132 Oligonucleotides Companies - Worldwide

San Diego, California, United States of America

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.

Cranbury, New Jersey, US

San Diego, California, United States of America

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.

Wuppertal, Germany

AiCuris (from 'Anti-infective Cures'​) is a pharmaceutical company focused on the discovery, research and development of novel antiviral agents for the treatment of severe and potentially life-threatening infectious diseases in immunocompromised patients. Founded in 2006 from Bayer's virology and bacteriology research divisions, AiCuris has a broad and innovative pipeline of novel anti-infectives and a team of internationally regarded scientists and drug developers. With its focus on specialist markets with high medical need, the company is perfectly positioned for growth and success.

Cambridge, Massachusetts, United States

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+.

Itasca, Illinois, United States

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.

Xiamen, China

Amoy Diagnostics (AmoyDx) is a visionary diagnostic company dedicated to transforming the landscape of cancer care. 'Breaking Barriers to Precision Oncology' reflects our commitment to revolutionizing cancer diagnosis and treatment. We are driven by four fundamental values: Innovation in Care, Accessibility and Equity, Collaboration and Partnership, and Integrity and Trust. These values guide our pursuit of developing groundbreaking diagnostic tools and fostering a more equitable, collaborative, and trustworthy approach to oncology. Join us as we pave the way to a future where precision cancer care is a reality for every patient, everywhere.

Tokyo, Japan

AnGes was founded In December 1999, with the aim of developing hepatocyte growth factor (HGF) gene therapy drugs which can help regenerate blood vessels. Originating from the word angiogenesis meaning the formation of new blood vessels in English, our corporate name AnGes encapsulates our desire to be an angel (ange in French) offering new drugs for patients with hard-to-treat and rare diseases. Both our founder and our employees are still motivated by this desire.

Milan, Italy

Reprogramming DNA methylation in disease conditions by RNA therapeutics APTADIR Contact us NEWS Press Release Reprogramming DNA methylation by RNA therapeutics A novel therapeutic approach for untreatable cancers and rare diseases Aberrant methylation by DNA methyltransferase 1 (DNMT1) of certain genes may cause abnormal cell development and lead to disease. Current therapies targeted to inhibit

Gyoungbuk, South Korea

Aptamer Sciences Inc. is a privately held biotech company focused on commercializing cutting-edge technologies for analysis of proteins, based on its proprietary aptamer technology platforms. Aptamer Sciences is dedicated to develop the aptamer technologies to generate high quality aptamer and in broad applications of research, diagnosis and therapeutics. Compared to conventional aptamer generating technologies, Aptamer Sciences has developed advanced aptamer technologies and thus can generate aptamers more efficiently with superior binding affinity. Aptamer Sciences has ongoing collaboration relationships with several industry partners. The Company also has research collaboration with several universities, hospitals and research organizations. Aptamer Sciences was founded in 2011 by researchers from POSTECH BIOTECH CENTER and is based in Pohang, South Korea. Aptamer Sciences is currently funded by aptamer service revenue and grants, as well as by investments from strategic investors.

Austin, Texas, United States

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).

2477 55th Street, Suite 202, Boulder, Colorado 80301, US

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

10355 Science Center Drive, Suite 130

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.

Philadelphia, Pennsylvania, United States

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.

Portland, Oregon, United States

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.

Aarhus, Denmark

Eurofins Genomics was founded in 1990 and is part of the Eurofins Scientific Group, a Life Science Company with laboratories in 61 countries and 61,000 employees. We are an international provider of genomic services around the core business lines next generation sequencing, genotyping, gene expression, custom DNA sequencing, oligonucleotides, siRNA and gene synthesis. Our services are tailored for pharma, diagnostics, agriculture, food, biotechnology and research applications worldwide. Imprint: https://www.eurofinsgenomics.eu/imprint/ Privacy Note: https://www.eurofinsgenomics.eu/privacy/

Paterna, Valencia, Spain

ARTHEx Biotech is a spin-off biotech company from the University of Valencia (Spain) focused on the research and development of novel oligonucleotide therapies that modulate microRNA to treat diseases with unmet medical needs. ARTHEx has developed its proprietary technology, the ENTRY ™ platform, that allows fast and flexible development of investigational miR-modulating oligonucleotides that are designed to enter relevant tissues in pharmacological quantities, in order to treat diseases in which microRNAs play a key role. The first investigational product from this platform is ATX-01, an antimiR in preclinical development for Myotonic Dystrophy Type 1 (DM1). DM1 is an orphan disease affecting more than 900,000 people worldwide (estimated prevalence 1/8,000) with no cure, nor approved treatment to date. ATX-01 is a first-in-class disease-modifying therapy that addresses the cause of DM1 with a dual and unique mechanism of action, which targets micro-RNA-23b a key factor in the disease pathogenesis. To learn more about Arthex Biotech, please visit us at www.arthexbiotech.com

Philadelphia, Pennsylvania, United States

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.

