Retinitis Pigmentosa – VJRegenMed

jCell: allogeneic cell therapy for retinitis pigmentosa

admin_vjr_user — Tue, 04 Jan 2022 15:29:59 +0000

Gil Carrasquinho, MD, MBA, Santen Pharmaceutical Co. Ltd., discusses the mechanism of action of jCell Therapy, an investigational allogeneic cell therapy for retinitis pigmentosa (RP), a degenerative retinal disease. Human retinal progenitor cells (RPCs) are administered via intravitreal injection causing the release neurotropic factors, resulting in a reduction in photoreceptor cell death and restoration of surviving photoreceptor and retinal ganglion cell function. A Phase IIb trial (NCT04604899) evaluating jCell in patients with RP demonstrated promising efficacy and tolerability data. This interview took place at the International Society for Stem Cell Research (ISSCR) 2021 Annual Meeting.

Advanced therapies for retinitis pigmentosa

admin_vjr_user — Tue, 04 Jan 2022 15:29:57 +0000

Gil Carrasquinho, MD, MBA, Santen Pharmaceutical Co. Ltd., provides an overview of the current research landscape in retinitis pigmentosa (RP), a degenerative retinal disease that leads to a loss of vision over time. While current treatment options for RP are limited, the gene therapy voretigene neparvovec was recently approved, however, this can only be used to treat a small subset of patients with RP. A number of cell and gene therapies are currently under investigation, however the high level of genotypic and phenotypic heterogeneity seen in patients with RP makes the design of clinical trials and therefore the development and approval of new therapies challenging. This interview took place at the International Society for Stem Cell Research (ISSCR) 2021 Annual Meeting.

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.

Cell therapies for cone-rod dystrophy

Simon Ng — Tue, 26 Oct 2021 09:53:04 +0000

Marie Csete, MD, PhD, ConeSight Therapeutics, Pasadena, CA, discusses the development of cone progenitor cell therapies. Whilst advanced therapies in the ophthalmological space have mainly focused on retinal pigment epithelium (RPE) cells, developing cone-based cell therapies remain behind due to difficulties in regenerating cones from stem cells. Current advancements in cone progenitor cell therapies have been designed for cone rod dystrophies, with the hope of expansion to other indications including retinitis pigmentosa (RP) and macular degeneration. This interview took place at Meeting on the Mesa 2021.