Ocular Toxicology SS

OTSS Webinar—Cell and Gene Therapy for Retinal Dystrophies: Models, Delivery, and Imaging in Preclinical Research

Date and Time: Monday, January 12, 2026, 11:00 AM to 1:00 PM (US EST, UTC -5)

Webinar Description:
Retinal dystrophies, or inherited retinal diseases (IRDs), are a diverse group of disorders marked by progressive loss of photoreceptors or retinal pigment epithelium, leading to retinal atrophy and blindness. Common IRDs include retinitis pigmentosa, Stargardt disease, choroideremia, and Leber congenital amaurosis. Recent research and various clinical trials indicate that cell and gene therapies hold promise as potential treatments for IRDs to recover loss of function caused by mutations in retinal and RPE layers. Before receiving US FDA approval for clinical use, these therapies must undergo rigorous evaluations for safety and toxicity potential and dose optimizations in animal models. In this webinar, we will cover an overview of cell- and gene-therapy approaches for IRDs; various types of cell and gene therapies; criteria for selection of appropriate animal models; delivery routes for targeting various ocular compartments; the role of non-invasive in vivo imaging modalities in preclinical studies; study design considerations based on regulatory guidelines; and illustrative case presentations demonstrating key preclinical strategies.

Speakers:
Ken Shafer, PhD, Greenfield Pathology
Jonathan Luisi, PhD, University of Texas Medical Branch
Kathleen Krenzer, PhD, Canyon Labs
Patrick McNutt, PhD, Wake Forest School of Medicine and US Army Reserves
Anran Ran, PhD, The Chinese University of Hong Kong
Napoleon Alejandro, PhD, Labcorp
Steven Truscott, PhD, Corewell Health

Webinar Description:
A networking event with a focus on mentoring and career development, featuring a 60-minute webinar with a panel of experts from diverse career paths.

OTSS Webinar—General Considerations of Using Non-Human Primate in Non-Clinical Ocular Drug Research

Speaker:
Eric (Wankun) Xie, MD, PhD, Director of Ophthalmology, JOINN Laboratories

Webinar Description:
Non-human primate (NHP) is widely used in the non-clinical research of ocular drugs due to its unique anatomy and physiology. In this webinar, we will discuss the basic biology of the NHP eye compared to human and other species (rabbit and rodents) in anatomy and physiology, as well as the advantages and concerns of using the NHP eye in preclinical ocular research and drug discovery. We will share major background findings in ocular toxicity studies in different types of test articles (biologics and gene therapy) in different species. The origin and principle of immunogenicity in preclinical ocular studies will be discussed, along with representative images of ocular immunogenicity findings, and experience in risk mitigation strategies. We will introduce NHP ocular disease models, and present examples of using these models for efficacy studies with different routes of administration. We will also discuss the ophthalmic endpoints for ocular efficacy and toxicity studies, and present typical ocular findings related to the immune and test articles. Additionally, we will briefly discuss the differences in toxicological study practices in the US and China. Lastly, we will summarize our ocular study capabilities and experience at both sites, including efficacy and toxicology studies.

OTSS Webinar—iPSC-Derived Retinal Pigment Epithelium Replacement Therapy for Macular Degeneration: From Bench to Bedside

Speaker:
Arvydas Maminishkis, PhD, MD, Director of Translational Research, CORE,
The Ophthalmic Genetics and Visual Function Branch (OGVFB) at The National Eye Institute (NEI),
National Institutes of Health (NIH)

Webinar Description:
Induced pluripotent stem cells (iPSCs) can provide autologous and allogeneic replacement tissues, potentially all degenerative diseases. Autologous tissues have the advantage of not requiring immune-suppressive drugs that are known for their side-effects. However, feasibility of autologous iPSC-based therapies hasn’t been established. Here, we developed an autologous iPSC-based therapy for age-related macular degeneration (AMD), a blinding eye disease that affects over thirty million people worldwide. AMD is caused by the progressive degeneration of retinal pigment epithelium (RPE), a monolayer tissue that maintains photoreceptor function and survival. Combining developmental biology with tissue engineering, we developed clinical-grade iPSC-derived RPE-patch on a biodegradable scaffold. This patch performs key RPE functions like photoreceptor phagocytosis, water transport, and polarized cytokine secretion. We confirmed the safety of this patch in an immune-compromised rat model and confirmed its efficacy in a swine RPE injury model. A phase I/IIa Investigational New Drug (IND)-application for iPSC-derived ocular tissue to treat AMD was recently cleared by the US FDA. This Phase I/IIa clinical trial will test safety, feasibility, and integration of an autologous iPSC-derived RPE-patch in twelve advanced AMD patients. This work is helping leverage other similar autologous cell therapies in various other degenerative diseases.

