RNA Clinical Trials Articles
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A (More) Mature mRNA Therapeutics Industry: 4 Best Practices To Get There
9/27/2022
Throughout the two-day mRNA Therapeutics Summit, there were four key takeaways that clearly demonstrated how the mRNA industry could incrementally achieve one speaker's industry prediction that, within the next 10 years, every person in the world will have had the opportunity to benefit from mRNA.
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Breakdown Of Cell & Gene Live: Advancements In Targeted mRNA Delivery
9/21/2022
During our Cell & Gene Live, Advancements in Targeted mRNA Delivery, Dr. Murat Kalayoglu, CEO of Cartesian Therapeutics, Walter Strapps, Ph.D., Co-Founder and CEO of Carver Biosciences, and I spent the hour talking about the delivery of mRNA-based therapeutics. Our expert panelists discussed the specific challenges and opportunities in delivering conventional RNA therapeutics clinically, as well as the specific challenges and opportunities in delivering the RNA cell therapies clinically. You can view the full-length presentation here. For your convenience, we’ve broken down the hour into bite-size chunks based on the important topics we discussed throughout the hour.
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Capstan Therapeutics' Next Steps For In Vivo CAR Therapies
9/19/2022
Capstan Therapeutics is a brand-new biotech company dedicated to developing and delivering precise in vivo cell engineering to patients. With $165 million in financing, Capstan Therapeutics aims to build on the foundational insights of leaders in mRNA and cell therapy from the University of Pennsylvania. Laura Shawver, Ph.D., leads the company as President and CEO, and on the heels of the biotech’s recent news, I caught up with her to learn more about the company’s plans for developing in vivo CAR therapies.
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Don't Get Uppity: Match mRNA Science To Your Capabilities — And Vice Versa
9/6/2022
This article is part one of a four-part series unpacking four of my biggest takeaways from the mRNA Therapeutics Summit, which took place in Boston on July 27 & 28, 2022.
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The Promise Of Gene Silencing To Treat Not-So-Rare Diseases
9/2/2022
In this last article in the series on siRNA, I examine its potential for treating not-so-rare and common diseases. Unlike rare diseases, which are often caused by pathological genetic mutations, common diseases may be associated with genetic variants that are not pathological. I look at cardiovascular disease and iron flux redux in particular.
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The Potential Of siRNA To Treat Rare Diseases
8/19/2022
siRNA is a precision, targeted medicine with the potential to treat many diseases that currently lack effective therapeutic options. In this second article of my series on the subject, I take a closer look at siRNA’s potential for treating rare diseases.
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The Promise Of Gene Silencing: A Primer On siRNA
8/5/2022
Small interfering RNAs (siRNAs) are short sequences of RNA that interfere with gene expression, reducing or eliminating the expression of a disease-related protein by targeting its corresponding mRNA. siRNAs could treat a wide array of diseases. How does it differ from other therapeutic modalities such as gene therapy and gene editing?
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How Can We Move RNA Forward In Our Therapeutics Arsenal?
7/18/2022
RNA technologies, including mRNA therapeutics, have emerged as a promising drug modality. However, they have unique challenges. BioPhorum has dedicated a team to investigate these challenges and this Q&A shares their practical considerations for how to move this field forward.
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Expanding RNA-Based Immune Profiling To Immune State
4/27/2022
A complete model of the tumor biology is showing promise to become a new standard for predicting patient response and helping more patients benefit from the power of immunotherapy. Expanding RNA-based immune profiling to immune state makes precision medicine more precise.
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mRNA Vaccines: Key Considerations For Development & Manufacturing
4/4/2022
As we wind down National Immunization Awareness Month, this article looks at the complexities around developing and manufacturing mRNA vaccines and how you should address those complexities. Topics include delivery, process validation, long-term effectiveness, GMP, regulatory approval, and more.