Knowledge Base
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Knowledge Base
Oligos In Action
BioSpring is at the forefront of therapeutic nucleic acid manufacturing and analysis, providing comprehensive solutions for drug developers with advanced manufacturing technologies and high-quality, scalable production.
Discover the diverse applications of nucleic acids, learn how BioSpring supports researchers worldwide, and get an overview of approved oligonucleotide therapeutics.
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Areas of Application of Nucleic Acids
Therapeutic Applications
Nucleic acids have revolutionized medicine by serving as the foundation for therapies targeting genetic conditions, cancers, and more. siRNA, guide RNA, and mRNA-based therapies are gaining significant attention for their precise ability to edit the genome, silence specific genes, and more. These nucleic acid therapeutics, and more, provide targeted approaches to treat diseases that were once considered untreatable.
Cancer Treatment
Therapeutic nucleic acids, such as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), have been widely applied in cancer therapies to silence genes responsible for tumor growth and progression. In addition to gene-silencing approaches, TLRmers like CpG oligonucleotides activate the immune response by targeting Toll-like receptor 9 (TLR9), which can stimulate the body’s immune cells to attack cancerous cells.
Genome Editing
Guide RNAs (gRNAs) are essential in precision genome-editing technologies like CRISPR/Cas systems. These guide RNAs enable targeting of specific genetic sequences, allowing for accurate gene editing to treat genetic disorders such as muscular dystrophy, cystic fibrosis, or certain cancers.
Vaccines and Immunotherapies
Nucleic acids are becoming a cornerstone in the development of next-generation vaccines. mRNA-based vaccines have revolutionized immunization strategies by delivering instructions to cells for producing antigens that trigger protective immune responses. Additionally, CpG oligonucleotides, like CpG 7909, act as potent vaccine adjuvants by boosting the immune response. By triggering cytokine release and enhancing antigen-specific antibody production, nucleic acid-based immunotherapies help provide enhanced protection against diseases.
Genetic Disorders and Rare Diseases
With advancements in therapeutic nucleic acids, treatments for rare genetic disorders are becoming a reality. Antisense oligonucleotides (ASOs) and siRNAs are designed to silence or modify disease-causing genes, offering hope for the eradication of diseases that were previously untreatable.
Cardiovascular and Neurological Diseases
Therapeutic nucleic acids are being applied in the treatment of cardiovascular and neurological diseases. RNA-based therapies, such as mRNAs and antisense RNAs, are developed to target genes associated with heart disease or neurodegenerative conditions like Alzheimer’s.
Early-Stage Research and Development Projects
In addition to well-established diagnostic applications, nucleic acids are pivotal in early-stage R&D for novel diagnostic methods. As personalized medicine advances, nucleic acids are driving the development of companion diagnostics – tests that identify the patients most likely to benefit from a specific therapy. This approach allows for more targeted and effective treatments based on individual genetic profiles.
At BioSpring, we partner with diagnostic companies to provide custom oligonucleotides that meet the specific needs of early-stage research, offering flexibility in design, length, and modification. Our capabilities enable the creation of innovative diagnostic solutions, driving forward the next generation of precision medicine.
See how other researchers have partnered with BioSpring for their research and clinical studies in our Featured in Research section below.
Diagnostics
Nucleic acids have transformed the field of diagnostics by enabling highly specific, sensitive, and rapid detection of a wide array of diseases and conditions. At BioSpring, we support the growing demand for oligonucleotides in diagnostics by offering tailored manufacturing services for commercial diagnostic applications such as forensic science, HLA-typing, microbial and viral testing, and more. A closer examination reveals how nucleic acids are shaping key diagnostic areas:
Forensic Science
Nucleic acid-based technologies have revolutionized forensic science, where DNA and RNA are used to identify individuals, analyze biological evidence, and even uncover historical data from ancient remains. Techniques such as polymerase chain reaction (PCR) and short tandem repeat (STR) analysis rely on the precise amplification of nucleic acids, allowing forensic experts to generate DNA profiles from tiny biological samples (such as hair, blood, or skin cells). These profiles can be compared against criminal databases, making it a powerful tool in criminal investigations.
Genomics and Ancestry Testing
Beyond criminal investigations, nucleic acid-based methods are also widely used in genomic ancestry testing. By analyzing mitochondrial DNA (mtDNA) or Y-chromosome sequences, scientists can trace an individual's lineage, revealing genetic relationships that span generations. This has applications in anthropology, genealogy, and population studies.
HLA-Typing (Human Leukocyte Antigen Typing)
HLA-typing is a critical diagnostic tool in organ transplantation and immune-related disease detection. Nucleic acids are used to identify specific HLA gene variants, which are essential for matching organ donors with recipients. Accurate HLA-typing minimizes the risk of organ rejection and improves the success rate of transplants.
Microbial and Viral Testing
Nucleic acids have become indispensable in microbial and viral detection, especially in the diagnosis of infectious diseases. Techniques such as real-time PCR (qPCR), next-generation sequencing (NGS), and isothermal amplification allow for the rapid identification of pathogens like bacteria and viruses with high specificity and sensitivity. The COVID-19 pandemic highlighted the importance of nucleic acids in diagnostics, as PCR-based testing became the gold standard for detecting the SARS-CoV-2 virus.
Other Applications
Agricultural Biotechnology
In agriculture, nucleic acids are driving innovations in crop protection and improvement. Techniques like RNA interference (RNAi) are being utilized to develop crops with improved resistance to pests and diseases, reduced need for chemical treatments, and enhanced nutritional profiles.
Environmental and Industrial Applications
Beyond health and agriculture, nucleic acids are used in environmental monitoring and industrial biotechnology. Biosensors based on nucleic acids can detect pollutants and pathogens in water, soil, and air, contributing to safer environments. Additionally, nucleic acids serve in the development of biocatalysts and enzymes used in industrial processes, enhancing efficiency and reducing reliance on harmful chemicals.
how we supported researchers worldwide
Featured in Research
At BioSpring, we are dedicated to driving scientific progress by providing high-quality nucleic acid services. Our products and solutions have supported pioneering research in various fields - helping scientists push the boundaries of innovation. From early-stage discovery to clinical development, BioSpring materials and expertise have played a crucial role in numerous peer-reviewed publications and breakthrough studies. Explore a selection of research where our contributions have helped shape the future of nucleic acid-based science.
