Drexel faculty researchers working on the cutting edge of cell and gene therapy shared insights and information about their groundbreaking work at Drexel’s Cell & Gene Therapy Symposium held in May 2023. Faculty from across the University spoke about their visions to treat or even cure devastating conditions ranging from neurodegenerative diseases to idiopathic pulmonary fibrosis to HIV, and debilitating injuries including heart attacks, spinal cord injury and acute kidney injury.

BATTLING NEURODEGENERATION

Among the symposium speakers was College of Medicine alumnus Liang Oscar Qiang, MD, PhD, assistant professor, Department of Neurobiology & Anatomy. Qiang, who completed his PhD at Drexel in the Molecular & Cell Biology & Genetics program in 2009, has extensive knowledge and expertise in the field of neurodegenerative disease research.

After completing his pivotal postdoctoral research on Alzheimer’s and Parkinson’s disease at Columbia University Medical Center, Qiang joined Angiocrine Bioscience in 2013 as a principal scientist. There he played a vital role in establishing an in vitro platform of the blood-brain barrier for drug screenings in the central nervous system, as well as a CNS-derived microvasculature environment to support neuronal reprogramming.

In 2015, Qiang returned to Drexel, where he now leads a research laboratory that focuses on using human induced pluripotent stem cell-based technologies to develop in vitro and ex vivo cellular and tissue models.

Qiang spoke about his gene and cell therapy research in neurodegenerative disease, specifically Alzheimer’s disease and Alzheimer’s-related dementia. “As a scientist, I believe in hypothesis-driven studies and mechanistic analyses,” he says. “I want to know the causative pathways, essential modules and molecules involved in the disease progression. In my research, we are trying to find the underlying mechanism that leads to tauopathies, which are a group of neurodegenerative disorders such as Alzheimer’s disease, Parkinson’s disease and frontotemporal dementia, characterized by the deposition of abnormal tau protein in the brain.”

He explains, “Our research seeks to understand how certain changes in the brain’s internal structure, specifically related to tiny structures called microtubules, are caused by two types of abnormal situations found in tauopathies. They are known as ‘reduced functional tau’ and ‘toxic oligomeric tau aggregates.’ By studying these two mechanisms, we hope to gain insight into the underlying causes of tauopathies, which could lead to better treatments for these neurological disorders.”

In addition to tauopathies, Qiang’s lab is using the human induced pluripotent stem cell-derived CNS organoid and assembloid models to study hereditary spastic paraplegia and Gulf War illness. “By employing this cutting-edge technology, we have the ability to extract human skin cells and reprogram/ develop them into fully functional brain cells and three-dimensional central nervous system tissues. This approach allows us to effectively model various neurological diseases in a laboratory setting,” he notes.

“Consequently, we can conduct extensive drug screening experiments and identify potential therapeutic candidates for treating these conditions. Additionally, the generated functional brain cells and central nervous system tissues hold immense promise as a potential source for transplantation, thus paving the way for personalized medicine strategies in the field of neurodegenerative disorders.”

Qiang emphasizes the importance of patient involvement in cell and gene therapy research efforts. “I want to inspire and encourage patients to be involved as much as possible — attend conferences, read articles, participate in clinical or preclinical studies,” he says. “For example, right now, I’m using human fibroblasts and turning them into stem cells for other diseases I’m studying. If we all put forth effort toward our research goals, we will be able to conquer diseases one day earlier.”

Collaboration among researchers is also of paramount importance, according to Qiang. “Events such as the Cell & Gene Therapy Symposium give us opportunities for face-to-face contact, which is invaluable,” he says. “To learn about what other researchers are doing in detail is quite eye-opening. And the opportunities for collaboration are enormous. Working together, we can save time and resources, and move the research forward faster.”

BREATHING NEW LIFE INTO SPINAL CORD INJURY RESEARCH

At the College of Medicine’s Marion Murray Spinal Cord Research Center, Michael Lane, PhD, associate professor in the Department of Neurobiology & Anatomy, leads efforts to develop novel and innovative cell therapy strategies for repairing damaged neural tissue, particularly spinal cord injury. He began his research in his native Australia during postdoctoral training at the University of Melbourne, and later at the University of Florida. He accepted his position at Drexel in 2013 to continue his research into spinal cord injury, neuroplasticity, and strategies to optimize repair and lasting functional recovery.

