drugs commonly used to treat psychiatric illnesses and blood disorders in humans may protect the brain cells that die in people with Huntington's disease, possibly delaying the onset and slowing the progression of the disease.
These findings, available online and in today's issue of Proceedings of the National Academy of Sciences, may offer new treatment options for Huntington's disease, which has no cure.
Huntington's disease is a neurological disorder in which the medium spiny striatal neurons, the nerve cells that control movement and certain mental functions die. Patients die within 10-15 years after onset of the disease.The disease is caused by a mutation in the gene that makes the protein huntingtin. The mutation creates a long chain of the amino acid glutamine at one end of the protein. The length of the chain directly correlates with age of onset of the disease, with longer chains leading to symptoms earlier in life.
In previous studies, Dr. Ilya Bezprozvanny, associate professor of physiology at UT Southwestern, established that one of the defects that leads to death of nerve cells with the mutant huntingtin protein is improper regulation of calcium due to errant signals in the cells. Calcium is inappropriately released from its storage area in the cells, and eventually the cells die.
"We have developed a model that links the mutation in huntingtin with degeneration of motor neurons," Dr. Bezprozvanny said. "The model connects all the dots between the Huntington's disease mutation, defective calcium signaling in the cell, and subsequent degeneration of medium spiny striatal neurons."
In the current study, using the medium spiny neurons of mice that carry a copy of the mutated human huntingtin gene, Dr. Bezprozvanny and colleagues found that treatment of the cells in culture with the drug enoxaparin prevented inappropriate calcium release, and prevented cell death. Enoxaparin is an anti-coagulant that is FDA-approved in humans for use in treating blood clots.
Because the signals that lead cells to die can come from multiple pathways, Dr. Bezprozvanny then determined which cell death pathway affected the nerve cells carrying mutant huntingtin. He found that the nerve cells' mitochondria, the parts of the cell that create energy, released a protein called cytochrome c through a pore just before dying. From other studies, it was known the drugs nortriptyline and desipramine, which are antidepressants, and trifluoperazine, an antipsychotic, block the mitochondrial pore through which cytochrome c and other death signals are released. By treating the mouse nerve cells containing the mutant huntingtin protein with these drugs, Dr. Bezprozvanny was able to block the nerve cells from dying.
The next step, according to Dr. Bezprozvanny, will be to work with other researchers to test these drugs in whole animal models of Huntington's disease, and see if cell death and loss of motor function observed in these models can be prevented.
In addition, the researchers would like to expand their drug search beyond molecules that block calcium release and the mitochondrial pore. "We're looking for drugs that will prevent the pathological association of mutant huntingtin protein with the calcium signaling proteins in striatal neurons," he said. "We have a nice model system set up where we can easily look for cell death of Huntington's disease neurons, so we can look for the most specific drug with the least side effects."
###
Other UT Southwestern contributors to this study were Dr. Tie-Shan Tang, assistant instructor of physiology and Dr. Vitalie Lupu, postdoctoral researcher. In addition, Dr. Rodolfo Llinas of New York University School of Medicine, Dr. Bruce Kristal of Cornell University, and Dr. Michael Hayden of the University of British Columbia, who provided the mice used in the study, and members of their laboratories also contributed.The study was funded by the Robert A. Welch Foundation, the Huntington's Disease Society of America, the Hereditary Disease Foundation, and the National Institute of Neurological Disorders and Stroke.
Posted by Dave at 03:54 PM | Comments (0) | TrackBack
January 25, 2005
RNAi Trials May Start Next Year
Sirna Therapeutics has provided more news on their promising RNAi treatment for Huntington's Disease.
It looks like Phase 1 human trials may start next year! For those of you who have been following this blog...I believe this treatment has an excellent chance of being the effective/treatment for HD that we've been waiting for.
Here's the key quote from the press release:
Sirna's second program in local delivery is focused on Huntington's Disease, a life-threatening brain disorder accounting for more than $2 billion in patient costs annually in the U.S. Sirna recently entered into a collaboration with Targeted Genetics for the development of adeno-associated viral (AAV) vector delivery of siRNAs that target the expression of Huntington's protein, which in its mutant form causes the disease. Sirna is also collaborating with the Huntington's Disease Society of America and with Dr. Beverly Davidson at the University of Iowa, who has published promising data in this area. Sirna expects to file its Investigational New Drug (IND) application with the FDA in 2006 and to begin the first trial in HD using siRNAs shortly thereafter.And here's the full press release:
Sirna Therapeutics Establishes Broad Product Pipeline in RNAi for Systemic, Local and Topical Therapies
Company Expects One Phase 2 Program, Three Phase 1 Programs in 2006
Tuesday - January 25, 2005
BOULDER, Colo., Jan. 25 /PRNewswire-FirstCall/ -- Sirna Therapeutics, Inc. announced that the Company is advancing six major programs in its broadly diversified, clinical-stage product pipeline. Sirna's pipeline demonstrates the vast opportunity of short interfering RNAs (siRNAs) in systemic, local and topical delivery, enabling the Company to address serious unmet medical needs (Huntington's Disease), major disease markets (asthma and diabetes) and novel commercial programs (hair removal). Key programs are also focused on age-related macular degeneration (AMD), Sirna's first program to enter Phase I clinical testing, and oncology, through a collaboration with Eli Lilly.
