from UC San Diego
PRNewswire - Repligen Corporation announced today that it has licensed
two patent applications from the University of California, San Diego (UCSD)
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 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, Ph.D., President and CEO of Repligen,
"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.
Purine Autism
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. 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 Corporation develops new drugs for debilitating pediatric
disorders including autism, cancer, and immune and metabolic disorders.
Additional information may be requested from http://www.repligen.com.
Brought to you for your information by the Autism Society of California, ASA. The Autism Society of America (ASA) does not endorse individuals, groups or programs. References regarding programs, meetings, resources, treatment, etc. should not be interpreted as an indication of endorsement by ASA. They are provided for informational purposes only.
Source: PRNewswire
