Industry insights, October 2025
Nucleic Acid Insights 2025; 2(9), 251–255
DOI: 10.18609/nuc.2025.033
This month has seen a number of exciting discoveries and noteworthy advancements in the nucleic acid therapeutics space. Along with our recent infographic—Oligotherapeutics: status quo and future outlook [1]—which discusses the current landscape and future directions in oligonucleotide-based therapies, our new Nucleic Acid Insights Industry Insights series will present the latest updates and developments from across the nucleic acids space, including new collaborations accelerating RNA-based drug development, innovative manufacturing platforms, progress in clinical trials for rare and cardiovascular diseases, emerging AI and gene-editing tools, and regulatory updates supporting novel nucleic acid therapies.
 | CLINICAL TRIALS AND RESEARCH |
CRISPR Therapeutics shows promise for one-time treatment of alpha-1 antitrypsin deficiency [2]CRISPR Therapeutics AG. CRISPR Therapeutics presents new preclinical data for CTX460™ demonstrating in vivo gene correction of Alpha-1 Antitrypsin Deficiency (AATD) utilizing novel SyNTase™ editing platform. Oct 10, 2025.
CRISPR Therapeutics has recently presented new preclinical data for CTX460™, an investigational in vivo gene-editing therapy targeting alpha-1 antitrypsin deficiency (AATD). Utilizing the company's proprietary SyNTase gene-editing platform, CTX460 demonstrated efficient correction of the Z allele mutation in AATD models. In the NSG-PiZ mouse model, a single dose of CTX460™ led to sustained expression of functional AAT protein in the liver, with no observed off-target effects. These results support the potential of CTX460 as a one-time treatment option for AATD.
Argo Biopharma to start Phase II trial of siRNA therapy for rare blood disorder [3]PR Newswire. Argo Biopharma receives IND approval from NMPA for Phase II clinical trial of siRNA therapy BW-40202 for the treatment of PNH. Sep 15, 2025.
Argo Biopharma has received IND approval from China's National Medical Products Administration for BW-40202, an investigational small interfering RNA (siRNA) therapy targeting complement factor B (CFB). The Phase II clinical trial is set to begin in January 2026. BW-40202 aims to treat paroxysmal nocturnal hemoglobinuria and other complement-mediated diseases by suppressing CFB expression in the liver, thereby inhibiting complement alternative pathway activity. Preclinical studies have shown that BW-40202 significantly reduces serum CFB protein levels with long-lasting effects and a favorable safety profile.
 | REGULATORY CHANGES AND UPDATES |
Ionis receives breakthrough therapy designation for ION582 in Angelman syndrome [4]Business Wire. Ionis receives U.S. FDA Breakthrough Therapy designation for ION582 in Angelman syndrome. Sep 9, 2025.
Ionis Pharmaceuticals has received the US FDA Breakthrough Therapy designation for its investigational antisense oligonucleotide (ASO) medicine, ION582, in the treatment of Angelman syndrome (AS). AS is a rare neurological disorder characterized by severe developmental delays, motor dysfunction, and seizures. ION582 is designed to inhibit the expression of the UBE3A antisense transcript (UBE3A-ATS), thereby reactivating the expression of the UBE3A gene in neurons.
Ribo’s siRNA therapeutic receives orphan drug designation for hepatitis delta virus infection [5]PR Newswire. Ribo granted EMA orphan drug designation for siRNA therapeutic RBD1016 for hepatitis delta virus (HDV) infection. Oct 24, 2025.
Ribo Life Science has received Orphan Drug Designation from the EMA for RBD1016, an investigational siRNA therapeutic targeting hepatitis delta virus (HDV) infection. HDV is a severe form of viral hepatitis that occurs only in individuals co-infected with hepatitis B virus, accelerating liver disease progression. RBD1016 is designed to selectively silence key viral factors involved in HDV infection. The efficacy of RBD1016 is currently being assessed in global Phase II clinical development, aiming to provide a new treatment option for patients affected by this rare disease.
Olezarsen is approved in the EU for familial chylomicronemia syndrome [6]PR Newswire. Tryngolza® (olezarsen) approved in the European Union for familial chylomicronemia syndrome (FCS) Sep 19, 2025.
Ionis Pharmaceuticals and Sobi announced that ASO therapeutic TRYNGOLZA® (olezarsen) has been approved in the EU as an adjunct to diet for the treatment of genetically confirmed familial chylomicronemia syndrome in adults. The approval follows a positive opinion from the Committee for Medicinal Products for Human Use and is based on data from the Phase 3 Balance study. The study demonstrated that 80 mg of olezarsen significantly reduced fasting triglyceride levels at six months, with sustained effects through 12 months, and substantially decreased acute pancreatitis events over 12 months.
Corsera Health combines RNAi and AI to advance cardiovascular disease prevention [7]Business Wire. Corsera Health launches to extend healthspan by predicting and preventing cardiovascular disease. Sep 2, 2025.
Corsera Health has recently developed Klotho Health, an AI-powered tool to assess lifetime cardiovascular risk and guide preventive measures [7]Business Wire. Corsera Health launches to extend healthspan by predicting and preventing cardiovascular disease. Sep 2, 2025.. Additionally, Corsera is advancing a preventive RNAi medicine targeting PCSK9 to lower LDL cholesterol and angiotensinogen to reduce blood pressure, with plans to enter clinical trials by the end of 2025. The company has raised over $50 million from founders and insiders to support these initiatives.
 | TOOLS AND TECHNOLOGIES |
Novel AI-powered CRISPR tool to accelerate gene-editing research [8]Kay C. AI-powered CRISPR could lead to faster gene therapies, Stanford Medicine study finds. Stanford Medicine News Center Sep 16, 2025.