2025
- Lipopolymers as the Basis of Non-Viral Delivery of Therapeutic siRNA Nanoparticles in a Leukemia (MOLM-13) Model
Yotsomnuk, P., Rajendran, A. P., Sundaram, D. N. M., Morales, L. C., Kucharski, C., Nasrullah, M., Skolpap, W., Jiang, X., Gibson, S. B., Brandwein, J., Uludağ, H.
Biomolecules (2025) - FORCE platform overcomes barriers of oligonucleotide delivery to muscle and corrects myotonic dystrophy features in preclinical models
Weeden, T., Picariello, T., Quinn, B., Spring, S., Shen, P.-Y., Qiu, Q., Vieira, B. F., Schlaefke, L., Russo, R. J., Chang, Y.-A., Cui, J., Yao, M., Wen, A., Hsia, N., Evron, T., Ovington, K., Tsai, P.-N., Yoder, N., Lan, B., Venkatesan, R., Hall, J., Desjardins, C. A., Qatanani, M., Hilderbrand, S., Najim, J., Tang, Z., Tanner, M. K., Subramanian, R., Thornton, C. A., Ibraghimov-Beskrovnaya, O., Zanotti, S.
Commun Med (2025) - Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy
Oppeneer, T., Qi, Y., Henshaw, J., Larimore, K., Melton, A., Puoliväli, J., Carter, C., Fant, P., Brennan, S., Wetzel, L. A., Sigg, M. A., Crawford, B. E., Magat, J., Froelich, S., Woloszynek, J. C., O’Neill, C. A.
Nucleic Acid Therapeutics (2025) - BMN 351-Induced Exon Skipping and Dystrophin Expression in Skeletal and Cardiac Muscle Lead to Preservation of Motor Function in a Mouse Model of Exon 51 Skip-Amenable Duchenne Muscular Dystrophy
Oppeneer, T., Qi, Y., Henshaw, J., Larimore, K., Puoliväli, J., Carter, C., Fant, P., Brennan, S., Wetzel, L. A., Sigg, M. A., O’Neill, C. A.
Nucleic Acid Therapeutics (2025) - Assessment of native mass spectrometry as a screening method to identify and characterize RNA-targeting small molecules
Sternicki, L. M., Klose, J. W., Poulsen, S.-A.
bioRxiv (2025) - A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levels
Tremblay, F., Xiong, Q., Shah, S. S., Ko, C.-W., Kelly, K., Morrison, M. S., Giancarlo, C., Ramirez, R. N., Hildebrand, E. M., Voytek, S. B., El Sebae, G. K., Wright, S. H., Lofgren, L., Clarkson, S., Waters, C., Linder, S. J., Liu, S., Eom, T., Parikh, S., Weber, Y., Martinez, S., Malyala, P., Abubucker, S., Friedland, A. E., Maeder, M. L., Lombardo, A., Myer, V. E., Jaffe, A. B.
Nat Med (2025) - A Simplified Guide RNA Synthesis Protocol for SNAP- and Halo-Tag-Based RNA Editing Tools
Hofacker, D. T., Kalkuhl, S., Schmid, J. F., Singh, S., Stafforst, T.
Molecules (2025) - BCL11A-deficient human erythropoiesis is impaired in vitro and after xenotransplantation into mice
Jang, Y., Feng, R., Palmer, L. E., Mayuranathan, T., Yao, Y., Mayberry, K., Zhou, S., Xu, J., Gossett, J., Kang, G., Cheng, Y., Yen, J. S., Weiss, M. J.
Blood Adv (2025)
2024
- A regulatory loop involving the cytochrome P450-soluble epoxide hydrolase axis and TGF-β signaling
Li, X., Kempf, S., Delgado Lagos, F., Ukan, Ü., Popp, R., Hu, J., Frömel, T., Günther, S., Weigert, A., Fleming, I.
iScience (2024) - Ionizable nanoemulsions for RNA delivery into the central nervous system – importance of diffusivity
Borrajo, M. L., Quijano, A., Lapuhs, P., Rodriguez-Perez, A. I., Anthiya, S., Labandeira-Garcia, J. L., Valenzuela, R., Alonso, M. J.
Journal of Controlled Release (2024)' - Introducing a hemoglobin G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice
Li, C., Georgakopoulou, A., Paschoudi, K., Anderson, A. K., Huang, L., Gil, S., Giannaki, M., Vlachaki, E., Newby, G. A., Liu, D. R., Yannaki, E., Kiem, H.-P., Lieber, A.
Molecular Therapy (2024) - An improved SNAP-ADAR tool enables efficient RNA base editing to interfere with post-translational protein modification
Kiran Kumar, K. D., Singh, S., Schmelzle, S. M., Vogel, P., Fruhner, C., Hanswillemenke, A., Brun, A., Wettengel, J., Füll, Y., Funk, L., Mast, V., Botsch, J. J., Reautschnig, P., Li, J. B., Stafforst, T.
Nat Commun (2024) - INT-1B3, an LNP formulated miR-193a-3p mimic, promotes anti-tumor immunity by enhancing T cell mediated immune responses via modulation of the tumor microenvironment and induction of immunogenic cell death
Duurland, C. L., de Gunst, T., den Boer, H. C., van den Bosch, M. T. J., Telford, B. J., Vos, R. M., Xie, X., Zang, M., Wang, F., Shao, Y., An, X., Wang, J., Cai, J., Bourré, L., van Pinxteren, L. A. H., Schaapveld, R. Q. J., Janicot, M., Yahyanejad, S.
Oncotarget (2024) - Antithrombotic but not anticoagulant activity of the thrombin-binding RNA aptamer Apta-1
Jedlina, L., Paramel, G., Soboleva, S., Chutna Olin, O., Haug, M., Fransén, K., Lindstam, M., Brewinska-Olchowik, M., Piwocka, K., Grenegård, M.
British Journal of Pharmacology (2024) - INT-1B3, an LNP formulated miR-193a-3p mimic, promotes anti-tumor immunity by enhancing T cell mediated immune responses via modulation of the tumor microenvironment and induction of immunogenic cell death
Duurland, C. L., de Gunst, T., den Boer, H. C., van den Bosch, M. T. J., Telford, B. J., Vos, R. M., Xie, X., Zang, M., Wang, F., Shao, Y., An, X., Wang, J., Cai, J., Bourré, L., van Pinxteren, L. A. H., Schaapveld, R. Q. J., Janicot, M., Yahyanejad, S.
Oncotarget (2024) - Role of the soluble epoxide hydrolase in keratinocyte proliferation and sensitivity of skin to inflammatory stimuli
Naeem, Z., Zukunft, S., Huard, A., Hu, J., Hammock, B. D., Weigert, A., Frömel, T., Fleming, I.
