FORDADISTROGENE MOVAPARVOVEC
UNII H4T32JIH01

Substance Identification & Data

This profile provides standardized clinical and technical data for Fordadistrogene Movaparvovec, uniquely identified by the FDA Unique Ingredient Identifier (UNII) H4T32JIH01.

Technical mappings include the Chemical Abstracts Service (CAS) Registry Number 2248111-37-5 and the RxNorm Concept ID (RxCUI) N/A. Explore the sections below for detailed nomenclature and a complete directory of NDC-listed products containing this ingredient.

FDA UNII Code

H4T32JIH01

CAS Registry Number

2248111-37-5

RxNorm Concept ID

N/A

Detailed Substance Profile

Preferred Name

FORDADISTROGENE MOVAPARVOVEC

Official standardized name for this substance within the FDA UNII nomenclature system.

NCI Thesaurus

C175785

National Cancer Institute reference terminology for clinical and research data.

INN ID

11362

Sequential identifier assigned via the WHO International Nonproprietary Name program.

USAN ID

GH-67

Identifier assigned by the United States Adopted Names Council.

Substance Type

structurallyDiverse

ISO 11238 classification category (e.g., Chemical, Polymer, Protein).

Synonyms and Nomenclature

This section provides a complete list of nomenclature and identifier mappings for Fordadistrogene Movaparvovec. Identifiers are organized into official regulatory terms, commercial trade names, and technical systematic synonyms used to ensure accurate identification across clinical pharmaceutical databases, regulatory filings, and electronic health records.

FDA Official Name

FORDADISTROGENE MOVAPARVOVEC

Common Names & Synonyms

AAV9.HCK.OPTI-DYS.DELTA.3978

DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS 9 VECTOR PF-06939926 HUMAN MUSCLE-SPECIFIC ENHANCER/PROMOTER PLUS HUMAN MINI-DYSTROPHIN GENE PLUS ADENO-ASSOCIATED VIRUS 9 INVERTED TERMINAL REPEAT FLANKS)

FORDADISTROGENE MOVAPARVOVEC [INN]

FORDADISTROGENE MOVAPARVOVEC [USAN]

FORDADISTROGENE MOVAPARVOVEC [WHO-DD]

PF-06939926 IS A RECOMBINANT AAV VECTOR CONTAINING THE AAV SEROTYPE 9 (AAV9) CAPSID PACKAGING A MINIATURIZED CODON-OPTIMIZED VERSION OF THE DMD GENE(CALLED OPTI-DYS.DELTA.3978) WHICH ENCODES THE DOMAINS MINIMALLY REQUIRED FOR FUNCTIONALITY OF THE DYSTROPHIN PROTEIN (MINI-DYSTROPHIN). A SYNTHETIC POLYADENYLATION (SPOLYA) SEQUENCE FOLLOWS THE OPTI-DYS.DELTA.3978 GENE. CARDIAC AND SKELETAL MUSCLE-SPECIFIC TRANSGENE EXPRESSION IS MEDIATED BY A SYNTHETIC ENHANCER AND PROMOTER. THE EXPRESSION CONSTRUCT IS FLANKED BY THE AAV2 INVERTED TERMINAL REPEATS (ITRS). THE 3978 BASEPAIR MINI-DYSTROPHIN OPEN READING FRAME ENCODES THE N-TERMINUSHINGE REGIONS H1, H3 AND H4SPECTRIN-LIKE REPEATS R1, R2 AND R22-24AND THE C-TERMINAL CYSTEINE-RICH REGION OF THE DYSTROPHIN PROTEIN.

Technical Codes

PF-06939926

External Clinical & Regulatory Resources

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