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Palvella Therapeutics was founded with the single-minded goal of serving individuals suffering from serious rare genetic diseases with no approved treatments.
Navitor Pharmaceuticals, Inc., is a biopharmaceutical company developing novel medicines by targeting cellular nutrient signaling pathways. The companys proprietary drug discovery platform targets mTORC1, which responds to and integrates the cells response to nutrient availability and plays a key role in protein synthesis and cellular growth. Navitors therapeutics are designed to selectively modulate the cellular signals that are aberrant in disease processes caused by the dysregulation of mTORC1 activity to address a wide range of diseases, including metabolic, neurodegenerative, autoimmune and musculoskeletal diseases, as well as several rare disorders. The companys founding intellectual property is based on the groundbreaking discoveries related to the mTORC1 pathway and nutrient signaling mechanisms by Dr. David Sabatini at The Whitehead Institute for Biomedical Research. The company is backed by leading financial and corporate investors, including Polaris Partners, Atlas Venture, Johnson and Johnson Development Corporation, SR One and The Longevity Fund.
Celula Inc is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
AlloVir, formerly ViraCyte, was founded in 2013 and is the leader in the development of novel cell therapies with a focus on restoring natural immunity against life-threatening virus-associated diseases in patients with severely weakened immune systems.
GenSight Biologics is is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics` pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics` lead product candidate, GS010, is in Phase III trials in Leber`s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics` product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.