Diverse portfolio of internal programs for CNS diseases and monogenic disorders as academic research hub in Japan. Because Dr. Muramatsu is one of the few researchers of AAV-vector in Japan, many researchers who have research seeds from all over Japan offer him joint research. Lead internal programs are novel treatments for Sporadic ALS and Parkinson's disease.
Broad intellectual property around two original AAV vectors (AAV.GTX and AAV.GT5) with in vivo proof of concept data.
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Gene therapy manufacturing expertise since 2017. First GCTP/cGMP/PICS GMP complied manufacturing facility with 200L bioreactor completed Mar 2017.
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Parkinson’s disease is a progressive neurodegenerative disease that usually develops at 40–70 years of age, with major signs of dyskinesia including tremor, hypokinesis, muscle rigidity, and postural reflex disorder. It affects about 140,000 patients in Japan.
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Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that selectively affects motor neurons and has no known cause or treatment. It is a refractory disease that mainly develops in late middle age without any obvious trigger, leading to death in several years.
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