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progressive cone dystrophy in pde6c associated achromatopsia with a likely pathogenic variant and a vus
Original title: “Progressive cone dystrophy in PDE6C-associated achromatopsia with a likely pathogenic variant and a VUS.”
Over the next 18 months, the ... phase 1 clinical trial and preparing the investigational new drug application for submission to the FDA. Blue Gen Therapeutics Foundation also licensed ADVM-082, an intravitreal gene therapy that encodes a functional CNGB3 gene for the treatment of achromatopsia, another rare disease that leads ... Over the next 18 months, the Children’s Hospital Los Angeles and the Blue Gen Therapeutics Foundation will conduct the work to prepare for clinical trials for BGTF-027, including developing a protocol for a phase 1 clinical trial and preparing the investigational new drug application for submission to the FDA. Blue Gen Therapeutics Foundation also licensed ADVM-082, an intravitreal gene therapy that encodes a functional CNGB3 gene for the treatment of achromatopsia, another rare disease that leads to vision that has no color. The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene therapy for boys with blue cone monochromacy, which impairs color vision and severely affects visual function. ... received a $4.7 million grant from the California Institute for Regenerative Medicine to study a gene therapy to treat blue cone monochromacy (BCM), a rare, inherited vision disorder linked to the X chromosome that occurs in boys. Currently, there is no treatment for this disease. Aaron Nagiel, M.D., Ph.D., an investigator conducting ophthalmology and neuroscience research at Children’s Hospital Los Angeles, will lead efforts on research of a gene therapy that aims to slow the progression of disease and restore vision loss. Nagiel is also an assistant professor of Clinical Ophthalmology at Keck School of Medicine of USC. licensed to Blue Gen Therapeutics Foundation (BGTF), a nonprofit organization developing gene therapies for rare inherited retinal diseases. Blue Gen Therapeutics Foundation was co-founded by John Cavitt, who has three children affected by blue cone monochromacy. BGTF-027 is an intravitreal gene therapy that encodes a functional copy of the long-wavelength-sensitive opsin protein for the treatment of blue cone monochromacy.
Original title: “Gene Therapy for Rare Eye Disease to Advance to Human Trial | Managed Healthcare Executive”