Lead or Senior Scientist - Genome Editing, Genomics, Biotechnology; Shengdar Q. Tsai Lab

Lead or Senior Scientist Opportunity

A Lead or Senior Scientist opportunity with the Tsai Lab to develop a therapeutic platform for in vivo base editing treatments and drug products to treat patients with bone marrow failure disorders e. Help us build an individualized therapeutic base editing platform to treat St. Jude patients with rare bone marrow failure disorders by correcting their disease‑causing mutations in their hematopoietic stem cells in vivo.

Dr. Tsai leads the recently launched DRIVE project (Delivering Rare‑disease In vivo Editors), funding a multi‑institutional, public‑private partnership to develop in vivo base editors for treatment of bone marrow failure disorders. Bring your expertise in genome editing drug development to a Lead Scientist role in the Tsai Lab, where you will help advance genome editing technologies toward individualized treatments for pediatric patients.

Our goal is to develop in vivo base editing drug products, complete IND‑enabling studies, and open clinical trials in the next 3 years.

We are seeking an outstanding and highly motivated Lead Scientist or Senior Scientist to help lead efforts to develop personalized genome editing treatments. We care deeply about the success of our scientists, and this is a unique opportunity to help lead development of an in vivo gene therapeutic gene editing platform and drug products e, with substantial room for career growth and advancement.

The Tsai lab is a highly collaborative, creative, and motivated group of scientists e Children’s Research Hospital. Our research centers on development of state‑of‑the‑art genomic methods for understanding the global activities of genome editors, protein engineering to improve capabilities for precise editing, and translation to autologous genome edited hematopoietic stem cell therapies. Dr. Tsai also co‑leads the PARADIGM (Partnership to Advance Development of Individualized Genomic Medicines) blue sky project.

• Design and develop in vivo base editing drug products to treat St. Jude patients with bone marrow failure disorders.
• Characterize the genotoxicity of candidate drug products using state‑of‑the‑art genomic assays such as CHANGE‑seq‑BE and CHANCE‑seq, including variant‑aware analysis.
• Optimize potency and specificity of a HSC‑targeted lipid nanoparticle gene editing approach.
• Co‑lead a multi‑disciplinary team to test a new streamlined platform approach for in vivo genome editing medicines.
• Coordinate a team of scientists working to develop a novel gene editing platform.
• Accelerate lead‑target discovery, genotoxicity characterization, and cargo optimization.
• Non‑clinical pharmacology studies of an HSC‑targeted lipid nanoparticle.
• Continuously assess and manage project technical and drug development risks.
• Analyze experimental outcomes and develop high‑quality summaries.
• Summarize results and present updates to the broader DRIVE and ARPA‑H teams.
• Draft manuscripts and figures for publication in high‑impact journals.

You would learn valuable skills that include:
Genome engineering, high‑throughput genomics, laboratory automation, IND‑enabling studies for genome editing drug development.

• PhD in relevant scientific area is required, such as genomics, molecular and cell biology.
• Expertise in genome editing, genomics, next‑generation sequencing.
• Excellent communication, organizational, and presentation skills.
• Four or more years of hands‑on research laboratory experience working with CRISPR genome editors and NGS library preparation.

• Twelve (12) years of relevant work experience is required with a Bachelor’s degree.
• Ten (10) years…