Summary

Bone marrow failure (BMF) disorders are catastrophic diseases of childhood that result in failure to produce hematopoietic cells leading to severe morbidity and eventually mortality. Treatment options are limited to supportive care and allogeneic bone marrow transplantation. We are interested in developing curative therapies for patients with these rare but severe disorders. Using a combination of disease model systems such as induced pluripotent stem cell-derived hematopoietic stem cells and engineered hematopoietic stem cells from healthy donors to model patient mutations, we have advanced development of gene therapy for Diamond-Blackfan anemia which is anticipated to move to clinic in the coming months. Now we are developing individualized genome editing-based treatments using base editors and prime editors for BMF disorders. As part of an academic/industry team, we recently received funding from ARPA-H to advance development of in vivo genome editing for BMF disorders. This translational project, called DRIVE which stands for Delivering Rare-disease In Vivo Editors, will involve development of preclinical models and pipelines to demonstrate efficacy and safety and accelerated development of personalized bespoke genomic medicines for patients with BMF disorders such as Diamond-Blackfan anemia and GATA2 deficiency. You will work with a multidisciplinary team of scientists with expertise in genome editing, high throughput screens, hematology, cell biology, LNP formulation, technology development and commercialization, cGMP practices, technical writing, regulatory affairs, clinical trial design and project management.

Job description

Project Description

Bone marrow failure (BMF) disorders are catastrophic diseases of childhood that result in failure to produce hematopoietic cells leading to severe morbidity and eventually mortality. Treatment options are limited to supportive care and allogeneic bone marrow transplantation. We are interested in developing curative therapies for patients with these rare but severe disorders. Using a combination of disease model systems such as induced pluripotent stem cell-derived hematopoietic stem cells and engineered hematopoietic stem cells from healthy donors to model patient mutations, we have advanced development of gene therapy for Diamond-Blackfan anemia which is anticipated to move to clinic in the coming months. Now we are developing individualized genome editing-based treatments using base editors and prime editors for BMF disorders. As part of an academic/industry team, we recently received funding from ARPA-H to advance development of in vivo genome editing for BMF disorders. This translational project, called DRIVE which stands for Delivering Rare-disease In Vivo Editors, will involve development of preclinical models and pipelines to demonstrate efficacy and safety and accelerated development of personalized bespoke genomic medicines for patients with BMF disorders such as Diamond-Blackfan anemia and GATA2 deficiency. You will work with a multidisciplinary team of scientists with expertise in genome editing, high throughput screens, hematology, cell biology, LNP formulation, technology development and commercialization, cGMP practices, technical writing, regulatory affairs, clinical trial design and project management.

Position Responsibilities

Develop novel research project(s) and idea(s) in collaboration with mentor
Design and perform experiments, interpret data, and communicate results with scientific community
Work with mice models of human disease
Work collaboratively with a multidisciplinary team and with collaborative labs inside and outside St. Jude
Generate initial draft for grants and research manuscripts
Professional development requirement, including grant writing

Minimum Education and/or Training

PhD

Special Skills, Knowledge, and Abilities

Experience working with mice
Strong background in cell biology and HSC biology
Exposure to high-throughput screens, CRISPR/Cas9, base editors will strengthen the application

About the lab and St. Jude

The Bhoopalan lab’s goal is to advance our mechanistic understanding of human hematopoiesis in health and disease and leverage these findings to develop curative therapies for human blood disorders. The Bhoopalan lab is a diverse multidisciplinary group that utilizes various tools from molecular biology, cell biology, computational data science and translational medicine. Trainees will have access to the deep technical expertise in the lab, and the opportunities to participate in wide range of collaborative efforts, lead projects, learn grantsmanship and develop a path towards independent research.

Requirements

PhD
Experience working with mice
Strong background in cell biology and HSC biology
Exposure to high-throughput screens, CRISPR/Cas9, base editors preferred

Benefits

Comprehensive benefits
Relocation assistance
Professional development allowance
Grant writing workshops
Individual development plans

Company overview

St. Jude Children's Research Hospital is a world-renowned pediatric research and treatment institution headquartered in Memphis, Tennessee, dedicated to advancing cures for childhood cancer and other catastrophic diseases. Founded in 1962 by entertainer Danny Thomas, it stands as the first and only National Cancer Institute-designated Comprehensive Cancer Center devoted exclusively to children. The hospital operates on a unique funding model where families never receive bills for treatment, travel, housing, or food, with costs exceeding $2 billion annually primarily supported by donations through its fundraising organization ALSAC. St. Jude treats approximately 8,600 patients each year from all 50 states and around the world, with a focus on acute lymphoblastic leukemia, brain tumors, sickle cell disease, and infectious diseases. The institution employs over 7,000 staff members and maintains eight affiliate clinics across the United States. Through its St. Jude Global initiative, the hospital shares research and treatment protocols internationally, partnering with more than 350 institutions in over 90 countries. St. Jude has been instrumental in increasing childhood cancer survival rates from 20% in 1962 to over 80% today, while maintaining a robust research program that includes gene therapy, bone marrow transplantation, and precision medicine approaches.