Hangzhou, China

AusperBio is a clinical-stage biopharmaceutical company with operations in the USA and China, dedicated to the development of innovative therapeutics for curing chronic hepatitis B. The company has developed a proprietary Med-Oligo™ ASO technology platform, substantially enhancing the potency of targeted therapies, not only for liver diseases, but also with the potential for expansion beyond the liver. AusperBio's strategy is to combine its leading oligonucleotide therapies with other medications including therapeutic antibodies and mRNA vaccines to address a broad range of unmet medical needs.

San Diego, California, United States

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.

Fujisawa, Japan

Axcelead Drug Discovery Partners, Inc. will combine scientific excellence, world-class IP protection and state-of-the art GLP accredited facilities and capabilities to deliver integrated end-to-end early discovery.

Kulmbach, Germany

Axolabs specialises in a new class of drugs whose active ingredients are chemically derived from DNA or RNA. They are called oligonucleotide or nucleic acid therapeutics. We use our many years of expertise to help our clients make them a success. Some of these drugs have already been approved and are being used successfully against diseases that were previously untreatable in some cases. New drugs from this class are added every year. Axolabs is part of the LGC Group. Its business unit for nucleic acid therapeutics is combined under the brand name Axolabs. Axolabs operates companies in the following locations: Axolabs, Kulmbach, Germany Axolab Kulmbach GmbH, is the world's leading custom research organization in the field of nucleic acid therapeutics with a wide range of services. Axolabs Berlin, Berlin, Germany Axolabs Berlin is currently establishing a new site in Berlin for GMP manufacturing of nucleic acid therapeutics. The site will be dedicated to medium to large scale and early to late phase clinical GMP manufacturing of nucleic acid therapeutics. The state-of-the-art facility will deliver integrated analytical and manufacturing solutions. The operational start date is scheduled for 2024. Axolabs Petaluma, Petaluma, California, USA Axolabs operates a cGMP production facility in Petaluma for oligonucleotide compounds for technical, toxicological and preclinical to late-stage clinical trials. Services: bioinformatics, oligonucleotide synthesis, analytics and in vitro / in vivo analysis.

Bubendorf, Switzerland

Bachem is a pharmaceuticals company that specializes in research chemicals, peptide synthesis, and other life sciences products.

Munich, Germany

At baseclick GmbH we believe that bioconjugation is not just a method to connect (bio)molecules, but a tool to develop valuable applications. So, a new strategy was needed: click chemistry! " Nobel Prize 2022, the basis of the baseclick patented nucleic acid modification technology". We are a dynamic, highly professional team and enabling life science researchers to overcome challenges and create better solutions in diagnostics, therapeutics, and vaccines development through our proprietary click chemistry. Our mission is to develop and provide the best nucleic acid labeling applications and reagents for the life science market. For further information please visit our website https://www.baseclick.eu/ https://www.baseclick.eu/our-vaccine-strategy-and-its-benefits/

Cambridge, Massachusetts, United States of America

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.

Novato, California, United States of America

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.

Oakland, California, United States

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.

Worcester, Massachusetts, United States

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

Römerberg, Germany

BIORON GmbH is a producer and worldwide acting trader of molecular biological and diagnostic products. Our main focus are ready-to-use solutions for professionals, i.e. high concentrated polymerases for lyophilisation.

Frankfurt, Germany

Shirley, New York, United States

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!

Providence, Rhode Island, United States

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.

Beverly Hills, California, United States of America

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.

Hanover, Germany

Cardior Pharmaceuticals is a leading clinical-stage biopharmaceutical company pioneering the discovery and development of RNA-based therapeutics designed to prevent, repair and reverse diseases of the heart. Cardior’s therapeutic approach uses distinctive non-coding RNAs as an innovative platform for addressing the root causes of cardiac dysfunctions. The company aspires to bring transformative therapeutics and diagnostics to patients and thereby make a lasting impact on the treatment of cardiac diseases worldwide.

Wilmington, Massachusetts, United States

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.

Chevy Chase, Maryland, United States

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.

Redwood City, California, United States of America

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.

Chenôve, France

CordenPharma is your full-service partner in the Contract Development & Manufacturing (CDMO) of APIs, Lipid Excipients, Drug Products and Packaging. Through a network of cGMP facilities across Europe and the US organized under six technology platforms – Peptides, Lipids & Carbohydrates, Oligonucleotides, Injectables, Highly Potent & Oncology, Small Molecules - CordenPharma experts translate complex ideas, processes and projects at any stage of development into high-value pharmaceutical products. Visit to learn more > cordenpharma.com

Plankstadt, Germany

CordenPharma is your full-service partner in the Contract Development & Manufacturing (CDMO) of APIs, Lipid Excipients, Drug Products and Packaging. Through a network of cGMP facilities across Europe and the US organized under six technology platforms – Peptides, Lipids & Carbohydrates, Oligonucleotides, Injectables, Highly Potent & Oncology, Small Molecules - CordenPharma experts translate complex ideas, processes and projects at any stage of development into high-value pharmaceutical products. Visit to learn more > cordenpharma.com

675 US Highway 1

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.

Sunnyvale, California, United States

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.

San Diego, California, United States

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

Bethesda, Maryland, US

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.

