560 RNA Therapies Companies - Worldwide

Portland, OR

13therapeutics is a research and drug development company identifying and characterizing novel anti-inflammatory peptide therapeutics for unmet medical needs with high commercial potential. The company has an innovative platform for discovering and enhancing peptides, derived from naturally occurring pathogens (e.g., bacteria, viruses, fungi), which modulate the human immune response. This platform has produced a portfolio of peptides with demonstrated immune-regulatory activity and with varying mechanisms of action. These peptides have potential application for treatment of both acute and chronic inflammatory diseases. Many of the company’s peptides are capable of multiple methods of delivery, have shown serum stability, and have other desired features clinical therapeutics. Target conditions include the treatment of acute inflammatory diseases such as Acute Otitis Media (AOM), Systemic Inflammation, Meningitis, Traumatic Brain Injury (TBI), Uveitis and Ischemia. We are also evaluating peptides in chronic models for Lupus, Rheumatoid Arthritis (RA), Multiple Sclerosis, Atherosclerosis, Crohn’s Disease and Psoriasis, among others. Using our evolution-based methodology, we envision rapid development across several disease states.

Emeryville, California, United States of America

4DMT is a clinical stage biopharma company inventing and developing innovative products to unlock the full potential of genetic medicine to treat large market and rare diseases. Our vector discovery platform Therapeutic Vector Evolution harnesses the power of directed evolution, a Nobel Prize-winning technology, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors with improved therapeutic profiles compared to wild-type vectors. Our product design, development and manufacturing engine empowers us to efficiently create our valuable and diverse product pipeline. This combination of bold innovation and relentless execution gives 4DMT the capability to revolutionize genetic medicines and to strive for potential curative therapies. We have built a deep portfolio of genetic medicine product candidates, with five product candidates in clinical trials for seven patient populations. We are taking genetic medicine to new heights and into new disease areas to overcome challenges not addressed by existing medicines. This incredible challenge drives our relentless and talented team. Our people are the essence of our inspiring, innovative and collaborative environment that encourages individual contributions and provides opportunities for career development. We seek the best in the industry who identify with our mission and our bold and innovative results-driven approach. Our 4 Guiding Principles drive the way we work together: - Dare to Cure - Break Boundaries - Beyond Yourself - Prepare & Execute Relentlessly Join us in creating the future of genetic medicine.

Heidelberg, Baden-Württemberg, Germany

AaviGen GmbH is a biotechnology start-up located in Heidelberg, Germany. Our primary purpose is the development and commercialization of precision gene therapies for cardiovascular and cardiopulmonary diseases. Our business model builds on the success of the InoCard GmbH, a predecessor that was acquired by uniQure N.V. and subsequently entered into a development alliance with Bristol-Myers-Squibb. Please find our imprint ("Impressum") and data privavy statement ("Datenschutzerklärung") at https://aavigen.com/content/imprint.html and https://aavigen.com/content/privacy.html

Rockville, Maryland, United States of America

Our mission is to make you stronger. The company was founded by a family directly impacted by three muscle wasting diseases: Duchenne muscular dystrophy, cancer cachexia and COPD. Our fight is personal and our commitment to developing better and more effective therapies is undaunted. AVGN7 prevents muscle wasting in different animal models and can restore muscle mass and strength even after wasting has already occurred. It works when injected directly into muscle or when administered systemically to all muscles. It was also designed to avoid the potentially serious side-effects of other "myostatin attenuating" therapies. AVGN7 for the durable maintenance of muscle mass and function.

Buffalo, New York, United States

Abcombi Biosciences is a pre-clinical vaccine and therapeutic development company that uses a biofilm model that mimics the natural progression of disease for colonizing bacteria such Streptococcus pneumoniae and Staphylococcus aureus in conjunction with bioinformatics helpto identify antigens associated with specific stages of disease progression.

New York, New York, United States of America

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases.