Biomedicine & Pharmacotherapy Vol. 171 (2024) - Profiling the interactome of oligonucleotide drugs by proximity biotinylation
Hanswillemenke, A., Hofacker, D. T., Sorgenfrei, M., Fruhner, C., Franz-Wachtel, M., Schwarzer, D., Maček, B., Stafforst, T.
Nat Chem Biol (2024)
- A pipeline for identifying guide RNA sequences that promote RNA editing of nonsense mutations that cause inherited retinal diseases
Schneider, N., Steinberg, R., Ben-David, A., Valensi, J., David-Kadoch, G., Rosenwasser, Z., Banin, E., Levanon, E. Y., Sharon, D., Ben-Aroya, S.
Molecular Therapy - Nucleic Acids (2024) - GNTI-122: an autologous antigen-specific engineered Treg cell therapy for type 1 diabetes
Uenishi, G. I., Repic, M., Yam, J. Y., Landuyt, A., Saikumar-Lakshmi, P., Guo, T., Zarin, P., Sassone-Corsi, M., Chicoine, A., Kellogg, H., Hunt, M., Drow, T., Tewari, R., Cook, P. J., Yang, S. J., Cerosaletti, K., Schweinoch, D., Guiastrennec, B., James, E., Patel, C., Chen, T. F., Buckner, J. H., Rawlings, D. J., Wickham, T. J., Mueller, K. T.
JCI Insight (2024) - Add-on multiple submucosal injections of the RNA oligonucleotide GUT-1 to anti-TNF antibody treatment in patients with moderate-to-severe ulcerative colitis: an open-label, proof-of concept study
Suzuki, K., Sameshima, Y., Yokoyama, J., Terai, S., Yoneyama, H., Atreya, R., Neurath, M. F., Hibi, T., Asakura, H.
Inflamm Regener (2024)
2023
- The tumor suppressor 5A2, a synthetic miR-7-5p mimic, targets oncogenic and metabolic pathways, as revealed by transcriptome-wide analysis
van den Bosch, M. T. J., Telford, B. J., Yahyanejad, S., de Gunst, T., den Boer, H. C., Vos, R. M., Duurland, C. L., Biemans, R., Dubois, L. J., van Pinxteren, L. A. H., Schaapveld, R. Q. J., Janicot, M.
Frontiers in Drug Discovery Vol. 3 (2023) - Cationic LNP-formulated mRNA expressing Tie2-agonist in the lung endothelium prevents pulmonary vascular leakage
Radloff, K., Gutbier, B., Dunne, C. M., Moradian, H., Schwestka, M., Gossen, M., Ahrens, K., Kneller, L., Wang, Y., Moga, A., Gkionis, L., Keil, O., Fehring, V., Tondera, D., Giese, K., Santel, A., Kaufmann, J., Witzenrath, M.
Molecular Therapy: Nucleic Acids Vol. 34 (2023) - Targeted siRNA lipid nanoparticles for the treatment of KRAS-mutant tumors
Anthiya, S., Öztürk, S.C., Yanik, H., Tavukcuoglu, E., Şahin, A., Datta, D., Charisse, K., Álvarez, D.M., Loza, M.I., Calvo, A., & Sulheim, E.
Journal of Controlled Release (2023) - ADAR1-mediated RNA editing promotes B cell lymphomagenesis
Pecori, R., Ren, W., Pirmoradian, M., Wang, X., Liu, D., Berglund, M., Li, W., Tasakis, R. N., Di Giorgio, S., Ye, X., Li, X., Arnold, A., Wüst, S., Schneider, M., Selvasaravanan, K.-D., Fuell, Y., Stafforst, T., Amini, R.-M., Sonnevi, K., Enblad, G., Sander, B., Wahlin, B. E., Wu, K., Zhang, H., Helm, D., Binder, M., Papavasiliou, F. N., Pan-Hammarström, Q.
iScience Volume 26 (2023) - Combination of protein and cell internalization SELEX identifies a potential RNA therapeutic and delivery platform to treat EphA2-expressing tumors
Santana-Viera, L., Dassie, J. P., Rosàs-Lapeña, M., Garcia-Monclús, S., Chicón-Bosch, M., Pérez-Capó, M., del Pozo, L., Sanchez-Serra, S., Almacellas-Rabaiget, O., Maqueda-Marcos, S., López-Alemany, R., Thiel, W. H., Giangrande, P. H., Tirado, O. M.
ACS Nano (2023) - Positron Emission Tomography Studies of the Biodistribution, Translocation, and Fate of Poly Allyl Amine-Based Carriers for siRNA Delivery by Systemic and Intratumoral Administration
Simo, C., Salvador, C., Andreozzi, P., Gomez-Vallejo, V., Romero, G., Dupin, D., Llop, J., Moya, S. E.
Small (2023) - Spacer Fidelity Assessments of Guide RNA by Top-Down Mass Spectrometry
Macias, L. A., Garcia, S. P., Back, K. M., Wu, Y., Johnson, G. H., Kathiresan, S., Bellinger, A. M., Rohde, E., Freitas, M. A., Madsen, J. A.
ACS Cent. Sci. (2023) - An Aptamer against MNK1 for Non-Small Cell Lung Cancer Treatment
Carrión-Marchante, R., Pinto-Díez, C., Klett-Mingo, J. I., Palacios, E., Barragán-Usero, M., Pérez-Morgado, M. I., Pascual-Mellado, M., Alcalá, S., Ruiz-Cañas, L., Sainz, B., Jr., González, V. M., Martín, M. E.
Pharmaceutics (2023) - Correlating the Structure and Gene Silencing Activity of Oligonucleotide-Loaded Lipid Nanoparticles Using Small-Angle X-ray Scattering
Hammel, M., Fan, Y., Sarode, A., Byrnes, A. E., Zang, N., Kou, P., Nagapudi, K., Leung, D., Hoogenraad, C. C., Chen, T., Yen, C.-W., Hura, G. L.
ACS Nano (2023)
2022
- Herpes simplex virus PCR in 2230 explanted corneal buttons
Tóth, G., Berkó-Göttel, B., Seitz, B., Langenbucher, A., Stachon, T., Pluzsik, M.T., Nagy, Z.Z., Smola, S., & Szentmáry, N.
Acta Ophthalmologica (2022) - Selective Targeting and Eradication of Various Human Non-Small Cell Lung Cancer Cell Lines Using Self-Assembled Aptamer-Decorated Nanoparticles
Barak, D., Engelberg, S., Assaraf, Y. G., Livney, Y. D.
