Huntington's Disease News and Research

Latest Huntington's Disease News and Research

Regulus Therapeutics, sanofi-aventis partner to develop and commercialize microRNA therapeutics

Regulus Therapeutics Inc. and sanofi-aventis announced today that they have entered into a global, strategic alliance to discover, develop, and commercialize microRNA therapeutics. The alliance represents the largest microRNA partnership formed to date, valued at potentially over $750 million, and includes a $25 million upfront fee, a $10 million future equity investment subject to mutual agreement on company valuation, and annual research support for three years with the option to extend two additional years.

22 Jun 2010

USPTO awards Alnylam Notice of Allowance for patent covering methods of inhibiting expression of mutant genes

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office (USPTO) has awarded a Notice of Allowance for the "John et al." patent (Application No. 10/384,463), which covers methods of inhibiting mutant genes with double-stranded RNAs from 19 to 30 nucleotides in length.

18 Jun 2010

Many Huntington's disease patients suffer from weight loss, sleeping problems

An aggregating protein causes many of the symptoms of Huntington's disease, an incurable and frequently fatal brain disorder. Mosaic winner Ahmad Aziz discovered that the abnormal protein also aggregates in the hypothalamus, the part of the brain that regulates the autonomic nervous system and the endocrine system.

16 Jun 2010

ISB, GIND collaborate on whole-genome sequencing to identify drug targets for Huntington's Disease

The Institute for Systems Biology (ISB) of Seattle, WA, is collaborating with the Gladstone Institute of Neurological Disease (GIND) and its Taube-Koret Center for Huntington's Disease Research to use whole-genome sequencing to identify genes and novel drug targets related to the onset and progression of Huntington's disease (HD). The research team, led by GIND associate director and senior investigator Steven Finkbeiner, MD, PhD, will also use induced pluripotent stem (iPS) cells from patients with HD to screen for drugs that might delay, prevent, or even reverse this devastating condition.

16 Jun 2010

Peter and Patricia Gruber Foundation to present 2010 Neuroscience Prize to Robert H. Wurtz

Robert H. Wurtz, PhD, a pioneer and leader in the field of neurophysiology, is the recipient of the 2010 Neuroscience Prize of The Peter and Patricia Gruber Foundation. His discoveries of how the brain processes visual information and controls eye movements laid the groundwork for subsequent research into the neurophysiology of visual cognition. This research has led scientists to a deeper understanding of how the brain is organized to produce behavior.

15 Jun 2010

EMA COMP grants orphan medicinal product designation for Raptor's DR Cysteamine for cystinosis

Raptor Pharmaceutical Corp., announced that the European Medicines Agency's ("EMA") Committee for Orphan Medicinal Products ("COMP") has issued a positive opinion recommending orphan medicinal product designation for DR Cysteamine for the potential treatment of cystinosis.

15 Jun 2010

Repligen's fiscal 2010 total revenue decreases to $20,971,000

Repligen Corporation today reported results for the fourth quarter and fiscal year 2010, ended March 31, 2010. Total revenue for the year was $20,971,000 compared to total revenue of $29,362,000 for fiscal year 2009 ended March 31, 2009.

9 Jun 2010

Alnylam presents interim data from ALN-VSP02 Phase I trial for liver cancer at ASCO 2010

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today preliminary clinical data from its ongoing Phase I trial with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of advanced solid tumors with liver involvement.

7 Jun 2010

Anti-seizure drug can be used to treat AT deficiency: Pitt researchers

The liver scarring of α1-antitrypsin deficiency, the most common genetic cause for which children undergo liver transplantation, might be reversed or prevented with a medication that has long been used to treat seizures, according to findings from Children's Hospital of Pittsburgh of UPMC and the University of Pittsburgh School of Medicine that will published in Science and are available online today through the Science Express website.

4 Jun 2010

Raptor Pharmaceutical acquires exclusive license to intellectual property for Huntington's Disease treatment

Raptor Pharmaceutical Corp., announced that the Company has acquired an exclusive worldwide license to intellectual property related to the potential treatment of Huntington's Disease from the Weizmann Institute of Science in Israel and Niigata University in Japan. In addition, Raptor has added Professor Lawrence Steinman, M.D., an inventor on the Weizmann patent, to its Advisory Board.

2 Jun 2010

Alnylam, Mount Sinai scientists discover svRNAs that play a critical role in influenza A virus replication

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the publication of new research findings in the journal Proceedings of the National Academy of Sciences (PNAS) by Alnylam scientists and collaborators from Mount Sinai School of Medicine. In the new study, Alnylam and Mount Sinai scientists discovered a novel class of virus-produced small non-coding RNAs, called small viral RNAs (svRNAs), which play a critical role in the replication of influenza A virus.

2 Jun 2010

Neuralstem updates progress of ongoing Phase I human clinical trial for ALS

Neuralstem, Inc. updated the progress of its ongoing Phase I human clinical trial to treat ALS (Amyotrophic Lateral Sclerosis, or Lou Gehrig's disease) at Emory University in Atlanta, Georgia. The company announced that, after reviewing the safety data from the first cohort of three patients, the Safety Monitoring Board has approved moving to the next cohort and transplantation of the fourth patient.

25 May 2010

Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

Repligen Corporation announced today that the Office of Orphan Products Development of the Food and Drug Administration has granted orphan drug designation to RG2833, a selective histone deacetylase 3 inhibitor for the treatment of Friedreich's ataxia. Orphan drug designation qualifies Repligen to receive seven years of marketing exclusivity in the United States if the company is the first to obtain marketing approval for RG2833 for the treatment of Friedreich's ataxia.