Howard Robin, Sirna's President and Chief Executive Officer, stated, "The Sirna product pipeline addresses multi-billion dollar global markets across diverse disease targets, allowing us to reduce risk while at the same time attract multiple partners. Notably, we have entered into collaborations with Eli Lilly in oncology and Targeted Genetics in Huntington's Disease, and acquired Skinetics Biosciences to form our dermatology division. Each relationship has contributed to our development of what we believe is the most advanced and comprehensive pipeline in RNAi."
Mr. Robin continued, "Sirna is at a unique and pivotal point in its history. Having initiated our first clinical trial in AMD in November of 2004, we now have many near-term opportunities for positive news flow and value creation. In 2006, we expect to be advancing our lead AMD program into Phase 2 clinical testing and to have progressed three additional programs into Phase 1 clinical trials. Coupled with additional corporate partnerships that we expect to announce in 2005, we are in a great position to continue to communicate our value to the investor community and other key audiences."
Sirna's proprietary pipeline is rapidly advancing products for local, systemic and topical delivery:
Local Delivery (Age-Related Macular Degeneration, Huntington's Disease, Asthma)
Sirna's first program to advance into the clinic targets age-related macular degeneration, a degenerative eye disease that is the leading cause of blindness in the elderly in the United States. By 2010, over $1.5 billion is expected to be spent annually in the U.S. alone for therapies that treat AMD. In November 2004, Sirna initiated Phase 1 testing of Sirna-027, an siRNA targeting Vascular Endothelial Growth Factor Receptor-1 (VEGFR-1), which is a key component of the clinically validated vascular endothelial growth factor (VEGF) pathway. Sirna expects to announce results of the Phase 1 study by the end of 2005 and to initiate a Phase 2 clinical trial in 2006.
Sirna's second program in local delivery is focused on Huntington's Disease, a life-threatening brain disorder accounting for more than $2 billion in patient costs annually in the U.S. Sirna recently entered into a collaboration with Targeted Genetics for the development of adeno-associated viral (AAV) vector delivery of siRNAs that target the expression of Huntington's protein, which in its mutant form causes the disease. Sirna is also collaborating with the Huntington's Disease Society of America and with Dr. Beverly Davidson at the University of Iowa, who has published promising data in this area. Sirna expects to file its Investigational New Drug (IND) application with the FDA in 2006 and to begin the first trial in HD using siRNAs shortly thereafter.
Sirna is also developing a drug candidate for local delivery to treat asthma, representing a $13.3 billion market opportunity in the U.S. Sirna is working with Dr. Erwin Gelfand of the National Jewish Medical & Research Center to test an siRNA that targets Th2 cytokines, which play a critical role in inflammation and bronchconstriction in the airways. Early preclinical findings have shown a statistically significant reduction of airway hyperresponsiveness (66%) in an RNAi treatment group. An IND filing for this product is anticipated in 2006.
Systemic Delivery (Diabetes, Oncology)
Type II diabetes is a $6.3 billion market in the United States, and the market is growing. Sirna researchers have shown that administration of a systemically delivered siRNA resulted in a 72% reduction of PTP-1B (phosphatase 1B), a validated target in diabetes that is associated with insulin resistance. Other pioneering work at Sirna has demonstrated that up to 30% of administered siRNAs get into hepatocytes, liver cells that are the main target in treating diabetes. The Company plans to file its IND in diabetes in 2007.
In January 2004, Sirna initiated a collaboration with Eli Lilly and Company to apply Sirna's RNAi technology against Lilly's proprietary models in oncology. To date, the collaboration has demonstrated target mRNA and protein knockout in three tumor cell lines and an effect of siRNAs at the molecular level. Demonstration of in vivo target knockdown is currently in progress.
