Updated: January 4, 2017
MA09-hRPE cell implantation is a regenerative approach to treating retinal disease being developed by Astellas and Ocata. The primary cause of Stargardt disease is the buildup of toxic vitamin A aggregates due to a genetic defect resulting in damage to retinal tissue. MA09-hRPE aims to correct this via injection of human stem cell derived RPE cells, with the goal of regenerating the damaged tissue.
Stargardt Disease: Phase II
Stargardt disease (or fundus flavimaculatus) is the most common form of inherited macular degeneration, with a prevalence of about 1 per 10,000 births. The disease causes uncorrectable loss of central vision, with most patients experiencing initial symptoms prior to age 20.
Stargardt disease has been shown to be the result of a mutation in the ABCA4 (or ABCR) gene. This mutation leads to production of a dysfunctional Rim protein, which is responsible for the intracellular transport of vitamin A. As a result, vitamin A accumulates and forms toxic aggregates within the photoreceptors, leading to eventual vision loss. Subretinal MA09-hRPE injections deliver healthy human stem cell-derived RPE cells into the retina, allowing repair of damaged photoreceptors.
There is currently no cure or treatment for Stargardt disease, but competitors are developing alternatives to MA09-hRPE such as ALK-001 by Alkeus and SAR 433459 by Sanofi and Oxford Biomedica.
Dry Age-Related Macular Degeneration (Dry AMD): Phase II
Age-related macular degeneration is a type of vision loss typically affecting older people. It results in blurring or blocking of the central visual field, but does not progress to complete blindness. Dry AMD is caused by retinal apathy.
Astellas has completed a Phase II clinical trial for the treatment of Dry AMD with MA09-hRPE cells. There are currently no other cures or treatments available for the disease.
Managed Care Segment
The managed care segment for MA09-hRPE is 2B.
Selected Sample of Epidemiology Sources Used in This Analysis
Bocquet B, Lacroux A, Surget MO, et al. Relative frequencies of inherited retinal dystrophies and optic neuropathies in Southern France: assessment of 21-year data management. Ophthalmic Epidemiol. 2013;20(1):13-25.
Chacón-camacho OF, Granillo-alvarez M, Ayala-ramírez R, Zenteno JC. ABCA4 mutational spectrum in Mexican patients with Stargardt disease: Identification of 12 novel mutations and evidence of a founder effect for the common p.A1773V mutation. Exp Eye Res. 2013;109:77-82.
(See full report for complete source list)
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