Adverum Biotechnologies Announces Publication of Preclinical Long-Term Expression and Efficacy Data in Molecular Therapy

Adverum Biotechnologies (Nasdaq:ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ophthalmology and rare diseases, today announced the publication of preclinical long-term expression and efficacy data in Molecular Therapy, the leading international journal for research on the development of molecular and cellular therapeutics to correct genetic and acquired diseases.

As quoted in the press release:

The multi-center, open-label, phase 1, dose-escalation trial is designed to assess the safety and tolerability of a single intravitreal (IVT) administration of ADVM-022 in patients with wet AMD who are responsive to anti-vascular endothelial growth factor (VEGF) treatment. At least six leading retinal centers across the United States are expected to participate in the phase 1 trial. The trial is expected to enroll 18 patients and evaluate three doses of ADVM-022; first dose: 6 x 10^11 vg/eye, second dose: 2 x 10^12 vg/eye, and third dose: 6 x 10^12 vg/eye. Patients will be administered a tapering prophylactic corticosteroid regimen. The primary endpoint of the trial is the safety and tolerability of ADVM-022 at 24 weeks after a single IVT administration. Secondary endpoints include changes in best-corrected visual acuity (BCVA), measurement of central retinal thickness (CRT), as well as mean number of rescue anti-VEGF injections and percentage of patients needing rescue anti-VEGF injections.

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