7 Promising RNA Biotech Companies To Watch in 2026
For many years the focus of therapeutic developments and scientific research was directed at the genome and proteins. Yet, there is a completely different level of cell regulation – transcriptome level, presented by Ribonucleic acids (RNAs). RNA molecules are not just intermediates between DNA and proteins, in fact, they also carry enzymatic functions, control gene expression, and perform other functions in cells.
Major breakthroughs in transcriptomics have been made since the beginning of the century: the discovery of RNA interference blew up the industry and the Nobel Prize in Physiology or Medicine 2006 was awarded jointly to Andrew Z. Fire and Craig C. Mello "for their discovery of RNA interference - gene silencing by double-stranded RNA."
RNA interference is a gene silencing mechanism promoted by small siRNA molecules that are also exploited in patisiran and inotersen—first-in-class RNA therapeutics. Both drugs target transthyretin, a protein involved in the pathogenesis of transthyretin-mediated amyloidosis, and reduce the expression of its mutant version.
Today, RNA therapies can be categorized into 3 broad groups: RNAs that modulate DNA, RNAs that target proteins, RNAs that encode proteins, and hybrids of three listed approaches. Among existing modalities – ASOs, single-stranded antisense oligonucleotides that block mRNA translation, siRNAs and microRNAs that degrade protein-encoding mRNAs, RNA aptamers that tightly bind specific spots on proteins and regulate their functioning. In addition to that, last year showed a real boom in mRNA vaccines, with coronavirus vaccines and cancer vaccines being the most prominent representatives.
While there are numerous companies developing RNA-based therapeutics and vaccines, including major corporations like Pfizer, BioNtech, Moderna, etc, below we decided to list several biotech startups to keep an eye on.
Kernal Bio
Kernal Bio is a Cambridge, Massachusetts company founded in 2019 by Yusuf Erkul, Burak Yilmaz, and Cafer “Jeff” Ozdemir to design cell-selective mRNA medicines paired with targeted lipid nanoparticles for in-vivo delivery. The team positions its work as “mRNA 2.0,” using machine-learning models to tune sequence features for translation, stability, and cell selectivity.
The lead internal program, KR-402, encodes a chimeric antigen receptor in a patient’s T cells using targeted LNPs; indications named publicly include multiple sclerosis and B-cell malignancies. In 2025, the Advanced Research Projects Agency for Health (ARPA-H) awarded Kernal up to $48 million to advance KR-402 under the EMBODY initiative.
In parallel, the company disclosed kernaLM, a proprietary large language model for predicting mRNA stability and translation efficiency, presented at the Computational RNA Design & Delivery Summit.
Recently, the company joined Lilly Gateway Labs in Boston’s Seaport and NVIDIA Inception, both initiatives framed as support for scaling the AI-driven design stack and in-vivo programs.
Since emerging, Kernal Bio has disclosed $25 million Series A equity in July 2022 led by Hummingbird Ventures with Amgen Ventures, among others.
Mana.bio
Mana.bio is a promising biotech startup that was established in 2021 and operates primarily out of the San Francisco Bay Area, though it also has a notable presence in Tel Aviv, Israel. The company uses an artificial intelligence-based approach to improve the development of lipid nanoparticles (LNPs) for oligonucleotide therapies, which encompass DNA and RNA-based therapeutics as well as vaccines. As delivery is a crucial bottle-neck for RNA/DNA therapies in general, Mana.bio can potentially unlock new therapeutic horizons and contribute to the advancement of personalized medicine.
In February 2024, Mana.bio joined the Ginkgo Technology Network, a consortium of over 25 companies collaborating to integrate AI, genetic medicine, and biomanufacturing capabilities into a unified R&D ecosystem led by Ginkgo Bioworks.
Another notable collaboration was Mana.bio joining the NVIDIA Inception program, which provided the company access to NVIDIA’s AI development resources and networking opportunities as it advances its machine learning–based platform for optimizing RNA delivery.
In September 2025, Mana.bio has launched Mina, a large language model tailored for lipid nanoparticle (LNP) design and optimization. The AI system, available publicly, is designed to assist researchers in developing and improving RNA delivery systems by integrating predictive modeling, curated data retrieval, and formulation optimization tools.
