Kyowa Kirin

Garuda Therapeutics is a biotechnology company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening blood, bone marrow, immune, and metabolic diseases. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants. Utilizing advanced technologies, including zebrafish, mice, and human-induced pluripotent stem cells, Garuda Therapeutics conducts research to clone and analyze novel genes and mechanisms critical for blood formation. This innovative approach is designed to provide patients with consistent, durable, and HLA-compatible transgene-free blood stem cell therapies, offering rapid and broad access to life-saving treatments.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Ring Therapeutics is a biotechnology company developing gene therapies using Anellovector, a redosable and targetable DNA therapy platform based on the biology of the human commensal virome. The platform aims to address limitations of current DNA therapies, including limited tissue access, inability to redose, potential genomic integration, and tolerability concerns. Founded in 2017 and based in Cambridge, Massachusetts, Ring Therapeutics was established by Flagship Pioneering to pursue a multi-product platform. It focuses on discovering and developing Anellovector therapeutics to unlock the full potential of gene therapy, moving beyond gene replacement to a broader set of modalities and mechanisms. The approach targets disease sites with tissue and cellular specificity, aiming to treat conditions across genetic disorders, ophthalmology, oncology, and metabolic disorders and to expand therapeutic options for previously inaccessible diseases.

Cellarity Inc is a therapeutics company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines through the analysis of cellular behaviors. Established in 2017, the company employs advanced genomic technologies, data science, and artificial intelligence to create a comprehensive platform that digitizes molecular information within cells. This approach allows Cellarity to investigate how cells function in both health and disease, aiming to address the complexities of various medical conditions. By leveraging single-cell technologies and machine learning, the company seeks to enhance the success rate and efficiency of drug discovery, ultimately designing therapies that effectively target the intricate dynamics of cellular behavior.

Ardelyx is a biopharmaceutical company developing first-in-class, minimally absorbed oral medicines to treat mineral metabolism and metabolic disorders. Based in Fremont, California, founded in 2007. It focuses on non-systemic therapies that act in the intestines to modulate transporters and receptors, aiming to limit systemic exposure. Its lead product Tenapanor targets irritable bowel syndrome with constipation and hyperphosphatemia in chronic kidney disease on dialysis; the program includes Phase 3 trials. The company is pursuing additional candidates such as RDX013 for hyperkalemia, and RDX002 and RDX009 in earlier stages. Ardelyx aims to address cardiorenal and metabolic conditions using a non-systemic platform.

ProStrakan is a rapidly growing specialty pharmaceutical company engaged in the development and commercialisation of prescription medicines for the treatment of unmet therapeutic needs in major markets. With more than 300 people employed by the Company, ProStrakan is headquartered in Scotland with commercial operations throughout the rest of the UK and continental Europe and a US head office. The Company's Development group is located in Galashiels, Scotland and Bedminster, New Jersey, US.

TargaZyme, Inc. is a clinical-stage biotechnology company specializing in the development and commercialization of innovative products in cell and immunotherapy. Founded in 2005 and headquartered in Floresville, Texas, with additional offices in San Diego, California, and Basel, Switzerland, TargaZyme's flagship products, TZ101 and TZ102, enhance the migration and retention of therapeutic cells to target tissues. TZ101 is designed for various applications, including cancer treatment, ischemic diseases, and trauma, by improving the attachment of therapeutic cells to selectin proteins associated with inflammation and tissue damage. TZ102 focuses on neurodegenerative diseases and hematological malignancies, enhancing the effectiveness of specific cell types. The company's technology aims to improve clinical outcomes in stem cell transplantation, immunotherapy, regenerative medicine, and gene therapy, targeting significant health issues such as heart failure, diabetic retinopathy, and multiple sclerosis. TargaZyme was previously known as American Stem Cell, Inc. before rebranding in December 2013.