Industry Insights: Early 2026 brings clinical progress, regulatory designations, and strategic partnerships in nucleic acid therapeutics
Nucleic Acid Insights, 2026; 12(1), 103–107
DOI: 10.18609/nuc.2026.012
| Across January and early February 2026, activity in the nucleic acid therapeutics field spanned early clinical readouts, regulatory designations, preclinical advances, and new strategic partnerships. ProQR reported initial Phase 1 data for its editing oligonucleotide AX-0810 in cholestatic diseases, while Providence Therapeutics supported the launch of a multi-site paediatric trial evaluating personalized mRNA cancer vaccines in Australia. Regulatory momentum continued, with the US FDA granting Fast Track designation to BioNTech’s mRNA immunotherapy BNT113 for HPV16-positive head and neck cancer, and Japan awarding Orphan Drug designation to Dyne’s antisense candidate DYNE-101 for myotonic dystrophy type 1. Preclinical studies highlighted mRNA and antisense approaches in infertility and metabolic disease, alongside new collaborations to expand access to individualized antisense therapies and advance mRNA treatment for propionic acidemia. |  FDA fast track designation advanced mRNA cancer immunotherapy targeting HPV16+ head and neck cancer; Regulatory Changes and Updates. Credit: stock.adobe.com |
CLINICAL TRIALS AND RESEARCH |
ProQR reported initial Phase 1 data for an oligonucleotide targeting cholestatic diseases [1]
ProQR reported initial Phase 1 data for AX-0810, selected two development candidates, and outlined 2026 milestones. AX-0810 is ProQR’s lead investigational editing oligonucleotide, being developed for the treatment of cholestatic diseases, including primary sclerosing cholangitis and biliary atresia. Early results from the ongoing single-center, randomized, placebo-controlled, multiple-dose-escalation study in healthy volunteers (n=33 planned) showed no serious adverse events or clinically meaningful laboratory abnormalities after 4 weeks of dosing at 3 mg/kg. Pharmacokinetics were consistent with non-clinical data, and target engagement readouts are expected in the first half of 2026, with a patient cohort planned thereafter. The company also advanced AX-2402 for Rett syndrome and AX-2911 for metabolic-associated steatohepatitis, supported by preclinical data, and reported $4.5M in 2025 milestones from its collaboration with Eli Lilly.
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ProQR reported initial Phase 1 data for an oligonucleotide targeting cholestatic diseases; Clinical Trials and Research. Credit: www.proqr.com |
Providence Therapeutics supported the initiation of a personalized paediatric mRNA cancer vaccine trial in Australia [2]
Providence Therapeutics announced support for the PaedNEO-VAX study, described as the first multi-site paediatric trial evaluating individualized mRNA cancer vaccines for children with relapsed or refractory high-grade brain tumors, including high-grade glioma and medulloblastoma. The trial, co-led by The University of Queensland and SAHMRI and sponsored by ANZCHOG, is scheduled to begin in March 2026 across seven Australian hospitals. Phase I will assess safety and dose, followed by Phase II evaluation of clinical outcomes, including disease progression and survival. Tumor-specific targets will be identified through genome sequencing, and personalized mRNA vaccines will be manufactured in Australia and delivered within approximately 10 weeks of enrollment.
REGULATORY CHANGES AND UPDATES |
FDA fast track designation advanced mRNA cancer immunotherapy targeting HPV16+ head and neck cancer [3]
BioNTech has received Fast Track designation from the FDA for BNT113, an investigational mRNA cancer immunotherapy targeting human papillomavirus type 16 positive (HPV16+) head and neck squamous cell carcinoma (HNSCC) expressing PD-L1. The designation is based on preliminary data from the ongoing Phase 2/3 AHEAD-MERIT trial evaluating BNT113 in combination with pembrolizumab as a first-line treatment for unresectable recurrent or metastatic disease. HNSCC is the seventh most common cancer globally, with rising incidence driven by HPV16-related tumors. There are currently no approved HPV-targeted therapies for HPV16+ HNSCC, underscoring the need for novel, chemotherapy-free treatment options.
