List of Antisense Oligonucleotides Companies in California - 11

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.

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.

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

Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation platform. The Encoded approach offers potentially unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders.

Enabling Nucleic Acid-Based Medicines to Treat Solid Tumor Cancers Ground-breaking Impilo Platform™ The Impilo Platform™ is designed to address key issues that have limited ability of emerging nucleic acid-based therapeutic modalities such as antisense, siRNA, tRNA, and mRNA and in vivo gene editing to effectively treat solid tumor cancers.

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.

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.

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.

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.

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.