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Allen Institute, BioMarin To Develop Gene Therapies For Rare Brain Diseases

Allen Institute, BioMarin To Develop Gene Therapies For Rare Brain Diseases
WASHINGTON (dpa-AFX) - The Allen Institute and BioMarin Pharmaceutical Inc. (BMRN) announced a collaboration today that will use technologies developed at the Allen Institute to create new gene therapies aimed at rare genetic diseases of the central nervous system. They did not disclose financial terms.
BioMarin will receive an exclusive license to each program for research, development and commercialization.
The Allen Institute for Brain Science, a division of the Allen Institute, has developed modified adeno-associated viruses, or AAVs, that have been engineered to impact specific classes of cells in the brain. ....

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PKU miracle drug: Jeremy Hunt backs Kuvan crusade 'families will get their lives back'

Now Matt Hancock’s predecessor has written a letter to James Lennertz, boss of BioMarin Europe, exclusively seen by the Express.
Mr Hunt, chairman of the Health and Social Care Select Committee, said: “For far too long people have been denied the normal life most of us take for granted.
“Now we hope and pray 2021 will be not just the year we defeat Covid, but the year many families can get back their lives by accessing this vital drug.
“But the manufacturer BioMarin needs to be flexible on price.
“What is the point in medical breakthroughs if most people can’t afford to benefit from them?” ....

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