Vaxx Research Incorporated
Vaxx Research Incorporated is developing non-viral prime editing therapeutics to conquer Limb-Girdle Muscular Dystrophy. By targeting the pathogenic root of the disease, we are engineering the future of permanent genetic medicine.
Limb-Girdle Muscular Dystrophy R1 (LGMDR1), also known as LGMD2A or calpainopathy, is a debilitating autosomal recessive neuromuscular disorder. It is driven by pathogenic variations in the CAPN3 gene, which paralyze the calpain-3 protease. This calcium-dependent cysteine protease is critical for controlling cellular pathways such as cytoskeletal rearrangements and sarcomeric remodeling.
Accounting for approximately 40% of all limb-girdle muscular dystrophy cases worldwide, patients suffer from progressive muscle weakness, atrophy, bilateral scapular winging, and Achilles tendon contracture. With over 440 different reported pathogenic variants—mostly missense mutations dispersed throughout the gene—a precision intervention is urgently required.
Current AAV gene therapies are plagued by systemic hepatotoxicity, ectopic cardiac fibrosis, and immunological barriers that prohibit critical re-dosing. We utilize Transferrin-targeted Lipid Nanoparticles (TfR-LNPs) to hijack natural iron-transport pathways, bypassing the liver sink entirely and delivering payloads directly to skeletal muscle cells.
Acting as a molecular word processor, our Prime Editing framework executes precise C-to-G transversions. This permanently corrects the mutation at the genomic level without causing dangerous, unpredictable double-strand DNA breaks. By remaining entirely non-viral, our system evades capsid-based immune responses, allowing for infinite, safe re-dosing in an outpatient setting.
This initiative is named for Khushboo, a 26-year-old patient currently battling LGMDR1. Her condition has reached a critical tipping point—she is now limited to walking just 30 steps and can no longer climb stairs. Because the disease's progression rapidly accelerates at these later stages, our timeline to deliver a permanent, non-viral genetic cure is extremely urgent.
Our initial beachhead is the endogamous Indian Agarwal community, where just two founder mutations (c.2338G>C and c.2051-1G>T) account for 89% of LGMDR1 cases. This hyper-concentration provides the perfect N=1 start for validating our precision non-viral gene editing framework.
Beyond our initial founder mutations, we are expanding our CAPN3 variant database and engineering customized mRNA-lipid nanoparticles to target additional variants, such as the c.1354+5G>A pathogenic variant, addressing a $600 Million Serviceable Available Market.
Project Khushboo spans six years from virtual laboratory infrastructure to GMP clinical dosing. With capital requirements of $26.5M, we are building the independent technical foundation for scalable, global therapeutics.
Paul oversees Vaxx’s corporate strategy, clinical commercialization roadmap, and intellectual property portfolios, translating advanced gene editing concepts into viable therapeutic pipelines. He brings an extensive academic background, including completing his Master of Theological Studies at the University of Toronto and his foundational biological studies (BSCH, Biology) at Queen's University, where he received the Gold Medal in Biology. He also studied at the University of Calgary (BSc, Cell/Cellular and Molecular Biology), earning Dean's List honors before pursuing medical studies at the University of Alberta and Queen's University.
View LinkedIn Profile →Dr. Wensel drives the technical architecture of Vaxx’s non-viral delivery systems and metabolic engineering pipelines. He holds a Ph.D. in Biological Systems Engineering from Washington State University and M.S. degrees in Chemical Engineering and Artificial Intelligence. His extensive background spans roles such as Chief Science Officer at Spira Inc., Manufacturing Systems Engineer at Merck, and Owner of Dronext, bringing deep expertise in bioprocess engineering, process development, and downstream processing crucial for our LNP manufacturing and clinical upscaling.
View LinkedIn Profile →Anupam drives Vaxx's commercial strategy, bridging the gap between our advanced RNA research and pharmaceutical industry development. He brings deep scientific experience from his roles at Millennium Pharmaceuticals, Acceleron Pharma, and Codexis, where he focused on transfection optimization with mammalian cell lines, cell-based reporter assays, directed evolution of enzymes, and generating vectors for transgenic and knockout construction. He combines this technical foundation with extensive commercial execution, having established and managed regulated pharmaceutical trading and B2B operations across Southeast Asia.
View LinkedIn Profile →Christian is a chemical engineer and biochemist focused on process development for cell and gene therapy, nanomedicine, and drug delivery. He previously served as Co-Founder and CTO at Ligandal, where he led platform development and process design for non-viral vector production. He also brings specialized expertise from his time as a Production Specialist at Bayer, focusing on the clinical stage manufacturing of allogeneic cell therapies.
View LinkedIn Profile →Dr. Kumaresan brings crucial expertise from her role as a Scientist in RNA Technical Operations at Aldevron, where she executed RNA Synthesis including In Vitro Transcription, enzymatic capping, and Poly(A) Tailing. Her extensive postdoctoral research includes isolating CD34+ hematopoietic stem cells at North Dakota State University and reprogramming PBMCs into induced pluripotent stem cells (iPSCs) for regenerative biology applications at Western University.
View LinkedIn Profile →Dr. Harrison provides critical scientific oversight, bridging advanced university research in cellular pathways and molecular microbiology with Vaxx’s therapeutic engineering processes. He serves as a Professor at the University of Calgary, where he previously held a Canada Research Chair and established a microbial genetics, genomics, and biofilm core. He holds a PhD in Microbial Biochemistry and dual BSc degrees from the University of Calgary, with additional experience as a Senior Fellow at the University of Washington.
View LinkedIn Profile →Pawan leads the physical development of our automated laboratory screening hardware. He holds an MSc (Distinction) in Aeronautical/Aerospace Engineering from the University of Leicester and a BTech in Aerospace Engineering from Sandip University. With experience in CFD, ANSYS, COMSOL, CATIA, and SolidWorks, his background includes gas turbine modeling, airship design, flight operations support at Star Air, and a DRDO Internship at the Advanced Centre for Energetic Materials.
View LinkedIn Profile →Rahul designs the genetic architectures at the core of our platform. He engineers the mRNA constructs and Prime Editing components necessary to execute precise transversions and restore CAPN3 functionality. He brings practical experience as a Research Assistant at the University of Cincinnati and as a Student Assistant in Neurology and Nephrology labs at the University of Alabama at Birmingham (UAB), where he conducted in-vivo studies using humanized mouse models and utilized techniques such as RT-PCR and Western Blotting. He holds a Master's degree in Biotechnology from UAB and a Bachelor's degree in Microbiology from Bhavans New Science Degree College.
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