Gene Therapy Nomenclature!

The FDA considers that the nomenclature system for gene therapy products needs to identify the product as a vector carrying a gene to be transferred, but that the indication should not be part of the name, nor should the name incorporate the finer details of the construct. !

Gene Therapy Nomenclature Guidelines  

The simpler the name the better, while avoiding the danger of over simplification. !

Elements of the Name!

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Elements of the Name

Gene Therapy Nomenclature Guidelines  

Indication of the drug’s mechanism of action or pharmacologic class.!

The vector used in transfection!

@Three elements have been identified that are important in distinguishing a gene therapy drug and conveying safety information to the user physician. These include:!  

Complete identification of the genes carried by the vector if there are 1 or 2 transgenes; for vectors containing > 3 transgenes, one or two genes will be chosen to be included in the gene qualifier based on an analysis of the impact that they have on preventing product confusion or conveying safety information.!U

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Additional nomenclature elements would be a fantasy syllable(s) that serves as a unique identifier for the molecular entity and a stem indicating the gene therapy class of products or the ability of the vector to replicate. !

Gene Therapy Nomenclature Guidelines   Elements of the Name!

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1st Word: Corresponds to the gene component !  

Prefix: Fantasy element to provide unique identification; to contribute to the distinct name!  

Infix: Element to denote the gene’s mechanism of action (pharmacologic class)!

Gene Therapy Nomenclature Guidelines  

Elements of the Name!

1st

infix prefix

-cima- [cytosine deaminase] -ermin- [growth factor] -kin- [interleukins] -lim- [immunomodulator] -lip- [human lipoprotein lipase]   -mul- [multiple gene]

-stim- [colony stimulating factor] -tima- [thymidine kinase] -tusu- [tumor supression]

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Stem: Element to indicate gene. -gene  

2nd Word: corresponds to the vector component  

Prefix: Fantasy element to provide unique identification; to contribute to the distinct name  

Gene Therapy Nomenclature Guidelines  

Elements of the Name

stem prefix

2nd word

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-adeno- [adenovirus] -cana- [canarypox virus] -foli- [fowlpox virus] -herpa- [herpes virus] -lenti- [lentivirus] -morbilli- [paramyxoviridae morbillivirus] -parvo- [adeno-associated virus (parvovirdae dependovirus)] -retro- [other retro viruses] -vaci- [vaccinia virus]

Infix: Element to denote the type of viral vector such as:  

infix

Gene Therapy Nomenclature Guidelines  

Elements of the Name

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Pharmaceutical Expertise We have 20+ years of experience in evaluating pharmaceutical names in compliance with regulatory standards and can assure complete confidence in our safety and submission strategies. We offer a range of services that ease and unburden this process for your team. .

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Clinical Trial Identity Programs Created

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Internal Innovation Brands Created

Proprietary Brand Names Created

Generic, Non-Proprietary USAN/INN Names Created

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