Most recently, his lab has been collaborating with Drexel alumna Lana Zholudeva, PhD, staff research scientist at the renowned Gladstone Institutes in San Francisco. Zholudeva completed her PhD in neuroscience at the College of Medicine in 2018, focusing on the study of spinal interneurons, the neurons that physically reside and communicate in the spinal cord. In particular, she studied how these cells change after injury to the central nervous system and ways that scientists may harness this plasticity for functional improvement. At Gladstone, she is engineering human spinal interneurons from pluripotent stem cells and testing their therapeutic efficacy for promoting repair and recovery after injury and neurodegenerative disease (see Winter 2022 Alumni Magazine).

“Using the human cells that Dr. Zholudeva is engineering has significantly advanced our research,” says Lane. “With this technology, we can engineer specific types of neurons and glia that will be most reparative, and that’s where our research teams are a bit ahead of the game for cell therapies being used in spinal cord injury,” he explains. “We’re focusing on a cell called the V2a spinal neuron, which contributes to the recovery of locomotive functions and breathing. It has repeatedly been shown to be beneficial in our preclinical testing, without any observed adverse effects.

“Impaired breathing for people with spinal cord injury is not only devastating to their quality of life, but it’s also life threatening,” Lane continues. “If we can improve their quality of breathing, it will make very significant differences to their longevity and quality of life. If this same cell type can improve arm function, for example, for someone who has quadriplegia, they might be able to use their arms more effectively for activities of daily living such as buttoning a shirt or brushing their teeth. To be able to give them that level of recovery and independence would be a huge improvement in their quality of life.”

Lane and Zholudeva aim to scale up cell production of the V2a spinal neuron to the point where they can translate it as a therapy for people with spinal cord injury. If successful, this therapy will provide an innovative and effective cell therapy for spinal cord injury, and one that could become more broadly applicable to other types of neural injury and disease, according to Lane. They hope that it can be applicable more broadly to treating other types of neural injury and disease.

“Our research team is looking at other reparative cell types as well,” Lane adds. “We have several more cell candidates that we are testing to see if they are equally beneficial for treating a range of other functions, such as bladder and bowel function and pain. Personally, I’m very invested in finding ways to improve the translational path to treatment. And it’s good to see an investment and interest across the entire University, and the city of Philadelphia overall, in the growing field of cell and gene therapy.”

CRISPR FOR A CURE

Another Drexel alum involved in cell and gene therapy is Will Dampier, PhD bioinformatics ’11, BS bioinformatics ’06, who is an associate professor in the Department of Microbiology & Immunology and the Center for Molecular Virology and Gene Therapy within the Institute for Molecular Medicine and Infectious Disease. In collaboration with Brian Wigdahl, PhD, professor and chair of microbiology and immunology, and Michael Nonnemacher, PhD, professor of microbiology and immunology, Dampier is engaged in HIV gene editing to utilize CRISPR-based technologies to work toward an HIV cure. Their strategy directly targets the virus hiding within an infected individual’s cells, unlike current therapies, which only suppress the virus. By delivering a properly targeted CRISPR-Cas9 protein to a patient’s genome, the protein will cut the viral genome and remove it. Dampier studies the genetic variability of the HIV virus, which enables him to target the therapy in a way that will be both safe and effective for a wide patient population. He does this using a population drawn from the Greater Philadelphia area who are patients at Drexel’s Partnership Comprehensive Care Practice. His work is part of a wider effort at the College of Medicine to study the impacts of HIV and develop a cure.

HARNESSING THE HIVE MIND

“At Drexel, we have researchers from diverse backgrounds and interests striving to advance cell and gene therapy,” notes Kara Spiller, PhD, a professor in the School of Biomedical Engineering, Science and Health Systems, who developed the recent Cell & Gene Therapy Symposium and leads tissue engineering research in her lab. “The next phase of research in this area will require all of their input. We must capitalize on their diversity of expertise to develop new, innovative research pathways, as well as educational initiatives.”

“Drexel researchers have great enthusiasm for cell and gene therapy because it makes miracles happen,” says Mary Genevieve Carty, MS, MHEd, director and senior program manager, Department of Microbiology & Immunology, who has played a key role in developing new cell and gene therapy graduate programs at Drexel. “It literally can make the blind see again. We are seeing this with new drugs such as Spark Therapeutics’ Luxterna. At Drexel, we’re working on some of the scientific underpinnings that support these revolutionary breakthroughs, and it’s really inspiring.

“The fast pace at which this is happening is really quite unbelievable,” Carty continues. “This time last year, there were eight FDA-approved gene therapies and now, one year later, there are more than 30. Recent advances are bringing to market therapies for conditions like hemophilia and sickle cell anemia, which will affect a huge swath of the population. The hope and promise that come from gene therapy cannot be overstated.”