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| FDA fast track designation advanced mRNA cancer immunotherapy targeting HPV16+ head and neck cancer; Regulatory Changes and Updates. Credit: stock.adobe.com |
Orphan drug designation in Japan strengthened the global path for DYNE-101 in myotonic dystrophy type 1 [4]
Dyne Therapeutics has received Orphan Drug designation in Japan for zeleciment basivarsen (DYNE-101) to treat myotonic dystrophy type 1, reinforcing its regulatory momentum across major markets. The investigational antisense oligonucleotide, conjugated to an antigen-binding fragment that binds to the transferrin receptor 1, enables delivery to muscle and the central nervous system. The drug is currently in the Phase 1/2 ACHIEVE trial and has demonstrated sustained improvements in myotonia, muscle strength, and functional measures, alongside a favorable safety profile. Enrollment in the registrational expansion cohort is expected to be completed in early Q2 2026. The Japanese designation provides development support and potential 10-year market exclusivity, complementing Breakthrough Therapy, Fast Track, and Orphan Drug designations previously granted in the US and EU.
RESEARCH AND DEVELOPMENT HIGHLIGHTS |
Endometrial mRNA therapy improved embryo implantation in a mouse infertility model [5]
Researchers at Johns Hopkins Medicine reported early preclinical success using lipid nanoparticle-delivered mRNA therapy to enhance embryo implantation in mice. Published in Nature Nanotechnology, the study demonstrated targeted intrauterine delivery of granulocyte-macrophage colony-stimulating factor mRNA to the endometrium using peptide-modified LNPs. In a mouse model of endometrial injury, the treatment restored implantation rates to levels comparable to healthy controls, while reducing systemic exposure and off-target toxicity compared with recombinant protein administration. The findings suggest that localized mRNA-LNP therapy may represent a potential future strategy for treating infertility linked to impaired endometrial receptivity.
CD47 antisense therapy was shown to improve metabolic health in aged mice [6]
A new study published in Aging-US reported that ASO–mediated knockdown of CD47 improves glucose homeostasis and lipid balance in aged male mice. Researchers from the University of Kentucky and the Lexington Veterans Affairs Medical Center treated 20-month-old mice with a CD47-targeting ASO for 10 weeks, observing reduced fasting glucose, improved insulin sensitivity, and lower circulating cholesterol and free fatty acids. Notably, these metabolic benefits occurred without changes in body weight but were associated with reduced visceral fat mass and enhanced brown adipose tissue activity. The findings position CD47 as a potential therapeutic target for addressing age-associated metabolic dysfunction.
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CD47 antisense therapy was shown to improve metabolic health in aged mice; Research and Development Highlights. Credit: depositphotos.com |
COLLABORATIONS AND PARTNERSHIPS |
n-Lorem and EspeRare announced a collaboration to broaden access to individualized ASO therapies [7]
The n-Lorem Foundation and the EspeRare Foundation have announced a strategic collaboration to expand access to individualized ASO therapies in Europe for patients with rare genetic diseases. Initially focused on Switzerland, the partnership aims to establish regulatory and clinical pathways to enable the treatment of nano-rare patients outside the US, where n-Lorem currently operates under FDA authorization. EspeRare will support patient identification, regulatory alignment, and coordination with academic medical centers to create a scalable framework for broader European expansion. The multi-year collaboration seeks to reduce access inequities and enable treatment initiation for up to three Swiss patients within the first year.
Recordati partnered with Moderna to advance mRNA therapy in propionic acidemia [8]
Recordati has entered a global collaboration and license agreement with Moderna to develop and commercialize mRNA-3927, an investigational mRNA therapy for propionic acidemia (PA). Moderna will continue to lead development, while Recordati will assume global commercialization responsibilities if the therapy is approved. mRNA-3927 is designed to restore propionyl-CoA carboxylase enzyme activity and represents a potential first disease-modifying treatment for PA, a rare inherited metabolic disorder with high morbidity and mortality. The candidate is currently being evaluated in a potential registrational study, with data expected by the end of 2026. The agreement includes $50M upfront and additional milestone payments.