Pharmaceutics (2022) - Targeting TGF-ß in the Central Nervous System: Assessment of Cynomolgus Monkey—Toxicity and Pharmacokinetics for an LNA-Antisense Oligonucleotide
Peters, S., Wirkert, E., Kuespert, S., Heydn, R., Korte, S., Mecklenburg, L., Aigner, L., Johannesen, S., Bruun, T.-H., Bogdahn, U.
Appl. Sci. (2022) - Predictive high-throughput screening of PEGylated lipids in oligonucleotide-loaded lipid nanoparticles for neuronal gene silencing
Sarode, A., Fan, Y., Byrnes, A. E., Hammel, M., Hura, G. L., Fu, Y., Kou, P., Hu, C., Hinz, F. I., Roberts, J., Koenig, S. G., Nagapudi, K., Hoogenraad, C. C., Chen, T., Leung, D., Yen, C.-W.
Nanoscale Adv. (2022) - Enhancement of protective efficacy of innate immunostimulant based formulations against yolk sac infection in young chicks
Nguyen, T. T. T., Shahin, K., Allan, B., Sarfraz, M., Wheler, C., Gerdts, V., Köster, W., Dar, A.
Poultry Science (2022) - Functional Downregulation of PD-L1 and PD-L2 by CpG and non-CpG Oligonucleotides in Melanoma Cells
Kleemann, J., Steinhorst, K., König, V., Zöller, N., Cinatl, J., Jr., Özistanbullu, D., Kaufmann, R., Meissner, M., Kippenberger, S.
Cancers (2022) - SAMHD1 controls innate immunity by regulating condensation of immunogenic self RNA
Maharana, S., Kretschmer, S., Hunger, S., Yan, X., Kuster, D., Traikov, S., Zillinger, T., Gentzel, M., Elangovan, S., Dasgupta, P., Chappidi, N., Lucas, N., Maser, K. I., Maatz, H., Rapp, A., Marchand, V., Chang, Y.-T., Motorin, Y., Hubner, N., Hartmann, G., Hyman, A. A., Alberti, S., Lee-Kirsch, M. A.
Molecular Cell (2022) - Optimized Hemolysin Type 1 Secretion System in Escherichia coli by Directed Evolution of the Hly Enhancer Fragment and Including a Terminator Region
Pourhassan N., Z., Cui, H., Khosa, S., Davari, M. D., Jaeger, K.-E., Smits, S. H. J., Schwaneberg, U., Schmitt, L.
ChemBioChem (2022) - In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration
Choi, E. H., Suh, S., Foik, A. T., Leinonen, H., Newby, G. A., Gao, X. D., Banskota, S., Hoang, T., Du, S. W., Dong, Z., Raguram, A., Kohli, S., Blackshaw, S., Lyon, D. C., Liu, D. R., Palczewski, K.
Nat Commun (2022) - RNA-Cholesterol Nanoparticles Function as Potent Immune Activators via TLR7 and TLR8
Obermann, H.-L., Lederbogen, I. I., Steele, J., Dorna, J., Sander, L. E., Engelhardt, K., Bakowsky, U., Kaufmann, A., Bauer, S.
Front. Immunol. (2022)
2021
- Base editing of hematopoietic stem cells rescues sickle cell disease in mice
Newby, G. A., Yen, J. S., Woodard, K. J., Mayuranathan, T., Lazzarotto, C. R., Li, Y., Sheppard-Tillman, H., Porter, S. N., Yao, Y., Mayberry, K., Everette, K. A., Jang, Y., Podracky, C. J., Thaman, E., Lechauve, C., Sharma, A., Henderson, J. M., Richter, M. F., Zhao, K. T., Miller, S. M., Wang, T., Koblan, L. W., McCaffrey, A. P., Tisdale, J. F., Kalfa, T. A., Pruett-Miller, S. M., Tsai, S. Q., Weiss, M. J., Liu, D. R.
Nature (2021) - Aptamer BC 007’s affinity to specific and less-specific anti-Sars-CoV-2 neutralizing antibodies
Haberland, A., Krylova, O., Nikolenko, H., Göttel, P., Dallmann, A., Müller, J., & Weisshoff, H.
Viruses (2021) - The consequences of soluble epoxide hydrolase deletion on tumorigenesis and metastasis in a mouse model of breast cancer
Kesavan, R., Frömel, T., Zukunft, S., Brüne, B., Weigert, A., Wittig, I., Popp, R., & Fleming, I.
International Journal of Molecular Sciences (2021) - Regulation of GC box activity by 8-oxoguanine.
Müller, N. & Khobta, A.
Redox Biology (2021) - Targeted Nanoparticles Harboring Jasmine-Oil-Entrapped Paclitaxel for Elimination of Lung Cancer Cells
Engelberg, S., Lin, Y., Assaraf, Y. G., Livney, Y. D.
Int. J. Mol. Sci. (2021) - Overcoming PD-1 Blockade Resistance with CpG-A Toll-Like Receptor 9 Agonist Vidutolimod in Patients with Metastatic Melanoma
Ribas, A., Medina, T., Kirkwood, J. M., Zakharia, Y., Gonzalez, R., Davar, D., Chmielowski, B., Campbell, K. M., Bao, R., Kelley, H., Morris, A., Mauro, D., Wooldridge, J. E., Luke, J. J., Weiner, G. J., Krieg, A. M., Milhem, M. M.
Cancer Discov (2021) - Multi-modal effects of 1B3, a novel synthetic miR-193a-3p mimic, support strong potential for therapeutic intervention in oncology
Telford, B. J., Yahyanejad, S., de Gunst, T., den Boer, H. C., Vos, R. M., Stegink, M., van den Bosch, M. T. J., Alemdehy, M. F., van Pinxteren, L. A. H., Schaapveld, R. Q. J., Janicot, M.
Oncotarget. (2021) - Transcriptome-wide analysis reveals insight into tumor suppressor functions of 1B3, a novel synthetic miR-193a-3p mimic
van den Bosch, M. T. J., Yahyanejad, S., Alemdehy, M. F., Telford, B. J., de Gunst, T., den Boer, H. C., Vos, R. M., Stegink, M., van Pinxteren, L. A. H., Schaapveld, R. Q. J., Janicot, M.
