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.

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.

Achromatopsia – VJRegenMed

Manufacturing challenges in the advanced therapies sector

Treating achromatopsia with AAV-based gene therapy