Gene Therapy Insights

Matthew Scholz is a serial entrepreneur with a background in computer science and immunology, bridging the best of the fields of physics, computing, genetics and medicine, he is on the forefront of anti-aging technologies and non-ordinary approaches to rare disease gene therapies. Matt is a Chief Executive Officer and co-founder of Oisín Bio, a company focused on mitigating the senolytic technology to combat age-related diseases, With a gene delivery technology based on non-viral proteo-lipid vehicles. Matt is the founder of Immusoft, a biotech developing a breakthrough technology that turns a patient's B cells into miniature drug factories. He is a Co-Founder and Board Director of Sigma Genetics, Inc is building a non-invasive device that can deliver charged molecules, such as DNA, RNA, and proteins into patient cells. He is a CEO and Board Member, OncoSenX, Inc. (a developer of transient gene therapies designed for solid tumors). Lastly, he is a Co-Founder and Chief Technical Officer of Aegis Life, a company that works to protect human health from infectious diseases through Fusogenix technology. In our conversation Matt shares his fascination with programming and immunology, especially the concept of immune memory and creation of the immune software, hence, Immusoft, where B cells are reprogrammed to become enzyme producing factories. Matt discusses the MPS1 program and the work it took to dose the first patient with this detrimental disease. We lead the discussion into the information theory of aging, treating genetic disease as misinformation and hence, its correction. We touch on the transposon systems and the mysteries of retroviral DNA. We then get into viral versus non viral approaches to gene therapy. You will be delighted to learn about the progress in follistatin gene therapy for enhancing muscle growth, senolytic gene therapy to tackle age related diseases and the continuous ever changing views that FDA takes on such uncharted territories. We are thrilled to bring you this episode and we hope it amuses you with new and groundbreaking ideas! As always, nothing on this podcast should be considered a medical advice, and opinions expressed are speakers' own. Like and Subscribe to support us! Leave a comment with your questions and suggestions for the future episodes!

In today's episode, we dive into the base editing approaches in Spinal Muscular Atrophy (SMA), a degenerative motor neuron disease caused by the loss of motor neurons in the spinal cord. Our guest is Dr. Mandana Arbab is the Lodish Family Assistant Professor of Neurology at Harvard Medical School and faculty member of the Rosamund Stone Zender Translational Neuroscience Center at Boston Children's Hospital. She received her Ph. D. in Regenerative Medicine at the Hubercht Institute in the Netherlands and completed her postdoctoral fellowship at the Broad Institute with Professor David Liu.  Dr. Arbab sheds light on the genetics of SMA, emphasizing the critical role of SMN genes and the potential of base editing to bring about transformative treatments. SMA stands out as the most common genetic cause of infant death worldwide, affecting patients within the first few months of life. The podcast delves into the existing therapeutics for SMA, such as antisense oligonucleotides and AAV gene therapy, examining their limitations and the quest for more precise and long-term solutions. The concept of base editing takes center stage, offering a promising approach to correct the genetic mutations associated with SMA. Join us as we discuss the challenges and opportunities of bringing base editing into clinical trials. Learn about the intricacies of delivery mechanisms, biodistribution, and the potential for combining base editing with existing therapies. The conversation extends to considerations for clinical trials, including addressing off-target effects and the complexities of patient eligibility. The podcast concludes with a glimpse into the future of CRISPR genome editing, and the exciting potential for transformative treatments in the realm of rare diseases. Tune in to “Gene Therapy Insights” and embark on a journey through the cutting-edge world of gene therapy, where science meets hope, and the future of medicine unfolds.

Tune in to hear from Dr. Shahaf Peleg, PhD, a co-founder of Luminova Biotech and a researcher at the Research Institute for Farm Animal Biology as we explore the exciting work by the pioneers in the field of tackling age-related disorders with a light-activated energy replacement tool. Our guest is Shahaf Peleg, PhD, a co-founder of Luminova Biotech and a researcher at the Institute for Farm Animal Biology.

The debut episode hosted by Elena Buglo, PhD features Josh Yoder, PhD, a medical affairs leader with expertise in virology and gene therapy, focusing on hemophilia. Our discussion delves into:

Embark on a journey into the future of medicine!