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On September 7, 2021, Roche's Spark Therapeutics and NeuExcell Therapeutics announced a collaboration to develop a safe and effective gene therapy for patients suffering from Huntington's disease (HD)
.
HD is an incurable inherited brain disease caused by a single gene defect on chromosome 4
.
Because the disease affects different areas of the brain, it affects movement, behavior, and cognitive function
According to the terms of the cooperation agreement, Spark will obtain the right to use NeuExcell's proprietary nerve regeneration gene therapy platform
.
NeuExcell's research team will work closely with Spark Therapeutics to advance the project
▲NeuExcell's nerve regeneration gene therapy platform can transform glial cells into new neurons (picture source: NeuExcell's official website)
The general assumption is that mature neurons in mammals cannot be replaced, so the treatment of brain diseases often focuses on slowing the progression of the disease
.
But NeuExcell may have found a new way to unlock regenerating nerve tissue
Although neurons cannot divide and regenerate themselves, glial cells can divide and regenerate at the injured site.
Therefore, they are a sustainable source of new neurons to achieve clinically meaningful therapeutic effects
.
NeuExcell is developing gene therapy for nerve regeneration based on adeno-associated viruses (AAVs) to regenerate a large number of functional new neurons in situ at the site of neurodegeneration
Reference materials:
[1] NeuExcell Therapeutics and Spark Therapeutics Announce Research Collaboration Agreement to Develop a Novel Gene Therapy for Huntington's Disease.
(The original text has been deleted)