53 Antisense Oligonucleotides Companies - Worldwide

San Diego, California, United States of America

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.

Wuppertal, Germany

AiCuris (from 'Anti-infective Cures'​) is a pharmaceutical company focused on the discovery, research and development of novel antiviral agents for the treatment of severe and potentially life-threatening infectious diseases in immunocompromised patients. Founded in 2006 from Bayer's virology and bacteriology research divisions, AiCuris has a broad and innovative pipeline of novel anti-infectives and a team of internationally regarded scientists and drug developers. With its focus on specialist markets with high medical need, the company is perfectly positioned for growth and success.

Melbourne, Victoria, Australia

Previously Antisense Percheron Therapeutics is an Australian publicly-listed biotechnology company, developing and commercializing antisense pharmaceuticals for large unmet markets in rare diseases. The products are in-licensed from Ionis Pharmaceuticals Inc. (NASDAQ: IONS), an established leader in antisense drug development.

10355 Science Center Drive, Suite 130

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.

Philadelphia, Pennsylvania, United States

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.

Portland, Oregon, United States

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.

Paterna, Valencia, Spain

ARTHEx Biotech is a spin-off biotech company from the University of Valencia (Spain) focused on the research and development of novel oligonucleotide therapies that modulate microRNA to treat diseases with unmet medical needs. ARTHEx has developed its proprietary technology, the ENTRY ™ platform, that allows fast and flexible development of investigational miR-modulating oligonucleotides that are designed to enter relevant tissues in pharmacological quantities, in order to treat diseases in which microRNAs play a key role. The first investigational product from this platform is ATX-01, an antimiR in preclinical development for Myotonic Dystrophy Type 1 (DM1). DM1 is an orphan disease affecting more than 900,000 people worldwide (estimated prevalence 1/8,000) with no cure, nor approved treatment to date. ATX-01 is a first-in-class disease-modifying therapy that addresses the cause of DM1 with a dual and unique mechanism of action, which targets micro-RNA-23b a key factor in the disease pathogenesis. To learn more about Arthex Biotech, please visit us at www.arthexbiotech.com

Philadelphia, Pennsylvania, United States

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.

Hangzhou, China

AusperBio is a clinical-stage biopharmaceutical company with operations in the USA and China, dedicated to the development of innovative therapeutics for curing chronic hepatitis B. The company has developed a proprietary Med-Oligo™ ASO technology platform, substantially enhancing the potency of targeted therapies, not only for liver diseases, but also with the potential for expansion beyond the liver. AusperBio's strategy is to combine its leading oligonucleotide therapies with other medications including therapeutic antibodies and mRNA vaccines to address a broad range of unmet medical needs.

Cambridge, Massachusetts, United States of America

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.

Bellaire, Texas, United States

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.

Providence, Rhode Island, United States

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.

Marlton, New Jersey, United States

Bound Therapeutics LLC creates next generation healthcare solutions by developing RNA-based drugs for various diseases, immersive haptic surgical simulation, and artificial intelligence automated medical imaging analysis.

Cambridge, Massachusetts, United States

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.

Chevy Chase, Maryland, United States

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.

675 US Highway 1

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.

San Diego, California, United States

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

Bethesda, Maryland, US

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.

Cedex, France

Dynacure is a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options. Dynacure is developing DYN101, an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein for the treatment of Myotubular and Centronuclear Myopathies, in strategic collaboration with Ionis Pharmaceuticals. Dynacure is also building a complementary research portfolio targeting other orphan disorders, including its DYN201 program for the treatment of Hereditary Spastic Paraplegias. Dynacure is headquartered in Strasbourg, France with a corporate office in Philadelphia, PA, USA.

Waltham, Massachusetts, United States of America

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

South San Francisco, California, United States of America

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.

New York, New York, United States

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.

Austin, Texas, United States

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.

Leuven, Vlaams-Brabant, Belgium

Flamingo is pioneering RNA-targeted therapies for oncology with state-of-the art chemistries and a clinical-stage pipeline targeting undruggable transcription factors and splice variants. Flamingo Therapeutics has a Phase II trial (PEMDA-HN) evaluating the STAT3 targeting agent danvatirsen, in combination with pembrolizumab, in patients with head and neck squamous cell carcinoma (HNSCC). Flamingo has an alliance with Ionis Pharmaceuticals and is supported by well-known biotechnology investors including Andera Partners, Bpifrance Large Venture, Bpifrance through its FABS and Fonds Biothérapies Innovantes et Maladies Rares funds, Eurazeo - Kurma Partners, Perceptive Advisors, PMV, Pontifax, Sphera, funds managed by Tekla Capital Management LLC and VIB.

