List of Oligonucleotides Companies with Phase 2 Active Clinical Trial - 18

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.

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

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.

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

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

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

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.

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

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.

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.

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.

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.

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.

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

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

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.

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.

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.