Sarepta Therapeutics

Arrowhead Pharmaceuticals is a biotechnology company focused on creating medicines for diseases with a genetic basis driven by overproduction of proteins. The company develops RNA interference therapeutics designed to silence disease-related genes and reduce pathogenic protein production. Its pipeline includes candidates such as ARO-AAT for liver disease caused by alpha-1 antitrypsin deficiency, ARO-APOC3 for high triglycerides, ARO-ANG3 to lower angiopoietin-like protein 3, ARO-HSD for liver disorders, ARO-ENaC to modulate airway sodium channels, ARO-Lung2 for chronic obstructive pulmonary disease, and ARO-COV for pulmonary infections, with ARO-HIF2 in earlier stage for certain kidney cancer. It is also pursuing programs like JNJ-3989 for chronic hepatitis B, Olpasiran for apolipoprotein A production, and ARO-AMG1 for cardiovascular targets. The company collaborates with Janssen and Takeda on RNAi therapeutics and was formerly Arrowhead Research Corporation, renamed Arrowhead Pharmaceuticals in 2016 after being established in 1989. It is headquartered in Pasadena, California.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, focused on developing innovative genetic medicines for rare diseases. Founded in 2015, the company utilizes a proprietary structure-inspired adeno-associated virus (AAV) vector engineering platform to create unique and differentiated vectors. These vectors are designed to improve upon naturally occurring AAV serotypes, addressing limitations of first-generation gene therapies. StrideBio's technologies facilitate gene addition, silencing, and editing, providing potential curative options for patients with severe medical conditions. By combining structural insights with accelerated evolution, StrideBio aims to produce novel AAV capsids capable of evading neutralizing antibodies, ultimately enhancing treatment outcomes for those affected by rare diseases.

AavantiBio, Inc. is a biopharmaceutical company focused on developing gene transfer and gene editing therapies aimed at treating rare genetic diseases. Incorporated in 2019 and based in Gainesville, Florida, the company primarily concentrates on Friedreich’s Ataxia, a rare inherited disorder that leads to significant cardiac and central nervous system dysfunction. AavantiBio is committed to transforming the lives of patients suffering from this condition by advancing innovative therapies that address unmet medical needs in the field of genetic disorders. The company’s efforts are directed toward both treatment development and research, with the goal of providing effective solutions for patients facing the challenges of rare diseases.

Myonexus Therapeutics is a clinical stage company focused on developing gene therapies for rare diseases, specifically targeting Limb-girdle muscular dystrophy (LGMD) Types 2D, 2B, 2E, 2L, and 2C. The company is rooted in research conducted at Nationwide Children’s Hospital, which is recognized for its expertise in muscular dystrophy gene therapy and translational research. Myonexus aims to pioneer the first effective treatments for these specific LGMD types, utilizing advanced precision genetic medicine approaches. In addition to LGMD, the company’s innovative therapies show potential for addressing other genetic disorders, including Charcot-Marie-Tooth disease, MPS IIIA, and Pompe disease, among others.

Lacerta Therapeutics, Inc. is a clinical-stage gene therapy company based in Alachua, Florida, focused on developing innovative treatments for central nervous system and lysosomal storage diseases. Established in 2017, the company leverages a unique array of proprietary adeno-associated virus (AAV) vector technologies to advance its therapeutic programs. Lacerta Therapeutics is distinguished by its expertise in the development, production, and application of AAV vectors, utilizing proprietary capsid variants and a scalable vector manufacturing platform. The founders and team possess extensive experience in AAV gene therapy, contributing to the company's capabilities in basic vector development and clinical trial execution. Through these advancements, Lacerta Therapeutics aims to provide effective treatment options for patients suffering from neurological and lysosomal storage disorders.

Myonexus Therapeutics is a clinical stage company focused on developing gene therapies for rare diseases, specifically targeting Limb-girdle muscular dystrophy (LGMD) Types 2D, 2B, 2E, 2L, and 2C. The company is rooted in research conducted at Nationwide Children’s Hospital, which is recognized for its expertise in muscular dystrophy gene therapy and translational research. Myonexus aims to pioneer the first effective treatments for these specific LGMD types, utilizing advanced precision genetic medicine approaches. In addition to LGMD, the company’s innovative therapies show potential for addressing other genetic disorders, including Charcot-Marie-Tooth disease, MPS IIIA, and Pompe disease, among others.

Eisai is a global human healthcare company specializing in integrated pharmaceutical businesses focused on oncology and neurology, dementia-related diseases, and neurodegenerative disorders. Established in Japan, Eisai operates globally with a commitment to patient-centric care and innovation.

Ercole Biotech, Inc. is a research-stage biopharmaceutical company based in Research Triangle Park, North Carolina. Founded in 2002, the company specializes in the development of oligonucleotide drugs that achieve therapeutic effects by directing the alternative splicing of target genes. Ercole Biotech focuses on manufacturing these innovative medicines to treat various diseases through the modulation of target genes. As of March 2008, it operates as a subsidiary of AVI Biopharma, Inc.