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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.
Chroma Medicine is pioneering a new class of epigenetic medicines to revolutionize treatment of genetically driven disease.
Appia Bio is an early stage biotechnology company based in Los Angeles, California. Founded in 2020, Appia Bio is focused on discovering and developing engineered allogeneic cell therapies across a broad array of indications with a scalable technology platform that increases access for patients. With its ACUA (Appia Cells Utilized for Allogeneic) technology platform, Appia Bio leverages the biology of lymphocyte development with chimeric antigen receptor (CAR) and T-cell receptor (TCR) gene engineering to generate CAR-engineered invariant natural killer T (CAR-iNKT) cell subtypes from hematopoietic stem cells (HSCs).
GRAIL`s mission is to detect cancer early, when it can be cured.
Samumed is a leader in medical research and development for tissue-level regeneration. With our platform`s origins in small molecule-based Wnt pathway modulation, we develop therapeutics to address a range of degenerative diseases, regenerative medicine and oncology.