Mina can predict properties such as LNP size and stability, identify trends across public datasets, and provide data-driven guidance on parameters like safety and tissue targeting. Mana.bio retains proprietary predictive models and datasets for internal and partner use but offers Mina’s open-access version as a resource to the broader scientific community.
In another collaboration, Mana.bio entered a research partnership with Sheba Medical Center and ARC Innovation to apply its AI-based programmable RNA delivery platform to T cell engineering for cancer and autoimmune disease therapies. Data generated from joint experiments will be used to refine Mana.bio’s predictive models and improve RNA transfection efficiency and safety, with the goal of accelerating personalized RNA therapeutics toward clinical application.
Financially, Mana.bio secured $19.5 million in a seed investment round from Andreessen Horowitz Bio + Health, Base4 Capital, NFX, LionBird, and Technio, underscoring a significant vote of confidence in the company's innovative approach.
Orna Therapeutics
Orna Therapeutics is a Cambridge-based biotech startup founded in 2019 whose main focus lies in the novel field of circular RNAs (oRNAs) which is the next step in RNA-based therapies. The company’s innovation relies on the groundbreaking results of Prof. Daniel Anderssons’s group (MIT) that investigated oRNAs and found its superiority over linear analogs in terms of stability, protein expression, and synthetic availability.
The potential applications of this technology are far-reaching, including Orna’s initial focus on developing in situ CAR-T delivery system which avoids costly and complicated procedures of the current immunotherapies. The company already managed to close the Series A round in February 2021 raising $80 million from a group of prominent healthcare investors including F2 Ventures, Taiho Ventures, and MPM Capital. A successful Series B financing round was closed in August, 2022, raising $221 million.
In May 2023, Orna Therapeutics disclosed advancements regarding its circular RNA platform. Specifically, ORN-101, an optimized development-stage in situ CAR program, shows tumor eradication in a mouse model at doses far lower than previously reported results. Orna's FoRCE screening platform has enabled the identification of oRNAs that drive high protein expression, creating broad value across all of Orna's programs.
To strengthen its position in circular RNA drug development, Orna Therapeutics acquired ReNAgade Therapeutics, combining Orna’s oRNA expression technology with ReNAgade’s lipid nanoparticle–based delivery systems and RNA editing programs.
Additionally, the company has entered a partnership with Merck to discover, develop, and commercialize multiple programs, including vaccines and therapeutics in the areas of infectious disease and oncology.
Through its subsidiary ReNAgade Therapeutics, Orna Therapeutics also entered a three-year collaboration with Vertex Pharmaceuticals to apply its lipid nanoparticle (LNP) delivery technology to gene-editing therapies for sickle cell disease and beta thalassemia. The deal includes $65 million upfront and potential milestones exceeding $600 million, combining Orna’s non-viral delivery platform with Vertex’s expertise in hemoglobinopathies to advance in vivo RNA-based treatments.
The company, now led by CEO Joseph Bolen, Ph.D.—formerly the company’s Chief Scientific Officer and R&D head at Moderna and Millennium Pharmaceuticals—is advancing toward clinical trials in 2026 for its in vivo panCAR RNA programs targeting autoimmune diseases and multiple myeloma, alongside ongoing collaborations with Merck and Vertex.
At the 2025 ASGCT Meeting, Orna Therapeutics reported that its CD19 panCAR circular RNA therapy achieved targeted immune-cell delivery and durable B cell depletion in preclinical models, supporting its oRNA and LNP platforms for in vivo treatment of B cell–driven autoimmune diseases.
Korro Bio
A US-based startup Korro Bio has a smart approach to gene editing that relies on the natural RNA editing system, already present in all human cells. The company utilizes the short synthetic RNA strands to activate the ADAR (Adenosine Deaminases Acting on RNA) and guide it to make a specific fix in the messenger RNA. In comparison to CRISP technology when DNA gene material is altered directly, RNA editing only alters the genetic messenger and could be stopped at any time in case of adverse reactions. It also eliminates the need for an intrusive injection of a thid-party enzyme, therefore decreasing the risk and optimizing the safety profile.
