Drug Profile

M 043

Alternative Names: AAV-NAGLU-S1S3; Lenti-NAGLU-S1S3; M-043; Sanfilippo B (MPSIIIB)

Latest Information Update: 28 Mar 2025

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At a glance

Originator M6P Therapeutics
Class Gene therapies
Mechanism of Action Alpha N acetyl D glucosaminidase replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No
Available For Licensing Yes

28 Mar 2025 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-III in USA (Parenteral)
09 Feb 2023 M 043 receives Rare pediatric drug for Enzyme replacement therapy status for in USA prior to February 2023 (M6P Therapeutics website, February 2023)
04 Feb 2021 M 043 is available for licensing as of 03 Feb 2021

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