RNA Therapies Clinical Trials and Pipeline 2025: EMA, PDMA, FDA Approvals, Medication, IND, NDA Approval, Therapies, Mechanism of Action, Route of Administration and Companies by DelveInsight

RNA therapies companies are Novartis, Arrowhead Pharmaceuticals, Arbutus Biopharm, Dicerna Pharmaceuticals, Inc., SanegeneBio, Alnylam Pharmaceuticals, Eli Lilly and Company, Janssen Pharmaceuticals, Moderna Therapeutics, OliX Pharmaceuticals, ProQR Therapeutics, Fujian Shengdi Pharmaceutical, Silence Therapeutics, WaVe life Sciences, Sirnaomics, ExoRNA Bioscience, Comanche Biopharma, Ractigen Therapeutics, GeneCare Research Institute, Sirana Pharma, DTx Pharma, Aptadel Therapeutics, and others.
(Albany, USA) DelveInsight's 'RNA Therapies Competitive Landscape 2025' report provides comprehensive global coverage of available, marketed, and pipeline RNA therapies in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space and future growth potential of the RNA therapies competitive domain.

With over 80 key companies active in the RNA therapies space, the market is projected to grow due to advancements in mRNA, siRNA, and antisense oligonucleotide-based therapies. Emerging RNA-based, especially in areas like oncology, infectious diseases, and genetic disorders, are fueling this expansion.

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Key Takeaways from the RNA Therapies Pipeline Report

* Over 80+ companies are evaluating 100+ RNA therapies in various stages of development, and their anticipated acceptance in the RNA therapies market would significantly increase market revenue.
* Leading RNA therapies companies such as Novartis, Arrowhead Pharmaceuticals, Arbutus Biopharm, Dicerna Pharmaceuticals, Inc., SanegeneBio, Alnylam Pharmaceuticals, Eli Lilly and Company, Janssen Pharmaceuticals, Moderna Therapeutics, OliX Pharmaceuticals, ProQR Therapeutics, Fujian Shengdi Pharmaceutical, Silence Therapeutics, WaVe life Sciences, Sirnaomics, ExoRNA Bioscience, Comanche Biopharma, Ractigen Therapeutics, GeneCare Research Institute, Sirana Pharma, DTx Pharma, Aptadel Therapeutics, and others are evaluating novel RNA therapies candidates to improve the treatment landscape.
* Key RNA therapies pipeline in various stages of development include ARO-APOC3, AB-729, ARO-C3, DCR-AUD, SGB-9768, Cemdisiran, LY3561774, JNJ-75220795, mRNA 1230, OLX 702A, Sepofarsen, HRS 5635, Divesiran , WVE 003, STP 122G, STP 707, ER 2001, CBP 4888, RAG 17, RECQL1 siRNA, Research programme: musculoskeletal disorders, Research programme: RNA therapeutics , ADEL 001, and others.
* In November 2024, RiboX's Circular RNA Therapy RXRG001 Cleared for Phase 1/2a Trial in Radiation-Induced Xerostomia and Hyposalivation
* In June 2024, Avidity Biosciences experimental RNA therapy for Facioscapulohumeral muscular dystrophy (FSHD) which is rare, progressive, life-long muscle weakening condition has shown promising results in reducing the expression of DUX4 mRNA and DUX4 protein in muscles that causes this rare disease. Their proprietary monoclonal antibody delpacibart braxlosiran or del-brax (AOC 1020) binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DUX4 mRNA. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted orphan designation for del-brax and the FDA has granted del-brax fast track designation.
* On 17 Dec 2024, Silexion Therapeutics Corp., a clinical-stage biotech developing RNAi therapies for KRAS-driven cancers declared its ongoing collaboration with Evonik, a global leader in specialty chemicals. This collaboration aims at developing an advanced long-acting siRNA PLGA microparticle formulation which has shown high potency for KRAS-mutated cancers in preclinical models.
* In Nov 2024, HuidaGene Therapeutics received the first-ever FDA clearance of CRISPR/Cas13 RNA-Editing HG202 investigational new drug (IND) application for neovascular age-related macular degeneration (nAMD) which is the only clinical-stage RNA-targeting therapy for nAMD.
* In Sep 2024, Carisma Therapeutics, a clinical stage biopharmaceutical company developing innovative immunotherapies declared the expansions of its in vivo chimeric antigen receptor macrophage and monocyte (CAR-M) therapy collaboration with Moderna Inc., for developing therapies for autoimmune diseases.
* In June 2024, Ascidian Therapeutics, a company developing RNA rewriting (or RNA exon editing) technology declared a research collaboration and licensing agreement with Roche in which Ascidian will receive $ 42 million in initial payment, and up to $ 1.8 billion in research, clinical and commercial milestones. The collaboration aims using RNA Exon Editors with Roche's CNS delivery capabilities to developing novel therapies targeting neurological diseases.
* In Sep 2024, Ractigen Therapeutics received orphan drug designation (ODD) from the FDA for its investigational small activating RNA (saRNA) which is currently under preclinical development and will be used for treatment of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) caused by any mutation in the DMD gene.

