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Neurology/psychiatric

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

May 15, 2025

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).

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).

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.

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.

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.

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.

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).

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.

Regulus heads to phase III after more positive kidney disease data

Jan. 29, 2025

By Jennifer Boggs

In an indication that has proved difficult for biopharma to conquer, Regulus Therapeutics Inc. disclosed further positive data from its ongoing phase Ib study testing RGLS-8429 in autosomal dominant polycystic kidney disease and laid out its plans to move straight into a phase III trial later this year, with the potential for an accelerated U.S. approval.

Dyne seeks accelerated path on positive dystrophy data; shares down

Jan. 10, 2025

By Lee Landenberger

Dyne Therapeutics Inc. is eyeing accelerated approval for its myotonic dystrophy type 1 treatment after reviewing new results from a phase I/II study. DYNE-101, an oligonucleotide antisense and DMPK gene modulator, produced results on disease biomarkers that included DMPK and splicing correction, disease progression reversal on several functional endpoints and a favorable safety profile. The accelerated approval submission could come in the first half of 2026.

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

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

Nissan Chemical, Sanwa Kagaku Kenkyusho to codevelop antisense drug candidate

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

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

Stereopure gapmer ASOs targeting tau show promising preclinical safety and activity

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

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

Regulus heads to phase III after more positive kidney disease data

Dyne seeks accelerated path on positive dystrophy data; shares down

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