公开(公告)号：US20240167031A1

公开(公告)日：2024-05-23

申请号：US18283925

申请日：2022-03-31

Applicant: Biogen MA Inc.

Inventor： Hasan M. Yildiz ,  Mandana Bornapour ,  Paul Peng ,  Vishwesh A. Patil ,  Brian R. Simler ,  William F. Kiesman

IPC: C12N15/113 ,  A61K9/00 ,  A61K9/51 ,  A61K47/59

CPC classification number: C12N15/113 ,  A61K9/0019 ,  A61K9/0085 ,  A61K9/5153 ,  A61K47/59 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/345

Abstract: Featured are polymeric nanocarriers (e.g., PLGA nanoparticles) with encapsulated nucleic acid (e.g., an antisense oligonucleotide) for delivery (e.g., intrathecally) to the central nervous system. These polymeric nanocarriers are useful in the treatment of central nervous system disorders. They are capable of delivering their cargo (e.g., an antisense oligonucleotide) in higher amounts, for a longer period of time, and into deeper regions of the brain than a free or unformulated antisense oligonucleotide. The efficient delivery and distribution of antisense oligonucleotides results in reducing the number of administrations and patient compliance and improves patient experience.