List of RNA Therapies Companies with Phase 3 Active Clinical Trial - 18

4DMT is a clinical stage biopharma company inventing and developing innovative products to unlock the full potential of genetic medicine to treat large market and rare diseases. Our vector discovery platform Therapeutic Vector Evolution harnesses the power of directed evolution, a Nobel Prize-winning technology, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors with improved therapeutic profiles compared to wild-type vectors. Our product design, development and manufacturing engine empowers us to efficiently create our valuable and diverse product pipeline. This combination of bold innovation and relentless execution gives 4DMT the capability to revolutionize genetic medicines and to strive for potential curative therapies. We have built a deep portfolio of genetic medicine product candidates, with five product candidates in clinical trials for seven patient populations. We are taking genetic medicine to new heights and into new disease areas to overcome challenges not addressed by existing medicines. This incredible challenge drives our relentless and talented team. Our people are the essence of our inspiring, innovative and collaborative environment that encourages individual contributions and provides opportunities for career development. We seek the best in the industry who identify with our mission and our bold and innovative results-driven approach. Our 4 Guiding Principles drive the way we work together: - Dare to Cure - Break Boundaries - Beyond Yourself - Prepare & Execute Relentlessly Join us in creating the future of genetic medicine.

ADARx Pharmaceuticals is a newly launched next-wave genetic medicine company focusing on oligonucleotide therapeutics for inhibition, degradation and base editing of mRNA transcripts. With RNA as the target of our drug discovery, we are capable of working on a wide range of diseases and are currently focusing on genetic, cardiometabolic, and central nervous system (CNS) diseases. We are driven by our mission to turn cutting-edge science into life-saving therapeutics and our desire to provide hope for patients with intractable disease.

Alnylam is the world's leading RNAi therapeutics company and the first and only company to bring RNAi-based medicines to market. We are developing RNAi (RNA interference) as an innovative, entirely new class of medicines to treat rare genetic, cardio metabolic, acute hepatic infectious and central nervous system (CNS) and ocular diseases. Alnylam was founded in 2002 based on a Nobel Prize-winning breakthrough in biology – the discovery of RNAi, and a bold vision that this discovery could be used to silence disease-causing genes upstream of today’s medicines. We are relentless in our pursuit of new treatments because we believe that RNAi therapeutics can be used to treat many diseases for which treatment options do not exist or are inadequate. Alnylam is turning scientific possibility into reality - in 2018, the FDA and EMA approved of our first product, ONPATTRO (patisiran) which is also the first-ever approved RNAi therapeutic. We now have three additional medicines on the market: GIVLAARI (givosiran), OXLUMO (lumasiran) and LEQVIO (inclisiran) in partnership with Novartis). Our robust pipeline of investigational medicines includes multiple programs in late-stage and early-stage clinical development. We are a global and diverse company of more than 1,700 people. We pride ourself on fostering a fun, inclusive and dynamic work environment where employees can recognize their fullest potential. That's why we've been named a Science Magazine Top Employer 3 years in a row ('19-'21), a Boston Globe Top Place to Work 7x in a row ('15-'21) and one of Fast Company's Best Workplaces for Innovators for 2021.

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

BeBetter Medicine Technology Co., LTD. is a rapid growth novel drug discovery and development company located in Guangzhou, China. It was founded in 2012 by overseas returnees and aims to create advanced novel medicines to treat cancer and other severe diseases. The company has strong expertise and outstanding facilities to conduct comprehensive pharmaceutical research from drug design, synthesis to clinical development. Currently there are eight innovative drug candidates with own IP rights are in clinical or IND stages. Among them, BEBT-908 has entered the pivotal phase II trial for the treatment of lymphoma. BEBT-209 and BEBT-109 are in the phase Ib/II trial for the treatment of breast cancer and lung cancer, respectively. The company is expected to have a number of new drugs on the market in the next few years.

BIOCAD is Russia's leading innovative biotechnology company; it combines a world-class research and development center, ultra-modern pharmaceutical and biotechnological manufacturing facilities, as well as preclinical and clinical research infrastructure compliant with international standards. BIOCAD is one of the world`s few full-cycle drug development and manufacturing companies, from new molecule discovery and genetic engineering to large-scale commercial production and marketing support. BIOCAD`s medicines are dedicated to treat complex health conditions such as cancer, HIV and Hepatitis C infections, multiple sclerosis and other disorders.

