List of Antisense Oligonucleotides Companies in United States - 35

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.

Aldebaran Therapeutics leverages human genetics and best-in-class antisense oligonucleotide (ASO) technology to deliver life-changing, precision ocular ASO medicines. Our focus on differentiated ASO technology, powered by Alloy Therapeutics' Genetic Medicines platform and novel AntiClastic™ ASO format, paves the way for a unique and rapid path to meaningful ocular solutions for patients.

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.

Based in Philadelphia, Aro Biotherapeutics is a biotechnology company pioneering the development of tissue-targeted genetic medicines with a platform based on a proprietary protein technology called Centyrins. The company is developing a wholly-owned pipeline of Centyrin-based therapeutic candidates and is working with industry partners to leverage Centyrins for tissue-specific targeting of therapeutics for a diverse set of diseases.

Aronora is a translational biotechnology company (aronorabio.com) engaged in the development of proprietary biologic therapeutics, including recombinant monolconal antibodies and enzymes. Rational design of our innovative therapeutic agents is expected to result in drug products that reduce the progression and growth of life-threatening blood clots without the detrimental bleeding side effects that characterize all currently marketed antithrombotic drugs. Our products are intended to be used in difficult-to-treat, severe, rapidly progressing, or catastrophic thrombotic blood clotting diseases.

AUM LifeTech is an American Biotechnology company focused on using next-generation RNA silencing technology to develop solutions in diverse life science verticals including biomedical research, medicine, agriculture, and aquaculture. Specifically, AUM's custom products include next generation of innovative genetic tools in the area of gene silencing and manipulation for biomedical research and therapeutic development. AUM’s non-GMO products are also being developed for broad-spectrum target-specific pest management and disease control in agriculture. RNA silencing products using FANA technology provide superior alternatives to conventional RNAi and gene editing approaches like CRISPR and provide a fast track target discovery. AUM's goal is to provide innovation at the genetic level for a better life.

Through cutting-edge science and medicine, Biogen discovers, develops and delivers innovative therapies worldwide for people living with serious neurological and neurodegenerative diseases. Founded in 1978, Biogen is a pioneer in biotechnology and today the Company has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first and only approved treatment for spinal muscular atrophy, and is at the forefront of neurology research for conditions including Alzheimer’s disease, Parkinson’s disease and amyotrophic lateral sclerosis. Biogen also manufactures and commercializes biosimilars of advanced biologics. With approximately 7000 people worldwide, we are truly a global organization, headquartered in Cambridge, Massachusetts, which is also home to our research operations. Our international headquarters are based in Zug, Switzerland and we have world-class manufacturing facilities in North Carolina and Denmark. We offer therapies globally through direct affiliate presence in 30 countries and a network of distribution partners in over 50 additional countries.

Bio-Path is a biotechnology company developing targeted therapies for acute myeloid leukemia (AML), lymphoma, chronic lymphocytic leukemia, gynecological cancer and other challenging cancers. The company’s lead product candidate, prexigebersen (Liposomal Grb2 Antisense), formerly BP1001, is currently being assessed in a Phase 2 clinical study in previously untreated AML patients, who are not eligible for or who have decided to forego intensive chemotherapy because of their fragile health, and in refractory/relapsed AML patients. Additionally, BP1001-A is being assessed in a Phase 1 study in refractory/relapsed patients with solid tumors. The Company’s second product BP1002 (Liposomal Bcl-2 Antisense) is being evaluated in Phase 1 trials in patients with refractory/relapsed lymphoma and AML.

Bolden Therapeutics is a biotechnology company developing first-in-class therapeutics to treat central nervous system diseases such as Alzheimer’s and ischemic stroke. The company’s scientific founders have identified a key molecular pathway to stimulate neural stem cells to promote the birth of new neurons (neurogenesis) in the adult brain. In a proprietary mouse model Bolden has established a proof-of-concept of this approach to increase neurogenesis and enhance hippocampal-dependent memory. The company is actively developing antisense oligonucleotide and monoclonal antibody therapeutics to modulate this pathway.

