FDA Research - Developing Methods Efficient Gene Editing in Hematopoietic Stem

Organizational Information

U.S. Food and Drug Administration (FDA)

The Division of Cell Therapy (DCT2), within the Office of Cellular Therapy and Human Tissue (OCTHT) at the Center for Biologics Evaluation and Research (CBER), located in Silver Spring, Maryland.

This research project focuses on advancing the development and manufacturing of CRISPR/Cas9-edited hematopoietic stem cell (HSC) therapeutics. Under the mentorship of the Principal Investigator, you will gain experience in cutting‑edge genome editing techniques applied to human HSCs and related cell types. You will engage in research at the intersection of stem cell biology, gene therapy, and nanomedicine, addressing one of the most significant challenges in the field: achieving safe and effective delivery of gene editing machinery to HSCs.

You will utilize state‑of-the-art manufacturing tools and methodologies to investigate and optimize ex‑vivo HSC-gene editing and expansion protocols. You will gain comprehensive training in the design, development, and optimization of novel lipid nanoparticle (LNP) formulations specifically engineered for efficient gene editing in HSCs. This training opportunity is ideal for individuals seeking to contribute to the advancement of non‑viral delivery platforms that could transform the landscape of HSC‑based gene therapies and cellular therapeutics.

• Develop proficiency in designing, executing, and validating CRISPR/Cas9‑mediated genetic modifications in HSCs and other relevant cell types, including understanding guide RNA design, delivery methods, and assessment of editing efficiency and specificity.
• Gain experience in the rational design and synthesis of lipid nanoparticle components for gene editing applications.
• Develop technical skills in LNP formulation techniques and optimization strategies.
• Understand the biological barriers to HSC gene editing and develop strategies to overcome them.
• Acquire skills in evaluating gene editing efficiency and safety in hematopoietic stem cells.

Principal Investigator:
Pankaj Kumar Mandal (pankaj.mandal.gov).

Start Date:

2026 (flexible). Duration:
Initially one year, may be renewed contingent on FDA funding. Participation:
Full‑time. Stipend:
Monthly stipend commensurate with educational level and experience.

This opportunity is available to U.S. citizens, lawful permanent residents (LPR), and foreign nationals. Non‑U.S. citizen applicants should refer to program guidelines for acceptable immigration statuses.

Completion of a successful background investigation by the Office of Personnel Management (OPM) is required for all applicants, including non‑U.S. citizens who have resided in the U.S. for at least three of the past five years.

ORISE Fellows, including their spouse and minor children, must report any significantly regulated organization or prohibited investment fund financial interest and revoke it or terminate the relationship. Additional requirements are detailed in FDA Ethics for non‑employee scientists.

Participants must read and sign the FDA Education and Training Agreement within 30 days of start date. The agreement covers obligations for non‑employee nature of the appointment, prohibition on performing inherently governmental functions, intellectual property and research material ownership, and confidentiality of non‑public information.

The qualified candidate should be currently pursuing or have received a Ph.D. or equivalent degree in molecular and cell biology, pharmacology, or drug discovery/development.

Lab skills in common molecular and cell biology techniques are preferred.

• Degree:
  Doctoral Degree.
• Disciplines:
  • Chemistry and Materials Sciences (Chemistry (General))
  • Engineering (2)
  • Life Health and Medical Sciences (11)
  • Science & Engineering‑related (1)

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.

Ashley