https://www.bioworld.com/rss en-us Researchers from McGill University and collaborating institutions aimed to investigate whether oligonucleotides are a viable drug class to prevent hydrocephalus. http://www.bioworld.com/articles/730996 Mon, 11 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/730996-aso-therapy-prevents-hydrocephalus-in-a-monogenic-syndrome-model Illustration of brain in child with hydrocephalus Three decades of trial-and-error, and the resulting safety data, in the oligonucleotide-based therapeutic space have paved way for the present-day innovations and the promise of “programmable,” precision medicine for patients, speakers at Bio Korea 2026 said April 28. http://www.bioworld.com/articles/730927 Tue, 05 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/730927-bio-korea-2026-kicks-off-with-spotlight-on-oligonucleotides AI generated image for researcher developing antisense oligonucleotides Three decades of trial-and-error, and the resulting safety data, in the oligonucleotide-based therapeutic space have paved way for the present-day innovations and the promise of “programmable,” precision medicine for patients, speakers at Bio Korea 2026 said April 28. http://www.bioworld.com/articles/730686 Wed, 29 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730686-bio-korea-2026-kicks-off-with-spotlight-on-oligonucleotides AI generated image for researcher developing antisense oligonucleotides Acurastem Inc. has been awarded $7.5 million in grant funding by the California Institute for Regenerative Medicine (CIRM) to support the advancement of lead clinical candidate AS-241 toward first-in-human testing. http://www.bioworld.com/articles/730740 Wed, 29 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730740-cirm-grant-to-help-advance-acurastems-as-241-toward-clinic Brain as light bulb filament Vanda Pharmaceuticals Inc. presented data this week at the annual American Academy of Neurology conference regarding allele-specific antisense oligonucleotides (ASOs) that specifically target the mutant p.A53T allele from the SNCA gene while preserving the expression of the wild-type allele. The mutant allele is associated with increased risk of early-onset Parkinson’s disease (PD) and current ASOs target SNCA regardless of its mutation status. SNCA gene while preserving the expression of the wild-type allele. The mutant allele is associated with increased risk of early-onset Parkinson’s disease (PD) and current ASOs target SNCA regardless of its mutation status.]]> http://www.bioworld.com/articles/730537 Tue, 21 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730537-aso-targeting-snca-pa53t-shows-promise-in-parkinsons Steadying hand while reaching for glass Quiver Bioscience Inc. has received a strategic investment from the Porta family office (Argentina) to advance the company’s antisense oligonucleotide (ASO) therapeutic program for chromosome 15q duplication (Dup15q) syndrome. The investment will fund preclinical activities, including safety studies for candidate selection as the program advances toward clinical development. http://www.bioworld.com/articles/730349 Tue, 14 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730349-investment-to-advance-quivers-aso-for-dup15q-syndrome Illustration of human brain and dna Alloy Therapeutics Inc. has entered into a collaboration and license agreement with Biogen Inc. for the use of Alloy’s Anticlastic ASO platform to accelerate the development of innovative oligonucleotide therapeutics. http://www.bioworld.com/articles/730189 Wed, 08 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730189-biogen-to-use-alloy-therapeutics-anticlastic-aso-platform AI generated image for researcher developing antisense oligonucleotides Pepgen Inc. is forging ahead with tests of PGN-EDODM1 in other territories after the U.S. FDA placed a partial hold on the Freedom2-DM1 phase II trial, a multiple ascending-dose, randomized, placebo-controlled experiment in myotonic dystrophy type 1 (DM1). http://www.bioworld.com/articles/729340 Thu, 05 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729340-fda-curtails-pepgens-freedom2-operate-in-dm1 Person holding weight with assistance Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant skeletal muscle disorder with a prevalence of approximately 1 in 8,000. The disorder is driven by aberrant expression of double homeodomain protein 4 (DUX4) within the D4Z4 macrosatellite array. Currently, effective treatments for FSHD are lacking. Strategies aimed at reducing DUX4 expression could hold promise as potential therapeutic approaches for FSHD. http://www.bioworld.com/articles/728306 Fri, 23 Jan 2026 08:00:00 -0500 https://www.bioworld.com/articles/728306-targeting-dux4-with-improves-muscle-function-in-fshd-models Illustration showing cross section of skeletal muscle Mainly expressed in the liver, microRNA‑122‑5p (miR‑122) exhibits increased circulating levels in the context of obesity. When elevated in the