List of RNA Therapies Companies with Phase 1 Active Clinical Trial - 12

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About

Comanche Biopharma

Comanche Biopharma

Concord, Massachusetts, United States of America

Comanche is a biopharmaceutical company developing novel siRNA molecules for the treatment of preeclampsia. Our purpose is to lower the risks of pregnancy and prematurity worldwide by safely sustaining natural pregnancy and allowing for fetal maturation. We envision a world where all pregnant women have access to safe and effective therapies that are evidence-based, cost-effective and sustainable. Our mission is to prevent or treat preeclampsia at its root cause.

CureVac

CureVac

Tübingen, Baden-Württemberg, Germany

CureVac (Nasdaq: CVAC) is a pioneering multinational biotech company founded in 2000 to advance the field of messenger RNA (mRNA) technology for application in human medicine. In more than two decades of developing, optimizing, and manufacturing this versatile biological molecule for medical purposes, CureVac has introduced and refined key underlying technologies that were essential to the production of mRNA vaccines against COVID-19, and is currently laying the groundwork for application of mRNA in new therapeutic areas of major unmet need. CureVac is leveraging mRNA technology, combined with advanced omics and computational tools, to design and develop off-the-shelf and personalized cancer vaccine product candidates. It also develops programs in prophylactic vaccines and in treatments that enable the human body to produce its own therapeutic proteins. Headquartered in Tübingen, Germany, CureVac also operates sites in the Netherlands, Belgium, Switzerland, and the U.S. Further information can be found at www.curevac.com.

Exai Bio

Exai Bio

3350 W Bayshore Rd, Palo Alto, California 94303, US

Exai Bio is a next-generation liquid biopsy company. Our mission is to enable a world where cancer can be detected early, diagnosed accurately, treated in a personalized and targeted way, and ultimately cured. The company's proprietary RNA and artificial intelligence-based liquid biopsy platform delivers clinical insights into cancer biology to enable the earliest, most accurate diagnosis of cancer. Exai Bio was founded with technology from the UCSF laboratory of Dr. Hani Goodarzi, and consists of veteran leaders in the RNA biology, liquid biopsy, and artificial intelligence/machine learning fields from both academia (UCSF, Stanford, MIT) and industry (Bluestar, CareDx, Evidation, Google, 23andMe, Deep Genomics). Exai Bio is supported by leading life sciences and tech investors including Section 32, Casdin Capital, and Two Sigma Ventures, who have been integral to advancing cancer diagnostics, and artificial intelligence and machine learning technologies. We know that people are our greatest asset and we are rapidly building a rock-star team of talent. Our people are driven by the mission, inspired by the vision of the future, and are committed to living our core values (integrity, openness, teamwork, exploration, and kindness) that connect us to our work and to one another. What We Value Our core values represent what we stand for as a team. This is what we believe in. Everyday, we strive to align our behaviors to these core values in everything we do. Integrity – Our thoughts and actions are true to science and to people. We do the right thing. Openness – We recognize and respect our differences – and embrace learning from them. Teamwork – We are team players who trust and respect each other. Exploration – We are explorers, putting curiosity into action. Kindness – We are kind whenever possible – and we believe it's always possible.

Excision BioTherapeutics

Excision BioTherapeutics

San Francisco, California, United States of America

Excision BioTherapeutics is a private company focused on developing advanced gene editing therapeutics to address the medical need for the treatment of life-threatening disease caused by viral infections. Our mission is to advance gene editing therapeutics for our indications into safe and efficacious medicines that will greatly improve the lives of infected individuals around the world.

Geneoscopy

Geneoscopy

2220 Welsch Industrial Ct, St. Louis, MO 63146, US

Geneoscopy is a life sciences company with the mission to empower patients and providers to transform gastrointestinal health through innovative diagnostics. Geneoscopy's lead diagnostic uses stool-derived eukaryotic RNA (seRNA) to detect colorectal cancer and precancerous adenomas. This device was awarded a Breakthrough Device Designation from the FDA for its ability to reduce morbidity associated with colorectal cancer through advanced adenoma detection. Our initial prospective clinical study demonstrates that the diagnostic can detect these lesions with greater sensitivity than all existing noninvasive screening tests, thereby, serve as a potential aid in early detection and prevention of cancer. Geneoscopy is also developing diagnostics for the diagnosis and management of inflammatory bowel disease in partnership with biopharmaceutical companies and leading academic research institutions.

Innovac Therapeutics

Innovac Therapeutics

Cambridge, Massachusetts, United States

Innovac Therapeutics is a biotechnology company dedicated to developing innovative vaccines for patients with unmet medical needs, using our proprietary RNA technology.