San Diego, California, United States

Driven by a bold founding vision: Empowering patients from within, Abintus is pioneering potentially curative, off-the-shelf genetic medicines that engineer cells directly inside the patient’s body, also termed in vivo. These therapies are based on Abintus' clinically proven Modular Delivery Platform (MDP). The company’s two initial, fit-for-purpose, In Vivo programs are: 1. Highly selective gene expression targeting cell type/state via synthetic biology (e.g., In Vivo CAR-NK/T/Treg, In Vivo TCR-T) with applications across multiple gene delivery systems (e.g., AAV, lenti, adeno, retro) and therapeutic areas including leukemia, solid tumors, autoimmune, infectious, and genetic disease. 2. Simultaneous engineering of a diverse repertoire of active innate and adaptive immune cells (e.g., In Vivo CAR-X) with applications in diseases such as lymphoma and solid tumors. Abintus is leveraging over 20 years and $275 million of prior technology advancement for in vivo engineering including the treatment of over 350 patients through Phase 3 registration trials. Our company name comes from the Latin words “ab intus”, which means “from within”. We chose this name for 3 reasons: 1) Patients: We are pursuing product candidates that are designed to directly empower patients’ own immune cells to fight their disease from within. 2) Science: Our team is committed to scientific excellence and continual learning. Our differentiated technologies have the potential to directly reprogram activated immune cells “in situ”, or from within. 3) Culture: We believe our ability to achieve our vision will come from within - by building a performance-driven culture that prioritizes career development and rewards outstanding contributions. Abintus’ initial funding syndicate included Takeda Ventures and The Leukemia & Lymphoma Society’s Therapy Acceleration Program® (LLS TAP). Abintus Bio, Inc. (“Abintus”) is headquartered in San Diego. www.abintusbio.com.

Kyoto, Japan

aceRNA Technologies Co., Ltd. is a biotech startup established in 2018 based on RNA design technology developed in synthetic RNA biology research by Prof. Hirohide Saito at Kyoto University. We are developing "smart gene therapy” (mRNA-based and virus-base) that enables control of therapeutic transgene expression in cell-type and -state specific manner. By that, we are committed to develop new therapeutic approach, such as in vivo cell programming.

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.

Rockville, Maryland

At Addimmune, our mission is to deliver the world’s first functional cure for HIV. Using viral vector delivery, we are seeking to give the immune system the tools it needs to resist and ultimately clear the virus.

Delray Beach, Florida, United States of America

Adolore Biotherapeutics, Inc., is a biotechnology company focused on developing novel therapies for the treatment of chronic pain and other pain and nervous system conditions or disorders. Our best-in-class lead programs are long-acting locally acting gene-therapies that are opioid-free Disease Modifying Anti-Pain therapies (DMAPs) for the treatment of a variety of chronic pain indications. The Company’s two current CA8* gene therapy programs are in preclinical development for treatment of patients suffering from erythromelalgia, a life-long heritable chronic pain condition representing an orphan drug disease with no approved therapy, and chronic osteoarthritis knee pain, affecting a large number of patients that is often treated with opioids due to the lack of alternatives, thus contributing to the ongoing opioid crisis.

Raleigh, North Carolina

Adrenas Therapeutics is a company that was created with a single mission: to work with scientists, physicians, and patients in developing a gene therapy for people affected by CAH. Adrenas is a subsidiary of BridgeBio, a public company dedicated to finding, developing, and delivering breakthrough medicines for genetic diseases to patients as quickly and safely as possible.

Redwood City, California, United States of America

Adverum Biotechnologies, Inc., a clinical-stage gene therapy company, engages in developing gene therapy product candidates to treat ocular and rare diseases. Its pipeline of product candidates includes ADVM-022, an adeno- associated virus (AAV). 7m8-aflibercept for the treatment of wet age-related macular degeneration; ADVM-043, an investigational gene therapy candidate for the treatment of alpha-1 antitrypsin deficiency; and ADVM-053, a preclinical gene therapy product candidate for the treatment of hereditary angioedema. The company has collaboration agreements with Editas Medicine, Inc. to leverage its proprietary AAV vectors for genome editing technologies to treat up to five inherited retinal diseases; and Regeneron Pharmaceuticals, Inc. for the development of up to eight distinct ocular therapeutic targets. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was founded in 2006 and is headquartered in Redwood City, California.

