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Speaker: Gene therapy well-suited for retinal disease treatment
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LAS VEGAS — Safety results in the phase 2a trial of AVA-101 support ocular gene therapy as a potential long-term treatment option for wet age-related macular degeneration, according to a presentation here. There is, however, still a need for anti-VEGF rescue therapy.
Jeffrey S. Heier, MD, related topline results from the phase 1 and 2a clinical trials of AVA-101 (Avalanche Biotechnologies) at Retina Subspecialty Day preceding the American Academy of Ophthalmology annual meeting.
Jeffrey S. Heier
Heier accounted for unsatisfactory phase 1 results that showed a difference in visual acuity between treatment and control groups, saying, “This was largely driven by poor performance by the control.” There was also an increase in retinal thickness in the treatment arm and a decrease in the control arm.
In the phase 2a clinical trial, in which 21 patients received AVA-101 with 0.5 mg of ranibizumab as a rescue therapy and 11 patients received 0.5 mg of ranibizumab as rescue therapy only, the gene therapy was well-tolerated with no significant drug-related safety concerns.
Heier said the five takeaway points he derived from the clinical trials are these: surgical trials for treatment of medical diseases are extremely difficult to design; the control arm of a surgical trial is difficult to implement; the variability of surgery is difficult to control; when the expected doesn’t occur, go back to the basics; and gene therapy remains well-suited for the management of retinal disease.
“The retina is ideally suited for developing gene-based therapies,” Heier said. “The cell types are readily accessible, the eye is relatively immune-privileged, and there are noninvasive assessments. And the rationale for sustained delivery is strong.” — by Kristie L. Kahl
Disclosure: Heier reports he is a consultant for Avalanche.