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MBX Biosciences is a preclinical-stage biotechnology company using validated molecular targets to create medicines for rare endocrine diseases where current therapies are inadequate.
ADMA Biologics, Inc. is a late-stage biopharmaceutical company that develops, manufactures and intends to market specialty plasma-based biologics for the treatment and prevention of infectious diseases. The Company is engaged in the development and com...
Abeona Therapeutics (Nasdaq: ABEO) is a fully-integrated gene and cell therapy company at the forefront of the rapidly-advancing field of genetic medicine. The Company`s multi-platform expertise across the manufacture, delivery, development, and discovery of novel gene and cell therapies has it uniquely positioned for success. Underpinning the Company`s robust pipeline is its fully-operational manufacturing facility producing therapies and vectors for preclinical and clinical studies. Abeona is also developing the AIM™ Vector Platform: 100+ next-generation AAV capsids for delivering gene therapies targeting a wide range of organs and multiple routes of delivery. A robust and diverse pipeline is led by a novel gene-corrected cell therapy poised to enter Phase 3 in mid-2019 and complemented by one-time gene therapy candidates across four lysosomal storage diseases. Several preclinical discoveries are led by an emerging program in cystic fibrosis that uses the AIM vector platform and a capsid that has shown potential across inherited and acquired retinal diseases.
Poseida Therapeutics is a clinical-stage biotechnology company translating best-in-class technology into lifesaving cell and gene therapies for patients with high unmet medical need. The company is developing CAR T-cell immunotherapies for multiple myeloma, prostate and other cancer types, as well as in vivo gene therapies for orphan genetic diseases. These cell therapies and the gene therapies soon to come are products of our proprietary suite of industry-leading gene engineering technologies, including the piggyBac® DNA Modification System, Cas-CLOVER™ and TAL-CLOVER™ site-specific nucleases.
Scorpion Therapeutics was founded to expand the reach of precision medicine to more people with cancer.