Genetic medicines are genetic materials such as DNA and RNA delivered into the body as a therapeutic. They are a promising new class of medicine that was not possible even a short time ago.

Advancements in science and technology are changing the way we define disease, develop drugs and prescribe treatments with an explosion of insights into the role of genetics in infectious diseases, cancer and rare diseases. Genetic medicines are an emerging technology with the potential to be developed as personalized medicines and for mass administration, by teams with a wide range of capabilities.

2020 Nobel Laureate Dr. Michael Houghton on Lipid Nanoparticle RNA Vaccines

Genetic vaccines are a new class of medicine that introduces new genes to the body to produce key antigens that help the body fight infections. Genetic vaccines can be broken down into two broad categories, viral vectors and non-viral vectors that use synthetic methods such as lipid nanoparticles deliver genes into cells. Non-viral vectorsoffer some advantages of viral vectorsincluding the ability to deliver larger genes, reduced biosafety concerns, and simplified synthetic production.

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Particularly, mRNA vaccines are a recent innovation in genetic medicine but are now at the forefront of the many vaccine technologies for the COVID-19 pandemic. mRNA vaccines use non-viral vectors to deliver the drug into the body which leads to safer and fast-to-develop vaccines. The mRNA vaccines at the forefront of the COVID-19 pandemic show how genetic vaccines have not only the potential for high potency, low-cost manufacturing and safe administration but also the capacity for rapid development.

A promising application for genetic vaccines is self-amplifying mRNA (saRNA), a vaccine platform that uses the human body toamplifythe vaccine and produce vaccine antigens. saRNA can offer a potent vaccine in extremely small doses due to its ability to amplify itself inside the body.

All these advantages of the mRNA vaccines translate into the development of other genetic medicines. The search for a pandemic vaccine has accelerated all genetic medicines by highlighting their advantages on the world stage.

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Samuel Clark, PNI Director of R&D, on how an mRNA gene therapy can work.

Gene therapy is the introduction of genetic material into the body to modify how proteins are expressed to treat a disease. Gene therapy is a promising application for genetic medicines for a number of diseases such as inherited disorders, viral infection and cancers. This technique allows doctors to treat a disorder by delivering a gene into a patient instead of using drugs or surgery.

Researchers are testing several approaches to gene therapy, including:

Replacing a disease-causing mutated gene with a healthy gene

Inactivating, or knocking out, a malfunctioning mutated gene

Introducing a new gene to help fight a disease

The amount of gene therapies being developed have continued to grow with 352 gene therapy in clinical trialsglobally at the end of last year. The FDA expects to see a doubling of new gene therapy applications each year. Scott Gottlieb, the former FDA commissioner, predicts that by 2025 the US would be approving between 10 and 20 gene therapies each year.

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In 1989, A National Institutes of Health (NIH) approved study provided evidence for the first time that human cells could be genetically modified and returned to the patient without harm. Since then, the industry has grown significantly with 22 gene and gene-modified cell therapies approved by regulatory bodies from various countries as of August 2019.

Precision NanoSystems can provide a customized, end-to-end pathway for your drug development programs, from lead candidate selection through early phase clinical trials to commercialization. Our proprietary technology platforms and comprehensive expertise enable researchers to work with a single, integrated partner to translate disease biology insights into non-viral genetic medicines. Minimizing handoffs throughout the process results in faster timelines and reduced costs.

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Publication - Abstract

July 01, 2020

Journal of Controlled Release

G. Lou, G. Anderluzzi, S.T. Schmidt, S. Woods, S. Gallorini, M. Brazzoli, F. Giusti, I. F...

Publication - Abstract

May 08, 2020

Vaccines

G. Anderluzzi, G. Lou, S. Gallorini, M. Brazzoli, R. Johnson, D.T. O'Hagan, B.C. Baudner a...

Publication - Abstract

April 02, 2020

Journal of Controlled Release

D. Chatzikleanthous, S.T. Schmidt, G. Buffi, I. Paciello, R. Cunliffe, F. Carboni, M.R. Ro...

Publication - Abstract

December 31, 2019

Vaccines

C.B. Roces, M.T. Hussain, S.T. Schmidt, D. Christensen and Y. Perrie

Publication - Abstract

May 31, 2019

Vaccine

R.A. Feldman, R. Fuhr, I. Smolenov, A. Ribeiro, L. Panther, M. Watson, J.J. Senn, M. Smith...

Publication - Summary

May 18, 2019

Annals of Hematology

N. Jyotsana, A. Sharma, A. Chaturvedi, M. Heuser et al.

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What is Genetic Medicine