List of RNA Therapies Companies in Massachusetts - 35

AIRNA, a biotech company pioneering RNA editing therapeutics to restore the health of patients with rare and common diseases. AIRNA’s experienced team is aiming to advance a pipeline of RNA editing therapeutics driven by its powerful and flexible RNA editing platform, RESTORE+.

Alltrna is the first transfer RNA (tRNA) platform company, and our mission is to unlock tRNA biology and pioneer tRNA therapeutics to regulate the protein universe and resolve disease. Founded at Flagship Labs in 2018, Alltrna has mapped tRNA biology to systematically design tRNA medicines and encode a completely new, unifying approach to treating both rare and common human diseases driven by shared genetic mutations. Alltrna’s platform combines internal expertise and proprietary machine learning tools to unlock the entire tRNA biology space. We have an unprecedented opportunity to advance a single tRNA medicine to restore disrupted protein production, regardless of target, for thousands of diseases with the same underlying genetic mutation.

Alnylam is the world's leading RNAi therapeutics company and the first and only company to bring RNAi-based medicines to market. We are developing RNAi (RNA interference) as an innovative, entirely new class of medicines to treat rare genetic, cardio metabolic, acute hepatic infectious and central nervous system (CNS) and ocular diseases. Alnylam was founded in 2002 based on a Nobel Prize-winning breakthrough in biology – the discovery of RNAi, and a bold vision that this discovery could be used to silence disease-causing genes upstream of today’s medicines. We are relentless in our pursuit of new treatments because we believe that RNAi therapeutics can be used to treat many diseases for which treatment options do not exist or are inadequate. Alnylam is turning scientific possibility into reality - in 2018, the FDA and EMA approved of our first product, ONPATTRO (patisiran) which is also the first-ever approved RNAi therapeutic. We now have three additional medicines on the market: GIVLAARI (givosiran), OXLUMO (lumasiran) and LEQVIO (inclisiran) in partnership with Novartis). Our robust pipeline of investigational medicines includes multiple programs in late-stage and early-stage clinical development. We are a global and diverse company of more than 1,700 people. We pride ourself on fostering a fun, inclusive and dynamic work environment where employees can recognize their fullest potential. That's why we've been named a Science Magazine Top Employer 3 years in a row ('19-'21), a Boston Globe Top Place to Work 7x in a row ('15-'21) and one of Fast Company's Best Workplaces for Innovators for 2021.

Launched in February 2024, Alys Pharmaceuticals is an innovation leader in immuno-dermatology, co-founded by Medixci and world-leading dermatology and scientific experts. Originating from the aggregation of six asset-centric Medixci compannies, Alys boasts a robust pipeline of innovative programs and platforms targeting multiple dermatological indications. With a vision to transform the treatment paradigm for several dermatology indications of significant prevalence and major unmet medical need, Alys aspires to redefine the landscape of dermatological treatments.

Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With active discovery and preclinical programs in ophthalmology, neurological and neuromuscular disorders, and other rare diseases, Ascidian is opening new therapeutic possibilities for patients in need of breakthroughs.

We are a biotechnology company pioneering new treatment options for neurodegenerative diseases by utilizing our proprietary RNA interference platform. We were founded by Anastasia Khvorova, Ph.D., Craig Mello, Ph.D., and Neil Aronin, M.D., of the RNA Therapeutics Institute at the University of Massachusetts Medical School, with a Series A funding exclusively by F-Prime Capital. We are headquartered in Boston, Mass

Founded by pioneering executives and scientists in RNAi, City Therapeutics is advancing next-generation engineering of small interfering RNAs (siRNAs) – the “trigger” molecules that mediate RNAi – to improve and expand the reach of RNAi-based medicines. City’s mission is to build the leading next-generation RNAi therapeutics company, unlocking RNAi’s transformative potential to help patients with a wide range of diseases.

Comanche is a biopharmaceutical company developing novel siRNA molecules for the treatment of preeclampsia. Our purpose is to lower the risks of pregnancy and prematurity worldwide by safely sustaining natural pregnancy and allowing for fetal maturation. We envision a world where all pregnant women have access to safe and effective therapies that are evidence-based, cost-effective and sustainable. Our mission is to prevent or treat preeclampsia at its root cause.

Fighting the world’s most devastating diseases through the innovative power of targeted oral mRNA vaccines.