Philomath, Oregon, US

Lausanne, Vaud, Switzerland

HAYA Therapeutics is a precision medicines company developing RNA-guided programmable therapeutics targeting the regulatory genome for serious health conditions including cardiovascular disease and cancer. The company is using its innovative cell-state modifying platform to gain novel insights into the biology of long non-coding RNAs (lncRNAs), the ‘dark genomes’ information processing features and ‘source code’. HAYA’s lead therapeutic candidate is HTX-001, an antisense oligonucleotide targeting Wisper, which is a tissue and cell-specific cardiac lncRNA known to play a role in heart failure. The company is also developing a pipeline of lncRNA-targeting candidates for the cell-specific treatment of diseases in other tissues, including the lungs and the microenvironment of solid tumor cancers. Headquartered at the life sciences park Biopôle in Lausanne, Switzerland with laboratory facilities at JLABS @ San Diego, HAYA is led by a world-class team of experts in programmable and precision RNA-targeted therapeutics and is supported by a strong investor consortium. HAYA’s mission is to identify and develop breakthrough disease-modifying therapeutics with the potential for greater efficacy, safety, and accessibility than existing treatments.

Leiden, Netherlands

Hybridize Therapeutics is a spin-off from the Leiden University Medical Center and is a therapeutic company focused on developing kidney-protecting modalities for untreated kidney diseases.

Philadelphia, Pennsylvania, United States

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.

Carlsbad, California, United States

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.

Munich, Bavaria, Germany

Isarna Therapeutics is a clinical stage biotech company, headquartered in Munich, Germany. Isarna develops ISTH0036, a next-generation antisense to block TGF-beta 2 production, a key molecule in the pathogenesis of retinal disease. Current therapies – approved and pipeline products – mostly target the edema, with several anti-VEGFs approved or in development offering only minor improvements over current SOC. While anti-VEGF is a very effective therapy – it does not stop the disease progression of retinal pathologies like w AMD. Patients progress to late-stage disease despite effective anti-edema therapy – and thus develop fibrosis. TGF-b is a key driver of fibrosis, so blocking the production of this molecule intracellularly, using elaborated antisense molecules, promises to be a game-changer in retina therapy. Animal data show comparable efficacy of ISTH0036 to block neovascularization as anti-VEGF – with the added benefit of suppressing fibrosis. This MOA could be an effective long-term treatment in several retinal pathologies including the blockbuster-indications wAMD, DME, RVO, with a significant upward potential in dry AMD. A Phase 1 study showed excellent safety, Isarna Therapeutics is currently preparing a Phase 2a study in wet AMD and in DME/DR. Isarna is led by a highly experienced management, the COO, Rene Rückert, MD, MBA led at Bayer/Novartis the global development of both approved blockbuster drugs for retinal diseases – Eylea and Lucentis. The CMO, Prof. Marion Munk, MD, PhD is a leading global retina specialist. Isarna is open to discuss future partnering for the development and is seeking investors to finance the clinical development and growth of the company.

New York, New York, United States

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

Osaka, Japan

Luxna Biotech is a preclinical antisense drug development company based on proprietary modified nucleic acids, unique bridged structure (AmNA, scpBNA, GuNA) and modified DNA (5'-CP) can eliminate phosphorothioated in antisense and siRNA. Currently, better modifications are being discovered that are suitable for applications in neurological diseases that minimize neurotoxicity while maintaining knockdown efficacy in vivo by Gapmer. Based on these findings we developed excellent antisense (ASO) drug discovery platform called “LuxiAPTM” enable to wide therapeutic index for CNS ASO drug. We have several pharmaceutical collaborations and original ASO pipeline including ALS, Spinal cord injury and Hearing loss. We are looking for the partners not only for platform collaboration with pharmaceuticals and biotech but early-stage co-development alliance with our original pipeline.

San Francisco, United States of America

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.

South San Francisco, California, United States of America

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.

A.C. Meyers Vænge 15, Copenhagen, Capital Region of Denmark 2450, DK

Developing RNA therapeutics for disease modification neurological disorders

Bay Harbor Islands, Florida, United States

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.

Princeton, New Jersey, United States

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

Boston, Massachusetts, United States

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.

San Diego, California, United States

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.

Bilthoven, Utrecht, Netherlands

Sapreme’s mission is to develop next-generation macromolecule therapeutics by circumventing endosomal entrapment, thereby enhancing target engagement. The company’s proprietary endosomal escape platform improves the therapeutic window by enabling access to intracellular targets with minimal toxicity. This approach is applied for Sapreme’s internal pipeline and is available for partnering, without limitation to biologic modality or indication. Our solution to endosomal entrapment has the potential to re-open myriad drug development pathways for macromolecule therapeutics lacking efficacy and to explore new targets considered “undruggable” by permeabilizing endo-lysosomal membranes, allowing trapped macromolecules to be released, increasing target engagement without negatively impacting the plasma membrane integrity.

