REGENXBIO is searching for a translational scientist to design and contribute to the inclusion of PK/PD-related endpoints in preclinical studies to support the development of gene therapy clinical candidates. The successful candidate will also provide scientific input and support to investigating biomarkers of drug action and disease progression/regression.
Responsible for the preclinical strategy to incorporate PK/PD-related endpoints for assessing pharmacodynamics, biodistribution and immunogenicity in preclinical studies.
Responsible for providing interpretation of relevant preclinical pharmacology studies, including PD studies, analysis of data using PK/PD modelling and simulation methodologies adapted for gene therapy, development and delivery of regulatory strategy (e.g. IND and BLA) and data analysis.
Function as the primary point of contact for PK/PD-related activities on preclinical and early clinical development programs, and influence team strategy using modelling expertise.
Involved in assessing and managing analyses of PK/PD data, integrating preclinical study simulation strategies and disseminating the relevant risk/benefit implications to the Company's Translational Strategy Teams (TST).
Interact closely with bioanalytical sciences team to ensure seamless and timely transfer of bioanalytical data to the Preclinical team.
Interact within the preclinical and bioanalytical sciences team and regulatory in the creation of preclinical PK/TK written and tabulated summaries for IND and BLA applications, as well as IBs, annual updates and other regulatory activities.
Help design and conduct translational PK/PD modelling and simulation to support gene therapy design goals, designs of PK/PD and safety assessment studies, and dosing justifications for first-in-human clinical studies
Collaborate within preclinical development on the design and reporting of nonclinical safety studies; prepare subreports; comply with good laboratory practice (GLP) regulations for GLP studies.
Comply with all pertinent SOPs and GLP regulations.
Perform PD, biodistribution and immunogenicity analyses for GLP studies, record data and analysis results in GLP-compliant manner, ensure timely and clear communication with GLP SD/PI on GLP studies, contribute to GLP study reports.
Supervise, assist, and mentor contributing scientists on gene therapy pharmacokinetic analysis, data interpretation, and report writing.
SKILLS, KNOWLEDGE AND ABILITIES REQUIRED
2+ years of experience with a PhD in pharmacokinetics or a biological or related discipline (e.g. pharmacology, toxicology, biology. biochemistry, pharmaceutical science) Equivalent experience may be accepted.
Experience in contributing to pharmacokinetic summaries for regulatory documents successfully submitted to regulatory agencies.
Advanced scientific writing skills.
Maintains a compliant work environment by staying current with SOPs and being up-to-date with existing GLP guidelines.
Good communication and scientific writing skills, and ability to independently work in a matrix environment.
Expertise in assays for pharmacodynamics, biodistribution (PCR) and immunogenicity included in pharmacology and toxicology studies.
Expertise with PK software and have an understanding of non-compartment & multi-compartment modeling.
Experience in animal PK/TK studies, including protocol design & study conduct.
Experience in formulating hypothesis testing for early clinical studies and executing on that using in vitro and in vivo preclinical studies as well as patient samples.
WORKING CONDITIONS AND/OR PHYSICAL REQUIREMENTS
Normal working conditions
Internal Number: RE-19-00011
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
Our gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Through a single administration, our gene therapy product candidates are designed to provide long-lasting effects, potentially significantly altering the course of disease and delivering improved patient outcomes. We are currently developing gene therapy product candidates for the treatment of retinal, metabolic, and neurodegenerative diseases. Our product candidates all utilize AAV viral vectors from our proprietary gene delivery platform, which we call our ...NAV Technology Platform. In addition to our internal product candidate programs, we also selectively license our NAV Vectors to other leading biotechnology companies.