Eterna Therapeutics is developing breakthrough mRNA cell engineering technologies to repair cellular dysfunction and treat a range of therapeutic indications. We and our strategic partners are advancing innovative nucleic acid and cell therapies that offer the hope of radically improving the health outcomes of patients with high unmet medical needs. We are committed to creating a world in which patients and their families have access to effective, life-changing treatments for serious illnesses.

Fluent BioSciences, an Illumina company, is a biotechnology company with a mission to accelerate the understanding of biology and disease through simple, cost effective and scalable solutions for every laboratory from research to the clinic. Fluent’s breakthrough Pre-templated Instant Partitions (PIPseq™) technology enables near-instantaneous self-assembly of individual cells or molecules into millions of uniform partitions without the need for complex instrumentation or expensive consumables. This powerful platform enables extremely sensitive and unbiased preparation of proteins and nucleic acids for a broad range of discovery and disease applications including single-cell RNA sequencing (scRNA-Seq).

hC Bioscience is an innovator and pioneer of protein editing using context agnostic engineered tRNA. Their First in Class approach for tRNA drug products includes a variety of delivery approaches, with the goal of improving the lives of patients with the development of first-in-class tRNA-based therapeutics targeting cancer and rare diseases.

Honeycomb Biotechnologies is an early-stage company focused on making scalable solutions for storage and single cell genomic analysis of precious clinical samples. We enable translation of the rich biological information encoded in clinical specimens into high-resolution digital information, which can be queried and analyzed with sequencing. The resulting exponential growth in knowledge about the state of each person’s health and disease will enable the identification of new therapies and better targeting of current medicines in the era of precision medicine. Our first product is the “HIVE” – a single-use disposable device that is used (without any Honeycomb-specific instrumentation or equipment) for capturing, storing, and analyzing biological samples with single-cell resolution.

Innovac Therapeutics is a biotechnology company dedicated to developing innovative vaccines for patients with unmet medical needs, using our proprietary RNA technology.

Judo Bio is a biotechnology company focused on developing precision therapeutics against genetic disorders. The company has raised a total funding of $20M over 1 round and is known for its innovative approach to drug development.

At Moderna, we believe messenger RNA, or mRNA, is the “software of life.” Every cell in the body uses mRNA to provide real-time instructions to make the proteins necessary to drive all aspects of biology, including in human health and disease. Given its essential role, we believe mRNA could be used to create a new category of medicines with significant potential to improve the lives of patients. We are pioneering a new class of medicines made of messenger RNA, or mRNA. The potential implications of using mRNA as a drug are significant and far-reaching and could meaningfully improve how medicines are discovered, developed and manufactured.

NextRNA focuses on lncRNAs, which account for the majority of ncRNAs. Their approach is motivated by observations where disregulated interactions between lncRNAs and RNA-binding proteins (RBPs) are implicated in multiple disease areas, including oncology, immunology, and neurology. They are elucidating the proteins that lncRNAs interact with and what cellular processes they affect and designing small molecules to drug these lncRNA-RBP interactions.

Orbital Therapeutics is building a first-in-kind platform at the intersection of RNA technology, delivery methods, data science and automation to develop an expansive portfolio of RNA-based medicines to treat disease in humans.

Orna Therapeutics is a biotechnology company reshaping RNA therapeutics. We are developing the leading circular RNA technology platform (oRNA®) and unprecedented lipid nanoparticle (LNP) delivery solutions – unlocking the therapeutic possibilities of circular RNA and pioneering the next chapter of safe and effective in vivo therapies for the treatment of disease. Website: www.ornatx.com

Phio Pharmaceuticals Corp. (Nasdaq: PHIO) is a clinical stage biotechnology company whose proprietary INTASYL™ RNAi technology makes immune cells more effective in killing tumor cells. INTASYL is the only self-delivering RNAi technology focused on immuno-oncology therapeutics. INTASYL drugs precisely target specific proteins that reduce the body’s ability to fight cancer, without the need for specialized formulations or drug delivery systems. Phio was co-founded by Nobel Laureate Craig Mello, who was awarded the Nobel prize in 2006 for his discovery of RNAi, which has broad therapeutic applications. Our founders opened up a whole new field of biology when they discovered RNAi, and we continue their pioneering efforts today through our work with INTASYL. We’re committed to discovering and developing innovative cancer treatments for patients, creating new pathways for a cancer-free future. For additional information, visit the Company's website, www.phiopharma.com. #INTASYL #siRNA #RNAi #Phio #immunooncology