Boston, MA

Aera was founded with a vision to unlock the potential of genetic medicines across a broad range of modalities and therapeutic areas. Our protein nanoparticle (PNP) delivery platform leverages the discovery of endogenous, human proteins that can self-assemble to form capsid-like structures and that can package and transfer nucleic acid cargo. This platform has the potential to address many of the limitations of today’s delivery technologies and to enable various genetic medicine modalities.

Waltham, Massachusetts

Affinia Therapeutics is an innovative gene therapy company focused on broadening the reach of gene therapy. Our proprietary Affinia Rationally-designed Therapies (ART) platform is created to address key limitations of conventional gene therapies, by developing novel capsids, novel promoters, and novel manufacturing approaches to developing gene therapies for rare and prevalent devastating diseases. We are backed by a strong syndicate of life science investors and have ambitious plans to have a dramatic impact on the lives of patients around the world.

Cambridge, Massachusetts, United States of America

Following the acquisition of Lung Therapeutics in October 2023, Aileron is shifting its disease focus to advancing a pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis diseases, including LTI-03 for idiopathic pulmonary fibrosis (IPF) and LTI-01 for loculated pleural effusion (LPE).

San Diego, California

Ainos Inc. is a diversified healthcare company focused on the development of novel point-of-care testing (POCT), low-dose VELDONA interferon therapeutics, women's health, pneumonia, Ainos Pen, AI Nose, and other medical technologies. The company was founded in 1984 and is engaged in developing innovative medical technologies for point-of-care testing and safe and effective treatments for various health conditions.

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+.

9276 Scranton Rd, San Diego, California 92121, US

We are creating a new category of medicines with the application of spatial biology and AI.

Boston, Massachusetts, United States of America

Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss.

Lowell, Massachusetts, United States

Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between FalconTM, the company’s proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and four novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome, and gene replacement for the treatment of IGHMBP2-related disorders including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S).

11535 sorrento valley road, san diego, california, united states

Alida Biosciences is an early-stage, VC backed startup in San Diego focused on developing novel genomic analysis tools. The company’s technology will advance the understanding of RNA biology and provide actionable information for improving human health.

South San Francisco, California, United States of America

Our strategy is to develop pharmacologically optimized drug candidates, which are first assessed in healthy volunteers and patients as monotherapy prior to their use in combination regimens designed to achieve improved treatment outcomes in patients. Our initial areas of focus are viral and liver diseases where our team can leverage their in-depth knowledge and expertise to develop potentially best-in-class combination regimens for each disease area with significant unmet medical need.

Cambridge, Massachusetts

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

Bellevue, WA

Allysta Pharmaceuticals is a private venture-backed clinical stage biopharmaceutical focused on rare diseases with high unmet needs. We are developing first-in-class therapeutic peptides with multiple biological actions, including anti-fibrotic, anti-inflammatory, cell regenerative, and beneficial metabolic effects that provides development opportunities across multiple diseases. Our lead drug, ALY688ER, an extended release formulation designed for extended delivery of ALY688 throughout the body, is a potent and specific activator of adiponectin signaling pathways. Adiponectin is a beneficial cytokine produced mainly by adipose cells and which protects against obesity-associated diseases. ALY688 is the first adiponectin receptor agonist to enter human testing. Our lead indication is Duchenne muscular dystrophy (DMD), a serious rare genetic disease of boys in which muscle tissue damage due to muscle cell injury, inflammation and replacement of muscle by fibrotic tissue results in progressive loss of skeletal muscle function. In relevant animal DMD models, ALY688 reduced muscle cell death, enhanced muscle regeneration, and reduced inflammation and fibrosis, resulting in preservation of muscle strength and endurance. In addition, ALY688 has shown similar benefits across a range of other inflammatory and fibrotic conditions potentially expanding its use in additional indications.

