Senior Scientist I- Ocular Research

Company Overview:

ADARx Pharmaceuticals, Inc., (the “Company” or “ADARx”) located in San Diego, is a clinical stage biotechnology company dedicated to transforming cutting-edge science into next-generation RNA therapeutics to treat a broad spectrum of diseases. Our goal is to control the expression of specific disease drivers with highly selective RNA targeted therapies, delivering life-changing treatments for patients with urgent unmet medical needs.

Position Summary

We are seeking a highly motivated Ocular Research Scientist to support the discovery and preclinical development of innovative therapies for retinal diseases. The successful candidate will have strong expertise in ocular biology, retinal disease models, functional and structural ophthalmic assessments, molecular profiling, and translational research.

This position will contribute to programs focused on age-related macular degeneration (AMD), ocular vascular diseases, inherited and rare retinal diseases, and other conditions affecting the retina and retinal pigment epithelium (RPE). The candidate will lead or support in vivo and in vitro studies evaluating oligonucleotide-based therapeutics, including siRNA therapy.

Key Responsibilities

Lead and participate in research efforts to discover, characterize, and validate novel approaches for extrahepatic siRNA and RNA editing activities, with a focus on ophthalmology targets.

Develop, optimize, and characterize ocular mouse models relevant to AMD including Geographic atrophy (dry AMD), neovascular or wet AMD, diabetic retinopathy and diabetic macular edema, retinal vascular leakage and neovascularization, inherited and rare retinal diseases, retinal inflammation, and neurodegeneration.

Establishment of ocular in vivo systems to screen siRNAs via ocular dosing and administration in rodents, including intravitreal, subretinal, topical, periocular, and systemic delivery, as appropriate for the study.

Perform animal monitoring, clinical observations, study scheduling, tissue collection, necropsy procedures, and sample processing in accordance with approved protocols.

Conduct detailed microdissection of ocular tissues, including isolation of the retina, RPE/choroid, optic nerve, vitreous, aqueous humor, and other relevant ocular compartments.

Perform functional and structural ophthalmic assessments, with hands-on expertise in electroretinography (ERG), optical coherence tomography (OCT), fundus imaging, fluorescein angiography, other relevant ocular imaging and functional endpoints

Design, perform, and interpret RNAscope data to evaluate oligonucleotide distribution, tissue localization, cellular uptake, and target RNA expression in ocular tissues from rodent and nonhuman primate (NHP) studies.

Design and execute single-cell RNA sequencing studies using retinal, RPE/choroid, and other ocular tissues. Support tissue dissociation, single-cell preparation, sample quality assessment, library preparation, and sequencing workflows. Analyze and interpret single-cell RNA sequencing datasets.

Integrate single-cell transcriptomic data with RNAscope, RT-qPCR, histology, immunohistochemistry, imaging, and functional data. Develop and apply molecular, cellular, biochemical, and imaging-based assays to evaluate therapeutic activity.

Design and perform in vitro studies using retinal cells, RPE cells, vascular endothelial cells, microglia, primary cells, iPSC-derived cells, and other disease-relevant cellular systems. Analyze gene-expression, protein-expression, RNAscope, single-cell sequencing, imaging, histology, and functional data using appropriate quantitative and statistical methods.

Collaborate with multidisciplinary teams in discovery biology, discovery oligo and chemistry, DMPK, and non-clinical safety teams

Maintain accurate experimental records and prepare study reports, presentations, manuscripts, and regulatory-supporting documents. Present research findings and recommendations to project teams and senior leadership.

Essential Physical Characteristics:

Reasonable accommodation(s) may be made to enable qualified individuals with disabilities to perform the essential functions of a job, on a case-by-case basis.

On-Site Protocol:

Physical presence at the ADARx Pharmaceuticals worksite is a necessary job function of this role, which the Company deems critical to collaboration, innovation, productivity, employee well-being and engagement, and it enhances the Company culture.

Required Qualifications

Ph.D. in Neuroscience, Ophthalmology/Vision Science, Molecular Biology, Cell Biology, Pharmacology, or closely related discipline.

Prior minimum 3-4 years biotechnology or pharmaceutical industry experience in retinal or ocular drug discovery.

Demonstrated experience designing and conducting preclinical ocular research. Strong knowledge of retinal anatomy, physiology, disease mechanisms, and ocular pharmacology.

Extensive hands-on experience with in vivo ocular rodent models, including animal handling, dosing, ocular injections (IVT and SR), ophthalmic examinations, demonstrated strong proficiency in ocular tissue microdissection including isolation of the retina and RPE/choroid tissue, and study execution.

Hands-on expertise in in-life ocular functional and structural assessments including ERG and OCT data acquisition, analysis, and interpretation of results.

Prior experience with single-cell RNA sequencing of ocular, neural, or other complex tissues. Experience analyzing single-cell RNA sequencing data, including quality control, clustering, cell-type annotation, differential expression, pathway analysis, and biological interpretation.

Prior experience with RNAscope image acquisition, quantitative image analysis, assay optimization, appropriate controls, and interpretation of spatial expression and biodistribution data.

Expertise in molecular biology techniques such as RNA isolation, RT-qPCR, gene-expression analysis, Western blotting, ELISA, immunohistochemistry, immunofluorescence, and imaging-based assays.

Ability to work independently while contributing effectively within cross-functional project teams. Excellent written and verbal communication skills, scientific writing, and problem-solving skills. Proficiency working with Microsoft Office Suite Products, Adobe Pro and Prism.

Preferred Qualifications

Experience with preclinical models of AMD, geographic atrophy, choroidal neovascularization, retinal vascular disease, diabetic retinopathy, retinal degeneration, or rare inherited retinal disorders.

Experience with in-life ocular functional and structural assessments using ERG and OCT, and data acquisition, interpretation and presentation of results.

Experience in ocular injections through intravitreal, and subretinal, periocular, topical, and systemic routes, and microdissection of ocular tissues.

Experience combining RNAscope with immunohistochemistry to identify oligonucleotide localization within specific retinal cell types. Expert in ocular histology, retinal imaging, and quantitative image analysis, biodistribution, PK/PD, and safety assessments for ocular therapeutics.

Previous experience with RNA/oligonucleotide therapeutics is preferred but not essential.

Required Key Attributes

The successful candidate will be scientifically rigorous, technically skilled, highly collaborative, detail-oriented, and passionate about developing transformative therapies for patients with vision-threatening diseases.

The individual should be comfortable working in a fast-paced research environment and capable of advancing programs from target validation through preclinical candidate selection, final lead candidate nomination and translational development.

Must be able to work independently with supervision as needed.

Adaptable / Flexible - willing and able to adjust to multiple demands and shifting priorities as well as an ability to meet day-to-day challenges with confidence and professionalism.

Strong problem-solving skills and a proactive attitude towards exploring new approaches.

Understanding of project timelines and deliverables and plans/coordinate project work accordingly with departmental, functional, and external stakeholders.

Self-motivated with excellent interpersonal, organizational and communication skills with the ability to take a hands-on approach to work effectively in a dynamic and collaborative, fast-paced environment.

ADARx Pharmaceuticals is an EEO employer committed to an exciting, diverse, and enriching work environment. We prohibit discrimination and harassment of any kind based on race, color, sex, religion, sexual orientation, national origin, disability, genetic information, pregnancy, or any other protected characteristic as outlined by federal, state, or local laws.