PORTALEXCELLENCE: MUNICIPAL SERVICE JOB PORTAL Job Advancement Cerdanyola - brokerage TRAINING AND CONSULTANCY "Fresh News" Esteve and UAB promoted the development of a treatment for Sanfilippo syndrome in
Esteve has announced the signing of two agreements that will further develop their gene therapy research to treat mucopolysaccharidosis paragon consulting group type IIIA of (MPSIIIA Sanfilippo Syndrome Type A) and initiate clinical trials Phase I / II in 2015. This is in accordance with U.S. biotech company REGENX Biosciences and the French non-profit organization GENETHON.
Thanks to the agreement with REGENX, Esteve paragon consulting group can use the NAV vector rAAV9 in the development and commercialization of its gene therapy for mucopolysaccharidosis type IIIA treatment. paragon consulting group The viral vector NAV rAAV9 part of this gene therapy research paragon consulting group and allow the human gene sulfamidasa absent or defective in patients with Sanfilippo A syndrome, reaches into and expressed in cells as neurons and hepatocytes, so that there would sulfamidasa steadily and offset the absence of this enzyme in patients. paragon consulting group
The purpose of the agreement with Généthon is the production of the product of gene therapy research for the administration of clinical trials. The agreement will address seamless security preclinical development, clinical development and subsequent commercial distribution.
Sanfilippo project started in the research group of Dr. Fatima Bosch (CBATEG), since 2009 part of the partnership public - private between Clarence and Autonomous University of Barcelona (UAB). The aim is to develop gene therapies paragon consulting group for the treatment of this syndrome and other related diseases, called mucopolysaccharidosis.
In this alliance, Esteve leads activities related to the management and protection of intellectual property, regulatory actions and procedures, coordination and supervision of GMP production, the development of preclinical safety studies and clinical trials. The research group CBATEG UAB brings his scientific knowledge to the foregoing activities while developed gene therapy, including viral vector design and preclinical studies of disease.
Gene therapy research is a viral vector rAAV9 NAV, licensed REGENX, which contains an optimized version of sulfamidasa human gene that increases its expression. The preclinical models of the disease made by CBATEG support the potential paragon consulting group efficacy of this therapeutic option. The treatment consists in the administration of gene therapy in the cerebrospinal fluid, the fluid that bathes the brain and spinal cord. The NAV rAAV9 viral vector has the advantage of its high affinity for the brain (the main organ affected by disease) and, like all adenoassociats virus is harmless, no knowledge of any illness caused by the virus in humans. Sulfamidasa human gene vector introduced by the nucleus of the neuron, and began producing sulfamidasa. Thus, it was producing the enzyme that is secreted in cerebrospinal fluid, allowing its distribution throughout the brain and spinal paragon consulting group cord neurons and reaching those in which it has been incorporated into viral vector. In addition, paragon consulting group a small part of the gene therapy from the cerebrospinal fluid passes into the bloodstream, reaching organs such as the liver, where it is incorporated into hepatocytes, producing and secreting sulfamidasa distributed throughout the body via the bloodstream .
The results of the preclinical model of the disease indicate that resets once the levels of enzyme activity paragon consulting group both in the brain and the rest of the body disappears accumulation of glycosaminoglycans (substances that metabolize sulfamidasa) in cells cells disappears both neuroinflammation and dysfunction of other organs such as the brain affected, the behavior of the animal is normalized and life expectancy continues to normal.
The research group led by Dr. Fatima Bosch has shown in preclinical models that the administration in the cerebrospinal fluid may be performed through a single puncture paragon consulting group intracranial such as those made for treatment of hydrocephalus in children, the which means an important innovation because it is common practice interventions that are of pediatric neurosurgeons and ensure even distribution of the product throughout the brain.
Currently, the project designated as orphan drug by the authorities both European (EMA) and American paragon consulting group (FDA)
Esteve has announced the signing of two agreements that will further develop their gene therapy research to treat mucopolysaccharidosis paragon consulting group type IIIA of (MPSIIIA Sanfilippo Syndrome Type A) and initiate clinical trials Phase I / II in 2015. This is in accordance with U.S. biotech company REGENX Biosciences and the French non-profit organization GENETHON.
Thanks to the agreement with REGENX, Esteve paragon consulting group can use the NAV vector rAAV9 in the development and commercialization of its gene therapy for mucopolysaccharidosis type IIIA treatment. paragon consulting group The viral vector NAV rAAV9 part of this gene therapy research paragon consulting group and allow the human gene sulfamidasa absent or defective in patients with Sanfilippo A syndrome, reaches into and expressed in cells as neurons and hepatocytes, so that there would sulfamidasa steadily and offset the absence of this enzyme in patients. paragon consulting group
The purpose of the agreement with Généthon is the production of the product of gene therapy research for the administration of clinical trials. The agreement will address seamless security preclinical development, clinical development and subsequent commercial distribution.
Sanfilippo project started in the research group of Dr. Fatima Bosch (CBATEG), since 2009 part of the partnership public - private between Clarence and Autonomous University of Barcelona (UAB). The aim is to develop gene therapies paragon consulting group for the treatment of this syndrome and other related diseases, called mucopolysaccharidosis.
In this alliance, Esteve leads activities related to the management and protection of intellectual property, regulatory actions and procedures, coordination and supervision of GMP production, the development of preclinical safety studies and clinical trials. The research group CBATEG UAB brings his scientific knowledge to the foregoing activities while developed gene therapy, including viral vector design and preclinical studies of disease.
Gene therapy research is a viral vector rAAV9 NAV, licensed REGENX, which contains an optimized version of sulfamidasa human gene that increases its expression. The preclinical models of the disease made by CBATEG support the potential paragon consulting group efficacy of this therapeutic option. The treatment consists in the administration of gene therapy in the cerebrospinal fluid, the fluid that bathes the brain and spinal cord. The NAV rAAV9 viral vector has the advantage of its high affinity for the brain (the main organ affected by disease) and, like all adenoassociats virus is harmless, no knowledge of any illness caused by the virus in humans. Sulfamidasa human gene vector introduced by the nucleus of the neuron, and began producing sulfamidasa. Thus, it was producing the enzyme that is secreted in cerebrospinal fluid, allowing its distribution throughout the brain and spinal paragon consulting group cord neurons and reaching those in which it has been incorporated into viral vector. In addition, paragon consulting group a small part of the gene therapy from the cerebrospinal fluid passes into the bloodstream, reaching organs such as the liver, where it is incorporated into hepatocytes, producing and secreting sulfamidasa distributed throughout the body via the bloodstream .
The results of the preclinical model of the disease indicate that resets once the levels of enzyme activity paragon consulting group both in the brain and the rest of the body disappears accumulation of glycosaminoglycans (substances that metabolize sulfamidasa) in cells cells disappears both neuroinflammation and dysfunction of other organs such as the brain affected, the behavior of the animal is normalized and life expectancy continues to normal.
The research group led by Dr. Fatima Bosch has shown in preclinical models that the administration in the cerebrospinal fluid may be performed through a single puncture paragon consulting group intracranial such as those made for treatment of hydrocephalus in children, the which means an important innovation because it is common practice interventions that are of pediatric neurosurgeons and ensure even distribution of the product throughout the brain.
Currently, the project designated as orphan drug by the authorities both European (EMA) and American paragon consulting group (FDA)
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