Industry Insights: Clinical and regulatory momentum for antisense and mRNA therapeutics

Dyne Therapeutics initiated a Phase 3 trial of DYNE-101 for myotonic dystrophy type 1 [1]Dyne Therapeutics. Dyne Therapeutics announces initiation of phase 3 HARMONIA trial of z-basivarsen. Mar 8, 2026.

Dyne Therapeutics announced the initiation of the global Phase 3 HARMONIA trial evaluating z-basivarsen (DYNE-101), an ASO conjugated to a transferrin receptor 1-binding fragment, in approximately 150 patients aged ≥16 years with myotonic dystrophy type 1. The randomized, double-blind, placebo-controlled study will assess intravenous dosing at 6.8 mg/kg every eight weeks over 48 weeks. The primary endpoint is the change from baseline in the five times sit to stand (5xSTS) test at week 49, with secondary endpoints including muscle function measures and patient-reported outcomes. The trial design has been aligned with the FDA and is intended to support confirmatory evidence for potential regulatory approval.

Vico Therapeutics dosed patients in a Phase 1/2 trial evaluating VO659 across polyglutamine disorders [2]Vico Therapeutics. Vico Therapeutics announces patient dosing in twice annual regimen of VO659 in Phase 1/2 trial in Huntington's disease, spinocerebellar ataxia type 3 and type 1. Feb 24, 2026.

Vico Therapeutics announced patient dosing in an expanded cohort of its ongoing Phase 1/2a trial of VO659, an ASO targeting CAG repeat expansions, in Huntington's disease, spinocerebellar ataxia type 3, and type 1. The study is evaluating a twice-yearly intrathecal dosing regimen over 12 months for safety, tolerability, pharmacokinetics, and pharmacodynamics. Early data showed a 38% reduction in cerebrospinal fluid mutant huntingtin and a 2.5% reduction in neurofilament light chain at four months. VO659 has been reported as well-tolerated with no serious adverse events to date. The program is ongoing in Europe, with FDA clearance of an Investigational New Drug (IND) application to support planned US trial initiation.

Stoke Therapeutics dosed the first patient in the Phase 1 trial of STK-002 for autosomal dominant optic atrophy [3]Stoke Therapeutics. Stoke Therapeutics announces first patient dosed in Phase 1 study. Feb 11, 2026.

Stoke Therapeutics announced the first patient dosing in the Phase 1 OSPREY study of STK-002, an ASO designed to upregulate OPA1 protein expression in autosomal dominant optic atrophy. The open-label, dose-escalation trial is enrolling patients aged 6–55 years with confirmed OPA1 mutations to evaluate safety, tolerability, and pharmacokinetics following single ascending doses. Secondary endpoints include changes in visual function, ocular structure, and quality of life. The study is recruiting in the UK and Germany, with additional European sites planned. STK-002 aims to address haploinsufficiency by increasing wild-type OPA1 expression to potentially modify disease progression.

Harness Therapeutics nominated HRN001, a first-in-class drug candidate for Huntington’s disease [4]Harness Therapeutics. Harness Therapeutics nominates HRN001, a first-in-class drug candidate for Huntington's disease and establishes clinical advisory. Feb 16, 2026.

Harness Therapeutics announced the nomination of HRN001, an ASO designed to upregulate FAN1, as its lead candidate for Huntington's disease. The therapy leverages the company's microRNA site-blocking ASO platform to modulate expression of a DNA repair nuclease implicated in suppressing somatic CAG repeat expansion. Preclinical studies demonstrated increased FAN1 expression, reduced somatic expansion, and favorable pharmacokinetic and tolerability profiles. The company also established a clinical advisory board to support advancement toward first-in-human studies, planned for 2027. Ongoing preclinical development is expected to continue through 2026, with broader applications of the platform under exploration in additional repeat expansion disorders.

Innorna received FDA clearance of an IND for mRNA therapy IN026 in refractory gout [5]Innorna. Innorna announces FDA clearance of IND for IN026. Mar 17, 2026. Innorna announced that the FDA cleared its IND application for IN026, an mRNA-based therapy for refractory gout, enabling the initiation of a Phase 1 clinical trial. IN026 uses a lipid nanoparticle (LNP) platform to deliver mRNA encoding urate oxidase to the liver, facilitating systemic uric acid breakdown. The upcoming study will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with limited treatment options. The candidate is positioned as a potential first-in-class mRNA protein-replacement therapy designed for repeat administration and long-term disease control in chronic metabolic disorders.

