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Ortho-McNeil Pharmaceutical, Inc. is a Raritan, NJ-based company in the Healthcare, Pharmaceuticals, & Biotech sector.
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Graphite Bio is a next-generation gene editing company focused on the development of first-in-class therapies for patients suffering from life-threatening genetic diseases. The company`s platform harnesses the natural cellular process of homology directed repair (HDR) and targeted DNA integration to precisely repair genetic defects at their source. Graphite Bio is leveraging its differentiated platform to advance a portfolio of transformative genetic treatments with potential for saving and dramatically improving patients` lives
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.
miRagen Therapeutics, Inc., is a biopharmaceutical company discovering and developing innovative microRNA (miRNA)-targeting therapies to improve human health, specifically in disease areas of high unmet medical need. The company leverages in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance best-in-class technologies and high-potential drug candidates for its own pipeline or in conjunction with strategic partners. From its founding, miRagen has developed ongoing, collaborative relationships with academic institutions that give the company unique access to the most promising early-stage discoveries. This access to groundbreaking science provides a significant strategic advantage in the rapidly-developing field of miRNA-targeting therapeutics.