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Arkuda Therapeutics is a biotechnology company applying insights into lysosomal biology to drive the development of medicines to change the trajectory of neurodegenerative disease. Arkuda`s lead program ARKD-104 aims to correct progranulin deficiency and lysosomal dysfunction in patients with GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the progranulin (GRN) gene. The company is further exploring the therapeutic potential of its molecules in other neurodegenerative diseases where genetic links to dysfunction in progranulin biology have been established, including Alzheimer`s Disease and Parkinson`s Disease. Arkuda is backed by leading investors including Atlas Venture, Cormorant Asset Management, Pfizer Ventures, funds managed by Tekla Capital Management LLC, Mission BioCapital, and Eli Lilly and Company.
Allyis Inc is a Bellevue, WA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
PCI Pharma Services is a global leader in Contract Development and Manufacturing Organization (CDMO) services, offering integrated solutions for drug development, manufacturing, and packaging. The company focuses on innovation and patient-centric approaches, supporting pharmaceutical and biopharmaceutical companies at various stages of drug development and commercialization. With 30 Good Manufacturing Practice (GMP) facilities across seven countries, PCI employs over 4,300 people dedicated to delivering life-changing therapies. The company provides a comprehensive range of services, including drug development and manufacturing for various dosage forms, advanced drug delivery systems, and complex packaging solutions. PCI also manages storage, distribution, and logistics to support the pharmaceutical supply chain and offers clinical trial supply chain management. Committed to growth, PCI has made significant investments, including a $100 million expansion in New England to enhance its global capacity and capabilities.
The mission of 4D Molecular Therapeutics is to design and develop transformative gene therapy products using our proprietary technology to create novel adeno-associated virus variants to be utilized as treatments for serious unmet medical conditions. Our products are unlocking the full potential of gene therapy to treat, and potentially cure, genetic diseases. A large number of patients and diseases, who previously were not treatable by gene therapy, will be addressable by 4D products. 4D has one of the deepest and most diverse product pipelines in the gene therapy field. Adeno-associated virus (AAV) vectors have emerged as a favored delivery vehicle for gene therapy in the human body. They can deliver the genes for therapeutic proteins to accessible tissues in the body and are generally considered safe. Several AAV gene therapy products are in late-stage clinical development, and one product is approved in the EU (Glybera, Uniqure). However, these first-generation AAV vectors have limited utility for the majority of diseases. Many of these common AAV were discovered as laboratory contaminants and monkey infections, for example; they have not been customized as targeted medicines. In contrast, 4D customized AAV vectors, and the products made from them, are designed to unlock the full potential of gene therapy. Through 4D`s Therapeutic Vector Evolution, we are able to create customized vectors solutions to overcome the hurdles identified with first-generation AAV vectors. 4D is creating the ultimate gene therapy products to cure genetic diseases using new customized delivery vehicles (vectors) to shuttle genes into the cells in any organ in the body. This 4D “library” of vectors will soon include several for the liver, several for the brain, several for the heart, the eye, muscle, and so on. Doctors will be able to hand pick a vector for any patient based on the organ that is diseased.
SuperGen Inc. is a Dublin, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.