Korro’s OPERA (Oligonucleotide Promoted Editing of RNA) platform is built to create synthetic RNA-editing molecules that guide the body’s natural ADAR enzyme to correct single-base errors in RNA. The system combines deep knowledge of ADAR biology, advanced oligonucleotide chemistry, and machine learning–based design to optimize each candidate for efficiency and precision, supported by tissue-targeted delivery methods that improve specificity and durability in different diseases.
Although the company was founded in 2020 it has already raised a Series A in Sep 2020, attracting $91.5 million with Wu Capital as the lead investor.
In a strategic move to harness the potential of RNA editing technology, Korro Bio and Frequency Therapeutics underwent a merger in July 2023. The merged company continues under the moniker of Korro Bio.
In September 2024, Novo Nordisk entered a collaboration worth up to $530 million with Korro Bio to develop RNA-editing therapies for cardiometabolic diseases, leveraging Korro’s OPERA platform.
The 2023 Korro Bio collaboration and license agreement with Genevant Sciences supplied the clinically validated lipid nanoparticle (LNP) delivery system used in KRRO-110, the company’s first development candidate for alpha-1 antitrypsin deficiency (AATD) announced on the same year. KRRO-110 is an RNA-editing therapy for AATD, a genetic condition that damages the liver and lungs.
Korro Bio has initiated dosing in its REWRITE Phase 1/2a trial evaluating KRRO-110, which uses the company’s OPERA platform to harness the body’s ADAR enzyme to correct a single-letter RNA error and restore normal alpha-1 antitrypsin production. By repairing RNA rather than DNA, KRRO-110 aims to reduce liver protein buildup and improve lung protection in patients with AATD. The trial has progressed with over 80% of healthy volunteers dosed and no serious adverse events observed, and KRRO-110 has received orphan drug designation from both the FDA and EMA for its potential to address this rare disorder.
Shape Therapeutics
Shape Therapeutics also leverages a natural ADAR RNA editing system and makes use of it in the number of their products, such as the RNA Fix system for single-mutation genetic diseases. They have also developed an RNA Skip to fix the mutations associated with premature stop codons and enable the production of fully corrected protein. In order to face the delivery challenges, they are developing an AAVid capsid delivery platform which through the screening between millions of possible AAV (Adeno Associated Viruses) selects the most suitable for specific disease tissues.
The company was founded in Seattle, USA in 2018 and gained attention from notable investors, including New Enterprise Associates, Tectonic Associates, and Mission BioCapital who took part in Series A totaling $35.5 million.
At ASGCT 2023, the company announced seven presentations that spotlight its groundbreaking programmable medicine technology, enabled by artificial intelligence. This technology harnesses the power of AI and RNA to address genetic diseases. Shape’s CEO, Francois Vigneault, shared some notable advances in CNS in vivo at the J.P. Morgan 41st Annual Healthcare Conference. Those included highlights across the ShapeTX technology platforms, including breakthrough results from ShapeTX's AI-driven AAVid capsid discovery platform and TruStable AAV manufacturing technology platform.
Additionally, Shape Therapeutics reported that its RNAfix RNA-editing platform achieved over 95% editing across the mouse brain and up to 92% in non-human primates, with effects lasting at least six months after a single AAV-based dose. Presented at the 2024 ASGCT Annual Meeting, the data highlight RNAfix’s potential for treating central nervous system disorders such as Rett syndrome, Parkinson’s, and Alzheimer’s disease.
Shape Therapeutics has also expanded its reach through strategic collaborations, for example a $1.5 billion partnership with Otsuka to develop gene therapies for eye diseases, integrating Shape’s AI-driven AAV platform with Otsuka’s expertise in ophthalmology.
Recently, VectorY Therapeutics have also entered an option and license agreement with Shape Therapeutics to evaluate and potentially license SHP-DB1, Shape’s deep-brain penetrating AAV5-derived capsid, for delivering vectorized antibodies targeting three neurodegenerative disease proteins. The deal, valued at up to $1.2 billion in total payments, will enable VectorY to incorporate Shape’s capsid into programs for ALS, Huntington’s disease, and Alzheimer’s disease, supporting intravenous delivery of genetic therapies that reach deep brain regions while minimizing off-target effects.
