Macular Degeneration – VJRegenMed

Challenges in targeting the eye with cell therapy

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

Marie Csete, MD, PhD, ConeSight Therapeutics, Pasadena, CA, describes issues associated with developing cell therapies for ophthalmological conditions. Subretinal delivery of cell therapies require a retinal tear, which can prolong recovery times and even in the case of successful delivery, therapeutic efficacy is not guaranteed. The level of scarring and degeneration in the eye can additionally affect treatment outcomes. Phenotyping patients with visual tests or advanced imaging techniques can address the aforementioned issues by defining a subset of patients who will most likely benefit from cell therapies. 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.

Future developments in advanced therapies for the eye

Simon Ng — Tue, 26 Oct 2021 09:53:03 +0000

Marie Csete, MD, PhD, ConeSight Therapeutics, Pasadena, CA, talks on advancements in advanced therapies that are impacting the ophthalmological space. Induced pluripotent stem cell (iPSC)-based retinal pigment epithelium (RPE) cell products have made large strides within the field, and genetic engineering has enabled allogeneic therapies to be successfully delivered in patients. Treatment of glaucoma is also on the horizon with the development of cell therapies based on retinal ganglion cells. This interview took place at Meeting on the Mesa 2021.

Challenges in translating iPSC-based AMD therapies to the clinic

admin_vjr_user — Wed, 18 Aug 2021 16:32:45 +0000

Masayo Takahashi, MD, PhD, RIKEN Center for Biosystems Dynamics Research & Kobe City Eye Hospital, Kobe, Japan, discusses the challenges associated with the translation of induced pluripotent stem cell (iPSC)-based therapies to the clinic. She highlights the importance of developing robust culture protocols to help reduce lot-to-lot variation and explains that purity control of retinal pigment epithelium (RPE) cell products can be achieved based on pigmentation colour, which helps to avoid contamination. Another key challenge is the selection of suitable patients with age-related macular degeneration that will benefit from specific cell therapies. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Neuraminidase could improve AAV9 gene therapy for Batten disease

admin_vjr_user — Thu, 12 Aug 2021 10:07:41 +0000

Kathrin Meyer, PhD, Nationwide Children’s Hospital, Columbus, OH, discusses the administration of neuraminidase prior to or in combination with AAV9 gene therapy in Batten disease, a neurological condition that can lead to macular degeneration. In a mouse model, intravitreal administration of neuraminidase resulted in enhanced delivery of AVV9 into the eye by removing certain sugar residues on entry receptors, improving penetration of the virus into deeper structures of the eye. While additional research is required to assess safety in larger models, neuraminidase pretreatment holds potential in a range of neurological conditions affecting the eye, particularly when targeting the inner nuclear layer. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Highlights from ASGCT 2021: iPSCs & the immunological barrier

admin_vjr_user — Thu, 12 Aug 2021 10:07:40 +0000

Sonja Schrepfer, MD, PhD, Sana Biotechnology, Seattle, WA, shares her highlights from the American Society for Cell & Gene Therapy (ASGCT) 24th Annual Meeting 2021. Recent developments include an improved focus and understanding of immunological barriers associated with allogeneic induced pluripotent stem cells (iPSCs) and chimeric antigen receptor (CAR) T-cells. Other highlights include advances seen in the development of iPSC-based therapies including iPSC-derived dopaminergic neurons for Parkinson’s disease and iPSC-derived retinal pigment epithelium (RPE) for macular degeneration. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

hESC-derived RPE for dry AMD

admin_vjr_user — Sat, 03 Jul 2021 09:00:56 +0000

Jane Lebkowski, PhD, Regenerative Patch Technologies, Portola Valley, CA, describes ongoing research investigating CPCB-RPE1, a parylene membrane implant coated with human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) cells, for the treatment of dry age-related macular degeneration (AMD). CPCB-RPE1 is implanted in the subretinal space and is designed to replace the damaged Bruch’s membrane and dystrophic RPE cells in dry AMD. A recent Phase I/IIa trial (NCT02590692) has demonstrated safety of implantation, as well as improvements in visual acuity in patients with dry AMD that are classed as legally blind. This interview took place during the Cell & Gene Meeting on the Mediterranean 2021 virtual conference.

Manufacturing advanced therapies for AMD

admin_vjr_user — Sat, 03 Jul 2021 09:00:56 +0000

Jane Lebkowski, PhD, Regenerative Patch Technologies, Portola Valley, CA, explores the challenges in manufacturing cell and gene therapies for age related macular degeneration (AMD) and other indications. Given the large number of patients with AMD, it is important for developers to scale manufacturing in order to meet the high demand for therapies while reducing costs. Dr. Lebkowski discusses the benefits of investing in manufacturing at an early stage in the development process, the implementation of automated manufacturing technologies and the importance of appropriate potency assays and cellular characterization. This interview took place during the Cell & Gene Meeting on the Mediterranean 2021 virtual conference.

Horizon scanning of cell therapy: the eye and beyond

admin_vjr_user — Sat, 03 Jul 2021 09:00:55 +0000

Jane Lebkowski, PhD, Regenerative Patch Technologies, Portola Valley, CA, describes some of the impactful advances in cell therapy that are helping drive the field forward, including the ability to produce relevant cell types and characterize their functionality and phenotypic properties. Novel technologies and platforms offer the potential to build layers of the retina which could be applied in the treatment of a range of retinal diseases in the future. Continued advances in cell- and tissue-based therapies also hold great potential for rebuilding other organs such as the kidneys and lungs. This interview took place during the Cell & Gene Meeting on the Mediterranean 2021 virtual conference.

iPSC-derived RPE for macular degeneration

admin_vjr_user — Thu, 03 Jun 2021 16:50:54 +0000

Masayo Takahashi, MD, PhD, RIKEN Center for Biosystems Dynamics Research & Kobe City Eye Hospital, Kobe, Japan, provides an overview of clinical research evaluating the safety of subretinal implantation of induced pluripotent stem cell (iPSC)-derived retinal pigment epithelium (RPE) in patients with age-related macular degeneration (AMD). In the first safety study, autologous iPSCs were derived from skin fibroblasts of patients and then differentiated and implanted as RPE cell sheets, while later studies have used human leukocyte antigen (HLA)-matched allogeneic iPSCs. Dr Takahashi also describes a planned Phase II clinical trial which will aim to evaluate the therapeutic efficacy of allogeneic iPSC-derived RPE in various diseases caused by damage to the RPE. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.