List of Oligonucleotides Companies in California - 27

ACADIA Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of small molecule drugs that address unmet medical needs in central nervous system disorders. The company offers NUPLAZID (pimavanserin) for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis; Trofinetide, a novel synthetic analog for the treatment of Rett syndrome; ACP-044, a novel first-in-class orally administered non-opioid analgesic for treating acute and chronic pain; and ACP-319, a positive allosteric modulator of the muscarinic receptor for treating cognition and schizophrenia. It is also developing pimavanserin as a treatment for dementia-related psychosis and as an adjunctive treatment for schizophrenia; and pimavanserin as an adjunctive treatment for major depressive disorder. ACADIA Pharmaceuticals Inc. was founded in 1993 and is headquartered in San Diego, California.

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.

Harnessing the power of RNA biology to find treatments for patients with unmet medical needs. Advance and utilize the cutting edge antisense technology (siRNA and ASO) to discover and develop next generation medicines for both large and rare disease indications.

Avidity Biosciences, Inc., a biopharmaceutical company, engages in the development of oligonucleotide-based therapies. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat a range of serious diseases. The company’s lead product candidate AOC 1001 is used for the treatment of myotonic dystrophy type 1, a rare monogenic muscle disease; and to design, engineer, and develop therapeutics that combine tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies to access previously undruggable tissue and cell types, and target underlying genetic drivers of diseases. In addition, it develops muscle programs, which focuses on the treatment of facioscapulohumeral muscular dystrophy, Duchenne muscular dystrophy, muscle atrophy, and Pompe diseases.

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.

Since its founding in 1992 Bioneer Corporation has established market leadership in Korea for most of the products it manufactures. Bioneer has built its business by providing researchers with superior, high quality products at excellent value. Over the years, Bioneer has developed a broad range of unique products that provide total genomic research solutions for life sciences research including genomics, nucleic acid-based molecular diagnostics and gene therapy studies. Bioneer has extensive R&D experience in developing new drugs as well as the use of siRNA and drug delivery systems, and provides unrivalled service and support for such tools. For example, through the creation, development, and commercialization of the S. pombe deletion mutant library, Bioneer provides an innovative tool for improving the development of new drugs. The S. pombe library accurately predicts the toxicity of drug candidates for anti-cancer, Hyperlipidemia and also provides molecular level mechanism analysis of natural compound drugs. The company is headquartered in South Korea with an oligonucleotide manufacturing facility in the U.S., a regional office in China, and is supported by a network of distributors throughout the world. For more information, please visit www.bioneer.com.

Capricor Therapeutics, Inc., a biotechnology company, focuses on the development of transformative cell- and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases. The company’s lead candidate, CAP-1002, an allogeneic cardiac-derived cell therapy, which has completed phase II clinical trial for the treatment of patients with late-stage Duchenne muscular dystrophy (DMD); CAP-1002, which is in Phase II clinical trial for the treatment of cytokine storm associated with COVID-19; and completed various trials investigating the use of CAP-1002 for the treatment of cardiac conditions, including heart failure and post myocardial infarction with cardiac dysfunction. It is also developing CAP-2003 that is in pre-clinical development for the treatment of trauma related injuries and conditions; and two vaccine candidates, which are in preclinical stage for the potential prevention of COVID-19. Capricor collaborates with Lonza Houston, Inc. for the development of CAP-1002, its cell therapy candidate for the treatment of DMD and other indications. The company was founded in 2005 and is headquartered in Beverly Hills, California.

Codexis is a leading enzyme engineering company leveraging its proprietary CodeEvolver® platform to discover, develop and enhance novel, high-performance enzymes and other classes of proteins. Codexis enzymes solve for real-world challenges associated with small molecule pharmaceuticals manufacturing and nucleic acid synthesis. The Company is currently developing its proprietary ECO Synthesis platform to enable the scaled manufacture of RNAi therapeutics through an enzymatic route. Codexis' unique enzymes can drive improvements such as higher yields, reduced energy usage and waste generation, improved efficiency in manufacturing and greater sensitivity in genomic and diagnostic applications.

