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According to Outlook, in the FDA’s recently issued CRL it concluded it could not approve the BLA during this review cycle due to several CMC issues, open observations from pre-approval manufacturing inspections, and a lack of substantial evidence.

The research will focus on the compound's impact on biomolecular condensates, which are implicated as a key driver of pathology in neurodegeneration and diseases impacting high metabolic organs. Because the retina is one of the highest energy consuming systems in the human body, deficits in energy supply can be catastrophic.