NEW YORK--(BUSINESS WIRE)--Agilis Biotherapeutics, LLC, a synthetic biology-based company focused on rare genetic diseases, announced today that the company has completed an $8 million dollar financing. Proceeds from the financing will be used for Agilis initial focus on developing a treatment for Friedreichs ataxia (FRDA) in collaboration with Intrexon Corporation (NYSE: XON), a leader in the field of synthetic biology. Agilis and Intrexon recently executed an Exclusive Channel Collaboration (ECC) through which Agilis intends to develop and commercialize novel DNA-based therapeutics for the treatment of FRDA. Under the ECC Agilis also has an option to expand its relationship with Intrexon by adding another rare genetic disease to the collaboration. About Friedreichs Ataxia Friedreichs ataxia is a rare genetic neurodegenerative disease that results in a physically debilitating, life shortening condition. FRDA is caused by defect in a gene (the FXN gene) that results in limited production of an important protein called frataxin which functions in the mitochondria (the powerhouses ) of the cell. FRDA is the most common hereditary ataxia with an estimated 5,000 to 10,000 patients in the US. Progression of this disease causes nervous system damage, problems with movement, and finally early death resulting from cardiac malfunction. There are … Continue reading →

Amid somewhat of a renaissance in drug development for orphan diseases, another gene therapy company announced funding today for a potential treatment for a rare neurodegenerative condition called Friedreichs ataxia (FRDA). Agilis Biotherapeutics said its raised $8 million and will work with synthetic biology company Intrexon Corp. to develop gene therapies and genetically modified cell therapies for FRDA. Whereas current treatments focus on minimizing symptoms of the disease, the partners expect their drugs to be able to target the underlying disease mechanisms. FRDA is caused by a genetic defect that results in limited production of frataxin, a protein thought to help assemble clusters of iron and sulfur molecules in cells that are necessary for the function of many other proteins. When cells are deficient of frataxin, they may not function properly, causing damage to the nervous system and problems with movement. Most people with the disease become wheelchair-bound within two decades of diagnosis, and many die early due to weakened heart muscles. The goal for Agilis and Intrexon is to use Intrexons gene switch technology to develop drugs that will repair the defective gene and enable increased production of the frataxin protein. Under the options of the deal, Agilis and … Continue reading →

Current ratings for: Genetic brain development 'peaks before birth and in adolescence' Public / Patient: 0 (0 votes) Health Professionals: 0 (0 votes) Genetic expression behind the development of our brains is most active before birth, in the early months of pregnancy, and during our teenage years, scientists have found. There is a quieter intervening "movement" in the three-part "symphony" of human brain development, but one that is more sensitive to environmental factors, say the researchers publishing in the journal Neuron. The initial surge of brain-developing genetic expression takes place during the first two-thirds of our gestation in the uterus, says the team led from the Yale School of Medicine in New Haven, CT. The middle intermission then lasts from the final trimester of pregnancy until adolescence, at which point the genetic activity surges again for the final phase of our brain's development. These two most active spurts relevant to human brain power, found to sandwich the childhood years, involve the development of the cerebral neocortex: See more here: Genetic brain development 'peaks before birth and in adolescence' … Continue reading →

James DeLittle, 49, of Broadway West in Fulford, to travel to Kiev for stem cell treatment 11:27am Wednesday 1st January 2014 in News By Mike Laycock, Chief reporter James DeLittle A PARKINSONS Disease sufferer from York is to travel to Kiev for pioneering stem cell treatment, in a last-ditch bid to tackle his worsening condition. James DeLittle, 49, hopes the two-day treatment in the Ukrainian capital will lessen his symptoms, which include poor balance, tremor, difficulties controlling his limbs and slurred speech. Jamess mother, relatives and customers at three pubs in the York and Selby area have scraped together about 7,000 to pay for foetal stem cells to be injected into his stomach and arms. His condition has worsened significantly in recent months, causing him to fall several times, suffering injuries including a broken nose, ribs and thumb joint. James, of Broadway West in Fulford, said: As far as I know, Im the first person from the UK to go to the clinic and I would love to come back with an improvement in my condition to show all the other people with Parkinsons how they might benefit too. The NHS doesnt support the treatment at this stage so weve … Continue reading →

22 hours ago by John Steeno (Phys.org) A team of engineers at the University of Wisconsin-Madison has created a process that may revolutionize stem cell research. The process, outlined in a paper published in Stem Cells on December 19, 2013, will improve the state of the art in the creation of synthetic neural stem cells for use in central nervous system research. Human pluripotent stem cells have been used to reproduce nervous-system cells for use in the study and treatment of spinal cord injuries and of diseases such as Parkinson's and Huntington's. Currently, most stem cells used in research have been cultured on mouse embryonic fibroblasts (MEFs), which require a high level of expertise to prepare. The expertise required has made scalability a problem, as there can be slight differences in the cells used from laboratory to laboratory, and the cells maintained on MEFs are also undesirable for clinical applications. Removing the high level of required skilland thereby increasing the translatability of stem cell technologyis one of the main reasons why Randolph Ashton, a UW-Madison assistant professor of biomedical engineering and co-author of the paper, wanted to create a new protocol. Rather than culturing stem cells on MEFs, the new … Continue reading →