List of Oligonucleotides Companies in Massachusetts - 18

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+.

Through cutting-edge science and medicine, Biogen discovers, develops and delivers innovative therapies worldwide for people living with serious neurological and neurodegenerative diseases. Founded in 1978, Biogen is a pioneer in biotechnology and today the Company has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first and only approved treatment for spinal muscular atrophy, and is at the forefront of neurology research for conditions including Alzheimer’s disease, Parkinson’s disease and amyotrophic lateral sclerosis. Biogen also manufactures and commercializes biosimilars of advanced biologics. With approximately 7000 people worldwide, we are truly a global organization, headquartered in Cambridge, Massachusetts, which is also home to our research operations. Our international headquarters are based in Zug, Switzerland and we have world-class manufacturing facilities in North Carolina and Denmark. We offer therapies globally through direct affiliate presence in 30 countries and a network of distribution partners in over 50 additional countries.

BioPAL is a biotechnology company that provides novel diagnostic tools to the life science community.

At ChemGenes, we redefine the limits of scientific discovery, development and commercialization by manufacturing the broadest range of modified chemistries available in today's market. We produce building blocks of life used by scientists and physicians in academic institutions, as well as pharmaceutical and biotechnology companies around the globe, to accelerate breakthroughs, spark innovation and develop lifesaving diagnostics and medicines. Established in 1981, ChemGenes is the world's most experienced DNA and RNA manufacturing company. As a trailblazer in nucleic acid research and synthesis for nearly half a century, ChemGenes has earned its position as a market leader in the field of nucleic acid chemistry. With our state-of-the-art manufacturing facility located just North of Boston, ChemGenes is customer centric, nimble and well positioned to scale alongside our partners. We've helped manufacturers introduce vital new medicines and – in an achievement with truly global implications – manufactured critical materials used in lifesaving COVID-19 vaccines and diagnostics. But beyond even these accomplishments, what sets us apart is our compassionate approach toward our customers, vendors and employees. We are a potent force for good, a close-knit and supportive team whose commitment to science, innovation and discovery is matched by – perhaps even exceeded by – our passion for helping people and changing lives for the better.

Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue. Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and a preclinical program for facioscapulohumeral muscular dystrophy (FSHD). For more information, please visit https://www.dyne-tx.com. To view our community guidelines, click here: https://bit.ly/3BYPnpK

Entrada Therapeutics is a biotechnology company dedicated totreatingdevastating diseases through the intracellular delivery of biologics

Harvard University is devoted to excellence in teaching, learning, and research, and to developing leaders in many disciplines who make a difference globally. Founded in 1636, Harvard is the oldest institution of higher learning in the United States. The official flagship Harvard social media channels are maintained by Harvard Public Affairs and Communications and aim to provide access to the people, places, events, news and research at our Institution. We ask that all visitors to Harvard's digital spaces be civil to one another and to the site editors. Personal attacks, profanity, commercial solicitations, spam, misinformation or other inappropriate contributions are grounds for comment removal. We ask that you stay on topic when contributing to a discussion and refrain from duplicate posts. Hateful or discriminatory comments regarding race, ethnicity, religion, gender, disability, sexual orientation, or political beliefs will not be tolerated. The page administrators reserve the right to delete inappropriate or abusive comments and to permanently ban or block users from the Harvard social media accounts.

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.

Korro Bio was founded to turn extraordinary scientific insights into life-altering new treatments for patients. We harness the body’s natural RNA editing machinery to make precise, single-base RNA edits to modulate protein function, addressing the medical needs of patients. Our mission is to rewrite the future for patients living with debilitating disease by repairing the underlying genetic cause.

Lytica Therapeutics is a Cambridge-based biotech company developing novel peptide conjugates for next-generation ADCs. The secret to unlocking more effective ADCs is in the...

