Sarepta Therapeutics

Arrowhead Pharmaceuticals, Inc. is a biotechnology company based in Pasadena, California, focused on developing innovative medicines for the treatment of intractable diseases, particularly those with a genetic origin characterized by the overproduction of specific proteins. The company's diverse pipeline includes RNA interference (RNAi) therapeutics such as ARO-AAT for liver diseases associated with alpha-1 antitrypsin deficiency, ARO-APOC3 for hypertriglyceridemia, and ARO-HIF2 for clear cell renal cell carcinoma, among others. Arrowhead is also exploring treatments for various conditions, including chronic hepatitis B virus infection and cardiovascular diseases. The company collaborates with prominent pharmaceutical firms, including Janssen Pharmaceuticals and Takeda Pharmaceuticals, to develop additional RNAi therapeutic candidates. Originally founded as Arrowhead Research Corporation in 1989, it adopted its current name in April 2016.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, established in 2015. The company focuses on developing innovative adeno-associated viral (AAV) vector technologies and therapeutics aimed at treating rare diseases. Utilizing a proprietary platform that combines structural insights with accelerated evolution, StrideBio creates novel AAV capsids designed to evade neutralizing antibodies. This approach allows for enhanced gene addition, gene silencing, and gene editing capabilities, addressing the limitations of first-generation gene therapies. By engineering unique and differentiated vectors, StrideBio aims to improve treatment outcomes for patients facing devastating conditions.

AavantiBio, Inc. is a biopharmaceutical company based in Gainesville, Florida, focused on developing gene transfer and gene editing therapies for rare genetic diseases. Established in 2019, the company primarily targets Friedreich’s Ataxia, a severe inherited condition that leads to significant cardiac and central nervous system dysfunction. In addition to addressing this specific disorder, AavantiBio is advancing a pipeline of innovative gene therapies aimed at treating various autoimmune diseases and other conditions with unmet medical needs. Through its commitment to research and development, the company seeks to improve the lives of patients affected by these debilitating genetic 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's innovative treatments are based on research conducted at Nationwide Children’s Hospital, which is recognized for its leadership in muscular dystrophy gene therapy discovery and translational research. In addition to LGMD, Myonexus is also engaged in developing therapies for other conditions, including Charcot-Marie-Tooth disease and various CNS-related disorders, utilizing advancements in precision genetic medicine to address unmet medical needs in these areas.

Lacerta Therapeutics is a clinical-stage gene therapy company based in Alachua, Florida, focused on developing treatments for central nervous system and lysosomal storage diseases. Established in 2017, the company utilizes a range of proprietary adeno-associated virus (AAV) vector technologies to advance its clinical programs. Lacerta's expertise in AAV vector development, production, and application allows for the identification of novel capsid variants and supports a robust, scalable vector manufacturing platform. The team's extensive experience in gene therapy and clinical trials enhances its capacity to create targeted treatments for neurological and lysosomal storage disorders, aiming to improve patient outcomes in these challenging medical areas.

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's innovative treatments are based on research conducted at Nationwide Children’s Hospital, which is recognized for its leadership in muscular dystrophy gene therapy discovery and translational research. In addition to LGMD, Myonexus is also engaged in developing therapies for other conditions, including Charcot-Marie-Tooth disease and various CNS-related disorders, utilizing advancements in precision genetic medicine to address unmet medical needs in these areas.

Eisai, established in 1941, is a global human healthcare company headquartered in Japan. It operates as an integrated pharmaceutical business, focusing on two core therapeutic areas: oncology and neurology, including dementia-related and neurodegenerative diseases. Eisai's business spans multiple regions, with significant operations in Japan, the Americas, China, Asia, and Europe, Middle East, and Africa. The company's portfolio comprises prescription medicines for various indications, including central nervous system disorders, metastatic breast cancer, and diabetes. Eisai India, a 100% subsidiary, was the first Japanese company to enter the Indian pharmaceutical market, offering a range of products and maintaining a dedicated field force. Eisai's manufacturing and research complex in Vizag, India, produces drug substances, drug products, and conducts developmental research, with certifications from regulatory bodies such as USFDA, PMDA, MHRA, and WHO. Eisai Inc., its U.S. subsidiary, is committed to addressing unmet healthcare needs and fostering strong alliances. Eisai Innovation, a strategic investment organization, seeks synergies between the scientific community and Eisai's network.

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 modulate gene expression through alternative splicing, aiming to treat various diseases. Ercole Biotech operates as a subsidiary of AVI Biopharma, Inc. and focuses on innovative therapeutic solutions by targeting specific genes to achieve desired medical outcomes.