Listings in Gene and Cell Therapy
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Expression Therapeutics is a biotechnology company based in Atlanta with manufacturing in Ohio. Our therapeutic pipeline includes advanced gene therapies for hemophilia A and B; neuroblastoma, T-cell leukemia/lymphoma, and acute myeloid leukemia (AML); and hematologic disorders such as hemophagocytic lymphohistiocytosis...
Forge Biologics is a gene therapy development engine, focused on enabling access to life-changing gene therapies and helping bring them from idea into reality. We partner with innovators in the gene therapy community: scientists, physicians, biotech/pharma companies, and patient groups....
GRAIL is a healthcare company whose mission is to detect cancer early, when it can be cured. GRAIL is focused on alleviating the global burden of cancer by using the power of next-generation sequencing, population-scale clinical studies, and state-of-the-art machine...
Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases. Our primary focus is on rapidly advancing Duchenne muscular dystrophy (DMD) therapies that are designed to treat the underlying...
Trailhead Biosystems is a biotechnology company headquartered in Greater Cleveland, Ohio. We develop industrial-grade human cells from stem cells at a much faster rate, at dramatically lower cost, and with less R&D risk than is currently the norm in the...
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) - a rare, life-threatening genetic...
AdopTracell is a biotech/pharmaceutical start-up company based upon patented foundational technologies. This university spinoff company primarily focuses on development of chimeric antigen receptor (CAR) regulatory T cells (Treg) capable of recognizing relevant human autoantigens resulting in effector T cell (Teff)...
BioHeights LLC is a consulting company that specializes in business strategy for novel therapeutics. We pride ourselves on deep knowledge of the gene and cell therapy space. We are here to help small-, mid-, and large-size biopharmaceutical companies, startups, and...
Cell X Technologies, Inc., founded in 2017, is a spin-out of the Cleveland Clinic by stem cell scientist and Orthopedic Surgeon, Dr. George Muschler. The company's initial product, "Cell X," is an automated robotic cell processing platform, designed and engineered...
Copernicus Therapeutics, Inc. is a trial phase biopharmaceutical company utilizing newly developed nanoparticle technologies to produce safe and effective DNA drugs. Copernicus' recent Phase I/II cystic fibrosis therapeutic clinical trial results demonstrate that DNA nanoparticles efficiently transfer their DNA payload...