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Explore our cutting edge research, world-class patient care, career opportunities and more.
St. Jude Children's Research Hospital Home
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GEMINI
A comprehensive gene-targeted therapy development platform bringing novel therapeutics into the clinic to treat patients with rare genetic disorders
GEMINI is a St. Jude initiative that tackles a critical reality: despite clear therapeutic potential, many genetic pediatric diseases are too rare to be considered commercially viable for traditional pharmaceutical industry development.
Launched in 2024, GEMINI drives the discovery, development and clinical delivery of precision, gene-targeted therapies for children with rare, catastrophic monogenic diseases.
GEMINI: The beginning
Born as a 2023 St. Jude Blue Sky Initiative project and led by the Pediatric Translational Neuroscience Initiative (PTNI), GEMINI is a highly coordinated cross-institutional, translational effort combining scientific innovation, clinical readiness and regulatory foresight to take on the urgent, unmet need of bringing novel therapeutics into the clinic to treat children with rare genetic disorders.
GEMINI spans the full therapy development pipeline from the earliest preclinical stages of gene identification through manufacturing and dosing patients at St. Jude. Initially focused on neurological disorders and the development of antisense oligonucleotide (ASO) therapies, GEMINI programs are carefully selected to maximize their potential impact. Selection criteria emphasize deep molecular insight into disease mechanisms, assessment of therapeutic potential and target engagement and the ability to deliver meaningful outcomes while generating broader scientific learning.
A collaborative effort
A fundamental element of GEMINI is collaboration—with both internal and external stakeholders. Internally, GEMINI leverages the infrastructure and resources to St. Jude, drawing expertise from teams spanning basic and translational research, clinical care and trials, biostatistics and data science, ethics and regulatory affairs, manufacturing, and institutional strategy and innovation.
GEMINI partners with the St. Jude GMP Facility for ASO manufacturing and works across campus to ensure discoveries can move efficiently from concept to clinic. At the same time, GEMINI collaborates with external stakeholders including academic investigators, patient advocacy groups, industry partners, and regulatory agencies to advance individualized genomic medicines.
Operations
GEMINI is directed by Peter McKinnon, PhD, under the strategic oversight of the St. Jude Office of the Scientific Director and in collaboration with the rest of the PTNI leadership team. The platform’s work is advanced via individual disease programs, each advanced by five coordinated working groups:
GEMINI working groups
Preclinical research
Clinical development
Manufacturing
Data science
External alliances
Each GEMINI disease program focuses on the development of one or more gene mutation-specific ASO projects within the same disease category. These disease programs are led by a PTNI champion and advanced by disease-specific preclinical and clinical working groups. Meanwhile, the manufacturing, data science and external alliances working groups operate in a disease-agnostic manner, providing support across all GEMINI projects.
By performing comprehensive genomic screening and conducting functional studies, GEMINI leverages hypothesis-driven research to transform the development of therapies for diseases that have often been approached via compassionate use interventions. Through this scientific approach, the initiative aims to establish best practices for development of gene-targeted therapies both for St. Jude and the broader field of precision medicine.
Expanding impact
GEMINI is expanding beyond genetic neurological disorders to include additional diseases of interest across St. Jude through the development of a scalable, adaptable program. The initiative will continue to strengthen internal capabilities in both laboratory and clinical research while continuing to develop drug development capabilities in collaboration with the GMP Facility.
Over time, GEMINI aims to increase scale to broaden its reach across disease categories, population sizes, and treatment modalities, positioning St. Jude to serve as a convenor and leader in developing therapies for rare monogenic disorders.
