Gene Therapy

Gene therapy uses viral and nonviral vectors, gene editing (CRISPR/Cas), and RNA platforms to correct, silence, or add genes for durable therapeutic effect. Applications include promoting tissue repair after blast and polytrauma, delivering antimicrobial or immune-modulatory genes, and enabling sustained care during delayed evacuation or prolonged-field-care.

Technical Challenges

  • Targeted, efficient and safe in vivo delivery to injured tissues in polytrauma and hemorrhagic environments

  • Immune responses to viral vectors and repeat dosing limitations in previously exposed personnel

  • Off-target effects and genomic safety concerns with gene editing in austere use-cases

  • Manufacturing scale, cold-chain dependence, and rapid deployability for forward medical units

Emerging Opportunities

  • Point-of-care deployable delivery systems (room-temperature stable formulations, field-ready devices) for gene modalities

  • Rapid, validated assays to monitor on-target activity, off-target edits, and biodistribution in austere settings

  • Strategies for transient, tunable gene expression suitable for acute trauma or infection without prolonged systemic exposure

  • Regulatory and clinical pathways tailored to combat-relevant indications and small-cohort military trials

Current and Emerging Technologies in Gene Therapy

Viral vectors (AAV, lentivirus)

Adeno-associated and lentiviral vectors provide efficient gene delivery for long-term expression or ex vivo modified cells; ongoing work focuses on tropism engineering and immune-evasive capsids for repeated dosing in Warfighters.

Nonviral delivery (LNPs, polymers, electroporation)

Lipid nanoparticles and synthetic carriers enable mRNA, siRNA, and DNA delivery with lower immunogenicity and faster manufacturing; electroporation and focused ultrasound offer localized uptake in injured tissues.

Gene editing (CRISPR/Cas, base & prime editors)

CRISPR-based approaches allow precise correction, gene disruption, or transcriptional modulation; base and prime editors reduce double-strand breaks, improving safety for clinical trauma and infection indications.

RNA therapeutics (mRNA, siRNA, antisense)

mRNA can transiently express protective or regenerative proteins; siRNA and antisense oligonucleotides permit rapid gene silencing (e.g., virulence factors, proinflammatory mediators) with applications in AMR and sepsis.

Advanced delivery platforms (microneedles, hydrogel depots, cell-based carriers)

Field-stable patches, injectable hydrogels, and engineered cell carriers (e.g., MSCs expressing therapeutic transgenes) support localized, sustained release and reduced systemic exposure suitable for prolonged-field-care.

Importance to Military Medicine

Durable regenerative solutions

Promotes tissue repair and functional recovery after blast, burns, and musculoskeletal trauma when conventional grafting is limited or delayed.

Combatting infection and AMR

Enables delivery of antimicrobial peptides, immune-modulators, or bacteriophage-enhancing genes to address biofilms and antimicrobial resistance in contaminated wounds.

Prolonged and austere-field care

Transient or sustained gene-based therapies can bridge physiological gaps during delayed evacuation, reducing morbidity when surgical resources are limited.

Force readiness and long-term recovery

Restoring function and preventing chronic disability preserves operational capability and long-term veteran health, aligning clinical outcomes with mission readiness.

Alignment with the MTEC Mission

Advances durable, deployable medical countermeasures that accelerate transition from research to fielded solutions for Warfighters.

Dual-Use (Military + Civilian) Applications

Dual-use impact: civilian benefit in trauma care, regenerative medicine, and AMR therapies—speeding adoption in both military and public health sectors.

Explore MTEC Members with Gene Therapy Capabilities

MTEC members bring multidisciplinary strengths—vector development, clinical trial infrastructure, GMP manufacturing, and point-of-care device integration—enabling end-to-end solutions from bench to austere bedside. Explore member profiles to identify partners for gene therapy projects, joint proposals, and capability demonstrations.