Toronto, Canada

Deep Genomics is using artificial intelligence to build a new universe of life-saving genetic therapies. The future of medicine will rely on artificial intelligence, because biology is too complex for humans to understand. At Deep Genomics, our geneticists, molecular biologists and chemists develop new ways of detecting and treating disease using our biologically accurate artificial intelligence technology.

Ann Arbor, Michigan, United States

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.

Los Angeles, California, United States

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.

Emeryville, California, United States of America

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.

Waltham, Massachusetts, United States of America

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

Cambridge, United Kingdom

Eleven Therapeutics is a biotechnology company leading the AI revolution in nucleic acid therapeutics. They harness the power of combinatorial chemistry and AI to transform RNAi drug development into a programmable process.

Boston, Massachusetts, United States

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

Louisville, Kentucky, United States

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.

Austin, Texas, United States

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.

Alachua, Florida, United States

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.

Leuven, Vlaams-Brabant, Belgium

Flamingo is pioneering RNA-targeted therapies for oncology with state-of-the art chemistries and a clinical-stage pipeline targeting undruggable transcription factors and splice variants. Flamingo Therapeutics has a Phase II trial (PEMDA-HN) evaluating the STAT3 targeting agent danvatirsen, in combination with pembrolizumab, in patients with head and neck squamous cell carcinoma (HNSCC). Flamingo has an alliance with Ionis Pharmaceuticals and is supported by well-known biotechnology investors including Andera Partners, Bpifrance Large Venture, Bpifrance through its FABS and Fonds Biothérapies Innovantes et Maladies Rares funds, Eurazeo - Kurma Partners, Perceptive Advisors, PMV, Pontifax, Sphera, funds managed by Tekla Capital Management LLC and VIB.

Philomath, Oregon, US

TW

Genomics Bioscience and Technology Co., Ltd. is Taiwan's leading commercial genome sequencing company. Our diverse services encompass comprehensive gene sequencing technology and applications and oligonucleotide and peptide synthesis, meeting standards from RUO to GMP compliance. Committed to excellence, we collaborate with prestigious academic institutions and biotech companies to advance precise medical genetic testing and champion personalized precision medicine. With over 20 years of expertise and academic experience in the biotechnology field, we are equipped with state-of-the-art technology, including the Illumina Novaseq X Plus. We offer a comprehensive NGS workflow from sample preparation to bioinformatics analysis. Our dedicated goal is to support our partners in advancing Taiwan's biotechnology sector onto the international stage and highlighting the robust strength of Taiwan's biological sciences to the world. With Taiwan as our home base, we are set to make strides on the global stage.

Brentford, United Kingdom

GSK plc, together with its subsidiaries, engages in the creation, discovery, development, manufacture, and marketing of pharmaceutical products, vaccines, over-the-counter medicines, and health-related consumer products in the United Kingdom, the United States, and internationally. It operates through four segments: Pharmaceuticals, Pharmaceuticals R&D, Vaccines, and Consumer Healthcare. The company offers pharmaceutical products comprising medicines in the therapeutic areas, such as respiratory, HIV, immuno-inflammation, oncology, anti-viral, central nervous system, cardiovascular and urogenital, metabolic, anti-bacterial, and dermatology. It also provides consumer healthcare products in wellness, oral health, nutrition, and skin health categories. The company offers its consumer healthcare products in the form of nasal sprays, tablets, syrups, lozenges, gum and trans-dermal patches, caplets, infant syrup drops, liquid filled suspension, wipes, gels, effervescents, toothpastes, toothbrushes, mouthwashes, denture adhesives and cleansers, topical creams and non-medicated patches, lip balm, gummies, and soft chews. It has collaboration agreements with 23andMe; Lyell Immunopharma, Inc.; Novartis; Sanofi SA; Surface Oncology; Progentec Diagnostics, Inc.; Alector, Inc.; and CureVac AG., as well as strategic partnership with IDEAYA Biosciences, Inc. and Vir Biotechnology, Inc. The company was formerly known as GlaxoSmithKline plc and changed its name to GSK plc in May 2022. GSK plc was founded in 1715 and is headquartered in Brentford, the United Kingdom.