Cambridge, Massachusetts, United States of America

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

Stony Brook, New York

Alpha-1 Biologics is a biotherapeutics company developing innovative treatments for cancers and immune deficiencies. The company is dedicated to their mission to develop patented therapeutics related to the generation of immune cells from stem cells within the body to treat immunodeficiency with disease applications including immune cell replenishment in cancer therapy, HIV/AIDS, patients with inherited deficiency of α1PI, and in the most prevalent cause of immune deficiency, malnutrition. Alpha-1 Biologics is currently testing their orally-available, small molecules in studies to determine their ability to increase the number of tumor infiltrating CD4+ T cells and coordinate with immune checkpoint inhibitors to eliminate tumors. The discoveries at the core of Alpha-1 Biologics therapeutic approach resulted from basic research conducted by Dr. Bristow and supported for many years by the non-profit research organization, Institute for Human Genetics and Biochemistry (IHGB) funded by the Harry Winston Research Foundation.

Hamilton, Bermuda

Altamira Therapeutics (former Auris Medical) is dedicated to developing therapeutics that address important unmet medical needs. The Company is currently active in three areas: • the development of RNA therapeutics for extrahepatic therapeutic targets (OligoPhore™ / SemaPhore™ platforms; preclinical), • nasal sprays for protection against airborne viruses and allergens (Bentrio™; commercial) or the treatment of vertigo (AM-125; Phase 2) • the development of therapeutics for intratympanic treatment of tinnitus or hearing loss (Keyzilen® and Sonsuvi®, Phase 3). The Company was founded in 2003, under the name Auris Medical, and is headquartered in Hamilton, Bermuda with its main operations in Basel, Switzerland. The shares of Altamira Therapeutics Ltd. trade on the NASDAQ Capital Market under the symbol “CYTO.”

Milan, Italy

Altheia Science is a pioneering start-up founded by two world renowned Italian scientists, Prof. Alessandra Biffi, MD and Prof. Paolo Fiorina, MD, PhD, together with AurorA-TT. Altheia Science’s pipeline exploits pioneering therapeutic tools for autoimmune diseases and cancer. The modulation of PD-L1 expression at the molecular and protein level is key to devise advanced treatments. This can be achieved by first-in-class molecules controlling the PD-L1 pathway and/or by lentiviral vector-based engineering of patients’ hematopoietic stem cells. Altheia Science’s approach intends to drastically modify the natural history of diseases with high clinical impact in autoimmunity and cancer by modulating PD-L1 expression, achieving tangible and durable benefit for each patient.

Austin, Texas, United States

Altogen Labs is a biology CRO company providing GLP preclinical research services, tox studies for IND applications, RNAi and gene silencing services, xenograft testing services (100+ tumorigenic cell lines), in vivo siRNA delivery and biodistribution, development of stable cell lines, and many more. Altogen Labs is a GLP compliant laboratory that provides preclinical research and biotechnology contract research services (CRO) to pharma, biotechnology companies, universities, and cancer research centers worldwide. Our services include pharmacology and toxicology assays (IC50), cell banking, generation of stable cell lines, RNAi gene silencing and other in vitro services. Altogen Labs provides following in vivo services for efficacy studies in the drug discovery phase: rodent xenograft models, teratoma formation and analysis services, cancer disease animal models, in vivo siRNA delivery and tissue targeting, pharmacokinetics (PK) and pharmacodynamics (PD) services, immune response and biomarker analysis. Altogen Labs recently developed an `active bioremediation`​ process based on an approach using the most potent oil-degrading bacteria found at multiple oil spill sites in Texas. This technology allows remediation of large amounts of hydrocarbon-contaminated liquids or soil. The genomes of both microorganisms were recently sequenced, which revealed that both strains were previously unreported in the literature. Altogen Labs filed bioremeddiation patent application in 6/2012.

Seattle, Washington

AltPep develops early disease-modifying treatments and detection tools for amyloid diseases by targeting one of the earliest molecular triggers – toxic soluble oligomers. These oligomers are associated with many diseases, including neurodegenerative diseases and type 2 diabetes. Decades of scientific research by the Daggett Research Group culminated in the discovery of a novel protein structure, alpha-sheet, the foundation of our innovative approach. Our lead program aims to identify Alzheimer’s Disease long before symptoms occur and then neutralize the toxic soluble oligomers associated with the disease.

