List of Antisense Oligonucleotides Companies with Phase 2 Active Clinical Trial - 11

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.

Bio-Path is a biotechnology company developing targeted therapies for acute myeloid leukemia (AML), lymphoma, chronic lymphocytic leukemia, gynecological cancer and other challenging cancers. The company’s lead product candidate, prexigebersen (Liposomal Grb2 Antisense), formerly BP1001, is currently being assessed in a Phase 2 clinical study in previously untreated AML patients, who are not eligible for or who have decided to forego intensive chemotherapy because of their fragile health, and in refractory/relapsed AML patients. Additionally, BP1001-A is being assessed in a Phase 1 study in refractory/relapsed patients with solid tumors. The Company’s second product BP1002 (Liposomal Bcl-2 Antisense) is being evaluated in Phase 1 trials in patients with refractory/relapsed lymphoma and AML.

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

Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation platform. The Encoded approach offers potentially unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders.

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.

Imvax is a clinical-stage biotechnology company developing personalized, whole tumor-derived immunotherapies. Imvax’s combination therapy uniquely captures the complete antigenic signature of a patient’s own tumor and converts it into a highly stimulatory ‘training program’ for the patient’s immune system. The comprehensive nature of this training is intended to result in both innate and adaptive immune stimulation to optimize long-term anti-tumor effects. The company’s lead program is in glioblastoma multiforme (GBM), an extremely aggressive form of brain cancer, with additional programs in other types of solid tumors.

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.

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.