NEW AND ONGOING PROGRAMS AND INITIATIVES

The College of Medicine offers a number of new and ongoing programs and initiatives designed to support and advance cell and gene therapy research and education.

Drexel’s Master of Science in Biomedicine and Cell and Gene Therapy. One of only two such master’s programs in the United States, this program offers a unique graduate-level opportunity for students at the intersection of medicine and bioengineering.

An ever-evolving global burden of disease requires new innovations in pharmaceuticals, biotech products such as antibodies and vaccines, and medical devices. This program empowers students to contribute to these critical areas of research and development. Students take courses in both the College of Medicine and the College of Engineering, providing a unique collaboration between medicine and engineering.

Certificate in the Regulatory Affairs of Cell & Gene Therapy. The only post-baccalaureate certificate program of its kind offered by a university, this program trains leaders for the unique challenges of this growing discipline and prepares them to bring new therapeutics to market. Program courses are taught by College of Medicine faculty as well as adjunct faculty who are currently working in cell and gene therapy, biotech and the pharmaceutical industry.

Molecular & Cell Biology & Genetics Program – MS/PhD. This interdisciplinary graduate program offers both MS and PhD degrees, focusing on the study of the structure, function and makeup of biologically important molecules within the context of living cells. Curriculum and research activities are tailored to students’ needs and interests in biomedical problems that cross disciplinary boundaries.

Program faculty are at the forefront of new advances in the biomedical sciences and new developments in techniques for understanding the genetic and molecular basis of developmental pathways and disease states such as cancer, aging, AIDS, malaria and neurological disorders. This intensive and research-oriented program provides students with opportunities to perform cutting-edge biomedical research employing multidisciplinary strategies.

$1 Million Curriculum Development Grant. The College of Medicine’s Department of Microbiology & Immunology and Drexel’s School of Biomedical Engineering, Science and Health Systems secured a joint department grant for almost $1 million dollars from Bristol Myers Squibb. The award, Curriculum Development for Cell & Gene Therapy Technology, Engineering, Analytics, Manufacturing & Science (CGT-TEAMS), is being used to support cell and gene therapy curriculum, co-ops, research, outreach and other related endeavors at Drexel.

Drexel’s Cell and Gene Therapy Institute. The University’s Cell and Gene Therapy Executing Team, which was run by the Drexel Solutions Institute and reported to President Fry, was charged with creating a blueprint for a university-wide Cell and Gene Therapy Institute, as well as an assessment and evaluation of CGT current and future initiatives, including potential custom training, courses, degrees and enhanced partnerships.

CELL & GENE THERAPY SYMPOSIUM

Speakers at the May 2023 symposium demonstrated the wide-ranging cell and gene therapy research being conducted at Drexel.

CELL THERAPY

Wendy Clemens Trigona, PhD, MS
Vice President, Bristol Myers Squibb

Kara Spiller, PhD
School of Biomedical Engineering, Science and Health Systems
Immune cell therapy for regenerative medicine

Shaoping Hou, MD
Department of Neurobiology & Anatomy, College of Medicine
Rebuilding supraspinal regulation of sympathetic input to improve cardio-electric disorders after spinal cord injury

Yinghui Zhong, PhD
School of Biomedical Engineering, Science and Health Systems
Biomaterial-enabled cell therapy for neuronal repair

Christopher Rodell, PhD
School of Biomedical Engineering, Science and Health Systems
Biomaterials for cell therapy

Masoud Soroush, PhD
Department of Chemical and Biological Engineering, College of Engineering
mRNA vaccine manufacturing

GENE THERAPY

Federico Mingozzi, PhD
Chief Science and Technology Officer, Spark Therapeutics

Liang Oscar Qiang, MD, PhD
Department of Neurobiology & Anatomy, College of Medicine
Anti-sense oligonucleotides for neurodegenerative disease

Emanuela Piermarini, PhD
Department of Neurobiology & Anatomy, College of Medicine
Gene therapy for neurodegenerative disease

William Dampier, PhD
Department of Microbiology & Immunology, College of Medicine
Gene editing to cure HIV

Catherine von Reyn, PhD
School of Biomedical Engineering, Science and Health Systems
Optogenetic control of neurons

Wei-Heng Shih, PhD
Department of Materials Science and Engineering, College of Engineering
RNA/DNA-based biosensors

Michele Kutzler, PhD
Departments of Medicine and Microbiology & Immunology, College of Medicine
DNA vaccines for infectious disease

 
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