Cambridge, Cambridgeshire, United Kingdom

At Harness Therapeutics we have one mission – physiological upregulation of targets that have the promise to provide disease-modifying therapeutics for neurodegenerative diseases. We use a range of mRNA-targeted oligonucleotide-based methodologies to alter the post-transcriptional regulation of protein synthesis and so increase the target protein in a controlled manner. Our focus on neurodegenerative disease is driven by our passion to develop truly disease-modifying therapies for diseases that affect so many people, yet for which there are few if any therapies that can slow or halt the progression of the disease. The ability to effect controlled increases of protein levels is essential in being able to safely drug targets with narrow therapeutic windows, something that existing platforms cannot achieve. This is critical for neurodegenerative disease as there are now a number of targets that have strong genetic validation linked to disease progression but require controlled increases rather than knock-down or over-expression. Our goal is to open up the target space for neurodegenerative disease and be able to advance effective treatment solutions for the tens of thousands of patients and their families who are facing the certainty of irreversible disease progression, loss of quality of life, and ultimately death. Our name reflects our approach, built and refined since our formation in 2020; harnessing the body’s endogenous cellular processes controlling protein synthesis to achieve controlled increases in protein levels in the safest possible manner. We use our deep expertise in RNA biology/bioinformatics and neuro cell biology to probe the intricate regulation of each target and identify multiple upregulation opportunities. Our multi-year experience focussed on physiological upregulation has allowed us to develop bespoke assays and analytical techniques to screen for constructs that drive small increases in protein, which is a key challenge in this space. Our sole focus on neurodegeneration has allowed us to build deep expertise in the biology of neurons and our targets, and the best possible models for disease – critical in being able to de-risk and translate candidates to the clinic. Neurodegenerative disorders are chronic conditions that destroy parts of the nervous system over time, especially the brain. They result in progressive loss of cognitive and motor function and eventually, death. The most common neurodegenerative diseases include Alzheimer’s disease, Parkinson’s disease, Amyotrophic lateral sclerosis (ALS), Motor neuron disease (MND), Huntington’s disease (HD), Spinal muscular atrophy (SMA), and Spinocerebellar ataxia (SCA). It is estimated that more than 50 million people worldwide are affected by neurodegenerative diseases and this number is increasing as overall life expectancy increases. Most of these conditions are much more likely to happen in people over 65, but some conditions like Huntington’s disease and ALS often appear much earlier.

Boston, Massachusetts, United States

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Lausanne, Vaud, Switzerland

HAYA Therapeutics is a precision medicines company developing RNA-guided programmable therapeutics targeting the regulatory genome for serious health conditions including cardiovascular disease and cancer. The company is using its innovative cell-state modifying platform to gain novel insights into the biology of long non-coding RNAs (lncRNAs), the ‘dark genomes’ information processing features and ‘source code’. HAYA’s lead therapeutic candidate is HTX-001, an antisense oligonucleotide targeting Wisper, which is a tissue and cell-specific cardiac lncRNA known to play a role in heart failure. The company is also developing a pipeline of lncRNA-targeting candidates for the cell-specific treatment of diseases in other tissues, including the lungs and the microenvironment of solid tumor cancers. Headquartered at the life sciences park Biopôle in Lausanne, Switzerland with laboratory facilities at JLABS @ San Diego, HAYA is led by a world-class team of experts in programmable and precision RNA-targeted therapeutics and is supported by a strong investor consortium. HAYA’s mission is to identify and develop breakthrough disease-modifying therapeutics with the potential for greater efficacy, safety, and accessibility than existing treatments.

Chengdu, Sichuan, China

HitGen Inc. (SSE: 688222.SH), founded in 2012, is headquartered in Chengdu, China, with subsidiaries in Cambridge, UK and Houston, USA. HitGen is committed to building a world-class innovative biopharmaceutical enterprise and contributing to the better life and health of mankind. Engaged in the discovery and optimization of small molecules and nucleic acid drugs, HitGen dedicates itself to cultivating an internationally leading drug discovery and optimization system centered on four key technology platforms, including DNA-encoded library technology (including DEL design, synthesis and screening, and application expansion), fragment-based drug discovery and structure-based drug design technologies (FBDD/SBDD), synthetic therapeutic oligonucleotide technology (STO), and targeted protein degradation technology (TPD). It provides new molecular entities (NMEs) at different stages of research and development for the pharmaceutical industry, through its diversified business models including research and development services, out-licensing of projects at different R&D stages, and new drug launches in the long term, with an aim to address unmet clinical needs with innovative therapeutic solutions. As a leader in the field of DEL technology, HitGen has been committed to the development, application and improvement of DEL technology since its establishment. HitGen has approximately 20 in-house drug discovery projects at different stages of research and development, among which 4 have obtained IND approvals and entered into clinical trials. HitGen is in collaboration with pharmaceutical, biotech and chemical companies, foundations and research institutes in the Americas, Asia, Europe, Africa, and Oceania. For more information, please call +86-28-85197385, +1-508-840-9646 or visit www.hitgen.com. For business development: bd@hitgen.com

Leiden, Netherlands

Hybridize Therapeutics is a spin-off from the Leiden University Medical Center and is a therapeutic company focused on developing kidney-protecting modalities for untreated kidney diseases.

Coralville, Iowa, US

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.

Carlsbad, California, United States

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.

Burnaby, Canada

We’re at the forefront of next-generation antibody drug conjugate discovery, thanks to our innovative, in-house developed Smart AntibodiesTM delivery technologies and utilization of the next-generation smart Payloads such as oligonucleotides, PROTAC, and peptides. Our mission? To unlock the vast therapeutic potential of the antibody drug conjugate modality. To achieve this, we’re engaged in expansive global collaborations with leaders in therapeutic research.

Irvine, California, United States

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.

