Industry Insights. From discovery to delivery: recent developments shaping nucleic acid therapeutics

Summary

Throughout November 2025, the nucleic acids field continued to advance through new partnerships, clinical progress, and technology development. Collaborations between Biovectra and Revolution Biomanufacturing, and between Sanegene Bio and Eli Lilly, reflected sustained investment in mRNA design and RNA interference (RNAi) platforms.

Multiple clinical programmes reported encouraging early findings, including antisense oligonucleotide (ASO), small interfering RNA (siRNA), and DNA-directed RNAi candidates from Ausper Biopharma, ADARx, Rona Therapeutics, and Benitec. Market activity featured significant financing events for PeptiSystems and Benitec. In tools and technologies, innovations included expanded enzymatic DNA architectures from Touchlight and next-generation lipid nanoparticle (LNP) systems from Acuitas Therapeutics. Collectively, these updates highlight continued diversification across modalities, delivery approaches, and manufacturing strategies in nucleic acid therapeutics.

COLLABORATIONS AND PARTNERSHIPS

Biovectra and Revolution Biomanufacturing announced a partnership for integrated mRNA design and GMP production services [1]Biovectra and Revolution Biomanufacturing. BIOVECTRA and Revolution Biomanufacturing partner to deliver integrated mRNA design and GMP production services—partnership combines sequence design expertise with end-to-end GMP manufacturing to streamline mRNA development and scale-up. Nov 6, 2025.

Biovectra and Revolution Biomanufacturing have recently formed a partnership to provide an integrated pathway from mRNA construct design through GMP manufacturing. Revolution Biomanufacturing will contribute computational design and sequence optimisation, while Biovectra will provide GMP mRNA synthesis and supporting quality and regulatory activities. The companies stated that the collaboration is intended to streamline development timelines for therapeutic and vaccine programs that rely on custom mRNA constructs.

Sanegene Bio entered RNAi licensing and research collaboration with Eli Lilly and Company [2]Sanegene Bio. Sanegene Bio announces RNAi licensing and research collaboration with Lilly. Nov 8, 2025.

Sanegene Bio entered a licensing and research collaboration with Eli Lilly and Company to advance RNAi therapeutics. The agreement gives Lilly global exclusive rights to develop and commercialise these programmes, while Sanegene Bio will continue contributing discovery-stage work. Financial terms were not disclosed, but the companies noted that the deal includes upfront, milestone, and royalty components.

CLINICAL TRIALS AND RESEARCH

Ausper Biopharma reported updates on its Phase 1 trial of a novel ASO for hepatitis B [3]Ausper Biopharma. AusperBio announces late-breaking 48-week Phase II data of AHB-137 in chronic hepatitis B at AASLD 2025. Nov 8, 205.

Ausper Biopharma provided an update on the Phase 1 clinical trial of AHB-137, an investigational ASO therapy for chronic hepatitis B. The company announced completion of the single-ascending-dose portion and progress toward multiple-ascending-dose evaluation. Preliminary safety findings showed no serious adverse events at the tested doses, and the company plans to advance the programme based on these early results.

Benitec Biopharma reported progress on DNA-directed RNAi therapy [4]Benitec Biopharma. Benitec Biopharma releases first quarter 2026 financial results and provides operational update. Nov 14, 2025.

Benitec reported interim results from its Phase 1b/2a trial of BB-301, a gene therapy designed using the company’s DNA-directed RNAi platform to silence mutant PABPN1 and deliver a codon-optimized replacement. All six patients in Cohort 1 met the statistical response criteria, with improvements in dysphagic symptom burden, post-swallow residue, swallowing capacity, and pharyngeal closure over 3–12 months of follow-up. The US FDA granted Fast Track designation for BB-301 for oculopharyngeal muscular dystrophy with dysphagia; BB-301 also holds Orphan Drug Designation from both the FDA and EMA. Benitec treated the first patient in Cohort 2 in late 2025 and plans to meet the US FDA in 2026 to confirm the pivotal study design.

ADARx Pharmaceuticals announced positive interim Phase 1 data for siRNA therapy targeting complement factor B [5]ADARx Pharmaceuticals. ADARx Pharmaceuticals announces positive interim Phase 1 clinical data for its novel siRNA targeting complement factor B (CFB). Nov 7, 2025.

ADARx Pharmaceuticals reported interim Phase 1 results for ADX-038, a siRNA therapeutic targeting complement factor B (CFB) for complement-mediated diseases. ADX-038 was well tolerated, with headache and upper respiratory tract infection as the most common adverse events. The company noted that selective and durable alternative pathway suppression may support semi-annual dosing. ADX-038 is being advanced into multiple Phase 2 studies for immunoglobulin A nephropathy, complement 3 glomerulopathy, geographic atrophy secondary to age-related macular degeneration, and paroxysmal nocturnal hemoglobinuria.

Rona Therapeutics reported Phase 1 results for novel siRNA therapy [6]Rona Therapeutics. Rona Therapeutics presents Phase 1 data for RN0361, a long-acting APOC3-targeting siRNA, at the American Heart Association 2025 Scientific Sessions. Nov 9, 2025.

