List of Oligonucleotides Companies in United States - 74

ACADIA Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of small molecule drugs that address unmet medical needs in central nervous system disorders. The company offers NUPLAZID (pimavanserin) for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis; Trofinetide, a novel synthetic analog for the treatment of Rett syndrome; ACP-044, a novel first-in-class orally administered non-opioid analgesic for treating acute and chronic pain; and ACP-319, a positive allosteric modulator of the muscarinic receptor for treating cognition and schizophrenia. It is also developing pimavanserin as a treatment for dementia-related psychosis and as an adjunctive treatment for schizophrenia; and pimavanserin as an adjunctive treatment for major depressive disorder. ACADIA Pharmaceuticals Inc. was founded in 1993 and is headquartered in San Diego, California.

ADARx Pharmaceuticals is a newly launched next-wave genetic medicine company focusing on oligonucleotide therapeutics for inhibition, degradation and base editing of mRNA transcripts. With RNA as the target of our drug discovery, we are capable of working on a wide range of diseases and are currently focusing on genetic, cardiometabolic, and central nervous system (CNS) diseases. We are driven by our mission to turn cutting-edge science into life-saving therapeutics and our desire to provide hope for patients with intractable disease.

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+.

Ajinomoto is a global leader in the research, development, manufacture and sale of amino acid-based products for the pharmaceutical, nutraceutical, sports nutrition, health and beauty industries as well as food ingredients and consumer food products.

AptamiR Therapeutics has created an innovative model of virtual and outsourced Drug Research & Development to cure human obesity by developing microRNA-based treatments that target peripheral adipose tissues (fat cells).

ArcherDX is developing and seeking regulatory clearances for next-generation sequencing (NGS) diagnostics to help solve for the underutilization of targeted cancer therapies.

Harnessing the power of RNA biology to find treatments for patients with unmet medical needs. Advance and utilize the cutting edge antisense technology (siRNA and ASO) to discover and develop next generation medicines for both large and rare disease indications.

Based in Philadelphia, Aro Biotherapeutics is a biotechnology company pioneering the development of tissue-targeted genetic medicines with a platform based on a proprietary protein technology called Centyrins. The company is developing a wholly-owned pipeline of Centyrin-based therapeutic candidates and is working with industry partners to leverage Centyrins for tissue-specific targeting of therapeutics for a diverse set of diseases.

Aronora is a translational biotechnology company (aronorabio.com) engaged in the development of proprietary biologic therapeutics, including recombinant monolconal antibodies and enzymes. Rational design of our innovative therapeutic agents is expected to result in drug products that reduce the progression and growth of life-threatening blood clots without the detrimental bleeding side effects that characterize all currently marketed antithrombotic drugs. Our products are intended to be used in difficult-to-treat, severe, rapidly progressing, or catastrophic thrombotic blood clotting diseases.

AUM LifeTech is an American Biotechnology company focused on using next-generation RNA silencing technology to develop solutions in diverse life science verticals including biomedical research, medicine, agriculture, and aquaculture. Specifically, AUM's custom products include next generation of innovative genetic tools in the area of gene silencing and manipulation for biomedical research and therapeutic development. AUM’s non-GMO products are also being developed for broad-spectrum target-specific pest management and disease control in agriculture. RNA silencing products using FANA technology provide superior alternatives to conventional RNAi and gene editing approaches like CRISPR and provide a fast track target discovery. AUM's goal is to provide innovation at the genetic level for a better life.

Avidity Biosciences, Inc., a biopharmaceutical company, engages in the development of oligonucleotide-based therapies. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat a range of serious diseases. The company’s lead product candidate AOC 1001 is used for the treatment of myotonic dystrophy type 1, a rare monogenic muscle disease; and to design, engineer, and develop therapeutics that combine tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies to access previously undruggable tissue and cell types, and target underlying genetic drivers of diseases. In addition, it develops muscle programs, which focuses on the treatment of facioscapulohumeral muscular dystrophy, Duchenne muscular dystrophy, muscle atrophy, and Pompe diseases.

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.

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.

