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.

120 Members with Gene Therapy capabilities.

The University of Texas System
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.

Georgia Southern University
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.

Stoic Bio
Stoic Bio

Stoic Bio is revolutionizing the cell culture media industry by integrating sustainability into its manufacturing processes. The company focuses on accelerating research and development while minimizing environmental impact through innovative solutions like the Krakatoa benchtop media maker. This approach not only enhances the efficiency of cell culture media production but also significantly reduces greenhouse gas emissions and plastic waste, aligning with Stoic Bio's mission to create a cleaner and greener future.

Humanetics Corporation
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.

The University of Texas at San Antonio
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.

Auburn University
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.

University of Arkansas
University of Arkansas

The Institute for Integrative & Innovative Research (I3R) at the University of Arkansas is dedicated to pioneering solutions to complex 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.

The University of Texas Medical Branch at Galveston
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.

Advanced BioScience Laboratories, Inc.
Advanced BioScience Laboratories, Inc.

Advanced BioScience Laboratories, Inc. (ABL) is a global biomedical contract research and manufacturing organization dedicated to advancing vaccines, therapeutics, and other biologic products. ABL leverages its expertise in product development, GMP manufacturing, and immunological testing to support the development of candidate vaccines and therapeutics through preclinical and clinical stages. The company is involved in producing and testing oncolytic viruses, gene therapies, immunotherapies, viral vaccines, and recombinant protein vaccines.

ATCC (American Type Culture Collection)
ATCC (American Type Culture Collection)

ATCC is a nonprofit biological resource center, with a nearly century-long legacy supporting public health, biodefense, and global health security. ATCC provides critical infrastructure for infectious disease research and CBR threat response. Our biorepository operations support federal agencies, academia, and industry through secure specimen handling, reagent authentication, and global distribution. ATCC manages over 22 million vials under federal contracts, including repositories for NIAID, CDC, BARDA, NCI, FDA, and USDA. These programs enable rapid deployment of validated biological materials for vaccine development, diagnostic assay verification, and therapeutic screening against high-consequence pathogens such as SARS-CoV-2. Certified under ISO 9001, 13485, 17025, and 17034, ATCC ensures quality, traceability, and regulatory compliance across its operations. Our high-containment laboratories (BSL-2 and BSL-3, with access to BSL-4 through partners) and Select Agent license position us to support MTEC-aligned efforts in emerging infectious diseases and medical countermeasure development for CBRN threats.

The University of Queensland
The University of Queensland

The University of Queensland (UQ) is one of Australia's leading research and teaching institutions, dedicated to transforming lives through education and research. UQ aims to create a better future by equipping students with the skills and knowledge necessary for their chosen careers, while also addressing some of the world's most complex challenges through innovative research.

The University of Chicago
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.

Boston University
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.

Cooper University Health Care
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.

Baylor University
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.

University of South Carolina
University of South Carolina

The Molinaroli College of Engineering and Computing at the University of South Carolina fosters innovation by preparing engineers and computing professionals to develop new technologies that will improve lives and the world. The college is committed to addressing real-world challenges through education, research, and community engagement.

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Arizona State University

ASU Crosscutting Technologies to Enhance Military Medical Readiness and Resilience

Dr. Rachel Cassalia leads MTEC-supported efforts at ASU to shift military health from reactive care to proactive readiness through data-driven prevention.

University of Pittsburgh

Topical Metformin Lotion for Tendinopathy Prevention

University of Pittsburgh study shows topical metformin prevents overuse tendon injury. This work was sponsored through MTEC project 22-02-MPAI-014.

Theradaptive

Theradaptive’s RESTORE Trial Initiation for OsteoAdapt™ DE

Theradaptive launches RESTORE Phase I/II trial of OsteoAdapt™ DE for faster, precision bone healing in craniofacial injuries. MTEC-funded study targets improved recovery for military and civilian patients.

Altec

Autonomous Rehabilitation Device for Improved Recovery in Warfighters

The OSCIR platform—developed by Altec Inc in collaboration with the Naval Health Research Center—is bringing clinical-grade, real-time movement analysis to the front lines. Funded under MTEC-22-02-MPAI

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