Antisense Therapy

Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). 

ASOs are capable of altering mRNA expression through a variety of mechanisms.

Several ASOs have been approved.

The common stem for antisense oligonucleotides drugs is -rsen. The substem -virsen designates antiviral antisense oligonucleotides.

ASO-based drugs have highly modified, single-stranded chains of synthetic nucleic acids that achieve wide tissue distribution with very long half-lives.

Phosphorothioate ASOs can be delivered to cells without the need of a delivery vehicle. 

ASOs do not penetrate the blood brain barrier when delivered systemically.

They can distribute across the neuraxis if injected in the cerebrospinal fluid typically by intrathecal administration. 

By using conjugated ligands it greatly enhances delivery efficiency and cell-type specific targeting.

Mmilasen, an antisense oligonucleotide drug for Batten disease.

Fomivirsen (Vitravene), was approved by as a treatment for cytomegalovirus retinitis.

Several oligonucleotides have been approved to treat specific groups of mutations causing Duchenne muscular dystrophy. 

Eteplirsen (ExonDys51) is approved for the treatment of cases that can benefit from skipping exon 51 of the dystrophin transcript. 

Golodirsen (Vyondys 53) is approved for the treatment of cases that can benefit from skipping exon 53 of the dystrophin transcript. 

Viltolarsen (Viltepso) is approved approval for the treatment of cases that can benefit from skipping exon 53 of the dystrophin transcript.

Familial chylomicronaemia syndrome has been treated with Volanesorsen.

Familial hypercholesterolemia can be treated with mipomersen (Kynamro).

Hereditary transthyretin-mediated amyloidosis can be treated with


Spinal muscular atrophy utilizes nusinersen an antisense therapy.

Tofersen is currently being tested in a phase 3 trial for amyotrophic lateral sclerosis (ALS) due to mutations in the SOD1 gene.

Bepirovirsen in chronic hepatitis B lowered hepatitis B antigen and HBV DNA loss in 9-10 % of participants.

Clinical trials are ongoing for several diseases and conditions including:

Acromegaly, age related macular degeneration, Alzheimer’s disease, amyotrophic lateral sclerosis, autosomal dominant retinitis pigmentosa, beta thalassemia, cardiovascular disease, elevated level of lipoprotein(a),centronuclear myopathy, coagulopathies, cystic fibrosis, Duchenne muscular dystrophy, diabetes, epidermolysis bullosa dystrophica, familial chylomicronemia syndrome, frontotemporal dementia, Fuchs’ dystrophy, hepatitis B, hereditary angioedema, hypertension, IgA nephropathy, Leber’s hereditary optic neuropathy, multiple system atrophy, non-alcoholic fatty liver disease, Parkinson’s disease, prostate cancer, Stargardt disease, STAT3-expressing cancers, Usher syndrome.

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