1
Country: South Korea | Funding: ₩27B
Aston Sci develops cancer vaccines based on mRNA, DNA and peptides, which are currently being evaluated for efficacy in breast and gastric cancer. The company has created a cloud-based Collaborative Drug Discovery platform, powered by the Th-Vac AI epitope screening system and supported by an antigen-based epitope prediction program. Using the platform's results, the company performs precise immunological screening, enabling the identification of highly selective epitopes specific to MHC class II, known for their ability to induce strong and sustained CD4⁺ T-cell immunity. By integrating multi-omics datasets with machine learning, Th-Vac overcomes key barriers in the development of off-the-shelf vaccines encoding TAAs (tumor-associated antigens) or TCAs (tumor-specific antigens), as well as personalized neoantigen vaccines, including tumor heterogeneity, lengthy manufacturing lead times, and limited patient applicability.
Aston Sci develops cancer vaccines based on mRNA, DNA and peptides, which are currently being evaluated for efficacy in breast and gastric cancer. The company has created a cloud-based Collaborative Drug Discovery platform, powered by the Th-Vac AI epitope screening system and supported by an antigen-based epitope prediction program. Using the platform's results, the company performs precise immunological screening, enabling the identification of highly selective epitopes specific to MHC class II, known for their ability to induce strong and sustained CD4⁺ T-cell immunity. By integrating multi-omics datasets with machine learning, Th-Vac overcomes key barriers in the development of off-the-shelf vaccines encoding TAAs (tumor-associated antigens) or TCAs (tumor-specific antigens), as well as personalized neoantigen vaccines, including tumor heterogeneity, lengthy manufacturing lead times, and limited patient applicability.
2
Country: China | Funding: $100M
OTR Therapeutics is a biotechnology company developing platform-based treatments using modular RNA and delivery systems. Its pipeline covers differentiated programs that target significant treatment gaps in immunology and inflammation, oncology and neurologу. The company is a member of Bayer’s Co.Lab life sciences incubator network.
OTR Therapeutics is a biotechnology company developing platform-based treatments using modular RNA and delivery systems. Its pipeline covers differentiated programs that target significant treatment gaps in immunology and inflammation, oncology and neurologу. The company is a member of Bayer’s Co.Lab life sciences incubator network.
3
Country: USA | Funding: $3.9B
Moderna is a pioneer in developing a new class of drugs based on messenger RNA (mRNA). This new drug platform is based on the discovery that in-vitro engeneered and injected mRNA can direct the body's cellular machinery to produce virtually any protein of interest, from native proteins to antibodies and other entirely new protein constructs with therapeutic activity both inside and outside cells. In other words, mRNA can guide the body to produce its own drugs. Moderna used mRNA to create the first COVID-19 vaccine and is now applying this technology to create vaccines for infectious diseases (influenza, RSV, HSV, etc.), immuno-oncology (melanoma, lung cancer, liver cancer, etc.), rare diseases and autoimmune diseases.
Moderna is a pioneer in developing a new class of drugs based on messenger RNA (mRNA). This new drug platform is based on the discovery that in-vitro engeneered and injected mRNA can direct the body's cellular machinery to produce virtually any protein of interest, from native proteins to antibodies and other entirely new protein constructs with therapeutic activity both inside and outside cells. In other words, mRNA can guide the body to produce its own drugs. Moderna used mRNA to create the first COVID-19 vaccine and is now applying this technology to create vaccines for infectious diseases (influenza, RSV, HSV, etc.), immuno-oncology (melanoma, lung cancer, liver cancer, etc.), rare diseases and autoimmune diseases.
4
Country: USA | Funding: $2.4B
Arrowhead Research has created the Arrowhead TRiM drug discovery and development platform that utilizes a suite of RNA interference (RNAi) delivery technologies, chemistry, structures, and manufacturing technologies. REDEMPLO (plozasiran) is the company's lead (FDA-approved) siRNA-based drug used to significantly lower triglyceride levels in adults with familial chylomicronemia syndrome (FCS). Arrowhead is also conducting clinical trials for drugs against coronary artery disease, atherosclerotic cardiovascular disease, obesity, COPD, idiopathic pulmonary fibrosis, Alzheimer's disease, Huntington's disease, alpha-1 antitrypsin deficiency and hepatitis B.
