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Passage Bio was launched with the vision to be a first in class fully-integrated biotech company developing life transforming AAV-delivered in vivo therapeutics for the treatment of rare CNS diseases. In collaboration with the University of Pennsylvania Gene Therapy Program, Philadelphia-based Passage Bio plans to advance a portfolio of 5 rare disease indications in the neuro/CNS genetic space through IND-enabling studies using best-in-class AAV technology and know-how. Subsequently, Passage Bio will be responsible for all clinical development and will collaborate with Penn`s Orphan Disease Center to support these efforts.
Aldeyra Therapeutics, Inc., is a biotechnology company focused primarily on the development of products to treat diseases thought to be related to endogenous free aldehydes, a naturally occurring class of toxic molecules. The company has developed NS2, a product candidate designed to trap free aldehydes. Aldeyra plans to begin clinical testing of NS2 in 2014 for the treatment of Sjögren-Larsson Syndrome and acute anterior uveitis. NS2 has not been approved for sale in the U.S. or elsewhere.
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum`s proprietary product engine, FulcrumSeek™, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.
Diasome Pharmaceuticals, Inc. is focused on the clinical and commercial development of breakthrough therapies for diabetes and obesity.
Targacept`s clinical pipeline is built on a unique class of therapeutic targets.