Topical Delivery (Dermatology)
In December 2004, Sirna launched its dermatology division following the acquisition of Skinetics Biosciences. Skinetics founder, Dr. Angela Christiano of Columbia University, is now an exclusive consultant to the Company. Sirna's first dermatology program is focused on the removal of unwanted hair, accounting for an estimated $4 billion dollar market in the U.S. (excluding shaving). The Company is in the preclinical stages of developing an siRNA targeting the "hairless" protein, so-called because people who have a nonfunctional version of this protein do not grow any hair. Sirna believes that infrequent treatments with an siRNA targeting the "hairless" protein could achieve results comparable to those seen in laser hair removal with less inconvenience and discomfort to the patient. An IND filing for the hair removal product is expected in 2006.
About Sirna Therapeutics
Sirna Therapeutics is a clinical-stage biotechnology company developing RNAi-based therapies for serious diseases and conditions, including age-related macular degeneration (AMD), Huntington's Disease, diabetes, asthma, oncology, and hair removal. Sirna has initiated a Phase 1 clinical trial for its most advanced compound, Sirna-027, a chemically modified siRNA targeting the clinically validated vascular endothelial growth factor pathway to treat AMD. The Company has strategic partnerships with Eli Lilly, Targeted Genetics and Archemix and a leading intellectual property portfolio in RNAi. More information on Sirna Therapeutics is available on the Company's web site at http://www.sirna.com/.
Statements in this press release which are not strictly historical are "forward-looking" statements which should be considered as subject to many risks and uncertainties. For example, there is a very significant risk that promising pre-clinical results cannot be reproduced in the clinic. Furthermore, Sirna's ability to develop products and to operate as a going concern are contingent upon having readily available cash to fund its operating programs and are subject to the escalating expenses and risks associated with the initiation of clinical trials and their potential outcomes. Other risks and uncertainties include Sirna's early stage of development and short operating history, whether Sirna can achieve and maintain profitability, whether Sirna can obtain and protect patents, the risk of third-party patent infringement claims, whether Sirna can engage collaborators and obtain regulatory approval for products, Sirna's concentration of stock ownership, and availability of materials for product manufacturing. These and additional risk factors are identified in Sirna's Securities and Exchange Commission filings, including the Forms 10-K and 10-Q and in other SEC filings. Sirna undertakes no obligation to revise or update any forward-looking statements in order to reflect events or circumstances that may arise after the date of this release.
CONTACT:
Martin E. Schmieg, Sr. Vice President & CFO of Sirna Therapeutics, Inc., +1.303.449.6500Web site:
http://www.sirna.com/Posted by Dave at 07:27 AM | Comments (0) | TrackBack
January 12, 2005
Targeted Genetics
Now that you've read the latest great news from Sirna Therapeutics, here is some on their agreement with Targeting Genetics. Key item from Targeted Genetics press release:
The focus of the collaboration will be the development of a therapeutic short interfering RNA (siRNA) targeting the gene that encodes the Huntington’s Disease protein. The siRNA will be expressed from an AAV-vector. RNAi is a mechanism used by cells to regulate the expression of genes and replication of viruses. The RNA interference mechanism uses siRNA to induce the destruction of target RNA using naturally occurring cellular protein machinery. Sirna’s scientific advisor and collaborator, Dr. Beverly Davidson at the University of Iowa, has published data demonstrating that the delivery of siRNA using an AAV vector efficiently inhibited gene expression in an animal model of spinocerebellar ataxia 1 (SCA1), a member of a class of inherited human neurodegenerative diseases that includes Huntington's Disease (Nature Medicine 10:816-820 [2004]).“There are no treatments for Huntington’s Disease, and we believe that vector expressed RNAi-based approaches may have significant potential in slowing or halting disease progression,” said Howard Robin, President and Chief Executive Officer at Sirna. “Dr. Davidson’s work using AAV vectors to deliver siRNA for the treatment of related neurodegenerative disorders is quite promising. Through the collaboration announced today we are gaining access to Targeted Genetics’ extensive capabilities in the development, clinical testing and manufacture of AAV-based products. We are excited to explore the potential of AAV-RNAi therapies for Huntington’s Disease, and are very pleased to be working with the leading AAV-based product development company.”
