RANARX.COM

RaNA

Programs & Pipeline. The basis of our drug development approach. Learn about careers at RaNA Therapeutics. Areas where we're making a difference. GO TO NEWS and EVENTS. RaNA Therapeutics is pioneering the discovery of a novel, multi-dimensional approach that targets RNA within cells in the body to selectively activate protein expression. LEARN ABOUT OUR PLATFORM. Meet the RaNA Team. We are innovative and fearless, taking bold steps and pushing boundaries to pioneer groundbreaking science.

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RaNA | ranarx.com Reviews

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Programs & Pipeline. The basis of our drug development approach. Learn about careers at RaNA Therapeutics. Areas where we're making a difference. GO TO NEWS and EVENTS. RaNA Therapeutics is pioneering the discovery of a novel, multi-dimensional approach that targets RNA within cells in the body to selectively activate protein expression. LEARN ABOUT OUR PLATFORM. Meet the RaNA Team. We are innovative and fearless, taking bold steps and pushing boundaries to pioneer groundbreaking science.

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RaNA | ranarx.com Reviews

https://ranarx.com

Programs & Pipeline. The basis of our drug development approach. Learn about careers at RaNA Therapeutics. Areas where we're making a difference. GO TO NEWS and EVENTS. RaNA Therapeutics is pioneering the discovery of a novel, multi-dimensional approach that targets RNA within cells in the body to selectively activate protein expression. LEARN ABOUT OUR PLATFORM. Meet the RaNA Team. We are innovative and fearless, taking bold steps and pushing boundaries to pioneer groundbreaking science.

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Therapeutic Potential | RaNA

http://ranarx.com/therapeutic-potential

Programs & Pipeline. RaNA’s multi-dimensional approach enables the activation of the vast majority of genes. Making our platform applicable to a broad range of diseases that have never had treatment options before now. The promise of RaNA’s platform. For patients who have otherwise have had few or no approved treatment options is what makes us so fearless in our pursuits. ACTIVATING RNA TO CHANGE THE TREATMENT PARADIGM.

2

Programs & Pipeline | RaNA

http://ranarx.com/programs-pipeline

Programs & Pipeline. Programs & Pipeline. Our technologies enable highly selective targeting and upregulation or increased expression of the vast majority of genes. RaNA is currently focused on developing treatments for a number of diseases, with lead programs in cystic fibrosis and ornithine transcarbamylase (OTC) deficiency, a urea cycle disorder. ACTIVATING RNA TO CHANGE THE TREATMENT PARADIGM.

3

Our Science | RaNA

http://ranarx.com/our-science-2

Programs & Pipeline. People and patients drive our science. RaNA is dedicated to advancing the treatment paradigm by spearheading the discovery of revolutionary RNA-targeted medicines that selectively activate gene expression within cells in the body. Our approach has broad therapeutic potential. Opening up a vast number of hard-to-treat disease targets with significant need. RaNA’s Multi-dimensional Approach. What’s so important about IncRNA? Vast majority of the genome is transcribed as lncRNA. The pro...

4

Partnerships | RaNA

http://ranarx.com/partnerships

Programs & Pipeline. Collaboration at the core. Drawing on our diverse backgrounds, we collaborate as a team with one goal in mind: to help people. RaNA’s technology has broad therapeutic potential to treat a wide range of diseases. Including rare genetic disorders. RaNA’s goal is to generate new, highly selective therapeutics, with lead programs in cystic fibrosis and ornithine transcarbamylase (OTC) deficiency, a urea cycle disorder. Please contact us at partnering@ranarx.com.

5

Our Science | RaNA

http://ranarx.com/our-science

Programs & Pipeline. People and patients drive our science. RaNA is dedicated to advancing the treatment paradigm by spearheading the discovery of revolutionary RNA-targeted medicines that selectively upregulate gene expression within cells in the body. Our approach has broad therapeutic potential. Opening up a vast number of hard-to-treat disease targets with significant need. What’s so important about IncRNA? Strong emerging evidence that lncRNA is associated with disease. In patients with certain dise...

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FARA - Scientific Conferences

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Provided by OpenGlobal E-commerce. Please wait while your page loads . News and Press Releases. Clinical Network and Trials. For the Pharmaceutical Professionals. Autopsy and Tissue Donation. Message for New Families. Basics of Drug Development. Participation in Clinical Trials. Family Support and Resources. What is Friedreich's Ataxia? News and Press Releases. Clinical Network and Trials. For the Pharmaceutical Professionals. Autopsy and Tissue Donation. Message for New Families. Friedreich’s Ataxia Res...

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Programs & Pipeline. The basis of our drug development approach. Learn about careers at RaNA Therapeutics. Areas where we're making a difference. GO TO NEWS and EVENTS. RaNA Therapeutics is pioneering the discovery of a novel, multi-dimensional approach that targets RNA within cells in the body to selectively activate protein expression. LEARN ABOUT OUR PLATFORM. Meet the RaNA Team. We are innovative and fearless, taking bold steps and pushing boundaries to pioneer groundbreaking science.

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