St. Jude Research
Genomic Medicine Initiative

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.

Two men at a table

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.

A man and woman in a hall way. The man is wearing a white coat. The woman is holding a clipboard

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:

Meeting in conference type room. People around table, 2 presenting at screen.

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.

Person clad in white coat working in the lab

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

GEMINI in Progress

Research

Genomic Medicine Initiative (GEMINI) targets unmet needs in rare disease treatment

Brian O'Flynn, PhD and Jake Hopkins, MBA

Learn how GEMINI addresses the challenges of rare genetic diseases

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