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Company Description (as filed with the SEC)
We are a leader in the field of gene therapy, seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. The goal of gene therapy is to overcome the effects of a malfunctioning, disease-causing gene. Our product candidates have the potential to provide long-lasting effects, dramatically and positively changing the lives of patients with conditions where no, or only palliative, therapies exist. Our initial focus is on treating orphan diseases, and we recently reported statistically significant results in a pivotal Phase 3 clinical trial of our first product candidate targeting rare genetic blinding conditions, which has received both breakthrough therapy and orphan product designation. Based on these positive results, we intend to submit a Biologics License Application, or BLA, for this product candidate with the U.S. ... More ...
Where does ONCE fit in the risk graph?
|Annual EPS Est:||$-2.1|
|Quarterly EPS Est:||-1.13|