ISSCR 2021 – 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.

Neural stem cells isolated from the CSF for a potential autologous cell therapy

admin_vjr_user — Tue, 04 Jan 2022 15:29:55 +0000

Beatriz Fernández-Muñoz, PhD, Andalusian Network of Design and Translation of Advanced Therapies, Seville, Spain, discusses the isolation of neural stem cells (NSCs) from the cerebrospinal fluid of preterm infants with intraventricular hemorrhage (IVH). Since this fluid is routinely collected during therapeutic neuroendoscopic lavage and typically discarded, there are fewer ethical concerns associated with this source of NSCs. Recent research has demonstrated that these NSCs can be expanded, cryopreserved and differentiated into neurons and glia, and do not for tumors when transplanted into mice. Ongoing pre-clinical research aims to evaluate the potential of this cell source in the development of cell therapies for preterm infants with IVH. This interview took place at the International Society for Stem Cell Research (ISSCR) 2021 Annual Meeting.

The importance of improving the cryopreservation of cell therapy products

admin_vjr_user — Tue, 04 Jan 2022 15:29:53 +0000

Beatriz Fernández-Muñoz, PhD, Andalusian Network of Design and Translation of Advanced Therapies, Seville, Spain, describes the remaining challenges associated with the cryopreservation of cell therapy products, which facilitates the distribution of cell therapies and can reduce manufacturing costs. Dr Fernández-Muñoz discusses the need to improve cryopreservation techniques to enhance cell recovery after thawing, maintain cell potency and reduce the use of dimethyl sulfoxide (DMSO) given its toxicity. This interview took place at the International Society for Stem Cell Research (ISSCR) 2021 Annual Meeting.