Targeted Genetics and Sirna Therapeutics Announce Huntington's Disease Collaboration
- Agreement Pairs Leaders in AAV-Based Gene Delivery and RNA Interference (RNAi) Technologies -
Date : Tuesday - January 11, 2005
SEATTLE, Jan. 11 /PRNewswire-FirstCall/ -- Targeted Genetics Corporation and Sirna Therapeutics, Inc. today announced that they have established a collaboration to develop novel therapies for the treatment of Huntington's Disease (HD), an incurable neurodegenerative disorder. The collaboration will combine Targeted Genetics' adeno-associated virus (AAV) delivery platform with Sirna's HD program and expertise in RNA silencing technologies."We are excited to continue expanding our AAV-based gene delivery platform into the area of RNA interference by working with Sirna, the leading company developing this innovative technology," said H. Stewart Parker, President and Chief Executive Officer of Targeted Genetics. "This collaboration is consistent with our strategy to leverage our gene delivery capabilities beyond gene replacement. Shutting down the production of disease-causing proteins by delivering expressed RNAi sequences complements the other applications of our technologies: providing a functional copy of the gene deficient in cystic fibrosis, delivering a gene encoding a therapeutic protein for rheumatoid arthritis and stimulating the immune system using a DNA-based vaccine against HIV."
The focus of the collaboration will be the development of a therapeutic short interfering RNA (siRNA) targeting the gene that encodes the Huntington's Disease protein. The siRNA will be expressed from an AAV-vector. RNAi is a mechanism used by cells to regulate the expression of genes and replication of viruses. The RNA interference mechanism uses siRNA to induce the destruction of target RNA using naturally occurring cellular protein machinery. Sirna's scientific advisor and collaborator, Dr. Beverly Davidson at the University of Iowa, has published data demonstrating that the delivery of siRNA using an AAV vector efficiently inhibited gene expression in an animal model of spinocerebellar ataxia 1 (SCA1), a member of a class of inherited human neurodegenerative diseases that includes Huntington's Disease (Nature Medicine 10:816-820 [2004]).
"There are no treatments for Huntington's Disease, and we believe that vector expressed RNAi-based approaches may have significant potential in slowing or halting disease progression," said Howard Robin, President and Chief Executive Officer at Sirna. "Dr. Davidson's work using AAV vectors to deliver siRNA for the treatment of related neurodegenerative disorders is quite promising. Through the collaboration announced today we are gaining access to Targeted Genetics' extensive capabilities in the development, clinical testing and manufacture of AAV-based products. We are excited to explore the potential of AAV-RNAi therapies for Huntington's Disease, and are very pleased to be working with the leading AAV-based product development company."
Under the terms of the agreement, development costs and revenue from partnering and sales of products developed under the collaboration will be shared between Sirna and Targeted Genetics.
About Huntington's Disease
Huntington's Disease (HD) is a devastating, degenerative brain disorder for which there is, at present, no effective treatment or cure. According to the National Institute of Neurological Disorders and Stroke, 30,000 people in the United States alone have HD, and at least another 150,000 are at risk for developing the disease. HD results from genetically programmed degeneration of neurons in certain areas of the brain. This degeneration causes uncontrolled movements, loss of intellectual faculties, and emotional disturbance.
HD typically begins in mid-life, between the ages of 30 and 45, though onset may occur as early as the age of 2. Children who develop the juvenile form of the disease rarely live to adulthood. Early symptoms of Huntington's Disease may affect cognitive ability or mobility and include depression, mood swings, forgetfulness, clumsiness, involuntary twitching and lack of co-ordination. As the disease progresses, concentration and short-term memory diminish and involuntary movements of the head, trunk and limbs increase. Walking, speaking and swallowing abilities deteriorate. Eventually the person is unable to care for him or herself. Death follows from complications such as choking, infection or heart failure.
About Targeted Genetics
Targeted Genetics Corporation develops gene-based products for preventing and treating acquired and inherited diseases. The Company has three clinical product development programs, targeting cystic fibrosis, AIDS prophylaxis and inflammatory arthritis. The Company also has a promising pipeline of product candidates focused on hemophilia and cancer, and a broad platform of gene delivery technologies for application in nucleic acid-based drug development. For more information about Targeted Genetics, visit its website at http://www.targetedgenetics.com/.
About Sirna Therapeutics
Sirna Therapeutics is a clinical-stage biotechnology company developing RNAi-based therapies for serious diseases and conditions, including age-related macular degeneration (AMD), hepatitis, oncology, and diseases of the central nervous system. Sirna has initiated a Phase 1 clinical trial for its most advanced compound, Sirna-027, a chemically modified siRNA targeting the clinically validated vascular endothelial growth factor pathway to treat AMD. The Company has strategic partnerships with Eli Lilly and Archemix and a leading intellectual property portfolio in RNAi. More information on Sirna Therapeutics is available on the Company's web site at http://www.sirna.com/ .
Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:
Targeted Genetics Corporation and Sirna Therapeutics, Inc. have included in this press release certain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 concerning their businesses, their collaboration, their development of an AAV-RNAi therapy for Huntington's Disease and their intellectual property, The words or phrases "can be," "expects," "may affect," "anticipates," "may depend," "believes," "estimates," "plans," "projects" and similar words and phrases are intended to identify such forward-looking statements. These forward-looking statements, involve current expectations, forecasts of future events and other statements that are not historical facts. Inaccurate assumptions and known and unknown risks and uncertainties can affect the accuracy of forward-looking statements. Actual results could differ materially from expectations for a number of reasons, including failure of a partner to provide funding or other resources for the collaboration, failure to obtain positive results from preclinical programs and clinical trials, failure to obtain or maintain regulatory approvals, failure to obtain, maintain or protect our intellectual property and the other risks described in the section entitled "Factors Affecting Our Operating Results, Our Business and Our Stock Price" in Targeted Genetics' Quarterly Report on Form 10-Q for the quarter ended September 30, 2004 and Sirna's Quarterly Report on Form 10-Q for the quarter ended September 30, 2004. You should not rely unduly on these forward-looking statements, which apply only as of the date of this release. We undertake no duty to publicly announce or report revisions to these statements as new information becomes available that may change our expectations.
Targeted Genetics Corporation
CONTACT: Stephanie Seiler, Ph.D. of Targeted Genetics Corporation,
+1-206-713-0124Web site: http://www.sirna.com/
Web site: http://www.targetedgenetics.com/
Posted by Dave at 06:26 AM | Comments (0) | TrackBack
SIRNA Takes A Big Step Forward For HD
Sirna Therapeutics has a potential treatment for Huntington's Disease that could end up being the effective treatment/cure we've been waiting for. They're doing everything we can hope for...
First they signed an agreement with the University of Iowa to collaborate with them on developing an HD RNAi treatment. As you might recall, they've had huge success on this.
Next they announce they are collaborating with HDSA and their "clinical investigators" (I assume they refer to the Centers of Excellence.)
Now Sirna has signed an agreement with Targeted Genetics Corporation to use their adeno-associated virus (AAV) delivery platform to deliver the RNAi treatment to the brain cells.
So in less than a year...Sirna has announced successes in RNAI, licenced the most promising treatment, made an agreement with the organization that has the resources to help do the clinical testing, is going to use the most promising delivery vehicle, and...they've already started human trials of an RNAi therapy for Macular Degeneration.
All indications are that Sirna is working aggressively to start human testing as soon as possible. Here's their press release on the latest agreement:
Sirna Therapeutics and Targeted Genetics Form Huntington's Disease Collaboration
- Agreement Pairs Leaders in RNA Interference (RNAi) and AAV-Based Delivery TechnologiesBOULDER, Colo. and SEATTLE, Jan. 11 /PRNewswire-FirstCall/ -- Sirna Therapeutics, Inc. (Nasdaq: RNAI) and Targeted Genetics Corporation (Nasdaq: TGEN) today announced that they have established a collaboration to develop a novel therapy for the treatment of Huntington's Disease (HD), an incurable neurodegenerative disorder. The collaboration significantly advances Sirna's HD program by using Targeted Genetics' adeno-associated virus (AAV) delivery platform to develop an HD product.
The focus of the collaboration will be the development of a therapeutic short interfering RNA (siRNA) targeting the gene that encodes the Huntington's Disease protein. The siRNA will be expressed from an AAV-vector. RNAi is a mechanism used by cells to regulate the expression of genes and replication of viruses. The RNA interference mechanism uses siRNA to induce the destruction of target RNA using naturally occurring cellular protein machinery. Sirna's scientific advisor and collaborator, Dr. Beverly Davidson at the University of Iowa, has published data demonstrating that the delivery of siRNA using an AAV vector efficiently inhibited gene expression in an animal model of spinocerebellar ataxia 1 (SCA1), a member of a class of inherited human neurodegenerative diseases that includes Huntington's Disease (Nature Medicine 10:816-820 [2004]).
"There are no treatments for Huntington's Disease, and we believe that vector expressed RNAi-based approaches may have significant potential in slowing or halting disease progression," said Howard Robin, President and Chief Executive Officer at Sirna. "Dr. Davidson's work using AAV vectors to deliver siRNA for the treatment of related neurodegenerative disorders is quite promising. Through the collaboration announced today we are gaining access to Targeted Genetics' extensive capabilities in the development, clinical testing and manufacture of AAV-based products. We are excited to explore the potential of AAV-RNAi therapies for Huntington's Disease, and are very pleased to be working with the leading AAV-based product development company."