Stanford Medicine researchers have developed CRISPR-GPT, an AI tool designed to streamline gene-editing experiments. Acting as a "copilot", CRISPR-GPT assists researchers in designing experiments, analyzing data, and troubleshooting. By automating much of the experimental design and refinement process, the tool aims to expedite the development of gene therapies. In a demonstration, a student used CRISPR-GPT to successfully turn off genes in lung cancer cells on the first attempt, a task that typically requires extensive trial and error. The goal is to reduce the time needed to develop new drugs from years to months.
bYoRNA raises €1.5 million to advance yeast-based mRNA manufacturing platform [9]EIN Presswire. bYoRNA raises €1.5M to revolutionize mRNA manufacturing. Sep 8, 2025.
bYoRNA, a French biotech company, has secured €1.5 million in pre-seed funding to advance its innovative yeast-based platform for producing therapeutic mRNA. This in vivo fermentation approach utilizes recombinant yeast cells to generate long, stable, and naturally matured mRNA molecules, offering a cost-effective and scalable alternative to traditional in vitro transcription methods. The technology aims to enhance the purity and reduce the immunogenicity of mRNA, potentially lowering production costs by up to 100 times. bYoRNA plans to apply this platform to the development of mRNA-based vaccines and therapies for oncology, gene editing, and infectious diseases.
 | COLLABORATIONS AND PARTNERSHIPS |
Arrowhead and Novartis partner on siRNA therapy targeting neurodegenerative disorders [10]Business Wire. Arrowhead Pharmaceuticals and Novartis enter into a global license and collaboration agreement. Sep 2, 2025.
Arrowhead Pharmaceuticals has struck a global licensing and collaboration agreement with Novartis for ARO-SNCA, its preclinical siRNA therapy targeting alpha-synuclein for synucleinopathies (e.g., Parkinson’s disease). Novartis will obtain exclusive worldwide rights to research, develop, manufacture, and commercialize ARO-SNCA, and the parties will also apply Arrowhead’s TRiM™ (Targeted RNAi Molecule) platform to additional collaboration targets.
Lundbeck and Contera Pharma collaborate to develop RNA-targeting therapies for neurological diseases [11]Cision News. Lundbeck and Contera Pharma announce research collaboration to advance RNA-targeting medicines for serious neurological conditions. Oct 20, 2025.
Lundbeck and Contera Pharma have entered a strategic research collaboration to develop RNA-targeting oligonucleotide-based medicines for serious neurological conditions with significant unmet medical needs. Leveraging Lundbeck's extensive neuroscience experience and Contera Pharma's proprietary RNA discovery platforms (AttackPoint discovery®, OligoDisc®, and SpliceMatrix®), the partnership aims to identify and optimize novel therapies targeting molecular factors involved in disease progression.
Secarna Pharmaceuticals and Scenic Biotech collaborate to develop a disease-modifying oligonucleotide therapy targeting novel drug target [12]Secarna. Secarna Pharmaceuticals enters into discovery and co-development agreement with Scenic Biotech to develop disease-modifying oligonucleotide therapy against a novel drug target. Oct 23, 2025.
Secarna Pharmaceuticals and Scenic Biotech have entered a discovery and co-development agreement to develop a first-in-class disease-modifying oligonucleotide therapy targeting a novel drug target identified by Scenic Biotech's proprietary Cell-Seq platform. Secarna will lead the oligonucleotide discovery efforts using its AI-powered OligoCreator® platform, while Scenic Biotech will contribute its expertise in target and disease biology. The collaboration aims to address rare genetic disorders and broader disease conditions by leveraging the synergies of both companies' platforms to bring new treatments to patients.
References
1. Oligotherapeutics: status quo and future outlook. Infographic. Nucleic Acid Insights 2025; published online Sep 15. Link
2. CRISPR Therapeutics AG. CRISPR Therapeutics presents new preclinical data for CTX460™ demonstrating in vivo gene correction of Alpha-1 Antitrypsin Deficiency (AATD) utilizing novel SyNTase™ editing platform. Oct 10, 2025.
3. PR Newswire. Argo Biopharma receives IND approval from NMPA for Phase II clinical trial of siRNA therapy BW-40202 for the treatment of PNH. Sep 15, 2025.
4. Business Wire. Ionis receives U.S. FDA Breakthrough Therapy designation for ION582 in Angelman syndrome. Sep 9, 2025.
5. PR Newswire. Ribo granted EMA orphan drug designation for siRNA therapeutic RBD1016 for hepatitis delta virus (HDV) infection. Oct 24, 2025.
6. PR Newswire. Tryngolza® (olezarsen) approved in the European Union for familial chylomicronemia syndrome (FCS) Sep 19, 2025.
7. Business Wire. Corsera Health launches to extend healthspan by predicting and preventing cardiovascular disease. Sep 2, 2025.
8. Kay C. AI-powered CRISPR could lead to faster gene therapies, Stanford Medicine study finds. Stanford Medicine News Center Sep 16, 2025.
9. EIN Presswire. bYoRNA raises €1.5M to revolutionize mRNA manufacturing. Sep 8, 2025.
10. Business Wire. Arrowhead Pharmaceuticals and Novartis enter into a global license and collaboration agreement. Sep 2, 2025.
11. Cision News. Lundbeck and Contera Pharma announce research collaboration to advance RNA-targeting medicines for serious neurological conditions. Oct 20, 2025.
12. Secarna. Secarna Pharmaceuticals enters into discovery and co-development agreement with Scenic Biotech to develop disease-modifying oligonucleotide therapy against a novel drug target. Oct 23, 2025.
Affiliation
Jokūbas Leikauskas is Commissioning Editor of Nucleic Acid Insights