Oxford, United Kingdom

AlveoGene is an innovative company focused on transforming rare respiratory disease outcomes using inhaled gene therapy. AlveoGene has been created by OSE, Harrington and OCC in partnership with six leading scientists from the world-renowned UK Respiratory Gene Therapy Consortium (GTC). The GTC was founded in 2001 to catalyse the application of pioneering research to gene therapy development and manufacturing related to cystic fibrosis and other respiratory diseases, and originated at Imperial College London and the Universities of Oxford and Edinburgh. AlveoGene has secured an exclusive licence to a proprietary and validated next-generation lentiviral delivery platform developed by the GTC for the treatment of respiratory diseases with high unmet need (excluding the use of the CFTR gene which is mutated in cystic fibrosis). Gene therapies developed using our “InGenuiTy™” platform can be delivered through a nebuliser, transducing lung epithelial cells with high efficiency and producing a long duration of action, and can achieve these effects following repeated administration. The platform has been developed over more than a decade, supported by approximately £72 million in grant funding, including from the Wellcome Trust, Department of Health and Social Care, Medical Research Council and the Cystic Fibrosis Trust.

Boston, MA, United States

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

Atlanta, Georgia, United States of America

Alzamend Neuro, Inc., a preclinical stage biopharmaceutical company, focuses on developing products for the treatment of neurodegenerative diseases and psychiatric disorders. The company’s lead product candidate is AL001 for the treatment of Alzheimer’s and other neurodegenerative diseases and psychiatric disorders. It is also developing AL002, a cell-based therapeutic vaccine, which seeks to restore the ability of the patient’s immunological system to Alzheimer’s. The company was incorporated in 2016 and is headquartered in Tampa, Florida.

Leiden, Netherlands

Amarna Therapeutics is a privately held, pre-clinical biotech company developing transformative, potentially curative gene therapies for a range of both rare and prevalent diseases. The company is pioneering the development of gene therapies based on simian virus 40 (SV40), a macaque polyoma virus to which humans are immunologically naïve. Amarna has created a proprietary production cell line (SuperVero™) that, for the first time, makes it possible to produce SV40-derived vectors suitable for therapeutic use. Combining SuperVero™ with its genetically engineered SVec viral vector, the company’s fully-integrated gene therapy platform is truly unique in its intrinsic capability to deliver transgenes without eliciting immune responses to the vector nor to the transgene product. Leveraging this ‘natural’ non-immunogenicity feature of its gene delivery system in humans, Amarna is developing a broad pipeline of safe, effective and durable gene therapies focused on genetic disorders, autoimmune diseases and chronic inflammation. Founded in 2008, Amarna’s head office is based in Leiden Bio Science Park, the Netherlands, with a research subsidiary located in Seville, Spain.

Auckland, New Zealand

Amaroq Therapeutics is a New Zealand-based biotechnology company focused on developing a new class of therapeutics that target lncRNA in cancer. Long non-coding RNAs are a relatively recently discovered class of molecules often referred to as “dark matter” of the human genome. These naturally occurring molecules do not code for proteins but fulfill important regulatory functions in the cell that could be exploited as a new therapeutic approach for multiple indications. Amaroq’s research team has discovered lncRNA molecules that are highly expressed in cancer cells, and once removed, cancer cell growth slows. Amaroq Therapeutics is developing innovative therapeutics to target lncRNAs for cancer treatment. Amaroq Therapeutics is funded by the Medical Research Commercialisation Fund, managed by life science venture capital firm Brandon Capital Partners, Otago Innovation Ltd, University of Otago, Cure Kids, and NZ Innovation Booster. The Company has been spun out of research from the University of Otago in Dunedin, New Zealand.

Miami, Florida, United States of America

Ambulero is a cell and gene therapy spin-out of the University of Miami focused on advancing new therapies to fight vascular disease. We are a platform company that can deliver the adhesion molecule E-selectin using different cell and synthetic vector technologies. We work on rare vascular diseases as well as more common indications effecting large patient populations.