Founded in 2001, we are the third largest peptide-focused contract research, development, and manufacturing organization (“CRDMO”) worldwide in terms of sales revenue in 2023, according to Frost & Sullivan. We are also considered one of the most comprehensive peptide-focused CRDMO globally (Frost & Sullivan), offering complete life-cycle support from early-discovery, pre-clinical research and clinical development to commercial production. We mainly provide (i) contract research organization (“CRO”) services, including peptide new chemical entity (“NCE”) discovery synthesis; (ii) contract development and manufacturing organization (“CDMO”) services, including peptide chemistry, manufacturing, and controls (“CMC”) development; and (iii) contract manufacturing organization (“CMO”) services, including peptide NCE and generic drug commercial manufacturing. We have established global operations, with projects covering over 50 countries, including major markets in the United States, China, Japan, Europe, South Korea, and Australia. We also provide customers wit peptide drug development, production, and CMC filing support services that meet regulatory requirements in major markets worldwide. In addition to peptides, we have developed a diverse project pipeline focusing on various types of complex peptide- and oligonucleotide-based new chemical entities (“NCEs”). Our peptide conjugation services seamlessly integrate our peptide and oligonucleotide platforms to produce a wide range peptide conjugates products. The introduction of our PeptiConjuXTM and PeptiNuclide LinkTechTM are dedicated to the development and large-scale manufacturing (i.e., kilogram-scale) of peptide-oligonucleotide conjugates (“POC”), peptide drug conjugates (“PDC”), and radionuclide drug conjugates (“RDC”). As of December 31, 2023, we had successfully synthesized approximately 1,800 molecules through these two platforms.

Reinventing drug development from the ground up and making on-demand oligonucleotide therapeutics possible.

Duet BioTherapeutics is an immunotherapy company creating a new category of bispecific oligonucleotide treatments designed to target dormant immune cells within the tumor microenvironment. Activation of these antigen-presenting cells removes the immunosuppressive shield around the tumor and jump-starts T cell-mediated immune responses to kill cancer cells.

Dynavax is a fully-integrated biopharmaceutical company focused on leveraging the power of the body’s innate and adaptive immune responses through Toll-like Receptor (TLR) stimulation. Dynavax develops, and commercializes novel vaccines.

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It is involved in developing neurology products that include Tominersen for Huntington’s diseases; Tofersen for amyotrophic lateral sclerosis (ALS); IONIS-MAPTRx for Alzheimer's diseases; IONIS-C9Rx for genetic form of ALS; ION859 for Parkinson’s disease; ION464 for multiple system atrophy and Parkinson’s disease; ION541 for amyotrophic lateral sclerosis; and IONIS-DNM2-2.5Rx for centronuclear myopathy. The company is also developing products for cardiometabolic disease, such as IONIS-TTR-LRx for transthyretin amyloidosis and IONIS-GHR-LRx for acromegaly; IONIS-APOCIII-LRx for familial chylomicronemia syndrome and TG- driven diseases; IONIS-APOCIII-LRx for treatment resistant hypertension; Pelacarsen for Lp(a)cardiovascular disease risk reduction; Vupanorsen for sHTG/CVDRR; IONIS-FXI-LRx for clotting disorders; ION449 for cardiovascular diseases; and IONIS-GCGRRx for diabetics. In addition, it develops IONIS-ENAC-2.5Rx for cystic fibrosis and chronic obstructive pulmonary disease; IONIS-PKK-LRx for hereditary angioedema; Danvatirsen for cancer; IONIS-TMPRSS6-LRx for beta-thalassemia and iron toxicity; IONIS-HBVRx for hepatitis B virus; IONIS-AR-2.5Rx for prostate cancer; IONIS-FB-LRx for age-related macular degeneration and IgA nephropathy; and ION357 for retinitis pigmentosa. It has a collaboration agreement with AstraZeneca; GlaxoSmithKline plc; Janssen Biotech, Inc.; Bayer AG; Novartis AG; Pfizer, Inc.; Biogen Inc.; and Roche. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.

Nitto Avecia Pharma Services is your single source for high-quality chemistry, manufacturing, and control (CMC) services for the pharma, biotech, and medical device industries. Our comprehensive suite of services streamlines processes to accelerate time-to-market. With more than 30 years’ experience, we have deep scientific expertise across a range of products, with a special focus on oligonucleotides. In partnership with Nitto Avecia Pharma Services, Nitto Avecia is the first CMO that offers a comprehensive, end-to-end oligonucleotide solution. This offers the advantage of a single, expert partner handling the entire process—from drug substance through drug product manufacturing—with flexible solutions and consistent reliability and quality.