Arrowhead Research has created the Arrowhead TRiM drug discovery and development platform that utilizes a suite of RNA interference (RNAi) delivery technologies, chemistry, structures, and manufacturing technologies. REDEMPLO (plozasiran) is the company's lead (FDA-approved) siRNA-based drug used to significantly lower triglyceride levels in adults with familial chylomicronemia syndrome (FCS). Arrowhead is also conducting clinical trials for drugs against coronary artery disease, atherosclerotic cardiovascular disease, obesity, COPD, idiopathic pulmonary fibrosis, Alzheimer's disease, Huntington's disease, alpha-1 antitrypsin deficiency and hepatitis B.
5
Country: Germany | Funding: $2B
BioNTech is a global leader in mRNA-based immunotherapy. The company was the first to invent and commercialize a COVID-19 vaccine (in partnership with Pfizer). BioNTech is currently developing several personalized immunotherapeutic agents (vaccines) against cancer and infectious diseases, including melanoma, human papillomavirus, non-small cell lung cancer, tuberculosis and malaria. The company's flagship platform, FixVac, enables the creation of ready-to-use mRNA cancer vaccines that can be used off-shelf to a specific indication (fixed-dose vaccines). These vaccines are a fixed combination of unmutated tumor antigens encoded by mRNA, which are frequently expressed in certain types of cancer. The mRNA is delivered using a proprietary RNA-lipoplex delivery system, which is designed to enhance mRNA stability in the body and target antigen-presenting dendritic cells (DCs), the training grounds of our immune system.
BioNTech is a global leader in mRNA-based immunotherapy. The company was the first to invent and commercialize a COVID-19 vaccine (in partnership with Pfizer). BioNTech is currently developing several personalized immunotherapeutic agents (vaccines) against cancer and infectious diseases, including melanoma, human papillomavirus, non-small cell lung cancer, tuberculosis and malaria. The company's flagship platform, FixVac, enables the creation of ready-to-use mRNA cancer vaccines that can be used off-shelf to a specific indication (fixed-dose vaccines). These vaccines are a fixed combination of unmutated tumor antigens encoded by mRNA, which are frequently expressed in certain types of cancer. The mRNA is delivered using a proprietary RNA-lipoplex delivery system, which is designed to enhance mRNA stability in the body and target antigen-presenting dendritic cells (DCs), the training grounds of our immune system.
6
Country: China | Funding: $1.4B
Abogen Biosciences is a discovery stage biotechnology company focuses on developing nucleic acid-based (RNA and DNA) therapeutics.
Abogen Biosciences is a discovery stage biotechnology company focuses on developing nucleic acid-based (RNA and DNA) therapeutics.
7
Country: USA | Funding: $510.6M
GreenLight Biosciences is using RNA to target some of the world’s biggest problems
GreenLight Biosciences is using RNA to target some of the world’s biggest problems
8
Country: USA | Funding: $360.1M
Stoke is developing antisense oligonucleotide medicines that target RNA splicing to increase gene expression for the treatment.
Stoke is developing antisense oligonucleotide medicines that target RNA splicing to increase gene expression for the treatment.
9
Country: USA | Funding: $332M
ADARx Pharmaceuticals is a genetic medicine company focusing on the base editing of mRNA transcripts.
ADARx Pharmaceuticals is a genetic medicine company focusing on the base editing of mRNA transcripts.
10
Country: USA | Funding: $321M
Orna Therapeutics is a biotechnology company that specializes in fully engineered circular RNA therapeutics to treat various diseases.
Orna Therapeutics is a biotechnology company that specializes in fully engineered circular RNA therapeutics to treat various diseases.
11
Country: USA | Funding: $293.3M
Sirnaomics is a biopharmaceutical company developing therapeutics for critical human diseases by using RNA interference (RNAi) technology.
Sirnaomics is a biopharmaceutical company developing therapeutics for critical human diseases by using RNA interference (RNAi) technology.
12
Country: USA | Funding: $277.5M
Korro Bio provides an efficient and selective method for RNA editing, leveraging natural processes common to all multi-cellular organisms.
Korro Bio provides an efficient and selective method for RNA editing, leveraging natural processes common to all multi-cellular organisms.
13
Country: USA | Funding: $270M
Orbital Therapeutics aims to enhance global health by unleashing the full potential of RNA-based medicines to treat human disease.
Orbital Therapeutics aims to enhance global health by unleashing the full potential of RNA-based medicines to treat human disease.
14
Country: USA | Funding: $270M
Orbital Therapeutics aims to enhance global health by unleashing the full potential of RNA-based medicines to treat human disease in ways that were not previously possible. It's also developing tools to treat medical conditions astronauts could suffer in space
Orbital Therapeutics aims to enhance global health by unleashing the full potential of RNA-based medicines to treat human disease in ways that were not previously possible. It's also developing tools to treat medical conditions astronauts could suffer in space
15
Country: USA | Funding: $241M
Nutcracker Therapeutics is a biotech company that develops and produces mRNA therapeutics on its proprietary and biochip-based platform.
