Fusion protein containing sulfoglucosamine sulfohydrolase enzyme and method thereof

A fusion protein with modified Fc polypeptides that bind to the transferrin receptor enhances enzyme delivery across the blood-brain barrier, effectively treating both peripheral and central nervous system symptoms of Sanfilippo syndrome A.

JP2026095522APending Publication Date: 2026-06-11DENALI THERAPEUTICS INC
0 Cites 0 Cited by

Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
DENALI THERAPEUTICS INC
Filing Date
2026-03-27
Publication Date
2026-06-11

Smart Images

  • Figure 1
    Figure 1
  • Figure 2
    Figure 2
  • Figure 3
    Figure 3
Patent Text Reader

Abstract

The present invention provides a fusion protein containing a sulfoglucosamine sulfohydrolase enzyme and a method for producing such a protein. [Solution] Provided herein are proteins comprising a sulfoglucosamine sulfohydrolase (SGSH) enzyme-Fc fusion polypeptide that can be transported across the blood-brain barrier (BBB). Certain embodiments also provide a method of using such proteins to treat Sanfilippo syndrome A. Sanfilippo syndrome, or MPSIII, is a rare neurodegenerative disorder resulting from specific defects in lysosomal function.
Need to check novelty before this filing date? Find Prior Art