Since its founding in 1992 Bioneer Corporation has established market leadership in Korea for most of the products it manufactures. Bioneer has built its business by providing researchers with superior, high quality products at excellent value. Over the years, Bioneer has developed a broad range of unique products that provide total genomic research solutions for life sciences research including genomics, nucleic acid-based molecular diagnostics and gene therapy studies. Bioneer has extensive R&D experience in developing new drugs as well as the use of siRNA and drug delivery systems, and provides unrivalled service and support for such tools. For example, through the creation, development, and commercialization of the S. pombe deletion mutant library, Bioneer provides an innovative tool for improving the development of new drugs. The S. pombe library accurately predicts the toxicity of drug candidates for anti-cancer, Hyperlipidemia and also provides molecular level mechanism analysis of natural compound drugs. The company is headquartered in South Korea with an oligonucleotide manufacturing facility in the U.S., a regional office in China, and is supported by a network of distributors throughout the world. For more information, please visit www.bioneer.com.

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

BOC Sciences provides a wide variety of custom services which range from bulk compounds to specialty species in the pharmaceutical, agrochemical, and biotechnology industries. We are committed to providing our customers with the best products and services at the most competitive prices. Given diverse packing options for most of our chemicals, you are welcome to order the desired compounds in any quantities from a few mg to several kg along with all the relevant technical reports. We sincerely hope that our unremitting pursuits of excellence, success, and professionalism will add immense value to your research and development projects!

Bolden Therapeutics is a biotechnology company developing first-in-class therapeutics to treat central nervous system diseases such as Alzheimer’s and ischemic stroke. The company’s scientific founders have identified a key molecular pathway to stimulate neural stem cells to promote the birth of new neurons (neurogenesis) in the adult brain. In a proprietary mouse model Bolden has established a proof-of-concept of this approach to increase neurogenesis and enhance hippocampal-dependent memory. The company is actively developing antisense oligonucleotide and monoclonal antibody therapeutics to modulate this pathway.

Capricor Therapeutics, Inc., a biotechnology company, focuses on the development of transformative cell- and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases. The company’s lead candidate, CAP-1002, an allogeneic cardiac-derived cell therapy, which has completed phase II clinical trial for the treatment of patients with late-stage Duchenne muscular dystrophy (DMD); CAP-1002, which is in Phase II clinical trial for the treatment of cytokine storm associated with COVID-19; and completed various trials investigating the use of CAP-1002 for the treatment of cardiac conditions, including heart failure and post myocardial infarction with cardiac dysfunction. It is also developing CAP-2003 that is in pre-clinical development for the treatment of trauma related injuries and conditions; and two vaccine candidates, which are in preclinical stage for the potential prevention of COVID-19. Capricor collaborates with Lonza Houston, Inc. for the development of CAP-1002, its cell therapy candidate for the treatment of DMD and other indications. The company was founded in 2005 and is headquartered in Beverly Hills, California.

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.

CiVi Biopharma Inc. is a US-based, privately-held biopharmaceutical company, founded in 2016. The Company's research and development activities are focused on creating novel therapies for cardiovascular, metabolic and related diseases. CiVi’s innovative pipeline includes CiVi007, a long-acting PCSK9 third-generation Locked Nucleic Acid antisense molecule being developed for the treatment of hypercholesterolemia and the prevention of cardiovascular disease. In addition, the Company currently has other clinical stage development program activity aimed at treating severe liver diseases.

Codexis is a leading enzyme engineering company leveraging its proprietary CodeEvolver® platform to discover, develop and enhance novel, high-performance enzymes and other classes of proteins. Codexis enzymes solve for real-world challenges associated with small molecule pharmaceuticals manufacturing and nucleic acid synthesis. The Company is currently developing its proprietary ECO Synthesis platform to enable the scaled manufacture of RNAi therapeutics through an enzymatic route. Codexis' unique enzymes can drive improvements such as higher yields, reduced energy usage and waste generation, improved efficiency in manufacturing and greater sensitivity in genomic and diagnostic applications.

Couragene believes that delivery of gene and biologic therapies should be safer and more efficient. Delivery of genome modification therapies to brain is further complicated by the physiological barriers. Couragene's STEP (Stimuli-responsive Traceless Engineering Platform) technology has demonstrated great potential to overcome the delivery hurdles for treatment of various diseases, including neurogenetic disorders.