bloodstream, miR-122 can act on extrahepatic tissues, including vascular endothelial cells, where it contributes to endothelial dysfunction and may promote the development of diabetic vascular complications. http://www.bioworld.com/articles/728094 Mon, 19 Jan 2026 08:00:00 -0500 https://www.bioworld.com/articles/728094-targeting-mir-122-prevents-obesity-linked-vascular-dysfunction Aperture Therapeutics Inc. has advanced its matrix metalloproteinase-9 (MMP-9) antisense oligonucleotide (ASO) program, APRTX-003, for the treatment of amyotrophic lateral sclerosis (ALS). This first-in-class RNA-targeting approach program targets chronic neuroinflammation and neurodegeneration. http://www.bioworld.com/articles/727665 Wed, 07 Jan 2026 08:00:00 -0500 https://www.bioworld.com/articles/727665-aperture-therapeutics-advances-aprtx-003-for-als Illustration demonstrating muscle contraction in amyotrophic lateral sclerosis. Alagille syndrome (ALGS) is a rare, multisystem genetic disorder most commonly caused by haploinsufficiency of the JAG1 gene, leading to reduced JAG1 protein function and impaired development of intrahepatic bile ducts. Researchers from Arnatar Therapeutics Inc. described the development of antisense oligonucleotides (ASOs) engineered using their proprietary ACT‑UP1 platform to upregulate endogenous JAG1 expression and thereby address the underlying genetic deficiency. JAG1 gene, leading to reduced JAG1 protein function and impaired development of intrahepatic bile ducts. Researchers from Arnatar Therapeutics Inc. described the development of antisense oligonucleotides (ASOs) engineered using their proprietary ACT‑UP1 platform to upregulate endogenous JAG1 expression and thereby address the underlying genetic deficiency.]]> http://www.bioworld.com/articles/727484 Tue, 30 Dec 2025 08:00:00 -0500 https://www.bioworld.com/articles/727484-jag1-boosting-asos-ameliorate-liver-pathology-in-alagille-syndrome Illustration of DNA, magnifying glass Scineuro Pharmaceuticals Holdings Ltd. has received a $5 million research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to accelerate the preclinical development of the company’s novel LRRK2-targeted antisense oligonucleotide (ASO) program, SNP-614, for Parkinson’s disease. http://www.bioworld.com/articles/726414 Fri, 21 Nov 2025 08:00:00 -0500 https://www.bioworld.com/articles/726414-mjff-grant-backs-scineuros-snp-614-for-parkinsons-disease Dollar sign in light bulb on yellow background Aperture Therapeutics Inc. has nominated APRTX-001 as a development candidate, with the program now advancing through IND-enabling studies. APRTX-001 is a CD33-targeting antisense oligonucleotide (ASO) designed for the treatment of frontotemporal dementia and amyotrophic lateral sclerosis, with potential for indication expansion into Alzheimer’s disease. http://www.bioworld.com/articles/726347 Thu, 20 Nov 2025 08:00:00 -0500 https://www.bioworld.com/articles/726347-aperture-therapeutics-nominates-aprtx-001-as-candidate Man piecing together a puzzle Cystic fibrosis (CF) is a genetic disorder affecting around 90,000 people worldwide. It is commonly caused by the ΔF508 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which results in a misfolded CFTR protein that is subsequently ubiquitinated and degraded. http://www.bioworld.com/articles/725913 Fri, 07 Nov 2025 08:00:00 -0500 https://www.bioworld.com/articles/725913-rffl-targeting-aso-enhances-cftr-modulator-efficacy-in-cystic-fibrosis-cells Aniridia-associated keratopathy (AAK) is a rare genetic eye condition caused by PAX6 haploinsufficiency, which leads to chronic inflammation, neovascularization and vision loss. Currently, there are limited therapeutic options for the treatment of AAK, which has been linked to limbal stem cell deficiency. http://www.bioworld.com/articles/725349 Tue, 21 Oct 2025 09:00:00 -0400 https://www.bioworld.com/articles/725349-aso-targets-irs-1-to-mitigate-keratopathy-progression-in-mice A team of scientists demonstrated the potential of a new antisense oligonucleotide. http://www.bioworld.com/articles/725284 Mon, 20 Oct 2025 09:00:00 -0400 https://www.bioworld.com/articles/725284-anti-igf-1r-aso-tested-for-graves-ophthalmopathy As the first – and so far only – drug to enter clinical testing for the rare neurogenetic disorder Alexander disease, there were some unknowns heading into the readout of the pivotal study testing Ionis Pharmaceuticals Inc.’s zilganersen in children and adults. But the top-line data yielded a clear win for the antisense oligonucleotide candidate, which demonstrated a disease-modifying impact, including statistical significance on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test vs. control at week 61. http://www.bioworld.com/articles/724373 Mon, 22 Sep 2025 11:00:00 -0400 https://www.bioworld.com/articles/724373-ionis-hits-goals-with-zilganersen-in-first-alexander-disease-trial Hearing loss is a major global health challenge, affecting over 5% of the population and