CONFERENCES AND EVENTS |
Special Interest Group on efficient manufacturing of oligonucleotides [9]
The inaugural Nucleic Acid Insights Special Interest Group on enhancing efficiency and sustainability in oligonucleotide manufacturing is an exclusive virtual meeting that will take place on April 28–30, 2026. A hand-picked group of 50 oligos manufacturing, analytical, and regulatory experts from across the global sector will tackle key topics, including the enhancement of solid-phase synthesis, removing barriers to the adoption of novel synthesis methods, and driving innovation in both downstream purification and the analytical toolkit. Click here for more information.
RNAi-Based Therapeutics Summit 2026 [10]
RNAi-Based Therapeutics Summit 2026 took place on January 27–29, 2026, in Boston, Massachusetts. The summit brought together the RNA interference (RNAi) ecosystem to advance the next wave of small interfering RNA (siRNA) and microRNA drugs from bench to bedside. It served as a forum for stakeholders across pharma, biotech, venture capital, and vendors to examine recent successes, translate them into actionable strategies, and address ongoing challenges in delivery, development, and manufacturing. The year’s discussions extended beyond hepatic targets, with a focus on strategies aimed at enabling extra-hepatic delivery in clinical settings.
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| Special Interest Group on efficient manufacturing of oligonucleotides; Conferences and Events. |
5th mRNA-Based Therapeutics Summit Europe [11]
The 5th mRNA-Based Therapeutics Summit Europe took place on January 27–29 in Berlin, Germany, bringing together more than 100 senior leaders from Moderna, AstraZeneca, CybernaX Bio, Avocet Bio, and other organizations. Over 3 days, participants discussed advances in mRNA, self-amplifying RNA (saRNA), and circular RNA design, alongside delivery innovations and CMC considerations. A dedicated Gene Editing Focus Day examined transient mRNA-based editing strategies, and investor-led sessions addressed funding expectations for mRNA platforms in 2026.
References
ProQR Therapeutics. ProQR announces encouraging AX-0810 Phase 1 safety and PK data, development candidate selections and 2026 outlook. 2026; ProQR Press Release.
Providence Therapeutics. Providence Therapeutics announces world-first personalized pediatric mRNA cancer vaccine trial. 2026; Providence Therapeutics Press Release.
BioNTech SE. BioNTech receives FDA Fast Track designation for mRNA cancer immunotherapy targeting HPV16+ head and neck cancer. 2026; BioNTech Press Release.
Dyne Therapeutics. Dyne Therapeutics receives Orphan Drug designation in Japan for DYNE-101. 2026; Dyne Therapeutics Press Release.
Johns Hopkins Medicine. Johns Hopkins Medicine researchers find early success using endometrial mRNA therapy to treat infertility. 2026; Johns Hopkins Medicine News Release, January.
Gwag T, Newcomb A, Otuagomah J, et al. CD47 antisense therapy improves metabolic health in aged mice. Aging 2026; 17(12), 2885–2901.
n-Lorem Foundation. n-Lorem and EspeRare announce European collaboration. 2026; n-Lorem News.
Recordati. Recordati announces strategic collaboration with Moderna to develop and commercialize worldwide mRNA-3927 for the treatment of propionic acidemia. 2026; Recordati Press Release.
Nucleic Acid Insights SIG Sponsorship opportunities NAI Sig – Oligos (accessed Feb 18 2026).
RNAi-Based Therapeutics Summit (accessed 11 February 2026).
mRNA-Based Therapeutics Summit Europe. 5th mRNA-Based Therapeutics Summit Europe (accessed: 11 February 2026).