Munich, Bavaria, Germany

Isarna Therapeutics is a clinical stage biotech company, headquartered in Munich, Germany. Isarna develops ISTH0036, a next-generation antisense to block TGF-beta 2 production, a key molecule in the pathogenesis of retinal disease. Current therapies – approved and pipeline products – mostly target the edema, with several anti-VEGFs approved or in development offering only minor improvements over current SOC. While anti-VEGF is a very effective therapy – it does not stop the disease progression of retinal pathologies like w AMD. Patients progress to late-stage disease despite effective anti-edema therapy – and thus develop fibrosis. TGF-b is a key driver of fibrosis, so blocking the production of this molecule intracellularly, using elaborated antisense molecules, promises to be a game-changer in retina therapy. Animal data show comparable efficacy of ISTH0036 to block neovascularization as anti-VEGF – with the added benefit of suppressing fibrosis. This MOA could be an effective long-term treatment in several retinal pathologies including the blockbuster-indications wAMD, DME, RVO, with a significant upward potential in dry AMD. A Phase 1 study showed excellent safety, Isarna Therapeutics is currently preparing a Phase 2a study in wet AMD and in DME/DR. Isarna is led by a highly experienced management, the COO, Rene Rückert, MD, MBA led at Bayer/Novartis the global development of both approved blockbuster drugs for retinal diseases – Eylea and Lucentis. The CMO, Prof. Marion Munk, MD, PhD is a leading global retina specialist. Isarna is open to discuss future partnering for the development and is seeking investors to finance the clinical development and growth of the company.

300 Technology Square, 8th Floor, Cambridge, MA 02139

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.

Oceanside, California, United States

Polymeric Supports for Nucleotide Synthesis

Cambridge, Massachusetts, United States of America

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.

New York, New York, United States

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

Birmingham, United Kingdom

Linear Diagnostics Limited (LDL) is a diagnostics company based on a novel technology that allows wide range of molecular diagnostics tests to be carried out using a simple optical system. The technology provides significant advantages over the current state of the art allowing a more rapid detection of multiple targets using a single simple reagent. Such a solution has applications in a number of high-value sectors including health, food, defence and security. LDL has also established development partnerships with a number of end users including clinical microbiology, food producers and defence medicine.

Osaka, Japan

Luxna Biotech is a preclinical antisense drug development company based on proprietary modified nucleic acids, unique bridged structure (AmNA, scpBNA, GuNA) and modified DNA (5'-CP) can eliminate phosphorothioated in antisense and siRNA. Currently, better modifications are being discovered that are suitable for applications in neurological diseases that minimize neurotoxicity while maintaining knockdown efficacy in vivo by Gapmer. Based on these findings we developed excellent antisense (ASO) drug discovery platform called “LuxiAPTM” enable to wide therapeutic index for CNS ASO drug. We have several pharmaceutical collaborations and original ASO pipeline including ALS, Spinal cord injury and Hearing loss. We are looking for the partners not only for platform collaboration with pharmaceuticals and biotech but early-stage co-development alliance with our original pipeline.

Boston, Massachusetts, United States

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...

San Francisco, United States of America

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.

Bedminster, New Jersey, United States

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.

South San Francisco, California, United States of America

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.

Tokyo, Japan

Mochida Pharmaceutical - By aiming to become a comprehensive healthcare company, Mochida Pharmaceutical has made significant contributions in the field of medical care through its pharmaceuticals, healthcare products, and medical equipment businesses. Several years back, to improve the profitability, competitiveness value of all its divisions, Mochida Pharmaceutical split off the Medical Electronics and Equipment Division in 2003, the Healthcare Division in 2004 and the Pharmaceutical Manufacturing Division in 2005. Currently, the Group consists of Mochida Pharmaceutical and five consolidated subsidiaries. We at the Mochida Pharmaceutical Group remain committed as ever to contributing to peoples' health and well-being.

9276 Scranton Rd, Suite 300, San Diego, California 92121, US

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.

South San Francisco, California, United States

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.

Seattle, Washington, United States

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.

A.C. Meyers Vænge 15, Copenhagen, Capital Region of Denmark 2450, DK

Developing RNA therapeutics for disease modification neurological disorders

Sunnyvale, California, United States

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.

Cincinnati, Ohio, United States

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.

11558 Sorrento Valley Road, Suite 3, San Diego, CA 92121, United States

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.

Norton Shores, Michigan, US

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

32 gallowgate, newcastle upon tyne, tyne and wear, united kingdom

NunaBio is a privately-owned specialist DNA synthesis and development company. We are engaged in CRO (Contract Research Organizations) and CDMO (Contract Development and Manufacturing Organisation) activities. The company has built a solid reputation for its R&D flexibility and innovative approach to complex and challenging projects. NunaBio's core expertise is research and development into cell-free DNA synthesis, process optimisation, route design, scale-up and small-scale manufacture for clinical studies.

Gyeonggi, Korea

OliX Pharmaceuticals is a clinical stage pharmaceutical company developing therapeutics based on the science of RNAi to address a wide variety of illnesses. The Company has developed core platforms (asymmetric siRNA, cell-penetrating asiRNA) targeting locally administrable diseases, such as hypertrophic scars, hair loss, and age-related macular degeneration (AMD). OliX's third RNAi therapeutic platform, GalNAc-asiRNA, targets multiple liver diseases, including NASH/diabetes and HBV. For the general welfare of human beings, OliX is constantly endeavoring to become a leader in the global biotechnology industry by innovating and expanding its pipeline, even to the undruggable diseases.

Bay Harbor Islands, Florida, United States

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.