At CAMP4 Therapeutics, we are pioneering a novel approach to programmable therapeutics. We combine a deep understanding of regulatory RNA and gene expression with a complementary and customizable oligonucleotide modality. Our platform’s proprietary insights enable us to harness the power of RNA to upregulate the expression of genes and unlock the potential to create treatments for hundreds of diseases affecting millions of patients. Join us at the forefront.

CiVi Biopharma Inc. is a US-based, privately-held biopharmaceutical company, founded in 2016. The Company's research and development activities are focused on creating novel therapies for cardiovascular, metabolic and related diseases. CiVi’s innovative pipeline includes CiVi007, a long-acting PCSK9 third-generation Locked Nucleic Acid antisense molecule being developed for the treatment of hypercholesterolemia and the prevention of cardiovascular disease. In addition, the Company currently has other clinical stage development program activity aimed at treating severe liver diseases.

Couragene believes that delivery of gene and biologic therapies should be safer and more efficient. Delivery of genome modification therapies to brain is further complicated by the physiological barriers. Couragene's STEP (Stimuli-responsive Traceless Engineering Platform) technology has demonstrated great potential to overcome the delivery hurdles for treatment of various diseases, including neurogenetic disorders.

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

The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for people with cystic fibrosis, a rare, genetic disease in which a defective gene causes a thick buildup of mucus in the lungs, pancreas, and other organs. The buildup of mucus can lead to extensive lung damage, respiratory failure, malnutrition, liver disease, and gastrointestinal issues, among many other complications. Recognized globally, the Cystic Fibrosis Foundation has led the way in the fight against cystic fibrosis, fueling extraordinary medical and scientific progress. Working alongside the CF community, the CF Foundation has fostered the development of more than a dozen CF treatments — an unprecedented number in a short span of time — and helped add decades of life for people with CF. Thanks to this work, the life expectancy of someone born with CF has doubled in the last 30 years. We are driven by a dream that one day every person with cystic fibrosis will have the chance to live a long, healthy life. Our vision is a cure for every person with cystic fibrosis and a life free from the burden of this disease. While our headquarters are in Bethesda, Maryland, we have additional offices in more than 60 locations across the country with positions in fundraising, marketing, digital, information technology, legal, finance and more – all supporting our mission to cure cystic fibrosis. At the Foundation, we are committed to creating an environment that is free from discrimination and provides a rewarding experience for all members of our team. We strive to be an organization where everyone is welcomed and where talented individuals from all backgrounds have the opportunity to thrive. The CF Foundation is a nonprofit, donor-supported organization and an accredited charity of the Better Business Bureau's Wise Giving Alliance. Please visit us at www.cff.org.

Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue. Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and a preclinical program for facioscapulohumeral muscular dystrophy (FSHD). For more information, please visit https://www.dyne-tx.com. To view our community guidelines, click here: https://bit.ly/3BYPnpK

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.

Envisagenics was founded in 2014 as a spinout of Cold Spring Harbor Laboratory. Our mission is to reduce the complexity of biomedical data to accelerate the development of innovative therapeutic solutions through RNA splicing analytics and artificial intelligence. We focus on the discovery of RNA therapeutics. Over 30 million people in the US suffer from genetic diseases or cancer that could be caused by mutations affecting RNA splicing. 370 human diseases are known to be caused by splicing errors, and more remain to be discovered. Our breakthrough technology, SpliceCore, is a cloud-based platform that is experimentally validated to predict drug targets and biomarkers through splicing discovery from RNA-seq data, using Artificial Intelligence. We are using our software to identify splicing errors causative of diseases, identify the right drug-targets and develop RNA therapeutics through partnerships and collaborations with Biopharma. Our in-silico RNA therapeutics discovery platform replaces expensive drug-target selection and lead design with efficient computer simulations, decreasing time, cost, and failure risk of drug development programs.

Everlum Bio is pioneering a novel approach to drug development for rare diseases. As opposed to working on a single disease we are doing "Drug Development as a Service". We are relentlessly focused on innovating the drug development process. Ultimately we believe this approach will usher in a personalized/precision medicine revolution.