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RNA Therapies Overview

RNA therapies represent a rapidly evolving field in biotechnology, offering innovative treatment approaches for a wide range of diseases. These therapies utilize RNA molecules to regulate gene expression, modify protein production, or replace faulty genetic instructions. The main types of RNA-based therapeutics include messenger RNA (mRNA), small interfering RNA (siRNA), microRNA (miRNA), antisense oligonucleotides (ASOs), and RNA aptamers.

mRNA therapies, exemplified by COVID-19 vaccines, instruct cells to produce specific proteins that trigger immune responses or treat genetic disorders. siRNA and miRNA therapies silence harmful genes by interfering with messenger RNA, making them promising for neurodegenerative diseases, cancers, and rare genetic disorders. ASOs function by binding to target RNA sequences to modulate gene expression, while RNA aptamers act as targeted inhibitors or delivery agents.

Advancements in delivery technologies, including lipid nanoparticles and conjugation strategies, have improved the stability and effectiveness of RNA-based drugs. Regulatory approvals for RNA therapies, such as those for spinal muscular atrophy (SMA) and hereditary transthyretin amyloidosis, have validated their therapeutic potential.

With ongoing research into optimizing stability, specificity, and safety, RNA therapies hold promise for revolutionizing personalized medicine, oncology, infectious diseases, and rare genetic disorders, paving the way for the next generation of precision therapeutics.

RNA Therapies Pipeline Analysis: Drug Profile

LEQVIO: Novartis

LEQVIO (inclisiran) is an injectable treatment given every six months to reduce low-density lipoprotein (LDL) cholesterol, often referred to as "bad" cholesterol. It is a small interfering RNA (siRNA) therapy that targets a liver protein called PCSK9 (proprotein convertase subtilisin kexin type 9), which normally limits the number of receptors that remove LDL cholesterol from the bloodstream. By blocking the translation of PCSK9 mRNA, LEQVIO decreases PCSK9 production, helping to lower LDL cholesterol levels. It is used alongside diet and statins to manage high LDL cholesterol in adults with elevated cholesterol or atherosclerosis. Novartis holds the global rights to develop, produce, and market LEQVIO through a partnership with Alnylam Pharmaceuticals, a pioneer in RNA interference (RNAi) therapies.

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RNA Therapies Market Dynamics

The RNA therapies market has seen rapid growth and significant interest in recent years, driven largely by advances in biotechnology, increasing investments in the development of novel therapies, and the success of mRNA vaccines during the COVID-19 pandemic. With the potential to treat genetic disorders, cancers, infectious diseases, and other conditions that were previously considered untreatable, the RNA therapy market is expected to expand rapidly.

One of the key drivers of the market is the increasing prevalence of chronic and rare genetic diseases. RNA therapies are particularly effective in treating rare genetic disorders by correcting or modulating the expression of specific genes. For instance, the approval of treatments like SPINRAZA (for spinal muscular atrophy) and ONPATTRO (for hereditary transthyretin-mediated amyloidosis) has validated the commercial viability and therapeutic impact of RNA-based drugs. Additionally, RNA therapeutics provide a promising approach for precision medicine, where treatments can be tailored to the genetic profile of an individual patient.

On the competitive front, the RNA therapy market is dominated by both established pharmaceutical companies and emerging biotech startups. Major pharmaceutical players such as Moderna, Alnylam Pharmaceuticals, and BioNTech have made significant strides in developing RNA therapies and are investing heavily in expanding their pipelines. This competitive landscape is further enriched by partnerships and collaborations between biotech companies and academic institutions, which are accelerating innovation and product development. However, challenges such as delivery mechanisms, safety concerns, and scalability of RNA-based therapies still remain critical hurdles that industry players need to address.

Regulatory advancements and supportive government policies also contribute to the market's positive outlook. Regulatory agencies like the FDA and EMA have shown a willingness to fast-track the approval of RNA-based treatments, particularly for life-threatening or rare diseases with limited treatment options. The success of mRNA vaccines has also built confidence in the safety and efficacy of RNA therapeutics, paving the way for a more streamlined regulatory process for future RNA-based drugs.