BioNTech is a global next generation immunotherapy company aspiring to translate science into survival. Around 6.100 pioneers currently work with us, employing a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. Our broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor (CAR) T cells, several protein-based therapeutics, including bispecific immune checkpoint modulators, targeted cancer antibodies and antibody-drug conjugate (ADC) therapeutics, as well as small molecules.   Based on our deep expertise in mRNA vaccine development and in-house manufacturing capabilities, we are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its diverse oncology pipeline. To learn more about us, please visit www.BioNTech.com. Our privacy statement can be found here:  https://www.biontech.com/int/en/home/data-privacy-statement.html Our imprint can be found: https://www.biontech.com/int/en/home/imprint.html

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.

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.

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 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.

We are ProQR, a biotechnology company dedicated to changing lives by developing RNA therapies for rare genetic diseases. We focus our drug development mainly on a group of blinding disorders affecting the retina, called inherited retinal diseases.

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 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.

Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases. The company is pursuing an entirely new approach to treating severe diseases. We are initially focused on diseases of the central nervous system and the eye, with an emphasis on haploinsufficiencies, which are diseases caused by a loss of about half of the normal protein function. Rather than replace, repair or edit faulty genes, we aim to increase – or stoke – protein output from healthy genes to restore functional protein levels using our proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform. In all, Stoke has identified ~6500 genes with a TANGO signature, representing broad potential for our approach to help people with severe diseases. The early results have been promising. Stoke’s first medicine in development, STK-001 has demonstrated positive Phase 1/2 results for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy that is associated with a number of debilitating non-seizure comorbidities. Behind that, is STK-002, a potential treatment for autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Clinical studies of STK-002 are anticipated to start in 2024. The company is pursuing potential treatments for three additional neurodevelopmental disorders, including Syngap1 and Rett syndrome, in collaboration with Acadia Pharmaceuticals. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts.

Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community

Vir Biotechnology, Inc. (in Europe, Humabs BioMed, https://bit.ly/46lQGN2) is an immunology company focused on powering the immune system to address unmet patient needs in infectious disease and beyond. Our powerful R&D engine combines our proprietary monoclonal antibody (mAb) platform with machine learning and artificial intelligence-enhanced capabilities, enabling us to engineer our rich database of human antibodies and deliver transformative medicines for diseases with significant global burden. Our current pipeline consists of multiple clinical candidates targeting hepatitis B and hepatitis delta viruses and human immunodeficiency virus, including a potential functional cure for chronic hepatitis B and treatment for chronic hepatitis delta. Our portfolio of broad, potent investigational monoclonal antibodies also targets influenza A and B viruses, COVID-19, respiratory syncytial virus/human metapneumovirus and human papillomavirus, and our investigational T cell vaccine aims to prevent HIV. Explore our full pipeline: https://bit.ly/3tr7FPm We collaborate with some of the world’s leaders in industry as well as government agencies and non-profit organizations enabling us to rapidly advance cutting-edge scientific discoveries and innovative technologies with the potential to benefit people around the world. We are proud to have been ranked the fastest-growing company in North America on the 2022 Deloitte Technology Fast 500™. Join us as we pursue our mission to transform lives. It has come to our attention that there have been various recruitment scams targeting job seekers on LinkedIn. Vir Biotechnology, Inc. is committed to your privacy and will never request sensitive personal information including banking details through email, text message, social media or video software platforms. We also will never request a financial commitment from a candidate as part of the application process. Learn more about Vir’s recruiting process: https://bit.ly/461EgJN

Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas. Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company is conducting two global Phase 3 clinical trials (THRIVE and THRIVE-2) to evaluate the safety and efficacy of VRDN-001 in patients with active and chronic TED. Viridian’s goal is to advance VRDN-001 as a potential best-in-class intravenous therapy followed by VRDN-003 as a potential first- and best-in-class subcutaneous therapy for the treatment of TED. In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has the potential to be developed in multiple autoimmune diseases.