Gene Therapy for Wet AMD Begins Dosing in Cohort 4 of Trial

As reported in Biospace, Adverum Biotechnologies, a gene therapy company focused on clinical stage trials for rare and ocular diseases with unmet needs, has successfully dosed a patient in their Cohort 4 phase 1 clinical trial for a treatment for wet age-related macular degeneration(AMD). In the study (ADVM-022), the nine patients received a single injection of the gene and use steroid eye drops for six weeks.

Age-Related Macular Degeneration

Wet age-related macular degeneration(AMD) is a rare disease in which blood vessels grow improperly beneath the retina and macula at times bleeding or leaking. The excess of fluid or blood can lift the macula which can rapidly damage or destroy central vision. While the patient may rapidly and severely lose their forward vision, they typically can continue to maintain their peripheral vision.

The Next Trial Phase

The disease’s current treatment method consists of routine anti-VEGF injections, which patients need to receive frequently to prevent vision loss. The clinical trial alternatively presents a treatment option that would be a one-time treatment. The treatment would be injected in the same way, but could save them the constant visits. This switch would be particularly notable in the time of COVID-19 when there is more risk in traveling for treatment. Dr. David Boyer, senior partner of Retina-Vitreous Associates Medical Group and Professor at USC, said that the treatment “Could transform the treatment paradigm for wet AMD, particularly at this time when it is more important than ever to reduce the need for frequent injections and clinic visits.”

The team at Adverum has announced they’ve enrolled their first patient in Cohort 4, meaning they patient will receive a higher dose of the treatment in conjunction with the steroid eye drop prophylaxis, which they predict will be more effective for their gene therapy. Aaron Osborne, chief medical officer of the company, said “Patients in this Cohort are receiving the higher dose of ADVM-022, which has demonstrated outstanding efficacy and durability in Cohort 1.” They are continuing trials through COVID-19 social distancing rules, with extreme measures to ensure safety in the labs. They believe now more than ever that the one time treatment would be a better option.


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