Company Highlights: Arcturus Therapeutics

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About the Company

Arcturus therapeutics is a leading clinical-stage messenger RNA medicines company focused on the discovery, development and commercialization of therapeutics for rare diseases and vaccines. They have proprietary technologies, validating partnerships, and an experienced team with deep expertise in delivery and RNA-based therapeutics.

Medicines

mRNA: Messenger RNA can be used as protein replacement therapy to treat diseases caused by a lack of protein, or by defective proteins, such as cystic fibrosis. If a gene has a mutation that stops it from producing protein or causes it to produce defective protein, mRNA medicine can provide a healthy version of the missing protein.

siRNA, miRNA, and ASO: RNA interference is a natural mechanism used by the body to turn off genes without permanently changing DNA, and it controls how and when genes are expressed into proteins. RNAi occurs when a small RNA, such as small interfering RNA, antisense RNA or microRNA, binds to a mRNA and stops a cell from translating that mRNA into a protein product.

CRISPR, TALEN, and meganucleases: Gene editing tools such as CRISPR, TALEN, and meganucleases, use a mixture of enzymes, RNA and DNA to cut out mutated DNA and replace it with a healthy sequence. This can treat genetic cause of a disease.

Replicon RNA, mRNA and DNA: RNA-based vaccines are a potential new class of RNA medicines. RNA vaccines can be developed more rapidly than traditional vaccines in response to infectious disease outbreaks. Unlike traditional vaccines, RNA vaccines can also be effective in infected patients. Therefore, these vaccines can be therapeutic as well as prophylactic.

Website: Arcturus Therapeutics

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