List of RNA Therapies Companies in California - 33

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.

Ainos Inc. is a diversified healthcare company focused on the development of novel point-of-care testing (POCT), low-dose VELDONA interferon therapeutics, women's health, pneumonia, Ainos Pen, AI Nose, and other medical technologies. The company was founded in 1984 and is engaged in developing innovative medical technologies for point-of-care testing and safe and effective treatments for various health conditions.

Alida Biosciences is an early-stage, VC backed startup in San Diego focused on developing novel genomic analysis tools. The company’s technology will advance the understanding of RNA biology and provide actionable information for improving human health.

Our strategy is to develop pharmacologically optimized drug candidates, which are first assessed in healthy volunteers and patients as monotherapy prior to their use in combination regimens designed to achieve improved treatment outcomes in patients. Our initial areas of focus are viral and liver diseases where our team can leverage their in-depth knowledge and expertise to develop potentially best-in-class combination regimens for each disease area with significant unmet medical need.

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR® mRNA Technology (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus developed Kostaive®, the first self-amplifying messenger RNA (sa-mRNA) COVID vaccine in the world to be approved. Arcturus has an ongoing global collaboration for innovative mRNA vaccines with CSL Seqirus, and a joint venture in Japan, ARCALIS, focused on the manufacture of mRNA vaccines and therapeutics. Arcturus’ pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF), along with its partnered mRNA vaccine programs for SARS-CoV-2 (COVID-19) and influenza. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus’ technologies are covered by its extensive patent portfolio (over 400 patents and patent applications in the U.S., Europe, Japan, China, and other countries).

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.

Developed a proprietary platform that holds the potential to discover novel structure targets across the transcriptome and for the design of RNA-targeted small molecules, RNA-based medicines and RNA tools.

Benitec is a biotechnology company developing a proprietary therapeutic technology platform that combines RNA interference (RNAi) with gene therapy with a goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration. Benitec is using its technology, called DNA-directed RNA interference (ddRNAi) to develop a pipeline of product candidates for the treatment of several chronic and life-threatening human diseases , including oculopharyngeal muscular dystrophy (OPMD) and hepatitis B. By combining the specificity and gene silencing effect of RNAi with gene therapy, ddRNAi has the potential to produce long-lasting silencing of disease-causing genes from a single administration, which could minimize the requirement for patients to take regular doses of medicine. Benitec’s objective is to become the leader in discovering, developing, clinically validating and commercializing ddRNAi-based therapeutics for a range of human diseases with high unmet clinical need or large patient populations and, as a result, provide a better life for patients with these diseases.

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of life-threatening genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology.

DxTerity is bringing the power of precision medicine to autoimmune disease. We are pioneering the use of RNA-based immune system profiling to better understand the root causes of immune mediated conditions. Our goal is to provide more personalized care for autoimmune sufferers and empower patients with more detailed information about their condition so they can better navigate their complex health journey.

Eclipsebio develops first-in-class technologies, analyses, and platforms for the development of tomorrow’s RNA-based and RNA-targeting therapies. With our extensive experience in supporting early-stage basic research to evaluating preclinical vaccines and gene therapies, we provide unparalleled support for obtaining deep insights into RNA and therapeutic biology. We offer our solutions as end-to-end partnerships, including custom assay development, for biopharma and biotech companies and as a la carte services. Our areas of support include: - The optimization and characterization of RNA-based vaccines and therapeutics through our eMERGE platform - Multiomic characterization of small molecule and small oligonucleotide targets - Robust validation of on-target, and the identification of off-target, effects from small molecule and small oligonucleotide drugs - Deep profiling of RNA for groundbreaking discoveries in academic research Contact us today to discover how we can provide you with the insights needed for RNA success.

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.

ExcepGen is a venture-backed, early stage biotechnology company located in San Francisco. The company is focused on solving fundamental underlying problems in drug discovery and applying those solutions to create a new generation of therapeutics.

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.

Junevity is a biotech rewinding complex metabolic disease. Based on 6 years of breakthrough research at UCSF, Junevity's REWIND platform identifies high-level regulators of metabolic disease with known pharmacological pathways. Junevity is advancing multiple therapeutic programs towards a new era of medicines.

Kerna Labs is unlocking the full potential of mRNA as the universal toolkit for genetic medicine.

Nitto BioPharma is a clinical stage biopharmaceutical company located in San Diego focused on the discovery and development of novel RNA-based therapeutics for the treatment of fibrosis and other unmet medical needs. The company offers unique drug and siRNA delivery solutions by designing novel biodegradable delivery vehicles with the combined ability to target specific cells.

Nutcracker Therapeutics is an RNA therapeutics company that has combined the power of advanced engineering with high-precision biosynthesis to deploy a complete RNA therapeutics platform. Armed with this high-tech advantage, we have developed a wholly owned pipeline of RNA therapeutic programs and established partnership initiatives with top clinical investigators at several leading institutions across the globe. With our platform’s ability to accelerate the development of life-changing RNA therapeutics, we seek to advance breakthrough RNA therapies at high velocity through all stages of development across a variety of indications. Our technology platform has the potential to significantly reduce costs and cycle times for RNA therapeutic development, with dramatic advantages in capacity scaling and point-of-care delivery over other RNA manufacturing approaches.