Molecular Therapy: Nucleic Acids (2021) - CroSR391, an ortholog of the λ Cro repressor, plays a major role in suppressing polVR391-dependent mutagenesis
McDonald, J. P., Quiros, D. R., Vaisman, A., Mendez, A. R., Reyelt, J., Schmidt, M., Gonzalez, M., Woodgate, R.
Molecular Microbiology (2021) - Direct and Base Excision Repair-Mediated Regulation of a GC-Rich cis-Element in Response to 5-Formylcytosine and 5-Carboxycytosine
Müller, N., Ponkkonen, E., Carell, T., Khobta, A.
Int. J. Mol. Sci. (2021) - Induced dendritic cells co-expressing GM-CSF/IFN-α/tWT1 priming T and B cells and automated manufacturing to boost GvL
Bialek-Waldmann, J. K., Domning, S., Esser, R., Glienke, W., Mertens, M., Aleksandrova, K., Arseniev, L., Kumar, S., Schneider, A., Koenig, J., Theobald, S. J., Tsay, H.-C., Cornelius, A. D. A., Bonifacius, A., Eiz-Vesper, B., Figueiredo, C., Schaudien, D., Talbot, S. R., Bleich, A., Spineli, L. M., von Kaisenberg, C., Clark, C., Blasczyk, R., Heuser, M., Ganser, A., Köhl, U., Farzaneh, F., Stripecke, R.
Molecular Therapy: Methods & Clinical Development (2021) - Sulfonated Amphiphilic Poly(α)glutamate Amine—A Potential siRNA Nanocarrier for the Treatment of Both Chemo-Sensitive and Chemo-Resistant Glioblastoma Tumors
Krivitsky, A., Pozzi, S., Yeini, E., Israeli Dangoor, S., Zur, T., Golan, S., Krivitsky, V., Albeck, N., Pisarevsky, E., Ofek, P., Madi, A., Satchi-Fainaro, R.
Pharmaceutics (2021) - Embryonic stem cells are devoid of macropinocytosis, a trafficking pathway for activin A in differentiated cells
Kostopoulou, N., Bellou, S., Bagli, E., Markou, M., Kostaras, E., Hyvönen, M., Kalaidzidis, Y., Papadopoulos, A., Chalmantzi, V., Kyrkou, A., Panopoulou, E., Fotsis, T., Murphy, C.
J Cell Sci (2021) - Endothelial Tpl2 regulates vascular barrier function via JNK-mediated degradation of claudin-5 promoting neuroinflammation or tumor metastasis
Nanou, A., Bourbouli, M., Vetrano, S., Schaeper, U., Ley, S., Kollias, G.
Cell Reports (2021)
2020
- A Bovine Enteric Mycobacterium Infection Model to Analyze Parenteral Vaccine-Induced Mucosal Immunity and Accelerate Vaccine Discovery
Facciuolo, A., Lee, A.H., Trimble, M.J., Rawlyk, N., Townsend, H.G., Bains, M., Arsic, N., Mutharia, L.M., Potter, A., Gerdts, V., & Napper, S.
Frontiers in Immunology (2020) - Antisense oligonucleotide in LNA-gapmer design targeting TGFBR2—a key single gene target for safe and effective inhibition of TGFβ signaling
Kuespert, S., Heydn, R., Peters, S., Wirkert, E., Meyer, A.L., Siebörger, M., Johannesen, S., Aigner, L., Bogdahn, U. and Bruun, T.H.
International Journal of Molecular Sciences (2020) - A three-part, randomised study to investigate the safety, tolerability, pharmacokinetics and mode of action of BC 007, neutraliser of pathogenic autoantibodies against G-protein coupled receptors in healthy, young and elderly subjects.
Becker, N.P., Haberland, A., Wenzel, K., Göttel, P., Wallukat, G., Davideit, H., Schulze-Rothe, S., Hönicke, A.S., Schimke, I., Bartel, S. & Grossmann, M.
Clinical Drug Investigation (2020) - EGFP reporters for direct and sensitive detection of mutagenic bypass of DNA lesions
Rodriguez-Alvarez, M., Kim, D., & Khobta, A.
Biomolecules (2020) - Macrophages produce and functionally respond to interleukin-34 in colon cancer
Franzè, E., Laudisi, F., Di Grazia, A., Marônek, M., Bellato, V., Sica, G., Monteleone, G.
Cell Death Discov. (2020) - Radiolabeling and PET–MRI microdosing of the experimental cancer therapeutic, MN-anti-miR10b, demonstrates delivery to metastatic lesions in a murine model of metastatic breast cancer
Le Fur, M., Ross, A., Pantazopoulos, P., Rotile, N., Zhou, I., Caravan, P., Medarova, Z., Yoo, B.
Cancer Nano (2021) - Aptamer BC 007 - Efficient binder of spreading-crucial SARS-CoV-2 proteins
Weisshoff, H., Krylova, O., Nikolenko, H., Düngen, H.-D., Dallmann, A., Becker, S., Göttel, P., Müller, J., Haberland, A.
Heliyon (2020) - Dose-Finding Study and Pharmacokinetics Profile of the Novel 13-Mer Antisense miR-221 Inhibitor in Sprague-Dawley Rats
Di Martino, M. T., Arbitrio, M., Caracciolo, D., Scionti, F., Tagliaferri, P., Tassone, P.
Molecular Therapy: Nucleic Acids (2020) - MRI-based molecular imaging of epicardium-derived stromal cells (EpiSC) by peptide-mediated active targeting
Straub, T., Nave, J., Bouvain, P., Akbarzadeh, M., Dasa, S. S. K., Kistner, J., Ding, Z., Marzoq, A., Stepanow, S., Becker, K., Hesse, J., Köhrer, K., Flögel, U., Ahmadian, M. R., French, B. A., Schrader, J., Temme, S.
Sci Rep (2020) - RNA and DNA Binding Epitopes of the Cold Shock Protein TmCsp from the Hyperthermophile Thermotoga maritima
von König, K., Kachel, N., Kalbitzer, H. R., Kremer, W.
Protein J (2020)
2019
- Allometric scaling approaches for predicting human pharmacokinetic of a locked nucleic acid oligonucleotide targeting cancer-associated miR-221
Di Martino, M.T., Arbitrio, M., Fonsi, M., Erratico, C.A., Scionti, F., Caracciolo, D., Tagliaferri, P. & Tassone, P.
Cancers (2019) - Effects of siRNA-mediated knockdown of GSK3β on retinal ganglion cell survival and neurite/axon growth
Ahmed, Z., Morgan-Warren, P.J., Berry, M., Scott, R.A.H., & Logan, A.