"We are excited to continue expanding our AAV-based gene delivery platform into the area of RNA interference by working with Sirna, the leading company developing this innovative technology," said H. Stewart Parker, President and Chief Executive Officer of Targeted Genetics. "This collaboration is consistent with our strategy to leverage our gene delivery capabilities beyond gene replacement. Shutting down the production of disease-causing proteins by delivering expressed RNAi sequences complements the other applications of our technologies: providing a functional copy of the gene deficient in cystic fibrosis, delivering a gene encoding a therapeutic protein for rheumatoid arthritis and stimulating the immune system using a DNA-based vaccine against HIV."
Under the terms of the agreement, development costs and revenue from partnering and sales of products developed under the collaboration will be shared between Sirna and Targeted Genetics.
About Huntington's Disease
Huntington's Disease (HD) is a devastating, degenerative brain disorder for which there is, at present, no effective treatment or cure. According to the National Institute of Neurological Disorders and Stroke, 30,000 people in the United States alone have HD, and at least another 150,000 are at risk for developing the disease. HD results from genetically programmed degeneration of neurons in certain areas of the brain. This degeneration causes uncontrolled movements, loss of intellectual faculties, and emotional disturbance.
HD typically begins in mid-life, between the ages of 30 and 45, though onset may occur as early as the age of 2. Children who develop the juvenile form of the disease rarely live to adulthood. Early symptoms of Huntington's Disease may affect cognitive ability or mobility and include depression, mood swings, forgetfulness, clumsiness, involuntary twitching and lack of co-ordination. As the disease progresses, concentration and short-term memory diminish and involuntary movements of the head, trunk and limbs increase. Walking, speaking and swallowing abilities deteriorate. Eventually the person is unable to care for him or herself. Death follows from complications such as choking, infection or heart failure.
About Sirna Therapeutics
Sirna Therapeutics is a clinical-stage biotechnology company developing RNAi-based therapies for serious diseases and conditions, including age-related macular degeneration (AMD), hepatitis, oncology, and diseases of the central nervous system. Sirna has initiated a Phase 1 clinical trial for its most advanced compound, Sirna-027, a chemically modified siRNA targeting the clinically validated vascular endothelial growth factor pathway to treat AMD. The Company has strategic partnerships with Eli Lilly and Archemix and a leading intellectual property portfolio in RNAi. More information on Sirna Therapeutics is available on the Company's web site at http://www.sirna.com.
Contact:
Martin E. Schmieg, Sr. Vice President & CFO of Sirna Therapeutics, Inc.,
+1.303.449.6500About Targeted Genetics
Targeted Genetics Corporation develops gene-based products for preventing and treating acquired and inherited diseases. The Company has three clinical product development programs, targeting cystic fibrosis, AIDS prophylaxis and inflammatory arthritis. The Company also has a promising pipeline of product candidates focused on hemophilia and cancer, and a broad platform of gene delivery technologies for application in nucleic acid-based drug development. For more information about Targeted Genetics, visit its website at www.targetedgenetics.com.Contact:
Stephanie Seiler, Ph.D.
206-713-0124Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:
Targeted Genetics Corporation and Sirna Therapeutics, Inc. have included in this press release certain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 concerning their businesses, their collaboration, their development of an AAV-RNAi therapy for Huntington's Disease and their intellectual property. The words or phrases "can be," "expects," "may affect," "anticipates," "may depend," "believes," "estimates," "plans," "projects" and similar words and phrases are intended to identify such forward-looking statements. These forward-looking statements involve current expectations, forecasts of future events and other statements that are not historical facts. Inaccurate assumptions and known and unknown risks and uncertainties can affect the accuracy of forward-looking statements. Actual results could differ materially from expectations for a number of reasons, including failure of a partner to provide funding or other resources for the collaboration, failure to obtain positive results from preclinical programs and clinical trials, failure to obtain or maintain regulatory approvals, failure to obtain, maintain or protect our intellectual property and the other risks described in the section entitled "Factors Affecting Our Operating Results, Our Business and Our Stock Price" in Targeted Genetics' Quarterly Report on Form 10-Q for the quarter ended September 30, 2004 and Sirna's Quarterly Report on Form 10-Q for the quarter ended September 30, 2004. You should not rely unduly on these forward-looking statements, which apply only as of the date of this release. We undertake no duty to publicly announce or report revisions to these statements as new information becomes available that may change our expectations.
Posted by Dave at 05:57 AM | Comments (1) | TrackBack
January 11, 2005
New HD Blog
Wow, what a way to start a new HD website...
and this isn't the only HD Blog anymore!
Gene Veritas has started a blog for those living at risk for Huntington's Disease and I highly recommend you go visit it (and don't forget to bookmark it). Here's a sample:
No, I am not angry, I told him. It’s been nine years this December 26 that I learned of my mother’s diagnosis with HD. I have come to accept HD as part of my life.I think a lot about death, I continued. I don’t know exactly when HD will strike. It could be as early as in the next five years, or it could take twenty years. I’m trying to squeeze as much life into my days as possible before I starting living life as a “vegetable,” I said.