GEMINI Leaders
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J. Paul Taylor, MD, PhD
Executive Vice President
Scientific Director
Chair, Cell & Molecular Biology Department
Director, Pediatric Translational Neuroscience Initiative
Edward F. Barry Endowed Chair in Cell and Molecular Biology
J. Paul Taylor, MD, PhD
Member, St. Jude Faculty
Executive Vice President
Scientific Director
Chair, Cell & Molecular Biology Department
Director, Pediatric Translational Neuroscience Initiative
Edward F. Barry Endowed Chair in Cell and Molecular Biology
Affiliations
Research Interests
- Neurogenetics
- Mechanisms of neurological diseases
- RNA and RNA-binding proteins
- Biomolecular condensates
Contact Information
J. Paul Taylor, MD, PhD
Cell & Molecular Biology
MS 340, Room 4063D
St. Jude Children's Research Hospital
262 Danny Thomas Place
Memphis, TN 38105-3678
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Peter J. McKinnon, PhD
Director, Genomic Medicine Initiative
Director, Center for Pediatric Neurological Disease Research
Endowed Chair in Pediatric Neurological Diseases
Peter J. McKinnon, PhD
Member, St. Jude Faculty
Director, Genomic Medicine Initiative
Director, Center for Pediatric Neurological Disease Research
Endowed Chair in Pediatric Neurological Diseases
Affiliations
Research Interests
- DNA damage response in the nervous system
- Molecular neurobiology
- Mouse models of human disease
- Brain tumors
- Neurodegeneration
- Tumor suppressors
Contact Information
Peter J. McKinnon, PhD
Genomic and Translational Neuroscience
MS 331, Room D3055F
St. Jude Children's Research Hospital
262 Danny Thomas Place
Memphis, TN 38105-3678
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Richard S. Finkel, MD
Director, Center for Experimental Neurotherapeutics
Director, Pediatric Translational Neuroscience Clinical Research Network, Powered by St. Jude
George J. Pedersen Endowed Chair in Neurotherapeutics
Richard S. Finkel, MD
Member, St. Jude Faculty
Director, Center for Experimental Neurotherapeutics
Director, Pediatric Translational Neuroscience Clinical Research Network, Powered by St. Jude
George J. Pedersen Endowed Chair in Neurotherapeutics
Affiliations
Research Interests
- Participation in clinical trials with investigational drugs for pediatric neurologic and metabolic diseases
- Development of outcome measures, biomarkers, and clinical trial design for pediatric neurologic diseases
- Understanding the trajectories of change in pediatric neuromuscular diseases: spinal muscular atrophy, Duchenne muscular dystrophy, and Charcot-Marie-Tooth inherited neuropathies.
Contact Information
Richard S. Finkel, MD
Center for Experimental Neurotherapeutics
MS 760
St. Jude Children's Research Hospital
262 Danny Thomas Place
Memphis, TN 38105-3678
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Kristin Stephenson, MHA, JD
Vice President and Director, Office of Strategy & Alliances
Pediatric Translational Neuroscience Initiative
Kristin Stephenson, MHA, JD
Vice President and Director, Office of Strategy & Alliances
Pediatric Translational Neuroscience Initiative
Short Biography
Kristin Stephenson joined St. Jude in 2021 to lead the Office of Strategy & Alliances in the Pediatric Translational Neuroscience Initiative (PTNI). Immediately before joining St. Jude, Kristin served as EVP, Chief Advocacy and Clinical Services Officer for the Muscular Dystrophy Association. Prior to her tenure in the non-profit space, Kristin served as an attorney in the health care litigation practice groups of Fulbright & Jaworski, LLP (now Norton Rose Fulbright) and Akin, Gump, Strauss, Hauer and Feld, LLP, and as a business consultant in the healthcare practice of Arthur Andersen, LLC.
Contact Information
Kristin Stephenson, MHA, JD
Pediatric Translational Neuroscience Initiative
MS 760, Room S3024
St. Jude Children's Research Hospital
262 Danny Thomas Place
Memphis, TN 38105-3678
GEMINI in Progress
Genomic Medicine Initiative (GEMINI) targets unmet needs in rare disease treatment
Learn how GEMINI addresses the challenges of rare genetic diseases