https://www.bioworld.com/rss en-us 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 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 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 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. 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 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 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 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 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 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 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 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 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 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 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. 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 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 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 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 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 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 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 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 http://www.bioworld.com/articles/718446 Thu, 20 Mar 2025 10:00:00 -0400 https://www.bioworld.com/articles/718446-quiver-bioscience-collaborates-with-dup15q-alliance-to-advance-asos-for-dup15q-syndrome Illustration of DNA, magnifying glass http://www.bioworld.com/articles/717517 Fri, 28 Feb 2025 09:00:00 -0500 https://www.bioworld.com/articles/717517-antisense-oligonucleotides-targeting-fancm-show-preclinical-promise-in-alt-positive-cancers http://www.bioworld.com/articles/717055 Fri, 07 Feb 2025 09:00:00 -0500 https://www.bioworld.com/articles/717055-stereopure-gapmer-asos-targeting-tau-show-promising-preclinical-safety-and-activity Illustration of tau proteins in Alzheimer's disease http://www.bioworld.com/articles/717054 Fri, 07 Feb 2025 09:00:00 -0500 https://www.bioworld.com/articles/717054-cirm-grant-supports-cure-rare-diseases-antisense-oligonucleotide-therapy-for-sca3 Dollar sign droplet above test tube 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.]]> http://www.bioworld.com/articles/716765 Mon, 03 Feb 2025 09:00:00 -0500 https://www.bioworld.com/articles/716765-targeting-recurrent-abca4-variant-with-antisense-oligonucleotides-as-new-strategy-for-stargardt-disease Illustration of human eye http://www.bioworld.com/articles/716735 Wed, 29 Jan 2025 12:00:00 -0500 https://www.bioworld.com/articles/716735-regulus-heads-to-phase-iii-after-more-positive-kidney-disease-data Illustration of polycystic kidney http://www.bioworld.com/articles/716167 Fri, 10 Jan 2025 12:00:00 -0500 https://www.bioworld.com/articles/716167-dyne-seeks-accelerated-path-on-positive-dystrophy-data-shares-down