Founded in 2001, we are the third largest peptide-focused contract research, development, and manufacturing organization (“CRDMO”) worldwide in terms of sales revenue in 2023, according to Frost & Sullivan. We are also considered one of the most comprehensive peptide-focused CRDMO globally (Frost & Sullivan), offering complete life-cycle support from early-discovery, pre-clinical research and clinical development to commercial production. We mainly provide (i) contract research organization (“CRO”) services, including peptide new chemical entity (“NCE”) discovery synthesis; (ii) contract development and manufacturing organization (“CDMO”) services, including peptide chemistry, manufacturing, and controls (“CMC”) development; and (iii) contract manufacturing organization (“CMO”) services, including peptide NCE and generic drug commercial manufacturing. We have established global operations, with projects covering over 50 countries, including major markets in the United States, China, Japan, Europe, South Korea, and Australia. We also provide customers wit peptide drug development, production, and CMC filing support services that meet regulatory requirements in major markets worldwide. In addition to peptides, we have developed a diverse project pipeline focusing on various types of complex peptide- and oligonucleotide-based new chemical entities (“NCEs”). Our peptide conjugation services seamlessly integrate our peptide and oligonucleotide platforms to produce a wide range peptide conjugates products. The introduction of our PeptiConjuXTM and PeptiNuclide LinkTechTM are dedicated to the development and large-scale manufacturing (i.e., kilogram-scale) of peptide-oligonucleotide conjugates (“POC”), peptide drug conjugates (“PDC”), and radionuclide drug conjugates (“RDC”). As of December 31, 2023, we had successfully synthesized approximately 1,800 molecules through these two platforms.

Reinventing drug development from the ground up and making on-demand oligonucleotide therapeutics possible.

The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for people with cystic fibrosis, a rare, genetic disease in which a defective gene causes a thick buildup of mucus in the lungs, pancreas, and other organs. The buildup of mucus can lead to extensive lung damage, respiratory failure, malnutrition, liver disease, and gastrointestinal issues, among many other complications. Recognized globally, the Cystic Fibrosis Foundation has led the way in the fight against cystic fibrosis, fueling extraordinary medical and scientific progress. Working alongside the CF community, the CF Foundation has fostered the development of more than a dozen CF treatments — an unprecedented number in a short span of time — and helped add decades of life for people with CF. Thanks to this work, the life expectancy of someone born with CF has doubled in the last 30 years. We are driven by a dream that one day every person with cystic fibrosis will have the chance to live a long, healthy life. Our vision is a cure for every person with cystic fibrosis and a life free from the burden of this disease. While our headquarters are in Bethesda, Maryland, we have additional offices in more than 60 locations across the country with positions in fundraising, marketing, digital, information technology, legal, finance and more – all supporting our mission to cure cystic fibrosis. At the Foundation, we are committed to creating an environment that is free from discrimination and provides a rewarding experience for all members of our team. We strive to be an organization where everyone is welcomed and where talented individuals from all backgrounds have the opportunity to thrive. The CF Foundation is a nonprofit, donor-supported organization and an accredited charity of the Better Business Bureau's Wise Giving Alliance. Please visit us at www.cff.org.

DNA Software is the preferred partner to solve your diagnostic design and analysis problems.Our customized software platform predicts the “sweet spots” in the target and designs with optimum sensitivity and specificity. DNA Software provides diagnostic solutions to our partners saving time, money and resources, including difficult multiplex panel design for the most challenging targets.

Duet BioTherapeutics is an immunotherapy company creating a new category of bispecific oligonucleotide treatments designed to target dormant immune cells within the tumor microenvironment. Activation of these antigen-presenting cells removes the immunosuppressive shield around the tumor and jump-starts T cell-mediated immune responses to kill cancer cells.

Dynavax is a fully-integrated biopharmaceutical company focused on leveraging the power of the body’s innate and adaptive immune responses through Toll-like Receptor (TLR) stimulation. Dynavax develops, and commercializes novel vaccines.

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

Eurofins Genomics LLC is a member of the Eurofins Group with major offices and production facilities in the United States, Europe, and Asia, is an international provider of DNA sequencing services, DNA synthesis products, and bioinformatic services for academic and industrial research. The company’s strengths are its strong customer orientation, fast service and high quality products including a series of advanced oligonucleotide design tools.

Everlum Bio is pioneering a novel approach to drug development for rare diseases. As opposed to working on a single disease we are doing "Drug Development as a Service". We are relentlessly focused on innovating the drug development process. Ultimately we believe this approach will usher in a personalized/precision medicine revolution.

Firebird Biomolecular Sciences, LLC. operates as a supplier of nucleic acid components, libraries, polymerases, and software to support human diagnostics, systhetic biology, and human therapy. The Company also offers naturally organized genomic databases, customized databases, and interpretive genomics services.