SanegeneBio is an emerging RNAi-based company developing innovative RNA interference (RNAi) therapeutics driven by the cutting-edge delivery technology. Founded in 2021, SanegeneBio was propelled by a team of industry-leading experts and has operations in both the US and China. Since its inception, SanegeneBio has successfully established proprietary chemical modification platform, hepatic and extrahepatic delivery platforms, enabling tissue-specific delivery of a wide range of RNA therapeutics to efficiently knock down disease-causing genes. Our fast-growing RNAi portfolio covers a broad range of therapeutic areas including cardiovascular and metabolic diseases, immunology-related diseases, and nervous system diseases. Among them, our first RNAi drug has entered the clinical stage, and several pipelines are in progress simultaneously. SanegeneBio is committed to creating transformational RNAi medicines through striving for scientific innovation to address unmet medical needs worldwide and change the lives of patients and families.

Scipher Medicine is building the future of patient treatment. Most patients who are prescribed blockbuster therapies today don’t respond to the treatment, costing the health care industry billions in wasted drugs while patients continue to suffer. Scipher's platform identifies which drug will work based on the patient's fundamental disease biology, and not based on symptoms, disease classification, or medical bias. A simple test predicts which drug he/she will respond to, ensuring that most optimal treatment is prescribed from day one. The molecular data generated by our tests is then used to fuel novel target discovery to address a clear unmet medical need in patients who do not respond to any existing therapy.

SeqLL is a biotechnology company that specializes in Quantitative RNA & Specialty DNA Sequencing, offering True Single Molecule Sequencing technology.

siRNAgen Therapeutics is a next-generation RNAi platform products company focused on immunology and central nervous system (CNS) diseases. Their proprietary SAMiRNA™ platform leverages modular chemistry to overcome the challenges around delivery. SAMiRNA™ platform has been instrumental in the creation of innovative drugs such as SRN-001, which holds immense promise in treating fibrotic diseases globally. With a firm commitment to innovation and collaboration, siRNAgen continues to push the boundaries of scientific discovery to change the lives of patients worldwide.

Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases. The company is pursuing an entirely new approach to treating severe diseases. We are initially focused on diseases of the central nervous system and the eye, with an emphasis on haploinsufficiencies, which are diseases caused by a loss of about half of the normal protein function. Rather than replace, repair or edit faulty genes, we aim to increase – or stoke – protein output from healthy genes to restore functional protein levels using our proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform. In all, Stoke has identified ~6500 genes with a TANGO signature, representing broad potential for our approach to help people with severe diseases. The early results have been promising. Stoke’s first medicine in development, STK-001 has demonstrated positive Phase 1/2 results for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy that is associated with a number of debilitating non-seizure comorbidities. Behind that, is STK-002, a potential treatment for autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Clinical studies of STK-002 are anticipated to start in 2024. The company is pursuing potential treatments for three additional neurodevelopmental disorders, including Syngap1 and Rett syndrome, in collaboration with Acadia Pharmaceuticals. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts.

SynerK's founders are RNA-targeted therapy industry veterans with decades of drug development experience. Our expertise covers from discovery, preclinical and clinical development to commercial launch. Millions suffer from life threatening diseases, yet only few benefits from current treatment. There is so much we understood but also much more to understand. We strive to find a way to treat disease where a genetic defect is present. One patient at a time, the number will add up.

SYTE.bio is a Synthetic Biology Start-Up company developing precision DNA & RNA-based therapeutics and vaccines. The company has been incorporated as a Delaware C Corp in 2021 (SYTE.bio, Inc), and has labs & offices in Argentina's headquarter, that has been operating since 2017. SYTE.bio is pioneering the development of life-changing treatments for severe diseases and vaccines to treat and prevent diseases at the molecular and cellular level using engineered DNA, improving life through innovation. SYTE.bio’s powerful redosable DNA & RNA delivery platforms will unleash the potential of personalized genetic medicine in multiple potential applications, such as Gene Therapy (Oncology, Monogenic Diseases, Cardiovascular Diseases, Vaccines, Orphan Diseases), Cell Therapy (CAR-T Therapy, Induced Pluripotent Stem Cells (iPSC)) and Gene Editing (CRISPR/Cas, TALEN, ZFN).

Employs an iterative biophysics and computational engine that addresses the challenges and limitations that have historically hampered efforts to drug RNA. Utilizes NMR (nuclear magnetic resonance spectroscopy)-derived ligand binding data to guide molecular dynamics simulations and build accurate structural models.

Pioneering tRNA therapeutics to modulate mRNA function and cure a broad range of genetic diseases.