Nutcracker Therapeutics is a biotech company that develops and produces mRNA therapeutics on its proprietary and biochip-based platform.
16
Country: USA | Funding: $211M
Remix Therapeutics is a biotechnology company that develops novel small molecule therapies.
Remix Therapeutics is a biotechnology company that develops novel small molecule therapies.
17
Country: USA | Funding: $207M
Atalanta Therapeutics is a biotechnology company pioneering new treatment options for neurodegenerative diseases by utilizing our proprietary RNA interference platform.
Atalanta Therapeutics is a biotechnology company pioneering new treatment options for neurodegenerative diseases by utilizing our proprietary RNA interference platform.
18
Country: USA | Funding: $200M
Soufflé Therapeutics has potent, precise, and safe medicines can be designed and developed for all diseases.
Soufflé Therapeutics has potent, precise, and safe medicines can be designed and developed for all diseases.
19
Country: USA | Funding: $178M
Accent Therapeutics is a biopharmaceutical company developing oncology-focused, small molecule therapies in the emerging field of epitranscriptomics. This field of biology encompasses post-transcriptional chemical modifications of RNA that provide cells with a unique mechanism for regulating proteins critical for cellular growth and differentiation. By targeting cancer-linked RNA-modifying proteins (RMPs) with precision therapies, they aim to translate extraordinary science into life-changing therapies for patients.
Accent Therapeutics is a biopharmaceutical company developing oncology-focused, small molecule therapies in the emerging field of epitranscriptomics. This field of biology encompasses post-transcriptional chemical modifications of RNA that provide cells with a unique mechanism for regulating proteins critical for cellular growth and differentiation. By targeting cancer-linked RNA-modifying proteins (RMPs) with precision therapies, they aim to translate extraordinary science into life-changing therapies for patients.
20
Country: USA | Funding: $159M
Alltrna is the tRNA platform company to decipher tRNA biology and pioneer tRNA therapeutics to treat thousands of diseases. tRNAs play a central role in the translation of mRNA into proteins. tRNA biology modulates the complex code of DNA and RNA across translation and transcription. Altrna platform incorporates AI/ML tools to learn the tRNA language and deliver diverse programmable molecules with broad therapeutic potential.
Alltrna is the tRNA platform company to decipher tRNA biology and pioneer tRNA therapeutics to treat thousands of diseases. tRNAs play a central role in the translation of mRNA into proteins. tRNA biology modulates the complex code of DNA and RNA across translation and transcription. Altrna platform incorporates AI/ML tools to learn the tRNA language and deliver diverse programmable molecules with broad therapeutic potential.
21
Country: USA | Funding: $135M
City Therapeutics is a biopharmaceutical company focused on developing next-generation small interfering RNAs.
City Therapeutics is a biopharmaceutical company focused on developing next-generation small interfering RNAs.
22
Country: USA | Funding: $104.2M
Strand is creating mRNA “programming language,” creating the world’s first platform for mRNA smart therapies.
Strand is creating mRNA “programming language,” creating the world’s first platform for mRNA smart therapies.
23
Country: UK | Funding: $89.5M
Minatx is a therapeutics company uses gene activation mechanisms to discover new medical treatments.
Minatx is a therapeutics company uses gene activation mechanisms to discover new medical treatments.
24
Country: USA | Funding: $82M
Exsilio develops genomic therapeutics that introduce therapeutic genes into safe shelters within the DNA.
Exsilio develops genomic therapeutics that introduce therapeutic genes into safe shelters within the DNA.
25
Country: USA | Funding: $80M
Twentyeight-Seven develops small molecules that can modulate levels of miRNAs by targeting proteins that interact with these miRNAs.
Twentyeight-Seven develops small molecules that can modulate levels of miRNAs by targeting proteins that interact with these miRNAs.
26
Country: USA | Funding: $73M
kernal biologics is mRNA vaccine and therapeutics company. It develops onco-selective mRNA immunotherapy
kernal biologics is mRNA vaccine and therapeutics company. It develops onco-selective mRNA immunotherapy
27
Country: USA | Funding: $64.3M
Arnatar Therapeutics is a biopharmaceutical company that specializes in the development of novel nucleic acid medications.
Arnatar Therapeutics is a biopharmaceutical company that specializes in the development of novel nucleic acid medications.