Cambridge, Massachusetts, United States

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

Marburg, Germany

Secarna Pharmaceuticals is the next generation antisense oligonucleotide (ASO) company addressing high unmet medical needs in the areas of immuno-oncology and immunology, as well as viral, neurodegenerative and cardiometabolic diseases. Secarna’s mission is to maximize the performance and output of its proprietary LNAplus™ antisense oligonucleotide discovery platform, as well as to develop highly specific, safe, and efficacious best-in-class antisense therapies for challenging or currently undruggable targets. With over 15 development programs focusing on targets in indications where antisense-based approaches have clear benefits over other therapeutic modalities, Secarna is the leading European antisense drug discovery and development company.

Exeter, Devon, England, United Kingdom

SENISCA is a biotech spinout company from the University of Exeter, founded in 2020 and dedicated to the development of new approaches to reverse cellular senescence (senotherapeutics). Our founders are world leaders in molecular and cellular biology and have patent-protected an innovation for the reversal of cellular senescence. This innovation works by restoring the ability of cells to ‘fine tune’ the expression of their genes to rejuvenate aged cells. At SENISCA, we are using this knowledge, concerning how and why cells become senescent, to develop a new generation of oligonucleotide-based interventions, to turn back the ageing clock in old cells and to target the diseases and aesthetic signs of ageing.

Ness Ziona, Israel

SKIP Therapeutics develops RNA-based therapies for rare genetic disorders and common diseases – utilizing a proprietary computational discovery engine to optimize target selection and therapeutic design. We employ sequence-based molecules, called Antisense Oligonucleotides (ASO), to restore function of mutated genes in rare genetic disorders or to modify functionality of key genes in major disease pathways. Convergence of SKIP’s novel computational discovery engine alongside strong experimental capabilities form a unique venture in the landscape of RNA-based therapies. Thousands of rare genetic disorders affect millions of people worldwide, however the small number of patients for each disease, further segregated by patient-specific mutations, has hindered development of dedicated treatments. Recent advancements in RNA-therapeutics offer an expanded molecular toolkit that can be deployed rapidly and at low costs to restore protein function of mutated genes. Identifying sub patient-populations that could potentially be suitable for treatment with RNA-based therapeutics and matching molecular treatment-strategies across the vast landscape of known disease-causing mutations could be assisted by computational methods. To facilitate development of dedicated treatments, Skip Therapeutics has developed a bioinformatic pipeline that analyzes disease-causing mutations and recognizes the best molecular strategy (if any) to restore protein function, using splice modulating antisense oligonucleotides (ASOs). Analyzing mutation data at scale, enables identification of treatable patient cohorts and optimizes target selection. Top candidates from the computational analyses are experimentally validated and pursued for therapeutic development.

Jerusalem, Israel

SpliSense Ltd., is a growing and exciting biotechnology company, developing novel therapies for genetic diseases by inhalation. The company is currently focusing on splicing mutations in the CFTR gene for treating Cystic Fibrosis (CF) patients.

Marburg, Germany

sterna biologicals is a clinical-stage immunology company developing novel treatments for chronic inflammatory diseases such as atopic dermatitis, ulcerative colitis, asthma and chronic obstructive pulmonary disease (COPD). By targeting GATA-3, a key master transcription factor that plays a central role in chronic inflammatory diseases, the company’s proprietary DNAzyme-based drug candidate, hgd40, antagonizes several inflammatory processes and mediators. sterna biologicals has completed multiple phase IIa clinical trials with hgd40 in a number of commercially attractive chronic inflammatory diseases with high unmet medical need. If you are interested in partnering with us, please reach out at partnering@sterna-biologicals.com.

Bedford, Massachusetts, United States

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.

San Francisco, California

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.

oxford, oxfordshire, united kingdom

SynaptixBio is a rare disease biotech company that is pioneering ground-breaking treatments for severe leukodystrophies and other childhood neurodegenerative diseases.    SynaptixBio's mission is to deliver gene therapies for fatal leukodystrophies in children caused by loss of function or mutation in a single gene. Using a breadth of platforms including antisense oligonucleotide (ASO)-based technology, we can silence the expression of the toxic gene to reverse disease progression. This approach will create life saving treatments for severe genetic diseases that affect the central nervous system.

Lyon, France

Theranexus is an innovative biopharmaceutical company that spun off from the French Alternative Energies and Atomic Energy Commission (CEA). It specializes in the treatment of rare central nervous system disorders and is a pioneer in the development of drug candidates targeting both neurons and glial cells. Theranexus has a unique advanced therapy candidate identification and characterization platform focused on rare neurological disorders and an initial drug candidate in clinical development for Batten disease.