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Home » Keywords » antisense oligonucleotides

Items Tagged with 'antisense oligonucleotides'

ARTICLES

Neurology/psychiatric

App- or Rab5-targeting ASOs reverse Down syndrome-linked Alzheimer’s disease effects in mouse model

May 21, 2025
No Comments
Down syndrome (DS) is the most prevalent genetic cause of Alzheimer’s disease (AD). Previous evidence suggests that increased dosage of the amyloid precursor protein (APP) gene plays a crucial role in AD in individuals with Down syndrome (DS-AD), making APP expression a crucial therapeutic target.
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Illustration of human brain and dna
Neurology/psychiatric

Quralis signs agreements to further fragile X syndrome research

May 16, 2025
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Quralis Corp. has entered into a number of agreements with the aim of advancing the treatment of fragile X syndrome, a genetic condition caused by a mutation of a single gene – fragile X messenger ribonucleoprotein 1 (FMR1) – on the X chromosome.
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Cerebellum, brain stem, spinal cord
Neurology/psychiatric

Cure Rare Disease’s CRD-002 awarded orphan drug designation for spinocerebellar ataxia type 3

May 15, 2025
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The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia type 3 (SCA3).
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Cancer

Antisense oligonucleotide targeting nicotinamide N-methyltransferase as a potential cancer therapy

May 12, 2025
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Researchers from Osaka University have developed a novel approach to target nicotinamide N-methyltransferase (NNMT), an enzyme implicated in cancer progression, using antisense oligonucleotides (ASOs).
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Woman and 3D brain

ABL lands £2B deal with GSK based on BBB passing drug platform

April 8, 2025
By Marian (YoonJee) Chu
ABL Bio Inc. announced April 7 that it sealed a potential £2.075 billion (US$2.65 billion) license deal with GSK plc, granting GSK global rights to use ABL’s blood-brain barrier (BBB) penetrating bispecific antibody platform, Grabody-B, to develop multiple programs in the neurodegenerative disease arena. Under the terms signed April 5, ABL agreed to transfer Grabody-B-related technology and know-how to GSK, upon which GSK will assume responsibility for preclinical and clinical development, manufacturing and commercialization.
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Woman and 3D brain

ABL lands £2B deal with GSK based on BBB passing drug platform

April 7, 2025
By Marian (YoonJee) Chu
ABL Bio Inc. announced April 7 that it sealed a potential £2.075 billion (US$2.65 billion) license deal with GSK plc, granting GSK global rights to use ABL’s blood-brain barrier (BBB) penetrating bispecific antibody platform, Grabody-B, to develop multiple programs in the neurodegenerative disease arena. Under the terms signed April 5, ABL agreed to transfer Grabody-B-related technology and know-how to GSK, upon which GSK will assume responsibility for preclinical and clinical development, manufacturing and commercialization.
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Cancer

Antisense oligonucleotides targeting FANCM show preclinical promise in ALT-positive cancers

Feb. 28, 2025
A recent study by researchers from Nanyang Technological University identified Fanconi anemia complementation group M (FANCM) as a crucial regulator of alternative lengthening of telomere (ALT), aiming to develop new antisense oligonucleotides (ASOs) to suppress its function.
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Illustration of tau proteins in Alzheimer's disease
Neurology/psychiatric

Stereopure gapmer ASOs targeting tau show promising preclinical safety and activity

Feb. 7, 2025
New gapmer antisense oligonucleotide (ASO) candidates have been designed at Eisai Co. Ltd. to reduce microtubule-associated protein tau.
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Dollar sign droplet above test tube
Neurology/psychiatric

CIRM grant supports Cure Rare Disease’s antisense oligonucleotide therapy for SCA3

Feb. 7, 2025
Cure Rare Disease has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for spinocerebellar ataxia type 3 (SCA3).
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Illustration of human eye
Ocular

Targeting recurrent ABCA4 variant with antisense oligonucleotides as new strategy for Stargardt disease

Feb. 3, 2025
Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon 6, c.768G>T. Due to its high prevalence, c.768G>T is an interesting therapeutic target for STGD1. Researchers from Radboud University developed a new antisense oligonucleotide (AON) therapy, designed to rescue the splicing defect caused by this variant.
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