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20250205363. Functional Aav Capsids (SHAPE THERAPEUTICS .)

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FUNCTIONAL AAV CAPSIDS FOR SYSTEMIC ADMINISTRATION

Abstract: disclosed herein are engineered aav vp capsid polypeptides with the ability to assemble into virus particles and having improved tissue tropism to, for example, cns tissues or muscle tissues. the capsids are engineered using the high throughput discovery system described herein. in certain embodiments, provided herein are recombinant adeno-associated virus (aav) vp capsid polypeptides having at least one mutation in a 581-589 region of the vp capsid polypeptide, corresponding to residues 581 to residue 589 of a vp1 polypeptide of seq id no: 1.

Inventor(s): Thomas PACKARD, David Jeffrey HUSS, Francois VIGNEAULT, Adrian Wrangham BRIGGS, Ronald James HAUSE, JR., Kevin Christopher STEIN, Yue JIANG

CPC Classification: A61K48/005 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)

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