Cells (2019) - Comparison of PEGylated and non-PEGylated proticles: An in vitro and in vivo study
Fresacher, K., Helbok, A., Reiser, M., Blass, S., Rangger, C., Mair, C., von Guggenberg, E., Decristoforo, C., Andreae, F., & Zimmer, A.
European Journal of Pharmaceutical Sciences (2019) - Nucleotide excision repair of abasic DNA lesions
Kitsera, N., Rodriguez-Alvarez, M., Emmert, S., Carell, T., & Khobta, A.
Nucleic Acids Research (2019) - Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect
Cideciyan, A.V., Jacobson, S.G., Drack, A.V., Ho, A.C., Charng, J., Garafalo, A.V., Roman, A.J., Sumaroka, A., Han, I.C., Hochstedler, M.D. & Pfeifer, W.L.
Nature Medicine (2019) - Selective eradication of human non-small cell lung cancer cells using aptamer-decorated nanoparticles harboring a cytotoxic drug cargo
Engelberg, S., Netzer, E., Assaraf, Y. G., Livney, Y. D.
Cell Death Discov. (2019) - EnanDIM - a novel family of L-nucleotide-protected TLR9 agonists for cancer immunotherapy
Kapp, K., Volz, B., Curran, M. A., Oswald, D., Wittig, B., Schmidt, M.
Immunotherapy Cancer (2019) - Macrophages Are a Potent Source of Streptococcus-Induced IFN-β
Feuerstein, R., Gres, V., Elias Perdigó, N., Baasch, S., Freudenhammer, M., Elling, R., Henneke, P.
J Immunol (2019) - Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis
Beumer, W., Swildens, J., Leal, T., Noel, S., Anthonijsz, H., van der Horst, G., Kuiperij-Boersma, H., Potman, M., van Putten, C., Biasutto, P., Platenburg, G., de Jonge, H., Henig, N., Ritsema, T.
PLOS ONE (2019) - Enzyme-free ligation of dimers and trimers to RNA primers
Sosson, M., Pfeffer, D., Richert, C.
Nucleic Acids Research (2019) - Epigenetic control of the angiotensin-converting enzyme in endothelial cells during inflammation
Mudersbach, T., Siuda, D., Kohlstedt, K., Fleming, I.
PLOS ONE (2019) - UGCG influences glutamine metabolism of breast cancer cells
Schömel, N., Hancock, S. E., Gruber, L., Olzomer, E. M., Byrne, F. L., Shah, D., Hoehn, K. L., Turner, N., Grösch, S., Geisslinger, G., Wegner, M.-S.
Sci Rep (2019) - Site-Specific Cleavage of RNAs Derived from the PIM1 3′-UTR by a Metal-Free Artificial Ribonuclease
Zellmann, F., Thomas, L., Scheffer, U., Hartmann, R. K., Göbel, M. W.
Molecules (2019) - Myeloid-Specific Deletion of the AMPKα2 Subunit Alters Monocyte Protein Expression and Atherogenesis
Fisslthaler, B., Zippel, N., Abdel Malik, R., Delgado Lagos, F., Zukunft, S., Thoele, J., Siuda, D., Soehnlein, O., Wittig, I., Heidler, J., Weigert, A., Fleming, I.
Int. J. Mol. Sci. (2019) - VEGF-Trap is a potent modulator of vasoregenerative responses and protects dopaminergic amacrine network integrity in degenerative ischemic neovascular retinopathy
Rojo Arias, J. E., Economopoulou, M., Juárez López, D. A., Kurzbach, A., Au Yeung, K. H., Englmaier, V., Merdausl, M., Schaarschmidt, M., Ader, M., Morawietz, H., Funk, R. H. W., Jászai, J.
J. Neurochem. (2019) - Stable depletion of RUNX1-ETO in Kasumi-1 cells induces expression and enhanced proteolytic activity of Cathepsin G and Neutrophil Elastase
Schoenherr, C., Wohlan, K., Dallmann, I., Pich, A., Hegermann, J., Ganser, A., Hilfiker-Kleiner, D., Heidenreich, O., Scherr, M., Eder, M.
PLOS ONE (2019)
giving hope - changing lifes
Approved Oligonucleotide Therapeutics
Nucleic acid-based therapeutics have emerged as a powerful class of medicines, revolutionizing the treatment of genetic disorders, cancers, and other diseases. To date, 26 oligonucleotide-based therapies have received regulatory approval, highlighting the growing impact of these innovative treatments. Notably, 6 of these approvals were granted in 2023 alone, reflecting significant advancements in the field and an accelerated pace of clinical translation.
The following list provides an overview of all currently approved nucleic acid-based therapies, demonstrating the versatility and potential of oligonucleotides to address diverse medical challenges.