PepGen Inc., a clinical-stage biotechnology company, focuses on the development of oligonucleotide therapeutics for use in the treatment of severe neuromuscular and neurologic diseases. Its lead product candidate is PGN-EDO51, an EDO peptide that id in Phase I clinical trial to treat individuals with Duchenne muscular dystrophy (DMD). The company is also developing PGN-EDODM1, an EDO peptide-conjugated PMO, which is in Phase 1 clinical trial for the treatment of myotonic dystrophy type 1, as well as EDO therapeutic candidates, such as PGN-EDO53, PGN-EDO45, and PGN-EDO44 for the treatment of DMD. PepGen Inc. was founded in 2018 and is headquartered in Boston, Massachusetts.

Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance. Praxis is applying insights into the genetic mutations that drive excitation-inhibition imbalance in diseases to select biological targets for rare and more prevalent neurological disorders. We share a common vision of reshaping the human condition into a more freeing and fulfilled existence by developing high impact medicines for patients and families affected by and living with complex brain disorders. Praxis is applying genetic insights to the discovery and development of therapies for rare and more prevalent neurological disorders through our proprietary small molecule platform, Cerebrum™, and antisense oligonucleotide (ASO) platform, Solidus™, using our understanding of shared biological targets and circuits in the brain.

Our Mission Stealth BioTherapeutics is an innovative biopharmaceutical company developing therapies to treat mitochondrial dysfunction associated with genetic mitochondrial diseases and common diseases of aging. Our team works with patients and advocacy organizations to better understand their journey and raise awareness of the unmet need our programs seek to address. We collaborate with top-tier academic and medical institutions, scientific thought leaders, and clinical key opinion leaders in developing the first generation of targeted therapies focusing on mitochondrial dysfunction as it presents in these rare genetic diseases and common diseases of aging. With these collaborative efforts, we continue to advance our platform of late-stage clinical programs and novel pipeline candidates. Please be aware of the potential for scams claiming to represent Stealth BioTherapeutics in recruitment activities. Stealth exclusively uses @stealthbt.com e-mail addresses for recruitment purposes and does not conduct interviews via social media or other third-party sites. Please report any suspicious recruiting activity to us at careers@stealthbt.com and to the Internet Crime Complaint Center.

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.

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.

Valerio Therapeutics is a clinical-stage biotechnology company based in France and the US that develops new drugs by targeting tumor DNA functions through unique mechanisms of action in the field of DNA Damage Response (DDR). DDR consists of a network of cellular pathways that detect, report and repair DNA damage. Applied to oncology, this new field of research aims to weaken or block the ability of tumor cells to repair damage to their DNA, either naturally or under the effect of cytotoxic treatments. Focusing exclusively on intracellular DNA-binding targets, we design and develop first-in-class treatments for oncology and inflammatory diseases precision therapeutics from preclinical (translational) research to human clinical proof-of-concept studies. It thus leads its programs to the most value-creating and attractive inflection points for potential partners. The Company's portfolio is based on platON™, Valerio Therapeutic's decoy oligonucleotide platform. PlatON™ is intended to generate new compounds based on an unparalleled DDR decoy mechanism and capitalizing on the expertise the Company has developed on this type of oligonucleotides. The Company's portfolio includes: - AsiDNA™, a first-in-class product interfering with tumor DNA break repair, based on a decoy agonist mechanism, unmatched in the DDR field, which could, among other things, combat tumor resistance. AsiDNA™ is in clinical development in several trials, in combination with PARP inhibitors or in combination with radiation therapy. - A new family of compounds are positioned as a new-generation PARP agonists that are designed not to induce resistance and to activate the immune response. VIO-01, is currently being optimized in a preclinical phase. The Company is convinced of the significant therapeutic potential of its technology and the disruptive innovation it represents, which could pave the way for a new paradigm in cancer and inflammatory disease treatments.

We are leading a new era of precision medicine in which rationally designed oligonucleotides are the key to delivering safer, more effective medicines. These include potentially transformational therapies for conditions where there are currently few or no treatment options. We aspire to help create a world in which every patient with a genetic disease will have access to life-changing treatments in their lifetime.

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.