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.

Imvax is a clinical-stage biotechnology company developing personalized, whole tumor-derived immunotherapies. Imvax’s combination therapy uniquely captures the complete antigenic signature of a patient’s own tumor and converts it into a highly stimulatory ‘training program’ for the patient’s immune system. The comprehensive nature of this training is intended to result in both innate and adaptive immune stimulation to optimize long-term anti-tumor effects. The company’s lead program is in glioblastoma multiforme (GBM), an extremely aggressive form of brain cancer, with additional programs in other types of solid tumors.

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.

Leal Therapeutics is a biopharmaceutical company that focuses on developing novel therapeutics for patients with major disorders of the central nervous system.

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.

Mitotherapeutix was created to take advantage of advancements in RNA chemistry and its use in developing siRNA based drugs that can target otherwise difficult to address disease targets. Our initial gene target is a novel protein that controls metabolic activity/efficiency in the mitochondria. The ability to control mitochondrial activity is significant because > 90% of the energy of the cell comes from the mitochondria. This gene target is a negative regulator. Thus, in the presence of the regulator, metabolism is slowed down and the removal of the regulator safely increases metabolism. We have explored the function of this metabolic regulator in different tissues and disease states and have found that turning off the regulator can have a positive effect in disease treatment. Targeting this gene product creates the opportunity of producing a drug that can treat a wide array of diseases.

Oncogenuity is a biopharmaceutical company committed to developing and commercializing new treatments for genetically driven diseases. Our lead asset targets the KRAS mutation G12D, which plays a significant role in pancreatic and colorectal cancers, as well as other “untreatable” cancers of considerable unmet need. Our core technology focuses on the delivery of PNA’s (peptide nucleic acids). The PNA acts as a targeting sequence for gene-silencing. These targeting sequences are interchangeable, and we plan to aggressively pursue both oncology and non-oncology genetic mutations where gene silencing can be effective. We are a partner of Fortress Biotech, and we have an exclusive licensing agreement with Columbia University to develop oligomers (ONCOlogues) that attack genetically driven diseases at the DNA level.

Optimeos Life Sciences is pioneering a novel targeted gene delivery approach to advance medical treatment and patient care.

PepGen Inc., a clinical-stage biotechnology company, focuses on the development of oligonucleotide therapeutics for use in the treatment of severe neuromuscular and neurologic diseases. Its lead product candidate is PGN-EDO51, an EDO peptide that id in Phase I clinical trial to treat individuals with Duchenne muscular dystrophy (DMD). The company is also developing PGN-EDODM1, an EDO peptide-conjugated PMO, which is in Phase 1 clinical trial for the treatment of myotonic dystrophy type 1, as well as EDO therapeutic candidates, such as PGN-EDO53, PGN-EDO45, and PGN-EDO44 for the treatment of DMD. PepGen Inc. was founded in 2018 and is headquartered in Boston, Massachusetts.

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.

Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For information on our Community Guidelines, please visit sarepta.com/community-guidelines. We want to share a reminder with all job seekers and candidates regarding the persistence of recruiting fraud. Please read a message about recruiting fraud and steps you can take to protect yourself here: https://www.sarepta.com/recruiting-fraud

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.

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.

La Laiterie du Berger est une entreprise familiale sénégalaise, créée en 2006. Notre mission est de collecter le lait frais des éleveurs de notre pays, pour offrir chaque jour aux consommateurs sénégalais des produits laitiers, au lait local et naturel, connus sous la marque Dolima. C’est ce qui fait que la Laiterie du Berger n’est pas une entreprise comme les autres, et que le yaourt Dolima n’est pas un yaourt comme les autres. Notre engagement s’incarne dans notre mission sociale : construire une filière sénégalaise plus forte, avec un élevage plus productif, en mesure d’approvisionner le marché local. Il s’agit aussi de plus en plus d’aller vers la nutrition, pour apporter la santé par une alimentation saine et accessible dans notre pays où cet enjeu est si grand.