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A snapshot of the pipeline RNA therapies mentioned in the report:

* ARO-APOC3: Arrowhead Pharmaceuticals
* Cemdisiran: Alnylam Pharmaceuticals
* Sepofarsen: ProQR Therapeutics
* AB-729: Arbutus Biopharm
* Solbinsiran: Eli Lilly and Company
* HRS 5635: Fujian Shengdi Pharmaceutical
* ARO-C3: Arrowhead Pharmaceuticals
* DCR-AUD Dicerna Pharmaceuticals, Inc
* JNJ-75220795 Johnson & Johnson Innovative Medicine
* OLX 702A: OliX Pharmaceuticals
* STP 122G: Sirnaomics
* SGB-9768: SanegeneBio

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Key Developments in the RNA Therapies Domain

* In July 2024, Rgenta Therapeutics announced the clearance of its IND by the FDA for RGT-61159, which is being developed for the potential treatment of adenoid cystic carcinoma, colorectal cancer, and other solid tumors as well as acute myeloid leukemia.
* In June 2024, Ascidian Therapeutics announced a research collaboration and licensing agreement with Roche for the discovery and development of RNA exon editing therapeutics targeting neurological diseases.
* In May 2024, Achilles Therapeutics plc announced a research collaboration with Arcturus Therapeutics Holdings Inc. to evaluate best-in-class, self-amplifying mRNA (sa-mRNA) personalized cancer vaccines (PCVs) targeting clonal neoantigens. The research collaboration will combine Achilles' best-in-class AI-driven, tumor-targeting technology with Arcturus' world-leading sa-mRNA platform.
* In April 2024, Ipsen and Skyhawk Therapeutics signed an exclusive worldwide collaboration to discover and develop novel small molecules that modulate RNA for rare neurological diseases. The agreement includes an option according to which Ipsen would acquire an exclusive license for the worldwide rights to develop successful development candidates (DC).
* In April 2024, MiNA Therapeutics announced a research collaboration and licensing agreement option with Nippon Shinyaku Co., Ltd. The collaboration will allow for the discovery and potential development and commercialization of RNAa therapeutic candidates targeting rare neurodegenerative diseases for which there are currently no treatment options.
* In January 2024, Boehringer Ingelheim agreed to collaborate with Suzhou Ribo Life Science Co and Ribocure Pharmaceuticals AB (Ribo), to develop new RNAi therapeutics for nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH). The deal exceeds a value of $2 billion and will utilize Ribo's expertise in small interfering RNA (siRNA) treatments.
* In January 2024, Ascidian Therapeutics announced that the FDA had cleared its application to begin the first RNA-editing clinical trial in the United States.

Scope of the RNA Therapies Competitive Landscape Report

* Coverage: Global
* Key RNA Therapies Companies: Novartis, Arrowhead Pharmaceuticals, Arbutus Biopharm, Dicerna Pharmaceuticals, Inc., SanegeneBio, Alnylam Pharmaceuticals, Eli Lilly and Company, Janssen Pharmaceuticals, Moderna Therapeutics, OliX Pharmaceuticals, ProQR Therapeutics, Fujian Shengdi Pharmaceutical, Silence Therapeutics, WaVe life Sciences, Sirnaomics, ExoRNA Bioscience, Comanche Biopharma, Ractigen Therapeutics, GeneCare Research Institute, Sirana Pharma, DTx Pharma, Aptadel Therapeutics, and others
* Key RNA Therapies in Pipeline: ARO-APOC3, AB-729, ARO-C3, DCR-AUD, SGB-9768, Cemdisiran, LY3561774, JNJ-75220795, mRNA 1230, OLX 702A, Sepofarsen, HRS 5635, Divesiran , WVE 003, STP 122G, STP 707, ER 2001, CBP 4888, RAG 17, RECQL1 siRNA, Research programme: musculoskeletal disorders, Research programme: RNA therapeutics , ADEL 001, and others.

Table of Contents

1. RNA Therapies Pipeline Report Introduction

2. RNA Therapies Pipeline Report Executive Summary

3. RNA Therapies Pipeline: Overview

4. RNA Therapies Marketed Drugs

4.1. LEQVIO: Novartis

5. RNA Therapies Clinical Trial Therapeutics

6. RNA Therapies Pipeline: Late-Stage Products (Pre-registration)

7. RNA Therapies Pipeline: Late-Stage Products (Phase III)

7.1. ARO-APOC3: Arrowhead Pharmaceuticals

8. RNA Therapies Pipeline: Mid-Stage Products (Phase II)

8.1. AB-729: Arbutus Biopharm

9. RNA Therapies Pipeline: Early-Stage Products (Phase I/II)

9.1. ARO-C3: Arrowhead Pharmaceuticals

10. RNA Therapies Pipeline: Preclinical and Discovery Stage Products

10.1. SGB-9768: SanegeneBio

11. RNA Therapies Pipeline Therapeutics Assessment

12. Inactive Products in the RNA Therapies Pipeline

13. Company-University Collaborations (Licensing/Partnering) Analysis

14. Unmet Needs

15. RNA Therapies Market Drivers and Barriers

16. Appendix

About DelveInsight

DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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