Rona Therapeutics released Phase 1 results for RN0361, a long-acting siRNA targeting APOC3, at the American Heart Association 2025 Scientific Sessions. In the study, RN0361 produced durable, dose-dependent reductions in APOC3 and triglyceride levels, and was reported as well-tolerated across evaluated cohorts. The company highlighted the potential for RN0361 to be developed for dyslipidaemia and cardiovascular risk reduction.

MARKET TRENDS

PeptiSystems announced growth equity investment from Rubicon Healthcare Partners [7]PR Newswire. PeptiSystems secures growth equity investment from Rubicon Healthcare Partners to accelerate commercial expansion. Nov 13, 2025.

PeptiSystems announced a growth investment from Rubicon Healthcare Partners to support the global expansion of its manufacturing technologies for therapeutic peptides and oligonucleotides. Rubicon will become the company’s largest shareholder following the transaction and aims to contribute strategic expertise and networks to PeptiSystems’ international development. The investment is intended to accelerate commercial activities, expand organizational capabilities, and advance large-scale industrial applications of the company’s flow-through manufacturing technology. PeptiSystems stated that the funding will also support customers transitioning to GMP production of peptide- and oligonucleotide-based therapeutics.

Benitec Biopharma announced pricing of $100M common stock offering [8]Benitec Biopharma. Benitec Biopharma Inc. announces pricing of $100 million common stock offering. Nov 6, 2025.

Benitec Biopharma announced the pricing of a $100M equity offering comprising an underwritten public sale of 5,930,000 common shares and a concurrent registered direct offering of 1,481,481 shares to Suvretta Capital, each priced at $13.50. Underwriters received a 30-day option to purchase up to 889,500 additional shares. Benitec stated that net proceeds will support development of its DNA-directed RNA interference ‘Silence and Replace’ platform and associated therapeutic programmes, along with working capital and general corporate purposes.

TOOLS AND TECHNOLOGIES

Touchlight expanded its doggybone DNA portfolio with new circular architectures [9]Touchlight. Touchlight expands mbDNA portfolio with new circular DNA architectures for advanced therapies. Nov 5, 2025.

Touchlight announced the expansion of its cell-free DNA platform with three new circular DNA format—sscDNA, hsscDNA, and dscDNA—designed to complement its existing megabulb DNA (mbDNA™) technology. mbDNA is a single-stranded DNA construct produced enzymatically, offering high purity, low immunogenicity, and knock-in efficiencies of 60–75% in primary T cells, with payload capacities up to 20 kb for applications such as homology-directed repair and episomal expression. The new architectures provide fully single-stranded, hybrid, or fully double-stranded circular DNA options, allowing user-defined sequences for systems incorporating transposases, recombinases, HITI, and episomal delivery. The expanded portfolio aims to improve stability, reduce immunogenicity, and enhance compatibility with diverse gene editing and expression platforms.

Acuitas Therapeutics unveiled next-generation LNP advancements at the 2025 mRNA Health Conference [10]Business Wire. Acuitas Therapeutics unveils next-generation lipid nanoparticle advancements at the 2025 mRNA Health Conference. Nov 17, 2025.

At the 13th International mRNA Health Conference, held in Berlin, Germany, Acuitas Therapeutics presented its next-generation LNP suite, designed to enhance potency, improve safety, and expand delivery beyond the liver for mRNA therapeutics. The company reported novel LNP candidates with up to four-fold increased potency in gene editing and vaccine applications, reduced liver exposure through optimized lipid structures, targeted immune-cell delivery using DARPin-conjugated LNPs, and mucous-penetrant formulations enabling airway delivery in cystic fibrosis models. Acuitas also highlighted a new pre-formed vesicle manufacturing approach that may reduce cost and improve flexibility for personalized mRNA-LNP therapies.

References

1. Biovectra and Revolution Biomanufacturing. BIOVECTRA and Revolution Biomanufacturing partner to deliver integrated mRNA design and GMP production services—partnership combines sequence design expertise with end-to-end GMP manufacturing to streamline mRNA development and scale-up. Nov 6, 2025. 

2. Sanegene Bio. Sanegene Bio announces RNAi licensing and research collaboration with Lilly. Nov 8, 2025. 

3. Ausper Biopharma. AusperBio announces late-breaking 48-week Phase II data of AHB-137 in chronic hepatitis B at AASLD 2025. Nov 8, 205.

4. Benitec Biopharma. Benitec Biopharma releases first quarter 2026 financial results and provides operational update. Nov 14, 2025.

5. ADARx Pharmaceuticals. ADARx Pharmaceuticals announces positive interim Phase 1 clinical data for its novel siRNA targeting complement factor B (CFB). Nov 7, 2025.

6. Rona Therapeutics. Rona Therapeutics presents Phase 1 data for RN0361, a long-acting APOC3-targeting siRNA, at the American Heart Association 2025 Scientific Sessions. Nov 9, 2025.

7. PR Newswire. PeptiSystems secures growth equity investment from Rubicon Healthcare Partners to accelerate commercial expansion. Nov 13, 2025. 

8. Benitec Biopharma. Benitec Biopharma Inc. announces pricing of $100 million common stock offering. Nov 6, 2025.

9. Touchlight. Touchlight expands mbDNA portfolio with new circular DNA architectures for advanced therapies. Nov 5, 2025.

10. Business Wire. Acuitas Therapeutics unveils next-generation lipid nanoparticle advancements at the 2025 mRNA Health Conference. Nov 17, 2025.