FDA Fellowship - Analysis of Codon Optimized FIX and FVIII Delivered by Adeno Associate Virus

Research Project

The successful candidate will join the research project that supports ongoing investigations into the functional and structural consequences of synonymous codon substitutions and codon optimization in the context of therapeutic biologics. You will be a part of studies examining how codon optimization and naturally occurring synonymous variants modulate gene expression, translational kinetics, RNA secondary structure, protein folding, post-translational modifications, and immunogenicity. Model systems include recombinant coagulation factors (Factor VIII, Factor IX, ADAMTS13/MDTCS) expressed across multiple platforms, including transient transfection, lentiviral delivery, AAV-mediated gene delivery, and targeted genomic integration. Techniques may include ribosome profiling, tRNA sequencing, proteomics, glycoproteomics, mass spectrometry, structural characterization assays, and high-throughput cell-based assays.

Mentor

The mentor for this opportunity is Chava Kimchi-Sarfaty (chava.kimchi-sarfaty@fda.hhs.gov). If you have questions about the nature of the research, please contact the mentor.

Learning Objectives

• Develop proficiency in molecular biology techniques relevant to recombinant protein expression, including cell culture, transient transfection, lentiviral and AAV-mediated gene delivery, and stable genomic integration systems.
• Learn to apply proteomics and glycoproteomics workflows, including mass spectrometry-based post-translational modification analysis, to evaluate protein quality attributes of therapeutic biologics.
• Develop skills in structural characterization of recombinant proteins using conformation-sensitive antibody panels, circular dichroism, and/or Ankyron-based assays.
• Gain experience in computational analysis of codon usage metrics (e.g., CAI, codon-pair optimization, RNA secondary structure modeling) and their integration with experimental data.

Mentor Information

The mentor for this opportunity is Chava Kimchi-Sarfaty (chava.kimchi-sarfaty@fda.hhs.gov). If you have questions about the nature of the research, please contact the mentor.

Citizenship Requirements

This opportunity is available to U.S. citizens only and Legal Permanent Residents (LPRs).

Qualifications

• The qualified candidate must have received or be currently pursuing a master's or doctoral degree in one of the related fields: pre-med, life/health/medical sciences, biochemistry, chemistry, and/or genetics. The degree must be received within the past five years or be currently pursuing.
• Preferred skills:
  • Basic knowledge of biology or biochemistry
  • Familiarity with codon optimization using in vitro methods
  • Experience with cell culture, protein purification, Western blots
  • Willingness to research with antibodies, recombinant proteins and gene therapy

Application Process

• To submit your application, scroll to the bottom of this opportunity and click APPLY.
• A complete application consists of:
• An application
• Transcripts – Click here for detailed information about acceptable transcripts
• A current resume/CV, including academic history, employment history, relevant experiences, and publication list
• One educational or professional recommendation

Documents Required

• All documents must be in English or include an official English translation.

Contact Information

• If you have questions, send an email to ORISE.FDA.CBER@orau.org. Please include the reference code for this opportunity in your email.
• *Applications will be reviewed on a rolling-basis.

Location

FDA Office and Location: A research opportunity is currently available at the Center for Biologics Evaluation and Research (CBER), Food and Drug Administration (FDA) in White Oak, Maryland

Background Investigation

Proof of health insurance is required for participation in this program. Participants do not become employees of FDA, DOE or the program administrator, and there are no employment-related benefits.

ORISE Education and Training Agreement

The participant will receive a monthly stipend commensurate with educational level and experience. Proof of health insurance is required for participation in this program. Participants do not become employees of FDA, DOE or the program administrator, and there are no employment-related benefits.

Background Investigation

The qualified candidate must have received or be currently pursuing a master's or doctoral degree in one of the related fields: pre-med, life/health/medical sciences, biochemistry, chemistry, and/or genetics. The degree must be received within the past five years or be currently pursuing.

Preferred Skills

• Basic knowledge of biology or biochemistry
• Familiarity with codon optimization using in vitro methods
• Experience with cell culture, protein purification, Western blots
• Willingness to research with antibodies, recombinant proteins and gene therapy

Additional Information

• I am a U.S. citizen, or I have lived in the United States for at least 36 out of the past 60 months. (36 months do not have to be consecutive.)
• I have read the FDA Ethics Requirements.