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Notable anticipated approvals for second quarter 2025 include: A cell-based gene therapy for epidermolysis bullosa, a rare genetic skin disorder First medicine for Menkes disease, a rare genetic metabolic disorder A new cellular therapy for patients with advanced melanoma By the end of summer of 2025, FDA decision is expected for six new gene and cellular therapies, four of which are seeking Accelerated Approval

None of these were symptomatic

Much of an administered dose is swallowed but not absorbed, as shown by fecal excretion studies

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