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Knowledge Base

Knowledge Base

BioSpring's 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 here, and get an overview of approved oligonucleotide therapeutics.

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Areas of Application of Nucleic Acids

Therapeutic Applications

Early-Stage Research and Development Projects

Diagnostics

Other Applications

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.

2026 

  • Cystine import and oxidative catabolism fuel vascular growth and repair via nutrient-responsive histone acetylation
    Drekolia, M.-K., Mettner, J., Wang, D., Delgado Lagos, F., Koch, C., Hecker, D., Eresch, J., Mao, Y., Bähr, M., Weichenhan, D., Cordero, J., Wittig, J., Zhang, B., Cui, H., Li, X., Oo, J. A., Weigert, A., Siragusa, M., Klatt, S., Fleming, I., Günther, S., Looso, M., Brandes, R. P., Langer, H. F., Papapetropoulos, A., Singhal, M., Schulz, M. H., Plass, C., Heineke, J., Dobreva, G., Hu, J., Bibli, S.-I.
    Cell Metabolism (2026)


  • Intestinal organoids as a platform for functional evaluation of ASO-mediated splicing modulation in cystic fibrosis
    Stanleigh, N., Irony-Tur Sinai, M., Oren, Y., Ozeri-Galai, E., Hart, G., Grunewald, M., Birimberg-Schwartz, L., Beekman, J. M., Kerem, E., Wilschanski, M., Kerem, B.
    Journal of Cystic Fibrosis (2026)

  • Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE
    Lazzarotto, C. R., Katta, V., Li, Y., Manquen, G., Wood, R. K., Chyr, J., Urbina, E., Matsubara, A., Lee, G., Wu, X., De Ravin, S. S., Tsai, S. Q.
    Nature Biotechnology (2026)

  • Therapeutic suppression of Tubb4a rescues H-ABC leukodystrophy
    Sase, S., Hacker, J. L., Napit, P. R., Bhagavatula, A., Woidill, S., D’Alessandro, A., Jeffries, M. A., Almad, A., Takanohashi, A., Padiath, Q. S., Grinspan, J. B., Marsh, E. D., Vanderver, A. 
    Molecular Therapy (2026) 

 

2025

2024

2023

2022

2021

2020

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, 27 oligonucleotide-based therapies have received regulatory approval, highlighting the growing impact of these innovative treatments. Notably, half of these approvals were granted in the last 5 years, 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.

Trade Name (Drug Name)

Year of Approval FDA/EMA

Modality

Disease Indication

REDEMPLO (Plozasiran)

2025/NA

Small Interfering RNA (siRNA)

Familial Chylomicronemia Syndrome (FCS) 

Dawnzera (Donidalorsen)

2025/2026

Antisense Oligonucleotide (ASO) 

Hereditary angioedema (HAE) 

QFITLIA (Fitusiran) 

2025/NA

Small Interfering RNA (siRNA) 

Hemophilia A and B 

Rytelo (Imetelstat)

2024/2025

Antisense Oligonucleotide (ASO)

Transfusion-dependent anaemia of MDS patients 

Tryngolza (Olezarsen)

2024/2025

Antisense Oligonucleotide (ASO)

Familial chylomicronemia syndrome (FCS) 

Wainua (Eplontersen)

2023/2025

Antisense Oligonucleotide (ASO) 

TTR-mediated polyneuropathy/TTR-mediated cardiomyopathy 

Casgevy (Exagamglogene autotemcel)

2023/2024

CRISPR-Cas9 Gene Editing

Sickle cell anemia

Rivfloza (Nedosiran)

2023/NA

Small Interfering RNA (siRNA) 

Primary hyperoxaluria 

Cyfendus (Anthrax Vaccine Absorbed, Adjuvanted) 

 2023/NA 

 CpG ODN 

Postexposure prophylaxis for anthrax  

Izervay (Avacincaptad pegol)

2023/2023

Aptamer

 Geographic atrophy of age-related macular degeneration 

Qualsody (Tofersen)

2023/2022

Antisense Oligonucleotide (ASO)

Amyotrophic lateral sclerosis 

Amvuttra (Vutrisiran)

2022/2022

Small Interfering RNA (siRNA)

Polyneuropathy of hereditary transthyretin amyloidosis 

Leqvio (Inclisiran)

2021/2020

Small Interfering RNA (siRNA) 

Hypercholesteremia, primary hyperlipidemia  

Amondys 45 (Casimersen) 

2021/NA

ASO (PMO) (Phosphorodiamidate morpholino oligomer)

Duchenne muscular dystrophy

Oxlumo (Lumasiran)

2020/2020

Small Interfering RNA (siRNA)

Primary hyperoxaluria type 1

Viltepso (Viltolarsen)

2020/2020

ASO (PMO) (Phosphorodiamidate morpholino oligomer) 

Duchenne muscular dystrophy

Vyondys 53 (Golodirsen)

2019/NA

ASO (PMO) (Phosphorodiamidate morpholino oligomer) 

Duchenne muscular dystrophy

Givlaari (Givosiran)

2019/2020

Small Interfering RNA (siRNA)

Acute hepatic porphyria

Waylivra (Volanesorsen)

NA/2019

Antisense Oligonucleotide (ASO)

Familial chylomicronemia syndrome

Tagsedi (Inotersen)

2018/2018

Antisense Oligonucleotide (ASO)

Polyneuropathy of hereditary transthyretin amyloidosis

Onpattro (Patisiran)

2018/2018

Small Interfering RNA (siRNA)

Polyneuropathy of hereditary transthyretin amyloidosis

Hepislav-B

2017/2021

CpG ODN

Hepatitis B

Spinraza (Nusinersen)

2016/2017

Antisense Oligonucleotide (ASO)

Spinal muscular atrophy

Exondys 51 (Eteplirsen)

2016/NA

ASO (PMO) (Phosphorodiamidate morpholino oligomer)

Duchenne muscular dystrophy

Kynamro (Mipomersen)(withdrawn) 

2013/NA

Antisense Oligonucleotide (ASO)

Homozygous familial hypercholesteremia 

Macugen (Pegaptanib)(withdrawn) 

2004/2006

Aptamer

 Age-related macular degeneration 

Vitravene (Fomivirsen)  (withdrawn)

1998/1999

Antisense Oligonucleotide (ASO)

 CMV retinitis 

 

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