About Tiba Biotech Tiba Biotech is a pre-clinical stage biotechnology company revolutionizing the design and delivery of a safer, more effective and affordable generation of nucleic acid products for human and animal health. Tiba’s innovative RNA vaccine platform disrupts existing design, delivery and bio-manufacturing processes while enabling the rapid development of highly effective vaccines against multiple diseases. Privately held, Tiba is headquartered in Cambridge, Massachusetts, emanated from the Massachusetts Institute of Technology’s Koch Institute, and has strategic partnerships with the U.S. Army Medical Research Institute of Infectious Diseases and a growing list of leading research institutions. To learn more about Tiba, visit www.tiba.bio.

TransCode's lofty goal is delivering a cancer-free future for all cancer patients. Our core belief is that cancer can be overcome through the intelligent design and efficient delivery of targeted therapeutics. Our lead therapeutic candidate, TTX-MC138, targets microRNA-10b, or miRNA-10b, considered the master regulator of metastatic cell viability in a range of cancers, including breast, pancreatic, ovarian, colon cancer, glioblastomas, and several others. Our other preclinical programs include two solid tumor programs, TTX-siPDL1, an siRNA-based modulator of programmed death-ligand 1, or PD-L1, and TTX-siLIN28B, an siRNA-based inhibitor of RNA-binding protein LIN28B. TransCode also has three cancer agnostic programs, TTX-RIGA, an RNA- based agonist of the retinoic acid-inducible gene I, or RIG-I, targeting activation of innate immunity in the tumor microenvironment; TTX-CRISPR, a CRISPR/Cas9-based therapy platform for the repair or elimination of cancer-causing genes inside tumor cells; and TTX-mRNA, an mRNA-based platform for the development of cancer vaccines that activate cytotoxic immune responses against tumor cells. Until now, targeting biomarkers that are the primary drivers of cancer onset, progression and recurrence have been locked behind the challenge of delivery – cytosolic delivery to engage these known targets. TransCode believes it has overcome this barrier, opening the door to a broad array of cancer therapies -- a disease with the highest unmet need that affects everyone. It is through the design and optimization of this technology that TransCode has devised an elegant solution -- TTX -- a proprietary delivery platform for the transport of targeted therapeutics to cancer cells. With their world class team and know how, TransCode has a rapidly expanding platform of drug candidates designed to target a variety of tumor indications with the goal of long-term treatment survival.

Our Therapies Restore Balance. We are explorers leading the revolution in RNA drug discovery to develop an entirely new class of therapies. We are identifying the chemical motifs to potently and selectively modulate RNA regulatory networks to treat the most challenging diseases.

Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas. Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company is conducting two global Phase 3 clinical trials (THRIVE and THRIVE-2) to evaluate the safety and efficacy of VRDN-001 in patients with active and chronic TED. Viridian’s goal is to advance VRDN-001 as a potential best-in-class intravenous therapy followed by VRDN-003 as a potential first- and best-in-class subcutaneous therapy for the treatment of TED. In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has the potential to be developed in multiple autoimmune diseases.

At the Wyss Institute, we leverage recent insights into how Nature builds, controls and manufactures to develop new engineering innovations - a new field of research we call Biologically Inspired Engineering. Our scientists, engineers and clinicians, who come from Harvard's Schools of Medicine, Engineering, Arts & Sciences, Design, and Education as well as 12 collaborating academic institutions and hospitals, work alongside staff with industrial experience in product development to engineer transformative solutions to some of the world’s greatest problems. By emulating biological principles of self assembly, organization and regulation, we are developing disruptive technology solutions for healthcare, energy, architecture, robotics, and manufacturing, which are translated into commercial products and therapies through formation of new startups and corporate alliances.

La Laiterie du Berger est une entreprise familiale sénégalaise, créée en 2006. Notre mission est de collecter le lait frais des éleveurs de notre pays, pour offrir chaque jour aux consommateurs sénégalais des produits laitiers, au lait local et naturel, connus sous la marque Dolima. C’est ce qui fait que la Laiterie du Berger n’est pas une entreprise comme les autres, et que le yaourt Dolima n’est pas un yaourt comme les autres. Notre engagement s’incarne dans notre mission sociale : construire une filière sénégalaise plus forte, avec un élevage plus productif, en mesure d’approvisionner le marché local. Il s’agit aussi de plus en plus d’aller vers la nutrition, pour apporter la santé par une alimentation saine et accessible dans notre pays où cet enjeu est si grand.