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Repligen licenses patent rights for treatment of mitochondrial disorders and purine autism.

Repligen Corporation (Needham, MA; 781-449-9560) announced that it has licensed two patent applications from the University of California (San Diego, CA) covering novel methods for the treatment of mitochondrial disease and for the treatment of autism in patients with abnormal purine metabolism ("purine autism"). Under the terms of the license agreement, Repligen receives exclusive commercial rights to both inventions and will pay UCSD an up-front fee, clinical development milestones and royalties on product sales. Repligen will also support two research projects at UCSD.

One patent application covers the use of analogs of uridine, a naturally occurring component of RNA and DNA, for the treatment of diseases characterized by defects in the function of mitochondria. Mitochondria are structures essential for many cellular functions including energy production. Inborn forms of mitochondrial disease affect approximately 20,000 people in the United States and result in symptoms including seizures, skeletal and heart muscle weakness and neurological and cognitive defects. Over the last three years, 15 patients with mitochondrial defects have been treated with uridine or an analog, triacetyl uridine (TAU), at the UCSD Mitochondrial and Metabolic Disease Center. These studies have shown that uridine and TAU are well tolerated in this population for more than two years. Some of these patients have also shown marked improvements in their symptoms including cognitive performance, muscle control and renal function.

A second patent application covers the use of uridine and TAU for the correction of defects in purine metabolism which produce the symptoms of autism or pervasive developmental disorder. A recently published study from UCSD reports that some autistic patients have evidence of a purine metabolism defect. A few of these patients have been treated with uridine over the past several years with improvements noted in cognitive performance and muscular function.

"These two initiatives are consistent with our goal of become the leading company in the development of therapies for pediatric developmental disorders," said Walter Herlihy, PhD, president and CEO of Repligen. "The Phase 1 clinical data obtained at UCSD suggest that uridine supplementation can produce clinical benefits in some patients with mitochondrial disease and that prolonged administration of uridine analogs appears to be well tolerated in pediatric patients."

"Recent data from UCSD researchers indicates that there is a subset of patients diagnosed with autism who have abnormal purine metabolism," continued Dr. Herlihy. "Through our research collaboration we will attempt to extend these observations and identify the enzyme responsible for these symptoms. This will allow us to develop diagnostic tools to identify patients who may benefit from uridine therapy."

Mitochondria are tiny structures in every cell which convert nutrients into the energy required for cellular functions. Patients with a mitochondrial defect display a wide range of symptoms including poor muscular and neurological performance. It has recently been recognized that the mitochondria are also the only source in the body for the production of uridine, an essential precursor for the synthesis of RNA and DNA as well as other cellular functions. This observation led researchers to evaluate synthetic uridine and triacetyl uridine as a therapy for a variety of mitochondrial disorders. Inborn errors in mitochondrial function occur in about 1/2000 live births. About half of the patients born with mitochondrial disease will develop symptoms in the first 5 years of life. The remaining half of patients will develop symptoms at an older age. Inborn forms of mitochondrial disease regularly lead to developmental delays in children, dementia in adults, and multi-organ system diseases in both children and adults. Mortality in the most severe forms of childhood mitochondrial disease can approach 50% per year. There are currently no FDA-approved therapies for mitochondrial disease.

Purines are key building blocks for the synthesis of DNA and RNA and are involved in a variety of other cellular processes. "Purine autism" was first characterized in the 1970's by a researcher who noted elevated levels of uric acid in the urine of some patients. Uric acid is the end product of purine metabolism and is elevated in other diseases of purine metabolism such as Lesch-Nyhan Syndrome. Recent studies at UCSD suggest that some of the autistic patients with elevated urate levels also have evidence of abnormally high rates of intracellular purine synthesis further indicating that they have a purine metabolism defect. A few of these patients have been treated with an analog of uridine for several years, with improvements observed in cognitive performance and muscular function. Autism spectrum disorders affect at least 500,000 patients in the United States. Additional research will define the patient subset with abnormal purine metabolism. No drugs are approved by the FDA for the treatment of autism spectrum disorders.

Repligen develops new drugs for debilitating pediatric disorders including autism, cancer, and immune and metabolic disorders. Repligen also manufactures and markets a set of patented products based on Protein A, which are used by the pharmaceutical industry to produce therapeutic antibodies.
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Comment:Repligen licenses patent rights for treatment of mitochondrial disorders and purine autism.
Publication:BIOTECH Patent News
Article Type:Brief Article
Geographic Code:1USA
Date:Dec 1, 2000
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