Sue Washer – VJRegenMed

The future of gene therapies for the eye

Simon Ng — Tue, 23 Nov 2021 15:28:47 +0000

Sue Washer, Applied Genetic Technologies Corporation, Alachua, FL, discusses future advancements within the field of ophthalmology gene therapies. With the advent of voretigene neparvovec, the first approved gene therapy for retinal diseases, the future of gene therapies treating vision loss is promising. Adeno-associated viruses (AAVs) additionally have potential uses other than as a vector, including delivering RNA and CRISPR-Cas 9 therapies. This interview took place at Meeting on the Mesa 2021.

Manufacturing challenges in the advanced therapies sector

Simon Ng — Tue, 23 Nov 2021 15:25:59 +0000

Sue Washer, Applied Genetic Technologies Corporation, Alachua, FL, outlines issues associated with manufacturing cell and gene therapies. The recent COVID-19 pandemic has affected the supply chain within the sector, as many materials and hardware are needed in the production of COVID-19 vaccines, limiting the supply for advanced therapy manufacturers. In the ophthalmological space, adeno-associated viruses (AAVs) cannot replicate themselves, limiting their capability as vectors in gene therapies for various conditions including X-linked retinitis pigmentosa (XLRP) and achromatopsia. This interview took place at Meeting on the Mesa 2021.

AAV-based gene therapy for X-linked retinitis pigmentosa

Simon Ng — Fri, 05 Nov 2021 09:09:29 +0000

Sue Washer, Applied Genetic Technologies Corporation, Alachua, FL, discusses the results of the Phase I/II and Phase II SKYLINE trials (NCT03316560) of a subretinal adeno-associated virus (AAV)-based gene therapy in patients with X-linked retinitis pigmentosa (XLRP). In the Phase I/II investigational portion of the trial, a majority of patients receiving a high dose reported an improvement in visual function and acuity, and in the SKYLINE trial, patients will receive further mobility tests to assess vision. An additional Phase III VISTA trial (NCT04850118) will be conducted to assess efficacy in another cohort. This interview took place at Meeting on the Mesa 2021.

Optogenetics in advanced retinitis pigmentosa

Simon Ng — Fri, 05 Nov 2021 09:09:28 +0000

Sue Washer, Applied Genetic Technologies Corporation, Alachua, FL, provides a brief overview of the development of an optogenetic adeno-associated virus (AAV)-based gene therapy in combination with a device that stimulates optic nerve cells for patients with advanced stage retinitis pigmentosa (RP). Patients with advanced RP may have no functioning photoreceptors and the gene therapy delivers via intravitreal injection channelrhodopsins to the optic nerve cells. In a promising Phase I/II trial (NCT04278131), an increased visual response and light sensitivity, as well as a favorable safety profile were reported. This interview took place at Meeting on the Mesa 2021.

Treating achromatopsia with AAV-based gene therapy

Simon Ng — Fri, 05 Nov 2021 09:09:28 +0000

Sue Washer, Applied Genetic Technologies Corporation, Alachua, FL, describes the results of the preliminary Phase I/II trials of a subretinal adeno-associated virus (AAV)-based gene therapy in patients with achromatopsia, an ophthalmological condition affecting cone cells. In the trial assessing patients with a mutation in the CNGB3 gene (NCT02599922), a significant improvement in visual function was reported, whereas in the trial with patients with mutations in the CNGA3 gene (NCT02935517), the gene therapy did not increase visual acuity. This interview took place at Meeting on the Mesa 2021.