Clinical Articles

Stem cell therapy clinical trial demonstrates safety and potential efficacy in small numbers of patients

Positive preliminary findings have been reported from Phase I and Phase 2 clinical research using retinal pigment epithelial (RPE) cells derived from embryonic stem cells. The studies have indicated that patients with AMD and Stargardt’s may have early signs of vision gain following the stem cell treatment. Dr. Eyal Banin, from Hadassah-Hebrew University Medical Centre, […]

Read full story

New geographic atrophy drug for AMD fails to meet primary endpoint in age-related macular degeneration

A new antibody drug (lampalizumab) targeting geographic atrophy in age related macular degeneration has missed its primary endpoint in two pivotal Phase III studies comprising >1,800 patients. The drug, sponsored by Genentech (Roche), was designed to target a key molecular component in the complement pathway, understood to be a critical pathway leading to the accumulation […]

Read full story

Patient reported outcomes in retinal gene therapy study make significant impact on regulatory assessment

An October meeting of the Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee has unanimously recommended (16-0) approval of a new treatment, “LUXTURNA” (voretigene neparvovec), a gene therapy for the treatment of patients with biallelic RPE65-mediated inherited retinal disease. The approval of the new treatment was supported by patient testimony, understood […]

Read full story

Related Clinical News