Drug Profile

M 043

Alternative Names: AAV-NAGLU-S1S3; Lenti-NAGLU-S1S3; M-042; M-043; Sanfilippo B (MPSIIIB)

Latest Information Update: 18 Jul 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

18 Jul 2025 M 043 is still in early research in Mucopolysaccharidosis-III in USA (Parenteral) (M6P Therapeutics pipeline, July 2025)
28 Mar 2025 No recent reports of development identified for early research 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)

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