Rockville, Maryland

American Gene Technologies® International Inc. (AGT™) is a private biotechnology company pursuing cures and treatments for infectious diseases, cancers, and monogenic disorders. AGT™'s emerging gene and cell therapeutics programs are designed to: ✅ Create immunotherapy for HIV disease with gene-modified T cells ✅ Activate innate T cell immunity capable of killing all tumor cells with its vector used in immuno-oncology ✅ Treat PKU by bringing a functional gene into cells that express a dysfunctional gene due to mutation ✅ Advance clinically to provide human proof of concept For over a decade, AGT™ has developed, tested, and banked thousands of lentiviral vectors that can be adapted to the specific needs of new target diseases. AGT™ has developed gene therapy for diseases using this rapid gene delivery platform. GENE THERAPIES AGT™’s platform allows it to pursue clinical “cures” for complex diseases. The company has developed patent-protected gene therapeutics that are breakthroughs in medicine. *** See our extensive award winning patents on our website *** HIV/AIDS Immunotherapy for HIV disease with gene-modified T cells IMMUNO-ONCOLOGY Activates innate T cell immunity capable of killing all tumor cells with its vector used in immuno-oncology PHENYLKETONURIA (PKU) Treats PKU by bringing a functional gene into cells that express a dysfunctional gene due to mutation AGT™'s Core Objectives ✔️ Leverage our lentivirus platform to create highly-effective targeted genetic medicines ✔️ Create solutions for patients across a spectrum of infectious diseases, cancers and inherited disorders ✔️ Collaborate with leading medical researchers from around the globe To learn more and get in touch with American Gene Technologies® Inc., contact us today at https://www.americangene.com/contact-us/

Ampersand Biomedicines, 245 First Street, Suite 1800, Cambridge, MA 02142

Ampersand Biomedicines enables a new way of designing programmable medicines that work precisely where needed in the body and nowhere else. The company’s state-of-the-art computationally powered Address, Navigate, Design (AND)™ Platform continually learns the rules for identifying optimal localizer targets and refines its ability to design across the complex space of localizers and actuators. The result is increasingly sophisticated AND-Body™ therapeutics that are engineered across the physical constraints of molecules and the biological characteristics of the targets to effectively target the site of disease without affecting healthy tissue or cells. Ampersand Biomedicines was founded in 2020 at Flagship Labs, a unit of Flagship Pioneering.

Towson, Maryland, United States of America

Amplo Biotechnology is a private Gene Therapy company focusing on therapy for diseases of the neuromuscular junction.

Philadelphia, Pennsylvania

Amyndas is a pharmaceutical company developing potent complement therapeutics for inflammatory diseases and disorders based on its patented technology for modulating the complement system.

Nashville, Tennessee, United States

Amytrx Therapeutics is advancing a new class of peptide therapies to overcome inflammatory diseases—with a vision of providing transformative medicines to patients. Drawing on 25 years of research, Amytrx is harnessing the power of novel anti-inflammatory peptide discoveries to advance new medicines aimed at the prevention and treatment of inflammatory diseases. Our lead therapeutic candidate, AMTX-100, is bioengineered from human protein sequences that modulate the immune system by a natural process, reducing pathogenesis in a range of chronic diseases mediated by excessive inflammation. Joining forces with institutions for clinical research, Amytrx Therapeutics is dedicated to transforming the conventional treatment of chronic inflammatory diseases using biological technology that is easily understood by the human body in a way that is safe, effective, and simple to use.

Schlieren, Zurich, Switzerland

Anjarium Biosciences is a cell and gene therapy tools and technology company headquartered in Switzerland. Our mission is to enable researchers and manufacturers to accelerate the discovery and delivery cycle of new and evolving genetic medicines. Our technology enables our partners to meet biology’s greatest challenges with increasingly flexible and customizable DNA constructs in pursuit of better human health. We offer a range of products to support research, development, and commercialization of genetic medicines. Our enzymatic, cell-free platform enables Anjarium to offer pure, infinitely customizable, stable synthetic DNA constructs at scale for use across AAV, mRNA, Lentivirus and other applications.