In October 2025, Shape Therapeutics reported preclinical data showing that its SHP-201 gene therapy, delivered with the SHP-DB1 brain-penetrating AAV5 vector, reduced production of alpha-synuclein, a key protein linked to Parkinson’s disease.
Remix Therapeutics
The majority of the diseases are caused by protein malfunction and therefore are the main targets in the current pharmaceutical world. Remix Therapeutics, a company founded by former Atlas Venture Executive in Residence Peter Smith in 2019 in Cambridge, USA, decided to get back to the origin of protein expression and alter at the level when the RNA sequences are produced.
They are using small molecule therapies which have advantages of simple dosage and increased stability inside the living organism. Their research team presented a REMaster technology for the development of new therapies which consists of three main parts: a database of RNA messengers, screenings techniques to validate the target, and a custom library of small molecules.
In February 2022, the company partnered with industry heavyweight Johnson & Johnson. Together, they aimed to develop small-molecule therapeutics with the capability to reprogram RNA processing, targeting up to three diseases in the realms of immunology and oncology.
In another notable update, Remix Therapeutics reached a milestone in its collaboration with Roche, which focuses on discovering small molecule drugs that control RNA processing using Remix’s REMaster platform. Remix has received an upfront $30 million and milestone payment as part of a deal worth up to $1 billion in potential milestones and royalties. So far, the collaboration has advanced early discovery programs, demonstrating the platform’s ability to uncover novel druggable RNA targets for future development.
Recently, Remix Therapeutics has begun dosing patients in two Phase 1 trials of REM-422, an oral small molecule drug that targets and destroys faulty MYB mRNA—a key driver of certain cancers. The drug, granted orphan status by the FDA, represents a new approach to treating cancers previously considered resistant to direct molecular targeting.
Financially, Remix has accumulated $211 million over three funding rounds, with the most recent financing led by The Column Group concluding $60 million.
Deep Genomics
Deep Genomics is developing personalized RNA therapeutics by leveraging AI to decode the vast complexities of RNA biology, poised to make significant advancements in the treatment of genetic disorders. Founded in 2015 by Brendan Frey, an internationally acclaimed entrepreneur, engineer, and scientist, Deep Genomics is headquartered in Toronto, Ontario, with additional facilities in Cambridge, Massachusetts. Brendan Frey, also a co-founder of the Vector Institute for Artificial Intelligence, has made fundamental contributions to deep learning, genomic medicine, and information technology.
Deep Genomics initially developed 40 different machine learning systems for each biological process, but the introduction of BigRNA has transformed their approach by allowing the model to train on everything simultaneously. The BigRNA platform is designed to tackle the complexities of RNA biology to identify novel targets, mechanisms, and molecules that traditional methods often overlook.
BigRNA stands out as the world's first RNA foundation model for RNA therapeutics, boasting 1.8 billion tunable parameters built from 1 trillion genomic signals. It enables rapid exploration of RNA biology at scale, encompassing species, tissues, cell models, variants, genes, oligonucleotides, and editing mechanisms, including splicing, polyadenylation, and protein/microRNA binding. As a result, it enables the development of steric-blocking oligonucleotides (SBOs), which are used to increase gene expression for the treatment of genetic diseases, including ultra-rare, rare, and common diseases.
In April 2025, Deep Genomics introduced REPRESS, a deep learning model that predicts microRNA binding and mRNA degradation directly from RNA sequences, expanding its AI foundation platform for decoding RNA biology. Trained on millions of human and mouse data points, REPRESS enables rapid analysis of RNA stability, a key factor in regulating protein production. The tool offers new insight into post-transcriptional gene regulation and supports the design of RNA-based therapeutics such as antisense oligonucleotides (ASOs), siRNAs, and mRNA therapies. REPRESS is now available for non-commercial research use via Deep Genomics’ GitHub.
Deep Genomics has secured $236.7 million across various rounds, with a $180 million Series C round led by SoftBank Vision Fund 2 in 2021.
Topic: Next-Gen Tools