“I envy you,” my friend said. “I feel immortal. I don’t believe I’m going to die. But you know you’re going to die, and so you can live your life more fully.”
You can read all of it at http://www.curehd.blogspot.com/
Posted by Dave at 06:26 AM | Comments (0) | TrackBack
January 10, 2005
HD Takes Another
It won't be recorded as a death due to Huntington's Disease, but it was...
He started his walk wearing a fleece and cycling shorts and had no food, map or compass, just a small amount of cash and a bottle of energy drink...Two fell runners and a resident urged him to turn back as the weather was terrible, but it is believed he ignored the warnings.
Inspector Mihajlo Milinkovic said: "The weather was horrendous on Tuesday. He had inappropriate clothing for those conditions and he has physical impairments."Posted by Dave at 06:34 AM | Comments (0) | TrackBack
January 09, 2005
Donations
Money is being raised at a furious pace for the victims of the Tsunami. It's a major disaster and the money is desperately needed.
Unfortunately, this will impact fundraising for Huntington's Disease. I'll be writing on this over the coming year.
Posted by Dave at 11:48 PM | Comments (0) | TrackBack
January 07, 2005
SIRNA Branching Out
SIRNA is working one of the most promising treatments for Huntington's Disease utilizing RNAi.
The technology must be promising, they are branching out and are now working to apply RNAi to the treatment of Asthma. You can read the press release here.
Posted by Dave at 05:31 AM | Comments (0) | TrackBack
Stem Cell Therapeutics
Stem Cell Therapeutics Corp. just raised $8.5 million to help develop a product that assists the brain in generating its own stem cells.
I hope they succeed, but they are going to need a lot more funding - research is expensive. Their press release:
Stem Cell Therapeutics announces closing of initial public offering
/THIS RELEASE IS INTENDED FOR DISTRIBUTION IN CANADA ONLY AND IS NOT TO
BE DISTRIBUTED TO UNITED STATES NEWSWIRE SERVICES OR DISSEMINATED IN THE
UNITED STATES/CALGARY, Jan. 6 /CNW/ - Stem Cell Therapeutics Corp. ("Stem Cell") is
pleased to announce that it has successfully completed its initial public
offering consisting of the maximum offering of 34,000,000 common shares at a
price of $0.25 per share, for gross proceeds of $8,500,000. Subsequent to the
closing, Stem Cell has 52,986,364 (63,711,364 on a diluted basis) common
shares outstanding. Dlouhy Merchant Group Inc. and First Associates
Investments Inc. co-led the financing.
Stem Cell received cash proceeds (net of agents' commissions and
expenses) of $7.64 million from the offering and intends to use these funds
for the research and development of new products, general and administrative
expenses including working capital, and possible acquisitions of additional
technology.About Stem Cell Therapeutics Corp.: Calgary-based Stem Cell Therapeutics
Corp. is a biotechnology company focused on the development of our technology
platform and intellectual property to selectively induce a patient's own stem
cells to proliferate in the brain. Our core technology, which includes our
lead therapeutic product NTx(TM)-265, has been demonstrated to increase the
number of innate adult stem cells that grow in place when our therapeutic
approach is applied to test animals. This fundamental technology will be
further developed to create specific disease treatments for stroke,
Huntington's disease, Alzheimer's disease and other neurodegenerative
conditions.
Stem Cell's shares will be listed on the TSX Venture Exchange, under the
symbol "SSS".The TSX Venture Exchange does not accept responsibility for the adequacy
or accuracy of this release.These securities have not been registered under the United States
Securities Act of 1933, as amended, or the securities laws of any state, and
may not be offered or sold within the United States or to, or for the account
or benefit of U.S. persons unless an applicable exemption from U.S.
registration requirements is available.Except for historical information, this press release may contain
forward-looking statements, which reflect the Company's current expectation
regarding future events. These forward-looking statements involve risk and
uncertainties, which may cause but are not limited to, changing market
conditions, the successful and timely completion of clinical studies, the
establishment of corporate alliances, the impact of competitive products and
pricing, new product development, uncertainties related to the regulatory
approval process and other risks detailed from time to time in the Company's
ongoing quarterly and annual reporting.For further information: on Stem Cell Therapeutics Corp., visit
www.stemcellthera.com; or contact Dr. Joseph Tucker, Chief Executive Officer,
Stem Cell Therapeutics Corp., Phone : (403) 245-5495,
info@stemcellthera.comPosted by Dave at 05:23 AM | Comments (0) | TrackBack
January 06, 2005
Thanks To HDDW Participants
I should have posted this sooner. Here's a thank you from Dr. LaVonne Goodman:
A PUBLIC SALUTE TO HDDW TRIAL PARTICIPANTS
As of January 1, 2005, Huntington's Disease Drug Works has thirty-five hardworking participants who are working with us in HDDW individual therapeutic trials, using a cocktail of safe agents. We salute these trailblazers as the real leaders in HDDW's patient-centered clinical trials. We also thank designated helpers, family members and local doctors; all of whom have provided support this past year.