Copenhagen, Denmark

Antag Therapeutics is a preclinical stage biotechnology company committed to the development of novel therapeutic peptides for metabolic – and cardiovascular autonomic diseases. Antag Therapeutics is based on decades of research from the University of Copenhagen focusing on understanding and targeting incretin physiology. Our therapeutic peptides are based on a novel endogenous peptide antagonist discovered by the research teams of Professors Jens Juul Holst and Mette Rosenkilde that has been characterized in several human intervention studies.

Plan-les-Ouates, Canton of Geneva, Switzerland

Antion Biosciences SA (Antion) is a Swiss biopharmaceutical company developing novel gene and gene-modified cell therapies to treat and cure diseases with significant unmet medical needs. Our proprietary technologies allow for the creation of multimodal treatments that aim to substantially enhance clinical safety and efficacy. We have a number of products in preclinical development, and it is our mission to translate our proprietary technologies into meaningful therapies for the benefit of patients. We are fully committed to creating high-value therapeutic assets, while developing strategies to ensure broad accessibility of our treatments, especially to resource poor communities. Our patent-protected technologies are versatile, highly effective and broadly applicable to various therapeutic areas. We believe that these technologies will allow us to create both first-in-class and best-in-class therapies. We recognize that strengthening our IP portfolio is central to the commercial success of Antion, and strive to enhance and protect our core technologies further. We actively explore collaborations and partnering opportunities to out-license our proprietary technologies and in-license novel technologies that are commercially important to us. Antion is founded by a team of leading researchers, entrepreneurs and financial executives. Our founders and researchers are recognized scientists and clinicians from the Universities of Geneva, Zurich and Pretoria, with proven track records in molecular medicine and gene engineering. The team has a long collaborative history and previous success in establishing other biotech ventures. Antion’s executive team has broad experience in financial administration, trading, fundraising, initial public offerings, and mergers and acquisitions.

Waltham, Massachusetts, United States of America

Apellis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the development of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases. Its lead product candidate is pegcetacoplan that is in Phase III clinical trials for the treatment of geographic atrophy in age-related macular degeneration and paroxysmal nocturnal hemoglobinuria diseases; and in Phase II clinical trials for the treatment of cold agglutinin disease, C3 glomerulopathy, IgA nephropathy, primary membranous nephropathy, and lupus nephritis. The company is also developing APL-9, which is in single ascending dose Phase I randomized, double-blind, and placebo-controlled clinical trials for the prevention of immune system activation coincident with adeno-associated virus for intravenous administration. Apellis Pharmaceuticals, Inc. has a strategic collaboration with Swedish Orphan Biovitrum AB (publ) for the advancement of pegcetacoplan, a C3 therapy for the treatment of multiple rare diseases, including paroxysmal nocturnal hemoglobinuria. The company was founded in 2009 and is based in Waltham, Massachusetts.

Vienna, Austria

APEPTICO is a privately-held biotechnology company based in Austria, developing peptide-based products targeting chronic and life-threatening diseases. The peptide molecules correspond to validated, pharmacodynamic active structures and domains of well-known proteins and biopharmaceuticals. By concentrating on synthetically produced protein structures APEPTICO avoids general risks associated with gene- and cell-technologies. APEPTICO makes use of its technology platforms PEPBASE(TM) and PEPSCREEN(TM) to significantly reduce cost and to shorten time to market.

New York, New York

Apertura is a biotechnology company opening opportunities in gene therapy for treating debilitating diseases with limited options. We are pioneering next-generation AAV gene therapies by simultaneously engineering capsids, regulatory elements, and payloads to overcome current limitations in genetic medicine. Founded on technologies from the Broad Institute and Harvard University, and with support from Deerfield Management Company, we are located at the Cure in New York City.

Rissa, Norway

APIM Therapeutics is a venture capital financed Norwegian biotech company focusing on the development of novel treatments in the fields of oncology and inflammation. Our lead drug, ATX-101, is a first-in-class peptide drug candidate targeting a novel therapeutic intervention point involved in cellular stress defense mechanisms. ATX-101 is currently being tested in a phase I study in patients with advanced solid tumors.

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