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Home » Topics » Drugs » Antisense

Antisense
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Veritas, Mitsubishi Gas Chemical partner for new ASO candidates

July 8, 2025
By Marian (YoonJee) Chu
No Comments
Veritas In Silico Inc. (VIS) entered a joint research agreement with Mitsubishi Gas Chemical Co. Inc. to use VIS’s mRNA structural motif analyzing platform technology, called Ibvis, to develop antisense oligonucleotide drug candidates.
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Dyne vHOT in DM1 but shares cool on endpoint switch

June 17, 2025
By Randy Osborne
No Comments
The revised trial protocol that means a delay in filing for U.S. approval of DYNE-101 to treat myotonic dystrophy type 1 (DM1) dented shares of Dyne Therapeutics Inc. (NASDAQ:DYN), which closed June 17 at $10.86, down $2.96, or 21%.
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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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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
No Comments
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
No Comments
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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Brain and DNA
Neurology/psychiatric

Nissan Chemical, Sanwa Kagaku Kenkyusho to codevelop antisense drug candidate

April 8, 2025
Nissan Chemical Corporation and Sanwa Kagaku Kenkyusho Co. Ltd. (SKK) have entered an agreement to codevelop SK-2407/SN-001 for the treatment of dentatorubral-pallidoluysian atrophy (DRPLA) in Japan.
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Illustration of DNA, magnifying glass
Neurology/psychiatric

Quiver Bioscience collaborates with Dup15q Alliance to advance ASOs for Dup15q syndrome

March 20, 2025
Quiver Bioscience Inc. is collaborating with the Dup15q Alliance to advance an antisense oligonucleotide (ASO) therapeutic program for chromosome 15q duplication (Dup15q) syndrome.
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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.
Read More
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).
Read More
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