More formal results of the first 6 months of data will be available by the end of January at www.HDdrugworks.org. We plan to update in similar many every 6 months as the trials progress. But I will share some early results with you now.There are now more than 20 participants with four (or more) months of data.
Twelve have reached the six month mark. The majority of participants show steady improvement in most of the testing parameters that are measured in the web-interactive program. This includes both motor and the cognitive portions of the program. This steady gain has continued for first participants up to the 6 month time period of study.Magnitude of improvement varies between individuals. The magnitude is lesser for individuals with late stage disease, but is still measurable. Any given individual may not improve on all tests, but in these cases, each individual testing pattern is consistent over time.
We realize that the patient numbers are small; and that the time interval is short. But we believe these pilot results are give ample reason to proceed and hope for the best in 2005.
LaVonne Veatch Goodman
Posted by Dave at 07:19 AM | Comments (0) | TrackBack
January 05, 2005
Discrimination Survey
If you have the HD gene or are at risk be sure to visit this page on the HDSA website.
HDSA is attempting to measure the impact of genetic discrimination on those in the HD community. As you take the test, be sure to keep the following in mind (from the instructions):
For the purpose of this study, we define genetic discrimination as any denial of rights, privileges, and/or opportunities based on one's real or perceived chance of developing Huntington's Disease.Spread the word.
Posted by Dave at 06:35 PM | Comments (0) | TrackBack
January 04, 2005
"...with acceptance came self love"
I hope you make the Huntington's Disease Advocacy Center (www.hdac.org) a regular part of your journey on the web.
Marsha Miller points to another wonderful article, this one by Kelly B. and you can read it here. A excerpt:
For the first year after testing I felt that having HD marked the end of my life. The urge to just curl up and wait for death was tempting, for awhile. Luckily the urge to put to good use the limited days I had left, took over.One day it just clicked, I believe it was the day that my focus shifted from what I had lost, to what I had left. And I had a whole lot left. Recognizing my "blessings" was the first step to turning things around inside my head. It is hard for the dark thoughts to hang out when you are focusing on positive things.
At the camp I attended last year, Dr. Jess told us that through loss is gain, that the two are entertwined. The loss is the cloud itself and the gain is the silverlinings that exist in all the challenges that life brings us.
Surviving losses can give us inner strength, understanding, compassion, a zest/appreciation for life, and personal growth. Show me a person who has never suffered a loss, and I will show you a person who has not evolved at all. Survivors are a special breed of person...
Finding out why I was the way I was and being diagnosed with HD inspired many brand new beginings for me, after the shock phase had passed and the dust settled. With knowledge came understanding, with understanding came acceptance, and with acceptance came self love.
Posted by Dave at 06:28 AM | Comments (1) | TrackBack
January 03, 2005
Yoga & HD Again In The News
Good news for Laura Jean and her DNA Wellness Studios. They are the recipient of another nice news article.
In case you don't remember or are new to this blog, Jean started taking yoga to combat the effects of Huntington's Disease. It's gone so well that she opened her own yoga studio. From the article:
She talked about the mind body connection, with regard to her running regimen. "I need it for the mental health" aspects, she said. She talked about Jean's work with a population of clients with medical conditions ranging from multiple sclerosis and cancer to brain injuries. "Through yoga," Dagle said, "they've been able to overcome a lot of obstacles."Posted by Dave at 11:14 PM | Comments (0) | TrackBack
January 02, 2005
Gene Article
As I read this excellent article on gene testing one thought came to mind...
"What about genetic discrimination?"
We need a white knight in congress who will push for a genetic anti-discrimination bill. Doing it sooner, rather than later, will save lives.
Posted by Dave at 05:30 AM | Comments (0) | TrackBack
January 01, 2005
Happy New Year!
We've survived another year!
2004 was a big year for Huntington's Disease research and 2005 looks even better. We may see two drugs approved this year for the treatment of Huntingon's Disease - no magic cures just yet ones that may help those with the disease.
Keep the faith!
Posted by Dave at 11:34 PM | Comments (0) | TrackBack