largely lacking effective biological therapies. Mutations in KCNQ4, which encodes the Kv7.4 potassium channel essential for outer hair cell function, are a leading cause of autosomal dominant, non-syndromic hearing loss (DFNA2). http://www.bioworld.com/articles/724002 Wed, 10 Sep 2025 09:00:00 -0400 https://www.bioworld.com/articles/724002-novel-antisense-oligonucleotide-therapy-for-dfna2-hearing-loss Illustration of ear next to DNA double helix Acute pancreatitis took center stage as Wall Street took heed of phase III data from Ionis Pharmaceuticals Inc. with olezarsen for severe hypertriglyceridemia (sHTG), while another player in the space, Arrowhead Pharmaceuticals Inc., signed a sizeable deal in a separate therapeutic area with Novartis AG. http://www.bioworld.com/articles/723787 Tue, 02 Sep 2025 11:00:00 -0400 https://www.bioworld.com/articles/723787-ionis-reports-shtg-win-while-arrowhead-inks-novartis-deal Triglycerides in blood vessel The FDA has granted orphan drug designation to Vanda Pharmaceuticals Inc.’s VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera (PV). http://www.bioworld.com/articles/723684 Fri, 29 Aug 2025 09:00:00 -0400 https://www.bioworld.com/articles/723684-vandas-vgt-1849b-designated-orphan-drug-for-polycythemia-vera White blood cells with red blood cells A few years after it was founded with the aim of taking RNA therapies to the next level, Arnatar Therapeutics Inc. emerged from stealth, disclosing a $52 million series A round raised in 2024 as well as U.S. FDA orphan and rare pediatric disease designations for ART-4, an antisense oligonucleotide candidate targeting the root cause of Alagille syndrome. http://www.bioworld.com/articles/723317 Tue, 26 Aug 2025 11:00:00 -0400 https://www.bioworld.com/articles/723317-arnatar-emerges-with-rna-approach-to-both-silence-and-restore Arnatar DARGER platform illustration Arnatar Therapeutics' DARGER platform combines gene silencing with the ability to upregulate protein expression using antisense oligonucleotides. Credit: Arnatar Therapeutics Inc. Arnatar Therapeutics Inc.’s ART-4, an upregulating antisense oligonucleotide (ASO) therapeutic candidate for the treatment of Alagille syndrome (ALGS), has been awarded U.S. orphan drug and rare pediatric disease designations by the FDA. Approximately 95% of ALGS cases are caused by haploinsufficient mutations in the Jagged-1 (JAG1) gene, leading to insufficient JAG1 protein levels and impaired liver bile duct development. http://www.bioworld.com/articles/723588 Tue, 26 Aug 2025 09:00:00 -0400 https://www.bioworld.com/articles/723588-arnatars-art-4-designated-orphan-drug-for-alagille-syndrome Liver and DNA In Ionis Pharmaceuticals Inc.’s second U.S. FDA approval in under a year, the agency approved Dawnzera (donidalorsen) as a prophylactic therapy in the rare and genetic disease hereditary angioedema (HAE). The approval came as scheduled as the NDA had a PDUFA date of Aug. 21. Dawnzera now joins a market with previously approved drugs for the rare, genetic, life-threatening condition, as well as other companies with HAE drugs in development. http://www.bioworld.com/articles/723272 Thu, 21 Aug 2025 11:00:00 -0400 https://www.bioworld.com/articles/723272-ionis-gets-approval-for-preventing-attacks-in-rare-and-genetic-hae FDA approved icons and medical professional 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. http://www.bioworld.com/articles/721913 Tue, 08 Jul 2025 11:00:00 -0400 https://www.bioworld.com/articles/721913-veritas-mitsubishi-gas-chemical-partner-for-new-aso-candidates 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%. http://www.bioworld.com/articles/721327 Tue, 17 Jun 2025 11:00:00 -0400 https://www.bioworld.com/articles/721327-dyne-vhot-in-dm1-but-shares-cool-on-endpoint-switch 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. http://www.bioworld.com/articles/720434 Wed, 21 May 2025 09:00:00 -0400 https://www.bioworld.com/articles/720434-app-or-rab5-targeting-asos-reverse-down-syndrome-linked-alzheimers-disease-effects-in-mouse-model 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). http://www.bioworld.com/articles/720210 Thu, 15 May 2025 09:00:00 -0400 https://www.bioworld.com/articles/720210-cure-rare-diseases-crd-002-awarded-orphan-drug-designation-for-spinocerebellar-ataxia-type-3 Cerebellum, brain stem, spinal cord 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). N-methyltransferase (NNMT), an enzyme implicated in cancer progression, using antisense oligonucleotides (ASOs).]]> http://www.bioworld.com/articles/720082 Mon, 12 May 2025 09:00:00 -0400 https://www.bioworld.com/articles/720082-antisense-oligonucleotide-targeting-nicotinamide-n-methyltransferase-as-a-potential-cancer-therapy 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. http://www.bioworld.com/articles/719029 Tue, 08 Apr 2025 09:00:00 -0400 https://www.bioworld.com/articles/719029-nissan-chemical-sanwa-kagaku-kenkyusho-to-codevelop-antisense-drug-candidate Brain and DNA