Polymeric Supports for Nucleotide Synthesis

The comapanies' research and development efforts are focused exclusively on rare genetic neurodevelopmental disorders, a group of serious diseases with few or no treatment options.

Maze Therapeutics is translating novel genetic insights into lifesaving medicines, through an approach that integrates human genetics and functional genomics. Advances in these areas have enabled us to decode the mysteries of genetic modifiers in a range of severe diseases, which we aim to turn into medicines that mimic protective mutations. We have assembled a world-class team that we need to realize this vision, including creative scientific pioneers in genetics, experienced company builders, and biotech industry leaders.

Molecular Assemblies, Inc. is a private life sciences company developing an enzymatic DNA synthesis technology designed to power the next generation of DNA-based products. The company's patented Fully Enzymatic Synthesis (or FES™), based on making DNA the way nature makes DNA, produces long, high quality, sequence-specific DNA reliably, affordably, and sustainably. FES technology will enable the reading and writing of DNA for many industries, including industrial synthetic biology and precision medicine, as well as emerging applications of nanomachines and bio-based electronics. Molecular Assemblies is headquartered in San Diego. *Fully Enzymatic Synthesis and FES are the trademarks of Molecular Assemblies, Inc.

Moonwalk Biosciences is an early-stage therapeutics company co-founded by Dr. Feng Zhang and leading experts in cell state epigenetics including Alex Aravanis, the former CTO of Illumina. The company has developed a differentiated approach to mapping the epigenome of cellular states at the whole genome, single cell resolution level, as well as proprietary strategies for precise epigenetic engineering. The combination enables both a novel discovery platform and a path to developing therapeutics that include both epigenetic engineering and other traditional modalities.

NinthBio is a software company serving Biotech. Our flagship product is "Homology Path", an application that designs the oligos for DNA synthesis. The value is how few oligos are needed to build a a group of similar DNA sequences. Think of the DNA you would need to test for a protein engineering campaign, or to optimize the affinity of an antibody. All require similar but ultimately unique DNA. Our customers can improve their Design, Build, Test Learn Cycle by 15X all while decreasing their DNA spend 10 fold. Researchers are rethinking where and what they are willing to explore given the power of Homology Path, opening the door for rapid and higher value discovery. Want to see how this can help your initiatives; request a demo token to access the capabilities of Homology Path.

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.

ParcelBio is developing a programmable technology to safely and specifically deliver mRNA to diverse cell types in the body. Our proprietary (nanoparticle-free) technology, which we call ‘STAmP’, stabilizes the mRNA and utilizes an interchangeable, lock-and-key-like targeting mechanism which enables fully customizable mRNA medicines.

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.

Tallac Therapeutics is a privately held biopharmaceutical company harnessing the power of innate and adaptive immunity to fight cancer. Tallac’s pipeline of immunotherapy candidates are derived from the company’s novel Toll-like Receptor Agonist Antibody Conjugate (TRAAC) platform to deliver a potent Toll-like receptor (TLR9) agonist (T-CpG) for targeted immune activation via systemic administration. Several TRAAC molecules are in various stages of discovery and preclinical development.

Telesis Bio is empowering researchers to accelerate the creation of novel synthetic biology solutions to address some of humanity's greatest challenges. With our breakthrough automation solutions for biological synthesis and expertise in DNA biology, we enable rapid, accurate, and reproducible writing of DNA and mRNA for wide-ranging biological applications. Around the world, innovators are leveraging Telesis Bio's technology to overcome current bottlenecks in synthetic biology workflows and advance the discovery of cutting-edge solutions for health and technology. We are proud of our growing collaborations with partners that include premier academic research institutions, emerging start-ups to nearly all of the Top 25 Biopharma companies. Telesis Bio is honored to play a part in several pioneering advances of our customers and collaborators ranging from novel infectious disease vaccines, precision immunotherapy for cancer and antibody therapeutics to creation of engineered meat substitutes and sustainable cellular agricultural products.

Launched in 2024, Trace Neuroscience is a biopharmaceutical company on a mission to expand the promise of genomic medicine for people living with neurodegenerative diseases. With an initial focus on ALS, the company is developing novel therapies to restore UNC13A protein function to re-establish healthy communication between nerves and muscle cells.