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.

Integrated DNA Technologies, Inc. (IDT) develops, manufactures, and markets nucleic acid products for the life sciences industry in the areas of academic and commercial research, agriculture, medical diagnostics, and pharmaceutical development. IDT has developed proprietary technologies for genomics applications such as next generation sequencing, CRISPR genome editing, synthetic biology, digital PCR, and RNA interference. Through its GMP services, IDT manufactures products used by scientists researching many forms of cancer and most inherited and infectious diseases. IDT is widely recognized as the industry leader in custom nucleic acid manufacture, serving over 130,000 life sciences researchers. IDT was founded in 1987 and has its manufacturing headquarters in Coralville, Iowa, USA, with additional manufacturing sites in San Diego, California, USA; Research Triangle Park, North Carolina, USA; Ann Arbor, Michigan, USA; Leuven, Belgium; and Singapore. For more information, please visit www.idtdna.com and follow on Twitter, LinkedIn, Facebook, YouTube, and Instagram.

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It is involved in developing neurology products that include Tominersen for Huntington’s diseases; Tofersen for amyotrophic lateral sclerosis (ALS); IONIS-MAPTRx for Alzheimer's diseases; IONIS-C9Rx for genetic form of ALS; ION859 for Parkinson’s disease; ION464 for multiple system atrophy and Parkinson’s disease; ION541 for amyotrophic lateral sclerosis; and IONIS-DNM2-2.5Rx for centronuclear myopathy. The company is also developing products for cardiometabolic disease, such as IONIS-TTR-LRx for transthyretin amyloidosis and IONIS-GHR-LRx for acromegaly; IONIS-APOCIII-LRx for familial chylomicronemia syndrome and TG- driven diseases; IONIS-APOCIII-LRx for treatment resistant hypertension; Pelacarsen for Lp(a)cardiovascular disease risk reduction; Vupanorsen for sHTG/CVDRR; IONIS-FXI-LRx for clotting disorders; ION449 for cardiovascular diseases; and IONIS-GCGRRx for diabetics. In addition, it develops IONIS-ENAC-2.5Rx for cystic fibrosis and chronic obstructive pulmonary disease; IONIS-PKK-LRx for hereditary angioedema; Danvatirsen for cancer; IONIS-TMPRSS6-LRx for beta-thalassemia and iron toxicity; IONIS-HBVRx for hepatitis B virus; IONIS-AR-2.5Rx for prostate cancer; IONIS-FB-LRx for age-related macular degeneration and IgA nephropathy; and ION357 for retinitis pigmentosa. It has a collaboration agreement with AstraZeneca; GlaxoSmithKline plc; Janssen Biotech, Inc.; Bayer AG; Novartis AG; Pfizer, Inc.; Biogen Inc.; and Roche. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.

Nitto Avecia Pharma Services is your single source for high-quality chemistry, manufacturing, and control (CMC) services for the pharma, biotech, and medical device industries. Our comprehensive suite of services streamlines processes to accelerate time-to-market. With more than 30 years’ experience, we have deep scientific expertise across a range of products, with a special focus on oligonucleotides. In partnership with Nitto Avecia Pharma Services, Nitto Avecia is the first CMO that offers a comprehensive, end-to-end oligonucleotide solution. This offers the advantage of a single, expert partner handling the entire process—from drug substance through drug product manufacturing—with flexible solutions and consistent reliability and quality.

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.

Polymeric Supports for Nucleotide Synthesis

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.

Leal Therapeutics is a biopharmaceutical company that focuses on developing novel therapeutics for patients with major disorders of the central nervous system.

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...

The comapanies' research and development efforts are focused exclusively on rare genetic neurodevelopmental disorders, a group of serious diseases with few or no treatment options.

Matinas BioPharma (NYSE AMER: MTNB) is a biopharmaceutical company focused on improving the intracellular delivery of nucleic acids and small molecules with its lipid nanocrystal (LNC) platform technology. The Company is developing its own internal portfolio of products as well as partnering with leading pharmaceutical companies to develop novel formulations that capitalize on the unique characteristics of the LNC platform. Preclinical and clinical data have demonstrated that this novel technology can provide solutions to many of the challenges in achieving safe and effective intracellular delivery, for both small molecules and larger, more complex molecules, such as mRNA, DNA plasmids, antisense oligonucleotides, and vaccines. The combination of a unique mechanism of action and flexibility with formulation and route of administration (including oral), positions Matinas’ LNC technology to potentially become the preferred next-generation intracellular drug delivery vehicle with distinct advantages over both lipid nanoparticles and viral vectors.