Princeton, New Jersey, United States

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

135 Mississippi Street, San Francisco, CA 94107

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.

Odense, Denmark

PentaBase is a knowledge-based Danish pharmacogenomics company founded and managed by researchers and located in the heart of Denmark. We specialise in custom oligonucleotide production and the development of in vitro diagnostic assays for real-time PCR with a special focus on the detection of somatic mutations in cancer and COVID-19. For more than 10 years, PentaBase has created products for researchers and medical professionals exploring new treatments and helping patients worldwide. Our products are given unique abilities in the analysis and manipulation of genetic material with our proprietary technology, INAs® (Intercalating Nucleic Acids). Medical professionals and researchers use these features to help treat diseases and acquire new knowledge. We believe in open-platform designs and strive to make our products compatible with basic instruments used in molecular biology facilities worldwide. For more information, please contact us at: Tel: +45 3696 9496 Mail: info@pentabase.com https://www.pentabase.com/

Boston, Massachusetts, United States

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.

London, England, United Kingdom

PolyModels Hub kicked off in late 2022 with a mission: make modeling in Life Sciences easier for everyone and boost innovation. We started right here on Linkedin, building a community by sharing the latest research and open-source tools for life sciences. We are strong advocates for open-source modeling – it's akin to the transformation we saw in Software Dev and AI. That's why we recently introduced our open-source Hub, where you can sign up to discover, build and connect with the open-source community! Fast forward to 2024, and we decided to level up. We expanded PolyModels Hub to offer modeling services and products to the pharmaceutical industry. Our focus is on helping companies speed up their innovation in developing new drugs. We use the best digital design tools available, both open-source and proprietary, through our digital design approach and platform. The goal? Empower companies to launch medicines faster, save costs, and improve the quality of their products. And trust us, there's a lot more to come in our story!

Illkirch-Graffenstaden, France

Polyplus, part of Sartorius, is a leading upstream solutions provider for advanced biologic and cell and gene therapy production from research to commercial scale. An innovator in nucleic acid delivery, the legacy portfolio features process-centric transfection reagents, kits, and support services for viral and non-viral delivery. Custom plasmid vector design and plasmid and protein manufacturing was integrated into the offer in 2022 to expand the products and services portfolio to help the industry optimize process economics while meeting strict scientific and regulatory standards.

Chongqing, China

Founded in 2005, Porton Pharma Solutions Ltd. is an internationally recognized pharmaceutical contract development and manufacturing organization (CDMO) in enabling our global clients to optimize drug development and manufacturing. We provide customer development and manufacturing services for small molecule, Large molecule and Gene and Cell Therapies from pre-clinical to commercialization. As of June 30, 2023, we have a proven record for the delivery of more than 2600 projects and established cooperation with more than 800 global clients. With our 5000+ skilled employees working across 20+ sites around the globe, Porton is committed to becoming the most open, innovative and reliable pharmaceutical service platform in the world and enabling the public's early access to good medicines. Porton is publicly listed on the Shenzhen Stock Exchange (Ticker: 300363). With over 5000 customer-centric employees, operations and commercial offices across the US, EU and China, Porton Pharma Solutions/J-Star Research provides global pharmaceutical companies with innovative, reliable and end-to-end process R&D and GMP manufacturing services across small molecule APIs, TIDES, biologics and Gene & Cell Therapies. We focus on the customer and have been recognized through awards by industry forums and global pharmaceutical companies for our process innovation, supply chain performance, and compliance with global Quality and EHS standards. We constantly strive for excellence and enable the public's early access to good medicines.

Boston, Massachusetts, United States of America

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.

Indore, Madhya Pradesh, India

PREMIER Biosoft is a leading bioinformatics company offering software products and services to government agencies, pharmaceutical and biotechnology companies and academia across the globe. PREMIER Biosoft specializes in software development for life science community. and has products for PCR, Real Time PCR, Mass spectrometry data analysis, Glycomics, Lipidomics, MALDI Imaging, Proteomics and Plasmid drawing. The company also offers custom informatics solutions to life science companies and laboratories. With all these services, the PREMIER Biosoft team promises to extend these benefits to companies, universities and laboratories.

Norwich, United Kingdom

Procarta Biosystems Ltdaims to revolutionise the treatment of serious and life-threatening infection through the development of innovative new oligonucleotide-based antimicrobials.

Leiden, Netherlands

We are ProQR, a biotechnology company dedicated to changing lives by developing RNA therapies for rare genetic diseases. We focus our drug development mainly on a group of blinding disorders affecting the retina, called inherited retinal diseases.

Suzhou, Jiangsu, China

Ractigen Therapeutics is an early-stage biotech company committed to bringing to market first-in-class therapies designed to selectively restore the expression of therapeutic genes silenced in diseased cells. Its core technology is based on a paradigm-shifting discovery known as RNA activation (RNAa). Founded in 2016 by the pioneers in the field, Ractigen is developing a rich pipeline of candidate medicines for patients with unmet need.

Åkandevej 21, 2700 Brønshøj, Denmark

Radiomer Therapeutics, Inc. is a Fannin-founded company that is developing targeted radiopharmaceuticals for cancer treatment.

San Diego, California, United States

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.