24 May 2010

Scientists identify new gene candidates that regulate aggregate formations in Huntington's disease

They might not be known for their big brains, but fruit flies are helping to make scientists and doctors smarter about what causes Huntington's disease and how to treat it. New research, published in the journal GENETICS (http://www.genetics.org) describes a laboratory test that allows scientists to evaluate large numbers of fruit fly genes for a possible role in the formation of plaque-like protein aggregates within cells.

24 May 2010

EPO rules in favor of Alnylam Pharmaceuticals’ Kreutzer-Limmer '945 patent

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the Board of Appeals of the European Patent Office (EPO) has ruled that claims of the Kreutzer-Limmer '945 (EP 1214945) patent meet needed requirements under European patent law. The '945 patent comprises broad claims covering the length and certain structural requirements for siRNAs that are important for advancement of RNAi therapeutics.

20 May 2010

Study confirms inverse association of increase in BMI with lower brain volumes

Researchers from Boston University School of Medicine determined that excess abdominal fat places otherwise healthy, middle-aged people at risk for dementia later in life. Preliminary findings suggest a relationship between obesity and dementia that could lead to promising prevention strategies in the future. Results of this study are published early online in Annals of Neurology, a journal of the American Neurological Association.

20 May 2010

Neurologix reports lower net loss of $3.5M for first-quarter 2010

Neurologix, Inc., a biotechnology company developing innovative gene therapies for the brain and central nervous system, today announced its financial results for the quarter ended March 31, 2010.

14 May 2010

Being physical and mental activity delays onset of Huntington’s disease: Research

Melbourne researchers report in a study that regular physical and mental activities can delay the onset of the dreaded genetic Huntington's disease. This disease is hereditary and it goes on to destroy the brain and nerve cells leading to loss of all or most faculties. It affects one in 10,000 people between ages 30 and 50 and there is no cure. Since it is passed on to the children at least 50% of the time the decision to have children also becomes an agonizing one for many sufferers.

13 May 2010

Repligen files IND for Phase 1 study of RG2833 selective HDAC-3 inhibitor for Friedreich's ataxia

Repligen Corporation announced today that it has filed an Investigational New Drug Application (IND) with the Food and Drug Administration (FDA) for a Phase 1 study of RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor. This is a double-blind, single ascending dose, Phase 1 study in healthy volunteers to evaluate the pharmacokinetic and safety profile of RG2833 in up to 40 subjects.

13 May 2010

Key mechanisms for delivery of LNP-encapsulated siRNAs to the liver

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the publication of pre-clinical research in the journal Molecular Therapy revealing key mechanisms related to the systemic delivery of RNAi therapeutics using lipid nanoparticles (LNPs).

12 May 2010

Huntington's Disease News and Research

Latest Huntington's Disease News and Research

Regulus Therapeutics, sanofi-aventis partner to develop and commercialize microRNA therapeutics

USPTO awards Alnylam Notice of Allowance for patent covering methods of inhibiting expression of mutant genes

Many Huntington's disease patients suffer from weight loss, sleeping problems

ISB, GIND collaborate on whole-genome sequencing to identify drug targets for Huntington's Disease

Peter and Patricia Gruber Foundation to present 2010 Neuroscience Prize to Robert H. Wurtz

EMA COMP grants orphan medicinal product designation for Raptor's DR Cysteamine for cystinosis

Repligen's fiscal 2010 total revenue decreases to $20,971,000

Alnylam presents interim data from ALN-VSP02 Phase I trial for liver cancer at ASCO 2010

Anti-seizure drug can be used to treat AT deficiency: Pitt researchers

Raptor Pharmaceutical acquires exclusive license to intellectual property for Huntington's Disease treatment

Alnylam, Mount Sinai scientists discover svRNAs that play a critical role in influenza A virus replication

Neuralstem updates progress of ongoing Phase I human clinical trial for ALS

Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

Scientists identify new gene candidates that regulate aggregate formations in Huntington's disease

EPO rules in favor of Alnylam Pharmaceuticals’ Kreutzer-Limmer '945 patent

Study confirms inverse association of increase in BMI with lower brain volumes

Neurologix reports lower net loss of $3.5M for first-quarter 2010

Being physical and mental activity delays onset of Huntington’s disease: Research

Repligen files IND for Phase 1 study of RG2833 selective HDAC-3 inhibitor for Friedreich's ataxia

Key mechanisms for delivery of LNP-encapsulated siRNAs to the liver

eBook: Advancing neurodegenerative research with iPSC-derived microglia models eBook

Shaping the Future of Neuroscience: A Conversation with Atlas Antibodies on the MolBoolean™ and the Impact of SfN 2024

How the Arts Reshape Brain Function: Susan Magsamen on the Future of Neuroaesthetics

Inside the Alzheimer's Association: Dr. Heather Snyder on Driving Research and Collaboration

Huntington's Disease News and Research

Latest Huntington's Disease News and Research

eBook: Advancing neurodegenerative research with iPSC-derived microglia models eBook

Shaping the Future of Neuroscience: A Conversation with Atlas Antibodies on the MolBoolean™ and the Impact of SfN 2024

How the Arts Reshape Brain Function: Susan Magsamen on the Future of Neuroaesthetics

Inside the Alzheimer's Association: Dr. Heather Snyder on Driving Research and Collaboration

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