28
Country: UK | Funding: $60M
Aerska develops RNA-based therapies to treat, slow and prevent brain diseases. The company uses blood-brain barrier drug delivery technology to deliver targeted RNA interference-based drugs to treat neurological diseases. This technology serves as a drug delivery platform that transports RNAi across the blood-brain barrier. The technology works by creating antibodies or molecules that bind to specific receptors on the surface of the blood-brain barrier. This action tricks the barrier, allowing the therapeutic agent to pass through endothelial cells and enter the brain parenchyma.
Aerska develops RNA-based therapies to treat, slow and prevent brain diseases. The company uses blood-brain barrier drug delivery technology to deliver targeted RNA interference-based drugs to treat neurological diseases. This technology serves as a drug delivery platform that transports RNAi across the blood-brain barrier. The technology works by creating antibodies or molecules that bind to specific receptors on the surface of the blood-brain barrier. This action tricks the barrier, allowing the therapeutic agent to pass through endothelial cells and enter the brain parenchyma.
29
Country: USA | Funding: $42M
Atomic AI is a developing the cutting-edge fusion of machine learning and structural biology to unlock RNA drug discovery.
Atomic AI is a developing the cutting-edge fusion of machine learning and structural biology to unlock RNA drug discovery.
30
Country: USA | Funding: $40M
Creyon Bio is a drug development company engineering RNA-based medicines and their components.
Creyon Bio is a drug development company engineering RNA-based medicines and their components.
31
Country: Israel | Funding: $20M
NeuroSense Therapeutics is a clinical stage drug development company offering ground-breaking treatment for ALS patients, as well as other neurodegenerative diseases. Focused on a combined therapy, targeting multiple pertinent MoAs in these complex diseases.
NeuroSense Therapeutics is a clinical stage drug development company offering ground-breaking treatment for ALS patients, as well as other neurodegenerative diseases. Focused on a combined therapy, targeting multiple pertinent MoAs in these complex diseases.
32
Country: Ireland | Funding: €13.4M
Developing first in class regenerative gene therapies. Proprietary OmniSomes technology uses bioengineered stem cell extracellular vesicles (exosomes) as novel delivery vehicles for RNA based advanced therapeutics with regenerative medicine properties.
Developing first in class regenerative gene therapies. Proprietary OmniSomes technology uses bioengineered stem cell extracellular vesicles (exosomes) as novel delivery vehicles for RNA based advanced therapeutics with regenerative medicine properties.
33
Country: Canada | Funding: $11.4M
Phenomic AI is a developer of a drug discovery platform designed to discovery and optimize therapeutics directly against phenotypic assays.
Phenomic AI is a developer of a drug discovery platform designed to discovery and optimize therapeutics directly against phenotypic assays.
34
Country: Italy | Funding: €1.3M
BrainDTech represents a disruptive approach to brain disease diagnosis because it detects a signal (MicroRNA in microvesicles) released by microglia years before any clinical manifestation
BrainDTech represents a disruptive approach to brain disease diagnosis because it detects a signal (MicroRNA in microvesicles) released by microglia years before any clinical manifestation
35
Country: UK
Roquefort Therapeutics is a biotech company focused on developing first in class medicines in the high value and high growth oncology market.
Roquefort Therapeutics is a biotech company focused on developing first in class medicines in the high value and high growth oncology market.
36
Country: Canada
Genevant brings world-class delivery platforms that include best-in-class lipid nanoparticle (LNP) technology and next generation ligand conjugate technology, the industry’s most robust and expansive LNP patent estate, decades of experience and expertise in nucleic acid drug development headed by the team that enabled delivery for the world’s only approved small interfering RNA (siRNA)-LNP product, and an innovative culture that positions us to maintain our leadership for years to come.
Genevant brings world-class delivery platforms that include best-in-class lipid nanoparticle (LNP) technology and next generation ligand conjugate technology, the industry’s most robust and expansive LNP patent estate, decades of experience and expertise in nucleic acid drug development headed by the team that enabled delivery for the world’s only approved small interfering RNA (siRNA)-LNP product, and an innovative culture that positions us to maintain our leadership for years to come.
37
Country: USA
SYTE.biotech is pioneering the development of life-changing RNA treatments for severe diseases with engineered viruses that harbor therapeutic genes to cure diseases at the molecular and cellular level and designing innovative vaccines.
SYTE.biotech is pioneering the development of life-changing RNA treatments for severe diseases with engineered viruses that harbor therapeutic genes to cure diseases at the molecular and cellular level and designing innovative vaccines.