Phalanx Biotech Group provides genomics research products and services worldwide to academic, pharmaceutical and biotech communities. We developed the OneArray® line of high-quality and affordable microarrays for expression profiling of whole genomes, microRNA, and custom targets. Our service specialists offer comprehensive services including sample preparation, expression analysis, and qPCR validation. In 2017, Phalanx is pleased to announce that it has added Next Generation Sequencing Services (NGS) and corresponding NGS Bioinformatics to its service offerings.

Neutralizing Antibody Discovery

Radar Therapeutics is a biotech company developing programmable precision therapeutics using molecular RNA sensors. The company has raised $13.4 million in seed funding to support its work in developing smart, programmable medicines. Radar Therapeutics is also the winner of the first-ever Amgen Bakar Labs Diversity, Inclusion, and Belonging Award. The company is building a business around medicines that use RNA sensors—mRNAs that gate their expression based on other RNAs in the cell.

RealSeq Biosciences’ core expertise includes innovative proprietary technologies for bias-free small RNA/miRNA NGS library construction, targeted NGS tools, and cf-RNA analysis (liquid biopsy) that form the basis of the Company’s life science research programs and product development.

Regulus Therapeutics Inc. (Nasdaq:RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkersSM biomarkers platform and a rich intellectual property estate to retain its leadership in the microRNA field. Regulus is located in San Diego, California and is led by a seasoned executive team experienced in corporate management, business, science, drug discovery and development. Regulus'​ scientific advisory board consists of world-class scientists and some of the foremost authorities in the field of microRNA research.

Rejuvenation Technologies is a biotechnology company that developed nucleoside-modified TERT mRNA to safely and rapidly extend telomeres. The company is focused on developing mRNA therapies to extend the healthspan by preventing and treating chronic age-related diseases.

Replicate Bioscience is amplifying the power of RNA therapeutics by pioneering novel self-replicating RNA (srRNA) technology. Replicate’s off-the-shelf srRNAs contain two components: virally derived genetic code to drive controlled and self-limiting amplification, and the RNA encoding therapeutic proteins. Our library of viral vectors, selected for driving robust and sustained protein expression and orders-of-magnitude improved performance over linear mRNA, allow for the development of treatments in applications including oncology, infectious disease, and autoimmunity. Differentiated by a team of srRNA experts, a customizable library of synthetic srRNA vectors, and end-to-end development capabilities, Replicate is uniquely positioned to finally expand the reach of RNA treatments toward widespread use in infectious disease, immuno-oncology, autoimmune disease, and more.

Sapu Bioscience is an immuno-oncology company dedicated to the development of first in class RNA therapeutics as well as small molecule drugs against cancer and infectious diseases. OT-101, the lead immuno-oncology drug candidate of Sapu Bioscience, is a first-in-class anti-TGF-βRNA therapeutic that exhibited single agent activity in relapsed/refractory cancer patients in clinical trial settings. OT-101 also has activity against SARS-CoV-2. Sapu Bioscience is seeking to leverage its deep expertise in drug development to improve treatment outcomes and survival of cancer patients with a special emphasis on rare pediatric cancers. Sapu Bioscience has rare pediatric designation for DIPG (OT-101).

Sirius is developing transformative siRNA therapeutics for patients with chronic diseases globally. Founded in 2021, Sirius established an innovation center in the United States and a translational medicine center in China dedicated to state-of-the-art solutions for the treatment and management of chronic diseases.

SomaGenics is a privately held biotech company with offices and laboratories located in Santa Cruz, CA. We specialize in small RNA technologies, including innovative RNAi-based therapeutics using sshRNA molecules, and diagnostics/biomarker detection (NGS, quantitative PCR) of small or fragmented RNA, including microRNA.

Switch Therapeutics is an emerging preclinical stage biotechnology company pioneering a new type of medicine that integrates nucleic acid nanotechnology and RNA interference (RNAi) science with the goal of treating a range of diseases – affecting the central nervous system and systemic indications – with significant unmet needs. Switch’s novel gene knockdown approach is based on technology developed by renowned researchers in the field of RNA from Caltech, Harvard and City of Hope. Based on the scientific discovery of Switch’s co-founders, the company has developed a novel proprietary platform known as CASi (Conditionally Activated siRNAs) that combines advantageous properties of both single and double-stranded RNAs in a single molecule, allowing for cell selective RNAi activity. To date, Switch has raised $52M in funding through Series A, and the company’s South San Francisco based team has continued to grow as its research has advanced.

Takara Bio USA, Inc. (formerly Clontech Laboratories, Inc.), a Takara Bio company, develops, manufactures, and distributes a wide range of life science reagents under the Takara®, Cellartis®, and Clontech® brands. Our products support applications including stem cell research; NGS; gene discovery, regulation, and function studies; protein expression and purification; RNAi and gene editing; and plant and food research.

We are a biotechnology company focused on restoring cells’ youthful vigor to repair damage caused by the aging process. Using a proprietary ERA™ Platform, we develop mRNA medicines that are specially formulated to instruct specific cells in the body to fight disease or repair damaged tissue. We do this by reprogramming the epigenome to restore cell function that people often lose as they age. This approach can be used to revitalize different cells throughout the body – we are researching its applicability across multiple therapeutic areas, including dermatologic conditions, immunology, osteoarthritis and ophthalmology.

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