Innorna received FDA clearance of an IND for mRNA therapy IN026 in refractory gout; Regulatory Changes and Updates. Credit: stock.adobe.com.

Argo Biopharma received FDA Fast Track designation for siRNA therapy BW-20805 in hereditary angioedema [6]Argo Biopharma. Argo Biopharma receives FDA fast track designation for BW-20805. Mar 16, 2026.

Argo Biopharma announced that the FDA granted Fast Track designation to BW-20805, an investigational small interfering RNA (siRNA) therapy targeting prekallikrein for the treatment of hereditary angioedema. The designation is intended to support accelerated development and regulatory review. BW-20805 is currently being evaluated in a global Phase 2 open-label study in adult patients, with primary completion expected in the second half of 2026 and plans for a subsequent Phase 3 trial. Early clinical data presented from open-label studies indicated reductions in plasma prekallikrein levels and decreases in time-normalized attack rates.

ARTHEx Biotech received FDA Fast Track designation for RNA therapy ATX-01 targeting myotonic dystrophy type 1 [7]ARTHEx Biotech. ARTHEx Biotech announces publication highlighting ATX-01 for myotonic dystrophy type 1. Feb 25, 2026.

ARTHEx Biotech announced that the FDA granted Fast Track designation to ATX-01, an investigational RNA-based therapy for myotonic dystrophy type 1. ATX-01 is designed to inhibit microRNA-23b, thereby increasing muscleblind-like protein levels and addressing RNA mis-splicing associated with disease pathology. The therapy has demonstrated increased protein expression and reduction of toxic RNA foci in preclinical models. ATX-01 is currently being evaluated in the Phase 1/2a ArthemiR study. The Fast Track designation enables enhanced regulatory interaction to support development in a condition with no approved disease-modifying treatments.

Novartis completed the acquisition of Avidity Biosciences to expand its RNA therapeutics pipeline [8]Novartis. Novartis successfully completes acquisition of Avidity Biosciences. Feb 27, 2026. Novartis announced the completion of its acquisition of Avidity Biosciences, adding its antibody–oligonucleotide conjugate platform and three late-stage neuromuscular programs to its pipeline. The transaction, valued at approximately $12B in equity and $11B enterprise value, results in Avidity becoming a wholly owned subsidiary. Avidity's platform enables targeted delivery of oligonucleotide therapies to muscle tissue, supporting Novartis' RNA therapeutics strategy. The deal includes a cash payment of $72.00 per share to Avidity shareholders. The acquisition is intended to strengthen Novartis' late-stage neuroscience portfolio and expand capabilities in developing therapies for genetic neuromuscular diseases.

Novartis completed the acquisition of Avidity Biosciences to expand its RNA therapeutics pipeline; Collaborations, Partnerships, and Acquisitions. Credit: stock.adobe.com.

Therna announced collaboration with Charles River to advance personalized RNA therapeutics for ultra-rare diseases [9]PR Newswire. Therna announces collaboration with Charles River to advance single-patient personalized RNA therapeutics. Mar 5, 2026.

Therna Biosciences announced a collaboration with Charles River to develop single-patient RNA therapeutics for ultra-rare disorders. Initial programs include an mRNA therapy targeting a rare lung fibrosis and ASOs designed to treat Lamb-Shaffer syndrome. Therna's AI-enabled platform was used to design and validate the mRNA candidate within months, while Charles River will lead preclinical development with the aim of submitting a single-patient IND application. The collaboration focuses on accelerating individualized treatment approaches where traditional clinical trials are not feasible, integrating RNA design with preclinical capabilities to support rapid development timelines.

BioNTech founders announced their departure and plans to establish a new mRNA-focused company [10]BioNTech. BioNTech and co-founders announce plan to pursue next-generation innovations. Mar 10, 2026.

BioNTech co-founders Ugur Sahin and Özlem Türeci announced plans to step down from their roles and establish a new biotechnology company focused on next-generation mRNA technologies. The transition is expected to occur by the end of 2026, with BioNTech initiating a search for successors. The new entity will receive certain mRNA technologies and related rights from BioNTech, which will retain a minority stake and may receive milestone and royalty payments. A binding agreement is anticipated in the first half of the year. The move comes as BioNTech advances its late-stage pipeline, including multiple Phase 3 oncology studies expected by the end of 2026.