94 Members with Gene Therapy capabilities.

Member
The University of Texas System

The University of Texas System is a leading public university system in the United States, dedicated to improving lives through education, health care, and research. With over 256,000 students enrolled across 14 institutions, the UT System is committed to providing affordable access to higher education and producing a skilled workforce to drive Texas's economy.

Member
Georgia Southern University

Georgia Southern University is a vibrant institution with over 26,100 students across three campuses in Statesboro, Savannah, and Hinesville. The university offers 132 degree programs at the associate, bachelor’s, master’s, and doctorate levels, emphasizing student success, community impact, and research excellence. With a commitment to inclusivity and engagement, Georgia Southern fosters a supportive environment for diverse learners, including military-affiliated and adult students. The university is dedicated to transforming lives through education and community engagement, aligning its programs with regional needs. Additionally, Georgia Southern provides accelerated pathways for students to fast-track their master's degrees, enhancing educational opportunities. The university also emphasizes career readiness and public impact research, preparing students for successful careers and contributing to community development.

Member
Humanetics Corporation

Humanetics Corporation is a clinical-stage pharmaceutical company based in Minneapolis, Minnesota, focused on developing and commercializing products to enhance human health and wellbeing. Founded in 1988, the company specializes in radiation modulators, adjunctive oncology therapies, and pulmonary protective therapies, particularly for COVID-19. Humanetics is known for its lead drug candidate, BIO 300, which is being developed as a radioprotectant for military and civilian use, as well as a treatment to improve outcomes in cancer patients receiving radiotherapy. The company is actively engaged in research programs for non-small cell lung cancer, prostate cancer, and head and neck cancers.

Member
The University of Texas at San Antonio

The University of Texas at San Antonio (UTSA) is dedicated to the advancement of knowledge through research, teaching, and community engagement. As a premier public research university, UTSA embraces multicultural traditions and serves as a catalyst for socioeconomic development, providing access to educational excellence and preparing citizen leaders for the global environment.

Member
Auburn University

Auburn University Research & Economic Development is dedicated to advancing research and economic growth through a collaborative effort among its various colleges and departments. The organization focuses on fostering innovation, supporting faculty and student research, and facilitating partnerships with industry and government to address societal challenges and enhance the quality of life. Recent initiatives include the launch of the Team Science Series to promote interdisciplinary collaboration and the development of the Gulf Coast Engineering Research Station to address coastal environmental challenges.

Member
Institute for Integrative & Innovative Research (I³R) at University of Arkansas

The Institute for Integrative & Innovative Research (I³R) at the University of Arkansas is dedicated to pioneering solutions to wicked problems and societal challenges through convergence research. With a mission to advance research excellence and stimulate economic development, I³R focuses on deploying innovations at scale through collaboration across various sectors, including academia, industry, and government.

Member
The University of Texas Medical Branch at Galveston

UTMB is dedicated to supporting research, education, and scholarly endeavors, with a focus on creating the future of healthcare through innovative research and community engagement.

Member
Revolution Biomanufacturing Inc.

Revolution Biomanufacturing Inc. (RBIx) is expanding beyond traditional CDMO Process/Analytical Development and Manufacturing services to pioneer distributed molecular manufacturing. A new class of digital biomanufacturing that enables the autonomous design, manufacture, quality verification, and deployment of Nucleic Acid therapeutics and molecular diagnostics at the point of need. By integrating AI-driven sequence optimization, enzymatic nucleic acid synthesis, Nanoparticle design and manufacturing (LNP & EV), closed-loop quality control, and field-deployable manufacturing platforms, RBIx is creating adaptive biological infrastructure capable of responding to emerging diseases, national security threats, and personalized medicine with unprecedented speed and flexibility.

Member
The University of Chicago

The University of Chicago is a prestigious urban research university committed to rigorous inquiry and intellectual freedom. Founded in 1890, it has produced numerous Nobel laureates and is known for its transformative education and groundbreaking research across various fields. The university fosters an inclusive and diverse learning environment, encouraging participation from all community members.