Maze Therapeutics is translating novel genetic insights into lifesaving medicines, through an approach that integrates human genetics and functional genomics. Advances in these areas have enabled us to decode the mysteries of genetic modifiers in a range of severe diseases, which we aim to turn into medicines that mimic protective mutations. We have assembled a world-class team that we need to realize this vision, including creative scientific pioneers in genetics, experienced company builders, and biotech industry leaders.

Molecular Assemblies, Inc. is a private life sciences company developing an enzymatic DNA synthesis technology designed to power the next generation of DNA-based products. The company's patented Fully Enzymatic Synthesis (or FES™), based on making DNA the way nature makes DNA, produces long, high quality, sequence-specific DNA reliably, affordably, and sustainably. FES technology will enable the reading and writing of DNA for many industries, including industrial synthetic biology and precision medicine, as well as emerging applications of nanomachines and bio-based electronics. Molecular Assemblies is headquartered in San Diego. *Fully Enzymatic Synthesis and FES are the trademarks of Molecular Assemblies, Inc.

Moonwalk Biosciences is an early-stage therapeutics company co-founded by Dr. Feng Zhang and leading experts in cell state epigenetics including Alex Aravanis, the former CTO of Illumina. The company has developed a differentiated approach to mapping the epigenome of cellular states at the whole genome, single cell resolution level, as well as proprietary strategies for precise epigenetic engineering. The combination enables both a novel discovery platform and a path to developing therapeutics that include both epigenetic engineering and other traditional modalities.

A pioneer in immuno-modulation by cell specific delivery of oligonucleotide drugs to antigen presenting cells completing IND enabling studies for a groundbreaking anti-GVHD drug for hematopoietic stem cell transplantation in hematological cancers.

NinthBio is a software company serving Biotech. Our flagship product is "Homology Path", an application that designs the oligos for DNA synthesis. The value is how few oligos are needed to build a a group of similar DNA sequences. Think of the DNA you would need to test for a protein engineering campaign, or to optimize the affinity of an antibody. All require similar but ultimately unique DNA. Our customers can improve their Design, Build, Test Learn Cycle by 15X all while decreasing their DNA spend 10 fold. Researchers are rethinking where and what they are willing to explore given the power of Homology Path, opening the door for rapid and higher value discovery. Want to see how this can help your initiatives; request a demo token to access the capabilities of Homology Path.

Oligonucleotides are giving rise to a new generation of therapeutics—and Nitto Avecia is helping lead the way. With more than 25 years' experience in oligonucleotide development and production, we are the partner of choice for pharmaceutical innovators creating new drugs with the power to transform treatment for a wide range of diseases. In partnership with Nitto Avecia Pharma Services, Nitto Avecia is the first CMO that offers a comprehensive, end-to-end oligonucleotide solution. This offers the advantage of a single, expert partner handling the entire process—from drug substance through drug product manufacturing—with flexible solutions and consistent reliability and quality.

Nitto BioPharma is a clinical stage biopharmaceutical company located in San Diego focused on the discovery and development of novel RNA-based therapeutics for the treatment of fibrosis and other unmet medical needs. The company offers unique drug and siRNA delivery solutions by designing novel biodegradable delivery vehicles with the combined ability to target specific cells.

Northern Biomedical Research is a non-clinical research organization focused on targeted delivery of therapeutics using advanced techniques. Our team is experienced in providing industry-leading research for targeted-delivery of drugs, biologics, gene/cell therapies, and medical devices. With a strong regulatory history and scientific background in neuroscience, cardiovascular research, ophthalmology, and orthopedics, our team works closely with Sponsors to design a non-clinical program to exedite the regulatory process. For more information, stop by Booth 250. Â

Oncogenuity is a biopharmaceutical company committed to developing and commercializing new treatments for genetically driven diseases. Our lead asset targets the KRAS mutation G12D, which plays a significant role in pancreatic and colorectal cancers, as well as other “untreatable” cancers of considerable unmet need. Our core technology focuses on the delivery of PNA’s (peptide nucleic acids). The PNA acts as a targeting sequence for gene-silencing. These targeting sequences are interchangeable, and we plan to aggressively pursue both oncology and non-oncology genetic mutations where gene silencing can be effective. We are a partner of Fortress Biotech, and we have an exclusive licensing agreement with Columbia University to develop oligomers (ONCOlogues) that attack genetically driven diseases at the DNA level.

