Sarepta Therapeutics

Arrowhead Pharmaceuticals is a biotechnology company that focuses on the development of medicine to treat diseases with a genetic origin, characterized by the overproduction of one or more proteins. Its medical solutions are aimed at the genes that trigger the diseases. Its portfolio includes drugs targeting hepatitis B, Alpha-1 Antitrypsin Deficiency, thrombosis and angioedema, clear-cell and renal cell carcinoma, and cardiovascular disease.

LEXEO Therapeutics is a biotechnology company that specializes in the development of gene therapies, particularly those utilizing adeno-associated virus (AAV) technology. The company’s pipeline includes both rare and non-rare monogenic diseases, with a strong emphasis on hereditary and acquired conditions that present significant unmet medical needs. LEXEO's lead programs and preclinical candidates are primarily developed in collaboration with Weill Cornell Medicine’s Department of Genetic Medicine. The company aims to advance its clinical programs toward commercialization while maintaining an ongoing research partnership with Weill Cornell to enhance its preclinical efforts. Through its innovative approach, LEXEO Therapeutics seeks to transform the treatment landscape for diseases that impose a substantial burden on patients and society.

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.

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.