Therapeutic |
Type of Nucleic Acid |
Year of Approval |
Disease / Condition |
Qfitlia (Fitusiran) |
Small Interfering RNA (siRNA) |
2025 |
Hemophilia A and B – rare genetic bleeding disorders caused by missing or defective clotting factors (factor VIII in Hemophilia A, factor IX in Hemophilia B). This leads to prolonged bleeding episodes, joint damage, and in severe cases, life-threatening internal bleeding. While traditional treatments involve replacing the missing factors, challenges remain—especially for patients who develop inhibitors. |
Rytelo (Imetelstat) |
Antisense Oligonucleotide (ASO) |
2024 |
Amyotrophic Lateral Sclerosis (ALS) - a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to muscle weakness, paralysis, and ultimately the loss of essential functions such as breathing and swallowing. While the exact cause is not always known, genetic and environmental factors play a role. Rytelo works by protecting nerve cells from further degeneration and reducing inflammation in the central nervous system, helping to slow disease progression and preserve motor function for a longer period. |
TRYNGOLZA (Olezarsen) |
Antisense Oligonucleotide (ASO) |
2024 |
Friedreich’s Ataxia (FA) - a rare genetic neurodegenerative disorder that leads to progressive loss of muscle coordination (ataxia), muscle weakness, and balance issues. It is caused by a mutation in the FXN gene, resulting in a deficiency of frataxin protein. This leads to mitochondrial dysfunction and cell damage, particularly affecting the nervous system and heart. Tryngolza aims to increase frataxin levels and improve cellular function, helping to slow disease progression and enhance patients’ quality of life. |
Cyfendus (Anthrax Vaccine Absorbed, Adjuvanted) |
CpG ODN |
2023 |
Anthrax caused by Bacillus anthracis, is a life-threatening infection that can affect the skin, lungs, and digestive system, often resulting in severe illness if untreated. Cyfendus is a specialized therapeutic developed to neutralize anthrax toxins in the body. By blocking these toxins, Cyfendus protects cells from damage, limits infection spread, and aids recovery from anthrax exposure, especially in cases of inhalation anthrax, which is the most dangerous form. It serves as a vital option for both preventing and treating anthrax in high-risk exposure scenarios. |
Exa-cel (Exagamglogene autotemcel) |
CRISPR-Cas9 Gene Editing |
2023 |
Sickle Cell Disease (a genetic disorder that affects the protein in reg blood cells that carries oxygen, leading to blockages, pain, and damage to organs, along with anemia) & Beta-Thalassemia (a genetic disorder where the body doesn’t produce enough hemoglobin, leading to a shortage of healthy red blood cells, causing anemia). Exa-cel is a gene editing treatment to edit the patient’s own stem cell, correcting the genes causing the diseases, allowing the body to produce enough healthy red blood cells and reduce disease symptoms. |
Izervay (Avacincaptad pegol) |
Aptamer |
2023 |
Geographic Atrophy (GA) - an advanced form of Age-Related Macular Degeneration (AMD), where parts of the retina deteriorate, leading to vision loss over time. This condition is marked by the gradual death of retinal cells, which impacts central vision, making it difficult for individuals to read, drive, or recognize faces. Izervay targets key proteins involved in the inflammation and cell damage seen in GA, reducing retinal cell death and slowing disease progression. By protecting the remaining retinal cells, Izervay helps maintain vision and delay further vision loss in people with GA. |
Qualsody (Tofersen) |
Antisense Oligonucleotide (ASO) |
2023 |
Amyotrophic Lateral Sclerosis (ALS) – a progressive neurological disorder that affects nerve cells in the brain and spinal cord, leading to loss of muscle control. Qualsody targets the genetic root of ALS by reducing the production of a toxic protein associated with nerve damage. By lowering levels of this protein, Qualsody aims to slow the progression of the disease and maintain muscle function for as long as possible. |
Rivfloza (Nedosiran) |
Small Interfering RNA (siRNA) |
2023 |
Myotonic Dystrophy Type 1 (DM1) - a rare genetic disorder characterized by progressive muscle wasting and weakness, particularly affecting the face, neck, and limbs. It can also lead to heart, breathing, and digestive problems. DM1 occurs due to a mutation that creates abnormal mRNA, leading to toxic clumps in muscle cells that disrupt normal functioning. Rivloza targets this abnormal mRNA, breaking down the toxic clumps and restoring normal cellular activity, which helps reduce muscle stiffness and other symptoms, improving overall muscle function and quality of life for patients with DM1. |
Wainua (Eplontersen) |
Antisense Oligonucleotide (ASO) |
2023 |
Huntington’s Disease (HD) - a genetic disorder that causes the progressive breakdown of nerve cells in the brain, affecting movement, cognition, and mental health. The disease is caused by mutations in the huntingtin (HTT) gene, leading to the production of a harmful, misfolded protein that builds up in nerve cells. Wainua works by targeting and reducing the levels of this toxic HTT protein. By doing so, it helps prevent further nerve cell damage, slowing down disease progression and alleviating some of the motor and cognitive symptoms associated with HD. |
Amvuttra (Vutrisiran) |
Small Interfering RNA (siRNA) |
2022 |
Hereditary transthyretin-mediated amyloidosis (hATTR) – a rare genetic disorder, where the protein transthyretin (TTR) becomes unstable and misfolds. This leads to the buildup of amyloid (abnormal protein deposits) in different tissues and organs, such as the nerves, heart, and digestive system. Over time, these amyloid deposits cause nerve damage, heart problems, and digestive issues. Amvuttra works by reducing the production of the defective TTR protein and thus prevent the buildup of amyloid deposits, slowing disease progression and improving symptoms related to nerve and organ damage. |
Amondys 45 (Casimersen) |
Antisense Oligonucleotide (ASO) |
2021 |
Duchenne muscular dystrophy (DMD) - a genetic disorder that mainly affects boys and causes progressive muscle weakness. This occurs because the body does not produce enough dystrophin, a protein necessary for maintaining muscle strength. Over time, the lack of dystrophin leads to muscle breakdown and loss of muscle function, impacting movement as well as the heart and lung. Amondys 45 works by allowing the body to skip over the defect part of the dystrophin gene and make a shorter but still functional version of the dystrophin protein. This helps slow down muscle damage and preserves muscle function for longer. |
Leqvio (Inclisiran) |
Small Interfering RNA (siRNA) |
2020 |
Hypercholesterolemia – a condition where there is too much cholesterol, especially “bad” LDL cholesterol, in the blood. High cholesterol can cause fatty deposits to build up in blood vessels, which increases the risk of heart disease, heart attacks, and strokes. Leqvio helps lowering LDL cholesterol levels by targeting a protein called PCSK9, which normally breaks down receptors that remove LDL cholesterol from the blood. Leqvio helps the liver keep more of these receptors, allowing the body to remove more LDL cholesterol and lower its levels over time. |
Oxlumo (Lumasiran) |
Small Interfering RNA (siRNA) |
2020 |
Primary hyperoxaluria type 1 (PH1) – a rare genetic disorder where the liver produces too much oxalate, a substance that the body usually gets rid of through urine. When too much oxalate builds up, it can combine with calcium to form kidney stones and crystals, leading to serious kidney damage. Over time, the excess oxalate can also affect other parts of the body, causing issues in bones, eyes, heart, and skin. Oxlumo works by reducing the production of oxalate in the liver by targeting the gene that produces the enzyme responsible for making too much oxalate, lowering its levels and preventing the buildup of harmful oxalate in the kidneys and other organs. |