Mirxes

Mirxes

Singapore, Singapore

Mirxes is a Singapore-headquartered biotechnology company whose mission is to save lives through early, actionable, and personalized diagnoses across the care continuum. We are Asia-centered, starting with Southeast Asia, and have global ambitions. We have research, development, manufacturing, and clinical diagnostic operations in Singapore, USA, Japan, and China. We have sales and distribution networks worldwide. Our flagship product is GASTROClear, the world’s first molecular blood test for early detection of gastric cancer, able to detect around 90% of patients with Stage I and II cancers. We continue to build a comprehensive cancer portfolio with early detection tests for lung and thyroid cancer (2022), colorectal and breast cancer (2023), liver and ovarian cancer (2024). We are concurrently building a precision oncology portfolio to provide clinical insights for the complete patient journey. MiRXES was spun off from A*STAR’s Bioprocessing Technology Institute in 2014 to commercialize an industry-leading qPCR-based technology for microRNA detection. Our ID3EAL discovery tool and workflow has been adopted by top academic and industry partners globally and is backed by the world’s first industry standard for microRNA-base diagnostics. Our versatile platform technology and capabilities have broad applications in the discovery of biomarkers and diagnosis of diseases. We are starting with early cancer detection, but we will also be addressing unmet clinical needs in cardiovascular, metabolic, and infectious diseases. We responded to COVID-19 by leveraging our capabilities to mass produce and distribute over 5 million units of the Fortitude COVID-19 RT-PCR test. This made-in-Singapore diagnostic test, developed by A*STAR and Tan Tock Seng Hospital, has been deployed in 13 Singapore hospitals and clinical labs and exported to over 45 countries worldwide. Post-COVID, we will leverage this network to commercialize multi-cancer early detection tests.

NeoCura

NeoCura

生命园路, 29号1幢3层, 北京, 102206, CN

NeoCura is a high-tech enterprise that relies on AI technology to create an RNA technology platform and conduct R&D of innovative drugs. The company has built multi-omics big data acquisition platforms and multiple bioomics databases. It uses AI and bioinformatics technology to conduct in-depth drug target mining and fully automated drug design for innovative RNA technology platform upgrades and drug research. It has established leading R&D centers and production centers in Beijing, Shenzhen, and Guangzhou to support pipelines development, platform upgrades, and clinical demand.

OliX Pharmaceuticals

OliX Pharmaceuticals

Gyeonggi, Korea

OliX Pharmaceuticals is a clinical stage pharmaceutical company developing therapeutics based on the science of RNAi to address a wide variety of illnesses. The Company has developed core platforms (asymmetric siRNA, cell-penetrating asiRNA) targeting locally administrable diseases, such as hypertrophic scars, hair loss, and age-related macular degeneration (AMD). OliX's third RNAi therapeutic platform, GalNAc-asiRNA, targets multiple liver diseases, including NASH/diabetes and HBV. For the general welfare of human beings, OliX is constantly endeavoring to become a leader in the global biotechnology industry by innovating and expanding its pipeline, even to the undruggable diseases.

Phio Pharmaceuticals

Phio Pharmaceuticals

Marlborough, Massachusetts, United States

Phio Pharmaceuticals Corp. (Nasdaq: PHIO) is a clinical stage biotechnology company whose proprietary INTASYL™ RNAi technology makes immune cells more effective in killing tumor cells. INTASYL is the only self-delivering RNAi technology focused on immuno-oncology therapeutics. INTASYL drugs precisely target specific proteins that reduce the body’s ability to fight cancer, without the need for specialized formulations or drug delivery systems. Phio was co-founded by Nobel Laureate Craig Mello, who was awarded the Nobel prize in 2006 for his discovery of RNAi, which has broad therapeutic applications. Our founders opened up a whole new field of biology when they discovered RNAi, and we continue their pioneering efforts today through our work with INTASYL. We’re committed to discovering and developing innovative cancer treatments for patients, creating new pathways for a cancer-free future. For additional information, visit the Company's website, www.phiopharma.com. #INTASYL #siRNA #RNAi #Phio #immunooncology

Ribonova

Ribonova

Wynnewood, Pennsylvania, United States

RiboNova is a privately-held biopharmaceutical company based in the Lankenau Institute for Medical Research near Philadelphia, Pennsylvania. Their product pipeline includes a Phase-2 ready small molecule drug for the treatment of all forms of genetically-confirmed mitochondrial disease and a proprietary drug discovery platform that targets transfer RNA with novel precision medicines for the treatment of mitochondrial and other diseases.

Scipher Medicine

Scipher Medicine

Waltham, Massachusetts, United States

Scipher Medicine is building the future of patient treatment. Most patients who are prescribed blockbuster therapies today don’t respond to the treatment, costing the health care industry billions in wasted drugs while patients continue to suffer. Scipher's platform identifies which drug will work based on the patient's fundamental disease biology, and not based on symptoms, disease classification, or medical bias. A simple test predicts which drug he/she will respond to, ensuring that most optimal treatment is prescribed from day one. The molecular data generated by our tests is then used to fuel novel target discovery to address a clear unmet medical need in patients who do not respond to any existing therapy.