Member
Boston University

Boston University is a major research institution committed to leading breakthroughs across various disciplines. The Office of Research supports and advances research excellence, fostering collaboration among researchers, industry partners, and government leaders to address significant societal challenges.

Member
Cooper University Health Care

Cooper University Health Care is a regional academic health system recognized for comprehensive clinical services, advanced multidisciplinary care, specialized surgery, and robust research in South Jersey. It provides integrated healthcare across general and specialty domains, including cancer, neurosciences, cardiology, trauma, genetics, addiction medicine, and complex surgical procedures, supported by leading technologies and a network of modern facilities.

Member
Baylor University

Baylor University is a preeminent Christian research university committed to addressing the world's most meaningful challenges through top-tier research and scholarship. Recognized as an R1 research institution, Baylor focuses on infusing its distinct Christian mission into its research initiatives, fostering a commitment to excellence and innovation.

Member
Alzheimer’s Treatment Centers of America

This organization specializes in integrated, science-based treatment and diagnostics for Alzheimer’s, dementia, and cognitive decline. Utilizing a precision medicine approach, the center combines advanced AI analysis, biomarker-driven planning, and multimodal therapies including brain imaging, neurocognitive testing, and over 50 clinical and supportive treatment modalities. The care model addresses both the root causes and neuropsychiatric aspects of cognitive impairment, delivered by a multidisciplinary team with expertise in functional, regenerative, and collaborative behavioral medicine.

Member
Base Pair Biotechnologies

Base Pair Biotechnologies specializes in custom aptamer discovery and development for research, diagnostics, therapeutics, and industrial applications. The company leverages proprietary multiplex selection, advanced bioinformatics, and chemical modification techniques to develop high-affinity and selective nucleic acid aptamers. Base Pair enables affinity reagent development, biosensor design, and molecular detection for a broad range of targets and partners across academia and industry.

Member
Blood Centers of America

Blood Centers of America (BCA) is the largest blood supply network in the United States, comprising over 60 independent community blood centers that collectively distribute approximately 50% of the nation's blood supply. BCA supports member organizations through supply chain solutions, donor recruitment, advocacy, disaster response, and purchasing power. In addition to providing whole blood, red blood cells, platelets, plasma, cells, and tissues for therapeutic, diagnostic, research, and advanced therapy applications, BCA and its network are engaged in the development and delivery of advanced therapies including cell and gene therapy starting materials, processing, and manufacturing. BCA actively mobilizes resources in response to national emergencies, pandemics, and technological advances in donor experience and biotherapeutics.

Member
The University of Texas at Arlington Research Institute

The UT Arlington Research Institute (UTARI) specializes in applying cutting-edge technologies to real-world engineering problems. Our collaborative endeavors reflect a commitment to excellence, integrity, and respect for all, bringing economic growth and fulfillment to our families, business partners, university, and community.

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Safi Biotherapeutics

Next-Generation Manufactured Red Blood Cells for Extended Battlefield Blood Supply: Safi Biotherapeutics

Safi Biotherapeutics is developing manufactured red blood cells (mRBCs) with extended shelf life to improve battlefield transfusion readiness and emergency trauma care.

Non-Invasive Glymphatic (GF) Monitoring and Recovery Optimization for Warfighter Cognitive Readiness

Applied Cognition is developing non-invasive glymphatic monitoring and recovery technologies to enhance warfighter cognitive readiness, resilience, and sleep recovery.

Moberg Analytics

Moberg Analytics – Advanced Brain Monitoring & Critical Care Informatic

Moberg’s platform unifies brain monitoring data for faster, precise treatment of severe brain injuries in military and civilian care.

University of California San Francisco

TemPredict: AI-Driven Wearable Monitoring for Early Illness Detection and Warfighter Readiness

TemPredict: AI wearables detect early illness and health risks to support warfighter readiness.

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