OTSS Webinar—Regulatory Requirements for Ocular Biologics Development

Coordinated global efforts over the last several decades have resulted in the development and adoption of alternatives to traditional animal tests for dermal hypersensitivity assessment. Following the adverse outcome pathway (AOP) framework, several new approach methodologies (NAMs) mapped to key molecular and cellular events in the AOP have been validated and combined to yield “defined approaches”, which were shown to provide superior performance to the existing animal tests when compared to human data for hazard and potency categorization. However, regulatory and industry needs extend beyond classification to deriving points of departure that will facilitate quantitative risk assessment. Next Generation Risk Assessment (NGRA) is an exposure-led, hypothesis-driven approach which integrates NAMs. This hypothetical skin allergy risk assessment case study of two consumer product exposures - 0.1% coumarin in a face cream and 1% coumarin in a deodorant – demonstrates the application of a skin allergy NGRA framework which incorporates the Skin Allergy Risk Assessment (SARA) Model. The SARA Model is a Bayesian approach which allows prediction of a human relevant point of departure (PoD) (the HRIPT dose with a 1% chance of sensitisation or ED01 [µg cm-2]) based upon any combination of HRIPT, LLNA, DPRA, KeratinoSens, h-CLAT or U-SENS data. Through incorporation of benchmark exposure information, the SARA Model can be used to calculate a risk metric for a consumer relevant exposure. This case study demonstrates how integrating a computational model and NAM data in a weight of evidence can build confidence in robust decision making.

Lighting in the Vivarium—The Spectrum and Intensity Is Crucial, The Source Is Not

The use of light-emitting diodes (LED) for vivarium lighting and in other laboratory spaces has come to the fore in the last several years, primarily driven by cost considerations, lower energy costs, and lessened environmental impact. However, as with any new technology, concerns have been raised about the light emitted from these devices, and confusion has arisen regarding the safety of their use in these environments. The purpose of this presentation is to present some fundamental information on lighting systems, the current safe lighting recommendations for vivariums, dispel the concerns for the use of LED for vivariums and provide anyone responsible for lighting to design the best systems for their vivariums or retrofit current facilities with this technology.

OTSS Reception Meeting

Nonclinical Safety Toxicology Strategies for the Development of Novel Ocular Biotherapeutics

OTSS/MDCPSS Virtual Career Panel—SOT Mentoring Event

Date and Time: Monday, September 21, 2020 | 90 minutes

The Ocular Toxicology Specialty Section (OTSS) and the Medical Device and Combination Product Specialty Section (MDCPSS) are holding a virtual career panel as a mentoring event for SOT members interested in career development within the ocular and medical device toxicology fields. The panel will consist of professionals at early, middle, and late stages of their career from Academia, Government, Industry, and Consulting. The panelists will introduce themselves, provide a brief overview of how they arrived at their current position, and address questions from the event moderators. Then the Q&A session will be opened to the audience to engage the panel on career advice, strategy, and goal setting. Please join MDCPSS for our upcoming virtual career panel.

Academia	Government	Industry	Consulting
Mindy Reynolds Washington College	Ron Brown Risk Science Consortium	Hiromi Hosako Alcon	Joel Cohen Gradient Corp.
Neera Tewari-Singh Michigan State Univ.	Alan Hood US FDA	James Kleinedler Boston Scientific	Andrea Rodrigues Tox Strategies
	Elissa Wong US FDA	Bob Przygoda* Johnson & Johnson	William Wustenberg Mycroft Medical LLC
		Ed Reverdy Johnson & Johnson

*Retired