Viltepso (Viltolarsen) |
Antisense Oligonucleotide (ASO) |
2020 |
Duchenne muscular dystrophy (DMD) - a genetic disorder that mainly affects boys and causes progressive muscle weakness. This occurs because the body does not produce enough dystrophin, a protein necessary for maintaining muscle strength. Over time, the lack of dystrophin leads to muscle breakdown and loss of muscle function, impacting movement as well as the heart and lung. Viltepso works by allowing the body to skip over the defect part of the dystrophin gene and make a shorter but still functional version of the dystrophin protein. This helps slow down muscle damage and preserves muscle function for longer. |
Givlaari (Givosiran) |
Small Interfering RNA (siRNA) |
2019 |
Acute hepatic porphyria (AHP) – a group of rare genetic disorders that affect the liver and lead to a buildup of toxic substances involved in the production of heme, a component of hemoglobin. This buildup can trigger severe, sudden attacks of abdominal pain, vomitin, and nervous system issues such as muscle weakness or seizures. Givlaari works by reducing the production of a specific enzyme in the liver that leads to the buildup of toxic substances. By lowering these toxins, Givlaari helps reduce the frequency and severity of acute attacks and improves the overall quality of life for people with AHP. |
Vyondys 53 (Golodirsen) |
Antisense Oligonucleotide (ASO) |
2019 |
Duchenne muscular dystrophy (DMD) - a genetic disorder that mainly affects boys and causes progressive muscle weakness. This occurs because the body does not produce enough dystrophin, a protein necessary for maintaining muscle strength. Over time, the lack of dystrophin leads to muscle breakdown and loss of muscle function, impacting movement as well as the heart and lung. Vyondys 53 is for people with a specific genetic mutation that affects a certain part of the dystrophin gene. The medicine helps the body skip over the mutated part of the gene, so it can make a shorter but still working version of the dystrophin protein. This helps slow down the muscle damage caused by DMD. |
Waylivra (Volanesorsen) |
Antisense Oligonucleotide (ASO) |
2019 |
Familial chylomicronemia syndrome (FCS) – a rare genetic disorder where the body cannot properly break the fats from the food you eat. This happens due to a deficiency of a key enzyme called lipoprotein lipase (LPL). As a result, fats build up in the blood, leading to extremely high triglyceride levels. This can cause symptoms like severe abdominal pain, pancreatitis, and fatty deposits in the skin. Waylivra targets the production of a protein called apolipoprotein CIII (apoC-III), which controls how triglycerides are processed in the body. By reducing apoC-III, Waylivra helps lower triglyceride levels in the blood, reducing the risk of pancreatits and other complications associated with FCS. |
Tagsedi (Inotersen) |
Antisense Oligonucleotide (ASO) |
2018 |
Hereditary transthyretin-mediated amyloidosis (hATTR) – a rare genetic disorder, where the protein transthyretin (TTR) becomes unstable and misfolds. This leads to the buildup of amyloid (abnormal protein deposits) in different tissues and organs, such as the nerves, heart, and digestive system. Over time, these amyloid deposits cause nerve damage, heart problems, and digestive issues. Tagsedi works by targeting the messenger RNA that directs the production of transthyretin. By blocking this RNA, Tagsedi reduces the amount of the abnormal transthyretin protein produced, helping to lower amyloid buildup and slow down disease progression. |
Onpattro (Patisiran) |
Small Interfering RNA (siRNA) |
2018 |
Hereditary transthyretin-mediated amyloidosis (hATTR) – a rare genetic disorder, where the protein transthyretin (TTR) becomes unstable and misfolds. This leads to the buildup of amyloid (abnormal protein deposits) in different tissues and organs, such as the nerves, heart, and digestive system. Over time, these amyloid deposits cause nerve damage, heart problems, and digestive issues. Onpattro works by silencing the gene that produces the defective transthyretin protein. This helps lowering the buildup of amyloid deposits, which can slow down or even improve symptoms related to nerve and organ damage. |
Hepislav |
CpG ODN |
2017 |
Hepatitis B - a serious liver infection caused by the hepatitis B virus, which can lead to chronic liver disease, cirrhosis, and liver cancer if untreated. HBV is highly infectious and can be transmitted through bodily fluids, posing a risk for people worldwide. Hepislav is an innovative vaccine designed to stimulate a strong and rapid immune response, offering protection with a two-dose schedule, compared to the traditional three. By boosting the body’s immune defenses, Hepislav helps prevent HBV infection, providing long-term protection and reducing the spread of this life-threatening virus. |
Exondys 51 (Eteplirsen) |
Antisense Oligonucleotide (ASO) |
2016 |
Duchenne muscular dystrophy (DMD) – a genetic disorder that mainly affects boys and causes progressive muscle weakness. This occurs because the body does not produce enough dystrophin, a protein necessary for maintaining muscle strength. Over time, the lack of dystrophin leads to muscle breakdown and loss of muscle function, impacting movement as well as the heart and lung. Exondys 51 works by skipping over the mutated part of the gene responsible for making dystrophin, allowing the body to produce a shorter but functional version of the protein. This helps slow down muscle damage in people with DMD. |
Spinraza (Nusinersen) |
Antisense Oligonucleotide (ASO) |
2016 |
Spinal muscular atrophy (SMA) – a genetic disorder that affects the nerve cells in the spinal cord, which control muscle movement. People with SMA lose motor neurons, the cells responsible for sending signals from the brain to the muscles. This leads to muscle weakness and can affect the ability to walk, eat, or even breathe. Spinraza works by helping the body produce more of the SMN protein, which is crucial for motor neurons to function properly. This helps slow down or even improve muscle weakness in people with SMA. |
Kynamro (Mipomersen) |
Antisense Oligonucleotide (ASO) |
2013 |
Homozygous familial hypercholesterolemia – a rare genetic disorder where the body is unable to remove bad cholesterol (LDL) from the blood properly, causing extremely high cholesterol levels from birth. This can lead to heart problems and clogged arteries at a very young age. Kynamro works by blocking the production of a specific protein, which is necessary for making LDL cholesterol. By reducing this protein, Kynamro helps lower the amount of bad cholesterol in the blood. |
Macugen (Pegaptanib) |
Aptamer |
2004 |
Age-related macular degeneration (AMD) – an eye disease that mostly affects older adults and causes damage to the central part of the retina called the macula, which helps by seeing fine details. There are two types of AMD: dry (more common) and wet (less common, but more serious). In wet AMD, abnormal blood vessels grow under the retina, causing rapid vision loss. Macugen treats wet AMD by blocking a specific protein, which is responsible for the growth of those abnormal blood vessels in the eye. By stopping the protein, Macugen helps slow down or even stop the damage to the eye and prevents further vision loss. |
Vitravene (Fomivirsen) |
Antisense Oligonucleotide (ASO) |
1998 |
CMV retinitis in AIDS patients – an eye infection caused by the cytomegalovirus (CMV) and most common in people with very weak immune systems, such as those with advanced AIDS. Without treatment, the virus damages vision quickly, leading to blind spots, blurry vision, or even complete blindness. Vitravene is an antisense therapy that targets the messenger RNA of the cytomegalovirus, stopping the virus from copying itself. |
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