November 2, 2015 7:33am
Achromatopsia results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.
AGTC has filed an IIND application with the U.S. Food and Drug Administration (FDA) to conduct a P1/2 clinical trial of its gene therapy product candidate for the treatment of achromatopsia caused by mutations in the CNGB3 gene.
The Bottom Line: This is another significant milestone for AGTC pending the FDA's acceptance of the IND application and approval from the sites' Institutional Review Boards.
AGTC had closed DOWN -$0.20 at $12.00 and has had a mixed number up days, weeks and months in the “pricing” category emerging as a trading vehicle versus appreciating to conform to the $20.62 investment by Biogen (BIIB) in July. A "coiled spring" of appreciation.
The incidence rate for achromatopsia is approximately one in 30,000 people, and it is estimated that there are approximately 10,000 people in the United States and 17,000 people in Europe with achromatopsia. Mutations in the CNGB3 gene cause the disease in approximately 50% of patients with achromatopsia.
