Genzyme, a Sanofi company (SNY: Quote), and Alnylam Pharmaceuticals, Inc. (ALNY: Quote) Monday said they have significantly expanded their strategic agreement to develop and commercialize treatments for rare genetic diseases. As part of the expanded relationship, Genzyme will invest $700 million to become a major Alnylam shareholder. Under the new agreement, Genzyme will have significant rights to Alnylam's portfolio of clinical and pre-clinical stage drug candidates. Alnylam will retain most product rights in North America and Western Europe, and will have significantly expanded development and commercial opportunities for its genetic medicine pipeline through Genzyme's established global infrastructure in rare diseases. David Meeker, Genzyme's President and CEO, said, "This collaboration is an important building block for our future. It strengthens our pipeline and provides us with the opportunity to meet the needs of patients with rare diseases around the world through our well-established global organization." In 2012, Alnylam and Genzyme formed an exclusive alliance to develop and commercialize Alnylam's lead product, patisiran, which is in Phase 3 development for the treatment of transthyretin or TTR-familial amyloid polyneuropathy, a rare life-threatening disease that damages the nervous system. In the new alliance, Alnylam benefits from Genzyme's proven global capabilities, enabling the firm to … Continue reading →

PUBLIC RELEASE DATE: 12-Jan-2014 Contact: Jessica Studeny jessica.studeny@case.edu 216-368-4692 Case Western Reserve University Geneticists from Ohio, California and Japan joined forces in a quest to correct a faulty chromosome through cellular reprogramming. Their study, published online today in Nature, used stem cells to correct a defective "ring chromosome" with a normal chromosome. Such therapy has the promise to correct chromosome abnormalities that give rise to birth defects, mental disabilities and growth limitations. "In the future, it may be possible to use this approach to take cells from a patient that has a defective chromosome with multiple missing or duplicated genes and rescue those cells by removing the defective chromosome and replacing it with a normal chromosome," said senior author Anthony Wynshaw-Boris, MD, PhD, James H. Jewell MD '34 Professor of Genetics and chair of Case Western Reserve School of Medicine Department of Genetics and Genome Sciences and University Hospitals Case Medical Center. Wynshaw-Boris led this research while a professor in pediatrics, the Institute for Human Genetics and the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UC, San Francisco (UCSF) before joining the faculty at Case Western Reserve in June 2013. Individuals with ring chromosomes … Continue reading →

PUBLIC RELEASE DATE: 12-Jan-2014 Contact: Sarah Dionne Sullivan ssullivan38@partners.org 617-726-6126 Massachusetts General Hospital Although antiviral therapy against HIV suppresses viral replication and allows infected individuals to live relatively healthy lives for many years, the virus persists in the body, and replication resumes if treatment is interrupted. Now investigators from Massachusetts General Hospital (MGH) and the Ragon Institute of MGH, MIT and Harvard may have found where the virus hides - in a small group of recently identified T cells with stem-cell-like properties. "Most human cells are short lived, so it has been unclear how HIV manages to stick around for decades in spite of very effective antiviral treatment," says Mathias Lichterfeld, MD, of the MGH Infectious Disease Division, corresponding author of the report receiving advance online publication in Nature Medicine. "This question led to the hypothesis that HIV might infect stem cells - the most long-lasting cells in the body - but traditional organ-specific stem cells, even those that give rise to all immune and blood cells, are resistant to HIV infection. We have discovered that a new group of T cells, called T memory stem cells, are susceptible to HIV and likely represent the longest lasting cellular niche for … Continue reading →

Nanomedicine: Challenges and opportunities Seminar from Alumni Weekend 2011 Nanotechnology is a new, exciting field that has the potential to transform the way that medical and healthcare solutions are developed and delivered. This... By: 21school … Continue reading →

January 12, 2014 Image Caption: Scanning electron microscopy of stem cells (yellow / green) in a scaffold structure (blue) serving as a basis for the artificial bone marrow. Credit: C. Lee-Thedieck/KIT Rebekah Eliason for redOrbit.com Your Universe Online An exciting breakthrough is offering hope for the treatment of blood diseases such as leukemia using artificial bone marrow. Specialized cells, known as hematopoietic stem cells, located within bone marrow, continuously replace and supply new blood cells such as red blood cells and white blood cells. Traditionally a blood disease like leukemia is treated with bone marrow transplants that supply the patient with new hematopoietic stem cells. Researchers have now discovered a way to artificially reproduce hematopoietic stem cells. Since not every leukemia patient can find a suitable transplant, there is a need for other forms of treatment. The lack of appropriate transplants could be solved by artificial reproduction of hematopoietic stem cells. Previously, reproduction of the cells has been impossible due to their inability to survive anywhere but in their natural environment. Hematopoietic stem cells are found in a special niche of the bone marrow. If the cells reside out of the bone marrow, the specialized properties are modified. Consequently, to … Continue reading →

CLINTON Alonna Dukeman was not one to sit. I always need something to do, the 59-year-old rural Clinton resident said. On the table in front of her were trays of cinnamon rolls and candies that she baked that morning. She spoke in a home that she and her fiance, Ron Deatrick, transformed from a farmhouse to a showpiece, including doors with stained-glass windows suspended from the dining room ceiling. There was no stopping me, she said. A co-worker called her Indy 500. Not anymore. As Dukeman motioned with her right arm, her left arm hung at her side. As she walked slowly, she concentrated so her left leg wouldnt drag behind her right leg. Ive learned that if youre going to do anything, its slow, she said. This was the hardest thing for me. Dukeman had a stroke on May 21, 2012. But shes doing OK, she said last week, and not only because of post-stroke rehabilitation, her exercises and Deatricks support. About a year ago, she and Deatrick began attending meetings of the Stroke/Brain Injury Support Group of the Bloomington-Normal Area. She learned she was not alone. Ive learned that I cant dwell on what I used to do, … Continue reading →

Current ratings for: Rare genetic mutation confirmed as a cause of Tourette Syndrome Public / Patient: 5 (1 vote) Health Professionals: 0 (0 votes) Brain researchers say they have confirmed for the first time that a rare genetic mutation can cause some cases of Tourette syndrome, with the fault disrupting production of histamine in the brain. The New Haven, CT, researchers at the Yale School of Medicine say the histamine effect "is a cause of the tics and other abnormalities of Tourette syndrome." Tics are repetitive movements and vocal sounds, and they are unwanted and involuntary - they cannot be controlled. Publishing their research on mice in the journal Neuron, the authors raise the question of investigating treatment of Tourette syndrome by drugs that target histamine receptors in the brain. Drugs with such a mode of action are already being explored by pharmaceutical companies for the treatment of separate brain disorders, schizophrenia and ADHD. Information from the national gene database about histamine describes the chemical's role - it is a messenger molecule released by nerves, among other functions. More here: Rare genetic mutation confirmed as a cause of Tourette Syndrome … Continue reading →