Kyowa Kirin

Garuda Therapeutics develops off-the-shelf hematopoietic stem cell therapies to treat life-threatening diseases. It seeks to eliminate dependency on donor or patient cells for blood stem cell transplants, leveraging a platform technology that generates off-the-shelf, self-renewing blood stem cells that are HLA-compatible and transgene-free, providing patients with rapid and broad access to durable therapies.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

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 is a therapeutics company that employs genomic technologies, data science, and artificial intelligence to develop medicines by digitizing and analyzing cellular molecular information and behaviors. The company's platform combines single-cell technologies with machine learning to quantify how cells behave in health and disease, map the network dynamics that govern those behaviors, and design medicines that direct them. Founded in 2017 and based in Cambridge, Massachusetts, Cellarity aims to advance drug discovery by targeting cellular and molecular complexity to improve the speed and success rate of therapies.

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.