Montreal, Quebec, Canada

Replicor Inc. is a Canadian biotechnology company focused on the development of its nucleic acid polymer (NAP) technology for the treatment of hepatitis B (HBV) and delta (HDV) infections. NAPs are the only antiviral agent to directly target the immunosuppressive protein (HBsAg) which drives chronic HBV infection and liver disease. Our lead clinical NAP drug product is REP 2139-Mg, a proprietary formulation of REP 2139 which makes once weekly subcutaneous injections easily tolerated. Replicor's latest clinical data demonstrates that NAP-based combination therapy can restore immune control of HBV infection in 78% of patients and eliminate HDV infection in 74% of patients. These effects persist in the absence of any therapy, allowing the liver to heal. REP 2139-Mg based combination promises to revolutionize the therapy of chronic HBV and HDV infection and play a pivotal role in the WHO's goal of eliminating viral hepatitis by 2030.

Limerick, Pennsylvania, United States

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.

London, United Kingdom

Roquefort Therapeutics is a LSE Main Market listed biotech company developing first in class drugs in the high value and high growth oncology segment prior to partnering or selling to big pharma. Since listing in March 2021, Roquefort Therapeutics has successfully acquired Lyramid Pty Limited, a leader in the development of medicines for a new therapeutic target, Midkine (a human growth factor associated with cancer progression), and most recently acquired Oncogeni Ltd, founded by Nobel Laureate Professor Sir Martin Evans, which has developed two families of innovative cell and RNA oncology medicines. Roquefort Therapeutics’ portfolio consists of four fully funded, novel patent-protected pre-clinical anti-cancer medicines. The highly complementary profile of four best-in-class medicines consists of: 1. Midkine antibodies with significant in vivo efficacy and toxicology studies; 2. Midkine RNA therapeutics with novel anti-cancer gene editing action; 3. MK cell therapy with direct and NK-mediated anti-cancer action; and 4. siRNA targeting novel STAT-6 target in solid tumours showing significant in vivo efficacy.

Bilthoven, Utrecht, Netherlands

Sapreme’s mission is to develop next-generation macromolecule therapeutics by circumventing endosomal entrapment, thereby enhancing target engagement. The company’s proprietary endosomal escape platform improves the therapeutic window by enabling access to intracellular targets with minimal toxicity. This approach is applied for Sapreme’s internal pipeline and is available for partnering, without limitation to biologic modality or indication. Our solution to endosomal entrapment has the potential to re-open myriad drug development pathways for macromolecule therapeutics lacking efficacy and to explore new targets considered “undruggable” by permeabilizing endo-lysosomal membranes, allowing trapped macromolecules to be released, increasing target engagement without negatively impacting the plasma membrane integrity.

New York, New York, United States

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

Marburg, Germany

Secarna Pharmaceuticals is the next generation antisense oligonucleotide (ASO) company addressing high unmet medical needs in the areas of immuno-oncology and immunology, as well as viral, neurodegenerative and cardiometabolic diseases. Secarna’s mission is to maximize the performance and output of its proprietary LNAplus™ antisense oligonucleotide discovery platform, as well as to develop highly specific, safe, and efficacious best-in-class antisense therapies for challenging or currently undruggable targets. With over 15 development programs focusing on targets in indications where antisense-based approaches have clear benefits over other therapeutic modalities, Secarna is the leading European antisense drug discovery and development company.

Exeter, Devon, England, United Kingdom

SENISCA is a biotech spinout company from the University of Exeter, founded in 2020 and dedicated to the development of new approaches to reverse cellular senescence (senotherapeutics). Our founders are world leaders in molecular and cellular biology and have patent-protected an innovation for the reversal of cellular senescence. This innovation works by restoring the ability of cells to ‘fine tune’ the expression of their genes to rejuvenate aged cells. At SENISCA, we are using this knowledge, concerning how and why cells become senescent, to develop a new generation of oligonucleotide-based interventions, to turn back the ageing clock in old cells and to target the diseases and aesthetic signs of ageing.

Ness Ziona, Israel

SKIP Therapeutics develops RNA-based therapies for rare genetic disorders and common diseases – utilizing a proprietary computational discovery engine to optimize target selection and therapeutic design. We employ sequence-based molecules, called Antisense Oligonucleotides (ASO), to restore function of mutated genes in rare genetic disorders or to modify functionality of key genes in major disease pathways. Convergence of SKIP’s novel computational discovery engine alongside strong experimental capabilities form a unique venture in the landscape of RNA-based therapies. Thousands of rare genetic disorders affect millions of people worldwide, however the small number of patients for each disease, further segregated by patient-specific mutations, has hindered development of dedicated treatments. Recent advancements in RNA-therapeutics offer an expanded molecular toolkit that can be deployed rapidly and at low costs to restore protein function of mutated genes. Identifying sub patient-populations that could potentially be suitable for treatment with RNA-based therapeutics and matching molecular treatment-strategies across the vast landscape of known disease-causing mutations could be assisted by computational methods. To facilitate development of dedicated treatments, Skip Therapeutics has developed a bioinformatic pipeline that analyzes disease-causing mutations and recognizes the best molecular strategy (if any) to restore protein function, using splice modulating antisense oligonucleotides (ASOs). Analyzing mutation data at scale, enables identification of treatable patient cohorts and optimizes target selection. Top candidates from the computational analyses are experimentally validated and pursued for therapeutic development.