BreezeBio raised $60M Series B to advance the mRNA-based diabetes candidate toward clinical development [11]BreezeBio. BreezeBio announces series B financing to advance pipeline of precision genetic medicines. Feb 25, 2026.

BreezeBio announced a $60M Series B financing to support the advancement of its lead mRNA therapeutic, BRZ-101, into IND-enabling studies for diabetes. The candidate delivers mRNA-encoded autoantigens and tolerogenic factors to antigen-presenting cells to induce antigen-specific regulatory T cells and address autoimmune drivers of disease. Proceeds will also support expansion of the company's NanoGalaxy delivery platform, comprising hydrophilic nanoparticles designed for targeted nucleic acid delivery. The funding round included new and existing investors and is intended to support manufacturing scale-up, preclinical development, and progression toward clinical evaluation, as well as additional pipeline programs in autoimmune disease and oncology.

UAlbany researchers developed a laser technique to assess mRNA encapsulation in LNPs [12]University at Albany. UAlbany researchers develop new laser technique to test mRNA-based therapeutics. Feb 25, 2026.

Researchers at the University at Albany reported the development of a Raman spectroscopy-based method to evaluate whether messenger RNA is properly encapsulated within LNPs. The non-destructive laser technique analyzes chemical composition to assess mRNA integrity and packaging, which are critical for therapeutic stability and delivery. In a study published in Analytical Chemistry, the method demonstrated potential for rapid quality assessment of mRNA-based therapeutics and vaccines. The approach is intended to support improved characterization of LNP formulations, addressing key challenges related to stability, delivery efficiency, and overall product performance.

etherna platform advanced to IND-enabling studies for mRNA/LNP cancer therapy [13]etherna. etherna's intratumoral mRNA therapy advances in non-melanoma skin cancer. Feb 19, 2026.

etherna announced that its intratumoral mRNA–LNP platform has advanced with the nomination of LAD116, a candidate targeting non-melanoma skin cancer, by its collaborator Almirall. The therapy is designed to induce an immune response within the tumor microenvironment. The program will progress into IND-enabling studies, with a focus on manufacturing GMP-grade material to support planned Phase 1 clinical trials. The advancement reflects the ongoing development of mRNA-based cancer immunotherapies using localized delivery approaches.

Jokübas Leikauskas, Commissioning Editor of Nucleic Acid Insights, holds a background in science communication and digital publishing, focusing on advancing the nucleic acid therapeutics field by commissioning and shaping high-impact, open access content for Nucleic Acid Insights. He leads the development of interviews, expert articles, and industry perspectives that highlight emerging advances across mRNA, DNA, oligonucleotide, and drug delivery modalities. Jokubas is driven to translate complex scientific topics into engaging, accessible content while maintaining strong connections across the nucleic acids community.

References

1. Dyne Therapeutics. Dyne Therapeutics announces initiation of phase 3 HARMONIA trial of z-basivarsen. Mar 8, 2026.

2. Vico Therapeutics. Vico Therapeutics announces patient dosing in twice annual regimen of VO659 in Phase 1/2 trial in Huntington's disease, spinocerebellar ataxia type 3 and type 1. Feb 24, 2026.

3. Stoke Therapeutics. Stoke Therapeutics announces first patient dosed in Phase 1 study. Feb 11, 2026.

4. Harness Therapeutics. Harness Therapeutics nominates HRN001, a first-in-class drug candidate for Huntington's disease and establishes clinical advisory. Feb 16, 2026.

5. Innorna. Innorna announces FDA clearance of IND for IN026. Mar 17, 2026.

6. Argo Biopharma. Argo Biopharma receives FDA fast track designation for BW-20805. Mar 16, 2026.

7. ARTHEx Biotech. ARTHEx Biotech announces publication highlighting ATX-01 for myotonic dystrophy type 1. Feb 25, 2026.

8. Novartis. Novartis successfully completes acquisition of Avidity Biosciences. Feb 27, 2026.

9. PR Newswire. Therna announces collaboration with Charles River to advance single-patient personalized RNA therapeutics. Mar 5, 2026.

10. BioNTech. BioNTech and co-founders announce plan to pursue next-generation innovations. Mar 10, 2026.

11. BreezeBio. BreezeBio announces series B financing to advance pipeline of precision genetic medicines. Feb 25, 2026.

12. University at Albany. UAlbany researchers develop new laser technique to test mRNA-based therapeutics. Feb 25, 2026.

13. etherna. etherna's intratumoral mRNA therapy advances in non-melanoma skin cancer. Feb 19, 2026.