Company Developing RNA for General Drug Use

In summary, Moderna Therapeutics has been quietly working on developing RNA for drug use and has received a significant amount of investment capital. Their goal is to create injectable RNAs that can be translated into specific proteins, which has potential for various applications. They have made notable technological advancements to make this a more feasible approach, including the hiring of Melissa Moore, a prominent researcher in the field. Despite the challenges faced by other RNA therapeutic developments, Moderna's technologies show promise in addressing these obstacles. They have already published successful trials for their mRNA vaccines.
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BillTre
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Moderna Therapeutics has apparently been working on developing RNA for drug uses with a minimum of publicity, publishing mostly just patents and getting a lot of investment capital.
Their goal is to make injectable RNAs that will be translated into specific proteins.
This approach could be useful for many purposes.
Seems they have made several technological developments to make this more feasible (news to me).

Here is a rather long and interesting Science magazine News article about it.
 
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I was unaware that Melissa Moore had left her position at UMass to work for Moderna. That's too bad–I enjoyed reading the papers from her lab.

I had heard of Moderna previously (maybe b/c I used to work in the Boston area), and there's definitely a lot of promise to what they're doing (as evidence by the amount of venture capital they have amassed). However, may other efforts to develop RNA therapeutics have faced significant challenges. For example, no one has really be able to develop RNAi into a useful therapy, and the one RNA-based therapeutic that I know of, eteplirsen from Sarepta, has looked like it lacks efficacy in the clinic (one reason to be skeptical that CRISPR will have immediate clinical applications). However, Moderna's technologies seem to be able to address some of the problems plaguing RNA therapeutics, so perhaps they will be able to succeed where so many others have failed.
 
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1. What is RNA and how does it differ from DNA?

RNA, or ribonucleic acid, is a type of genetic material found in all living cells. It is similar to DNA in that it is made up of nucleotides, but it differs in its structure and function. RNA is single-stranded, while DNA is double-stranded, and RNA is involved in protein synthesis while DNA stores genetic information.

2. How does RNA-based drug development work?

RNA-based drug development involves using RNA molecules to target and treat specific diseases or conditions. These molecules can be designed to bind to specific proteins or genes, either blocking their function or promoting it. This approach can be more targeted and precise compared to traditional drug development methods.

3. What potential benefits does RNA-based drug development offer?

RNA-based drug development has several potential benefits, including increased specificity and efficacy, reduced side effects, and faster development time. Additionally, RNA-based drugs can target a wider range of diseases, including those that are difficult to treat with traditional methods.

4. What challenges does RNA-based drug development face?

While RNA-based drug development shows promise, there are still several challenges to overcome. One of the main challenges is delivering the RNA molecules to the target cells without degradation. Additionally, the high cost of production and potential off-target effects are also areas of concern.

5. What are some potential future developments in RNA-based drug development?

Scientists are continually researching and developing new methods and techniques for RNA-based drug development. Some potential future developments include improved delivery methods, the use of CRISPR technology to target specific genes, and the development of personalized RNA-based treatments based on an individual's genetic profile.

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