Optimeos Life Sciences is pioneering a novel targeted gene delivery approach to advance medical treatment and patient care.

ParcelBio is developing a programmable technology to safely and specifically deliver mRNA to diverse cell types in the body. Our proprietary (nanoparticle-free) technology, which we call ‘STAmP’, stabilizes the mRNA and utilizes an interchangeable, lock-and-key-like targeting mechanism which enables fully customizable mRNA medicines.

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.

Regulus Therapeutics Inc. (Nasdaq:RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkersSM biomarkers platform and a rich intellectual property estate to retain its leadership in the microRNA field. Regulus is located in San Diego, California and is led by a seasoned executive team experienced in corporate management, business, science, drug discovery and development. Regulus'​ scientific advisory board consists of world-class scientists and some of the foremost authorities in the field of microRNA research.

Rockland Antibodies is a biotechnology company that offers a wide range of antibodies for academic, biopharma, and diagnostic professionals, catering to various research and development needs.

Scopus BioPharma is a biopharmaceutical company developing transformational therapeutics targeting serious diseases with significant unmet medical needs.

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.

Synbio Technologies aims to become the most trusted provider of DNA solutions that empower scientific discoveries by providing highly-accurate and cost-effective synthetic biology services and products for researchers around the world. We have developed a full range of DNA reading (sequencing), DNA writing (synthesis), and DNA editing (engineering) capabilities for various applications, including diagnostic DNA probes, precision medicine, protein production, antibody discovery, vaccine development, novel enzymes, molecular breeding, biofuel implication, and more. Our scientific capabilities cover all facets of DNA synthesis and engineering, offering a full range of support for molecular biology work, including but not limited to, oligo synthesis, gene synthesis, subcloning & PCR cloning, plasmid preparation, mutagenesis, DNA variant libraries, CRISPR sgRNA, protein expression and purification in multiple systems. Relying on our strong DNA sequencing, synthesis, engineering technology platform, and expertise in bioinformatics analysis, we have developed proprietary algorithms to facilitate and expedite the antibody discovery process. Our antibody discovery services include hybridoma sequencing, immune repertoire sequencing, antibody design and production, AI-guided heavy chain and light chain pairing, antibody humanization, affinity maturation, and sdAb.

Tallac Therapeutics is a privately held biopharmaceutical company harnessing the power of innate and adaptive immunity to fight cancer. Tallac’s pipeline of immunotherapy candidates are derived from the company’s novel Toll-like Receptor Agonist Antibody Conjugate (TRAAC) platform to deliver a potent Toll-like receptor (TLR9) agonist (T-CpG) for targeted immune activation via systemic administration. Several TRAAC molecules are in various stages of discovery and preclinical development.

Telesis Bio is empowering researchers to accelerate the creation of novel synthetic biology solutions to address some of humanity's greatest challenges. With our breakthrough automation solutions for biological synthesis and expertise in DNA biology, we enable rapid, accurate, and reproducible writing of DNA and mRNA for wide-ranging biological applications. Around the world, innovators are leveraging Telesis Bio's technology to overcome current bottlenecks in synthetic biology workflows and advance the discovery of cutting-edge solutions for health and technology. We are proud of our growing collaborations with partners that include premier academic research institutions, emerging start-ups to nearly all of the Top 25 Biopharma companies. Telesis Bio is honored to play a part in several pioneering advances of our customers and collaborators ranging from novel infectious disease vaccines, precision immunotherapy for cancer and antibody therapeutics to creation of engineered meat substitutes and sustainable cellular agricultural products.

Thera Neuropharmainvestigates the use of small molecules and RNA-based compounds as therapeutics to treat Amyotrophic Lateral Sclerosis (ALS)andTraumatic Brain Injury (TBI).

Launched in 2024, Trace Neuroscience is a biopharmaceutical company on a mission to expand the promise of genomic medicine for people living with neurodegenerative diseases. With an initial focus on ALS, the company is developing novel therapies to restore UNC13A protein function to re-establish healthy communication between nerves and muscle cells.

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.