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Plasmid Vaccines and Therapeutics: From Design to Applications

  • Marston Manthorpe
  • Peter Hobart
  • Gary Hermanson
  • Marilyn Ferrari
  • Andrew Geall
  • Blake Goff
  • Alain RollandEmail author
Chapter
Part of the Advances in Biochemical Engineering/Biotechnology book series (ABE, volume 99)

Abstract

In the late 1980s, Vical and collaborators discovered that the injection into tissues of unformulated plasmid encoding various proteins resulted in the uptake of the plasmid by cells and expression of the encoded proteins. After this discovery, a period of technological improvements in plasmid delivery and expression and in pharmaceutical and manufacturing development was quickly followed by a plethora of human clinical trials testing the ability of injected plasmid to provide therapeutic benefits. In this chapter, we summarize in detail the technologies used in the most recent company-sponsored clinical trials and discuss the potential for future improvements in plasmid design, manufacturing, delivery, formulation and administration. A generic path for the clinical development of plasmid-based products is outlined and then exemplified using a case study on the development of a plasmid vaccine from concept to clinical trial.

Anthrax Anthrax vaccine Biologics License Application Clinical trial Company-sponsored clinical trial DNA vaccine Formulation Gene delivery Gene expression systems Gene therapy Genetic immunization Genetic vaccination Investigational New Drug Application Needle-free devices Plasmid Plasmid design Plasmid DNA Plasmid formulation Plasmid manufacture Vaccination Vaccine 

Abbreviations

AD

autodisable or autodestruct

Ad

adenovirus vector

AIDS

acquired immunodeficiency syndrome

APC

antigen presenting cell

arg

arginine auxotroph gene

AVA

Anthrax Vaccine Adsorbed

BAK

benzalkonium chloride

BD

Becton Dickinson and Company

BGH

bovine growth hormone

BLA

Biologics License Application

CAD

coronary artery disease

CCC

covalently closed circular

CDC

The Centers for Disease Control and Prevention

CF

cystic fibrosis

CFTR

cystic fibrosis transmembrane conductance regulator

cGMP

current good manufacturing practice

CITE

cap-independent translational entry

CMV

cytomegalovirus

CTAB

cetyltrimethylammonium bromide

CTL

cytotoxic T lymphocytes

DC-Chol

3-N-(N′,N′-dimethylaminoethane)-carbamoyl cholesterol

DDAB-PC

dodecylammonium bromide-phosphatidyl choline

DEL-1

development-regulated endothelial locus-1

DMRIE

(+/ −)-N-(2-hydroxyethyl)-N,N-dimethyl-2,3-bis(tetradecyloxy)-1-propanaminium bromide

DODAC

N,N-Dioleyl-N,N-dimethylammonium chloride

DOPE

1,2-dioleoyl-sn-glycero-3-phosphoethanolamine

DOSPER

1,3-di-oleoyloxy-2-(6-carboxy-spermyl)-propylamide

DOTMA

N-[1-(2,3-dioleyloxy)propyl]-N,N,N-trimethylammonium chloride

DPyPE

1,2-diphytanoyl-sn-glycero-3-phosphoethanolamine

E. coli

Escherichia coli

EF

Bacillus anthracis edema factor

ELISA

enzyme-linked immunosorbant assay

FACS

fluorescence-activated cell sorting

FDA

U.S. Food and Drug Administration

FGF

fibroblast growth factor

FHIT

fragile histidine triad

Flu

influenza

GAP-DLRIE

aminopropyl-dimethyl-bis-dodecyloxy-propanaminium bromide

GAP-DMORIE

(+/ −)-N-(3-aminopropyl)-N,N-dimethyl-2,3-bis(cis-9-tetradecenyloxy)-1-propanaminium bromide)

GLP

good laboratory practices

GM-CSF

granulocyte-macrophage colony stimulating factor

GMP

good manufacturing practices

HBsAg

hepatitis B surface antigen

HIV-1

human immunodeficiency virus-1

HPLC

high pressure liquid chromatography

HPV

human papillomavirus

HSP

heat shock protein

HSV-TK

Herpes simplex virus-thymidine kinase

ID

intradermal

IFN

interferon

IL

interleukin

IN

intranasal

IP

intraperitoneal

IRES

internal ribosomal entry site

IT

intratumoral

IV

intravenous

IVT

intraventricular

JE

Japanese encephalitis

KDa

kilodaltons

LAL

Limulusamoebocyte lysate assay

LF

Bacillus anthracis lethal factor

MHC

major histocompatibility complex

MLV

multilamellar vesicles

mRBG

modified-rabbit beta-globin

Naked DNA

unformulated plasmid (e.g., in saline or phosphate-buffered saline)

NIAID

National Institute of Allergy and Infectious Diseases

NLS

nuclear localization signal

NP

influenza nucleoprotein

ORF

open reading frame

ori

bacterial origin of replication

OTC

ornithine transcarbamylase

PA

Bacillus anthracis protective antigen

PAD

peripheral arterial disease

PEG

polyethylene glycol

PEG-DSPE

monoethoxyl polyethylene-glycol-distearoylphosphatidylethanolamine

PINC

protective, interactive, noncondensing

PLG

poly(lactide-co-glycolide)

POC

proof of concept

POE

polyoxyethylene

POP

polyoxypropylene

Pulm

pulmonary

PVP

poly(vinyl pyrrolidone)

RSV

respiratory syncytial virus

RT-PCR

reverse transcription polymerase chain reaction

SC

subcutaneous

SCID

severe combined immunodeficiency

SPLP

stabilized plasmid-lipid particles

SV40

Simian virus-40

TA

taurocholic acid

TAP

transporter associated with processing

TFA

trifluoroacetic acid

TLR

toll-like receptor

TM-TPS

CellFECTIN ®; N, NI, NII, NIII-Tetramethyl-N, NI, NII, NIII-tetrapalmitylspermine

TPA

tissue plasminogen activator

UNICEF

United Nations Children's Fund

UTR

untranslated region

Vaxfectin™

GAP-DMORIE + DPyPE

VCL-6292

Vical plasmid 6292 encoding Bacillus anthracis protective antigen

VCL-62952

Vical plasmid 62952 encoding Bacillus anthracis lethal factor

VEGF

vascular endothelial growth factor

VTEU

Vaccine and Treatment Evaluation Unit

WHO

World Health Organization

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Notes

Acknowledgments

The authors wish to thank Drs. Thomas G. Evans, David C. Kaslow, Ruth Vahle, Jukka Hartikka and Mr. Alan Engbring for their helpful editorial comments, and we thank Dr. Rohit Mahajan for contributing Fig. 1.

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Authors and Affiliations

  • Marston Manthorpe
    • 1
  • Peter Hobart
    • 1
  • Gary Hermanson
    • 1
  • Marilyn Ferrari
    • 1
  • Andrew Geall
    • 1
  • Blake Goff
    • 1
  • Alain Rolland
    • 1
    Email author
  1. 1.Vical IncorporatedSan DiegoUSA

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