Oligonucleotide– tag –
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Ionis’ ASO Therapy Zilganersen Awaits FDA Approval for Alexander Disease: Achieves Primary Endpoints and Granted Priority Review
BioXconomy USA Overview Ionis Pharmaceuticals' antisense oligonucleotide (ASO), zilganersen, is awaiting FDA approval for the ultra-rare neurological disorder, Alexander disease. The pivotal Phase 1-3 trial (NCT04849741) met its primary ... -
FDA Approves Expanded Indication for Ionis’ ASO Tryngolza in Severe Hypertriglyceridemia, Achieving <500 mg/dL TG Levels in 86% of Patients
Fierce Pharma USA Overview The FDA has approved an expanded indication for Ionis Pharmaceuticals’ antisense oligonucleotide (ASO) Tryngolza (olezarsen) for severe hypertriglyceridemia. Clinical trials demonstrated remarkable efficacy, wi... -
Codexis Pioneers Enzymatic RNA Manufacturing to Scale Beyond Solid-Phase Synthesis, Meeting Demand Exceeding 30 Metric Tons of Oligonucleotide API Annually
Codexis USA Overview Codexis is advancing enzymatic RNA manufacturing to overcome limitations of traditional solid-phase oligonucleotide synthesis (SPOS), significantly enhancing scalability and product quality for RNA therapeutics. This... -
Ionis Forges $30M Ex-US Rare Disease Pact with Recordati, Accelerating Global ASO Reach
Fierce Biotech USA Overview Ionis Pharmaceuticals has secured a $30 million upfront payment from Recordati, granting the Italian firm exclusive ex-US development and commercialization rights for specific rare disease therapies. This stra... -
Elixirgen Therapeutics and Nippon Shinyaku Forge Alliance for Next-Gen DMD Therapy EXG-7001
GlobeNewswire Japan Overview Elixirgen Therapeutics and Nippon Shinyaku have signed an option agreement for the development and commercialization of EXG-7001, a promising therapeutic candidate for Duchenne Muscular Dystrophy (DMD). This ... -
Novartis’ Acquired RNA Therapeutic del-brax Achieves Phase 1/2 Success for Muscle-Wasting Diseases
BioPharma Dive USA Overview Novartis' RNA therapeutic del-brax, acquired through its $12 billion purchase of Avidity Pharmaceuticals, successfully completed a Phase 1/2 clinical trial for muscle-wasting diseases, achieving its primary en... -
Precision Science Fuels Biopharma M&A Surge: Q1 2026 Hits $65B+, Marking Industry’s ‘Full Recovery’
BioSpace / PwC, Pharmaceutical Technology / PwC USA Overview PwC's latest report declares the biopharma ecosystem 'fully recovered,' with Q1 2026 M&A deal values exceeding $65 billion, marking the largest quarter since 2020. This rob... -
Recipharm Infuses Millions into US Sterile Fill-Finish, Bolstering Advanced Therapy Manufacturing
Recipharm (プレスリリース), Fierce Pharma スウェーデン Overview Global CDMO Recipharm is making a multi-million dollar strategic investment to significantly enhance its US sterile fill-finish capabilities, addressing the surging demand f... -
Closing the Innovation Gap: Early CMC Risk Management and Robust Conjugation Strategies for ADCs and AOCs
Industry Publication Unknown Overview As Antibody-Drug Conjugates (ADCs) and Oligonucleotide-Drug Conjugates (AOCs) rapidly expand beyond oncology, early identification of Chemistry, Manufacturing, and Controls (CMC) risks and establishi... -
Hongene Biotech Leverages Chemoenzymatic Ligation to Scale CRISPR sgRNA Manufacturing
Industry Publication / Hongene Biotech Unknown Overview Hongene Biotech is pioneering a chemoenzymatic ligation technology to address the critical scalability challenges in producing single-guide RNA (sgRNA) for CRISPR gene editing. This...