Jerusalem, Israel

SpliSense Ltd., is a growing and exciting biotechnology company, developing novel therapies for genetic diseases by inhalation. The company is currently focusing on splicing mutations in the CFTR gene for treating Cystic Fibrosis (CF) patients.

123 Highland Avenue, Suite 201, Needham, MA 02494

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.

Bedford, Massachusetts, United States

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.

I Park Tower Bldg., Yeongdongdae-ro 520, Seoul, Seoul Teugbyeolsi, KR, 06170

"ST Pharm provides reliable and timely custom manufacturing services for API following stringent quality system and close communication with the clients." Founded in 1983, ST Pharm (formerly known as Samchully Pharmaceutical) has been offering excellent custom manufacturing services of APIs and their intermediates to meet clients' high expectation for their use in pharmaceutical development in compliant to cGMP requirements. Our reliability in service, which is well-proven through decades of business relationship with many big pharmas and biotech clients, is the essence of our strength along with our expertise in process development and scale-up optimization. Historically, ST Pharm has been focusing our expertise on nucleoside based active pharmaceuticals in various antiviral drug applications; we became the world largest supplier of Thymidine in the late 1990s and Zidovudine in the mid 2000s and protected nucleosides in the early 2000s. ST Pharm has continuously been expanding its service offering, including synthesis of therapeutic oligonucleotides. In June 2010, ST Pharm became a subsidiary of Dong-A Pharmaceutical, the top pharmaceutical company in Korea. ST Pharm is committed to expanding its business scope into generic APIs and their intermediates with full support and collaboration with parent company Dong-A Pharmaceutical.

Anhui, China

Established in 2007, Suzhou Ribo Life Science Co., Ltd. (Ribo) is a clinical stage company devoted to the development of innovative RNAi technologies and oligonucleotide therapeutics as an effort to address unmet clinical needs in the world with the tools, capacity and RNA therapeutics that we build up in China. Fueled by our mission to make China's oligonucleotide therapeutics serve the world, we seek. Based on its cutting edge RNAi technologies, Ribo has established oligonucleotide therapeutic platform that vertically integrates the complete set of technologies to support the full life cycle of oligonucleotide therapeutics from early R&D to commercialization.

oxford, oxfordshire, united kingdom

SynaptixBio is a rare disease biotech company that is pioneering ground-breaking treatments for severe leukodystrophies and other childhood neurodegenerative diseases.    SynaptixBio's mission is to deliver gene therapies for fatal leukodystrophies in children caused by loss of function or mutation in a single gene. Using a breadth of platforms including antisense oligonucleotide (ASO)-based technology, we can silence the expression of the toxic gene to reverse disease progression. This approach will create life saving treatments for severe genetic diseases that affect the central nervous system.

Monmouth Junction, New Jersey, US

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.

Tokyo, Japan

TAGCyx’s scientific team has established its proprietary Xenoligo® technology platform, allowing high functional oligonucletide drug discovery. The Xenoligo® platform utilizes genetic alphabet expansion by unnatural base-pairs to generate customized nucleic acid suited for therapeutic applications.

Burlingame, California, United States

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.

10431 Wateridge Circle, Ste 150, San Diego, California 92121, US

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.

Berywyn, Pennsylvania, United States

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

Lyon, France

Theranexus is an innovative biopharmaceutical company that spun off from the French Alternative Energies and Atomic Energy Commission (CEA). It specializes in the treatment of rare central nervous system disorders and is a pioneer in the development of drug candidates targeting both neurons and glial cells. Theranexus has a unique advanced therapy candidate identification and characterization platform focused on rare neurological disorders and an initial drug candidate in clinical development for Batten disease.

Berlin, Germany

TIB Molbiol is a medium-sized biotech company based out of Berlin, Germany who has supplied the global market for over 30 years with reagents for research and medical diagnostics. We were recently bought by Roche, one of the largest pharma and biotech companies in the world. Roche has committed to keep the start -up character of the company so that the company can benefit from the advantages of a small biotech and the muscle and power of a large pharma/biotech company such as Roche. Our customers are mainly diagnostic and research labs, servicing patients every day, selecting from any of our more than 2,500 diagnostic kits and LightSNiP assays. We fulfill many special orders for custom design of individual assays, for patient need.

South San Francisco, California, United States

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.

Newton, Massachusetts, United States

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.

San Diego, California, US

Allschwil Basel-Country, Switzerland

Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community

Cambridge, Massachusetts, United States

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.

Leiden, South Holland, Netherlands

Vico Therapeutics is committed to advancing RNA modulating therapies for patients with severe, genetic neurological disorders. Vico is initially targeting spinocerebellar ataxia types 3 and 1 and Huntington’s disease. Our VICOMER platform designs antisense oligonucleotides for various genetic defects by modulating or editing RNA.

Cambridge, Massachusetts, United States

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.

Worcester, Massachusetts, United States

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.