Kenneth Liechty, M.D.: Prevention and Treatment of Pulmonary Fibrosis
Dr. Kenneth Liechty is the Sandy Wolf Chair in Maternal Fetal Surgery and Director of the Colorado Fetal Care Center for Children’s Hospital at CU Anschutz. Dr. Liechty was awarded a second Grubstake Award to develop his platform technology for the treatment of acute lung injury leading to Acute Respiratory Distress Syndrome (ARDS). ARDS causes 150,000 adult ICU admissions and 75,000 deaths annually in the US. Currently, treatment for ARDS is palliative, and does not address the underlying causes. Dr. Liechty and his team have shown that their nanoparticle therapy is able to regenerate healthy lung tissue in a mouse model of acute lung injury. The Grubstake Award will allow them to develop an inhaled version of the nanoparticle therapy and do the additional safety studies required prior to a clinical trial in ARDS patients. Dr. Liechty is in the process of exclusively licensing this technology into Ceria Therapeutics, Inc.
Kunhua Song, Ph.D.: Heart Regeneration by Conversion of Non-Myocytes into Functional Cardiomyocytes
Dr. Kunhua Song, an Assistant Professor at CU Anschutz, was awarded a Grubstake Award to develop a genetic therapy for the treatment of heart failure. Eight million Americans suffer a heart attack annually; that is 3% of adults in the US. The annual cost for treating heart attacks is $12 billion, making it the most expensive condition treated in hospitals in the US. Even worse, 30-40% of heart attack patients develop heart failure, accounting for about 50% of all heart failure patients. The five-year survival rate for heart failure patients is only 50%. Despite the development of various therapies and medical devices such as the Left Ventricular Assist Device, the five-year survival rate has not changed in the past 20 years. Dr. Kunhua Song and his team have discovered a way to regenerate healthy cardiac cells from the fibrotic tissue causing heart failure using a gene therapy approach. The Grubstake Award will allow them to complete their animal studies and begin preparing to file an IND to enter clinical trials.
Michael Verneris, M.D.: Generation of Engraftable Hematopoietic Stem Cells from Induced Pluripotent Stem Cells
Dr. Michael Verneris is the Director of Bone Marrow Transplantation and Cellular Therapy and the Barton Family Endowed Chair of Bone Marrow Transplant at CU Anschutz. Dr. Verneris was awarded a Grubstake Award to development his technology for use in bone marrow transplantation. Bone marrow transplants are a high risk, expensive procedure (>$13 billion) used to treat leukemia, lymphoma, and genetic diseases. Two thirds of patients receive transplants from unrelated donors, which causes delays to find matching donors, and increases the complications and cost associated with the procedure. Recently, scientists have discovered how to create induced pluripotent stem cells (iPS cells) from a patient’s own cells, expand them, and convert them to bone marrow cells. However, these cells have not been successful at engrafting into the bone marrow in mouse models, indicating they would not be effective in a bone marrow transplant in patients. Dr. Verneris and his team have discovered that adding a small molecule during the conversion of the iPS cells allows engraftment of the cells in the mouse model. The Grubstake Award will allow them to confirm their initial finding in animal models, and to determine the most promising small molecule for clinical and commercial use.
Valeria Canto-Soler, Ph.D.: Stem Cell-Derived Retinal Transplant to Treat Dry-AMD
Dr. Valeria Canto-Soler is the director of CellSight, the Ocular Stem Cell and Regenerative Research Program in partnership with the Gates Center and Department of Ophthalmology at CU Anschutz. Dr. Canto-Soler was awarded a Grubstake Award for a stem cell-derived retinal transplant to treat dry, age-related macular degeneration. She hopes that one day her research will uncover the cure to blindness through transplanting retinal patches in patients’ eyes.
Karin Payne, Ph.D.: Bioresorbable 3-D Printed Personalized Implant for Cartilage Regeneration in Pediatric Growth Plate Injuries
Dr. Karen Payne is an assistant professor within the CU Anschutz Department of Orthopedics and is also the lab director for the Payne Regenerative Orthopedics Laboratory. Her lab focuses on the regenerative potential of stem cells for bone and articular cartilage tissue engineering to enhance bone fracture repair, improve spine function, and treat articular cartilage defects. Dr. Payne was awarded a Gates Grubstake Award for a bioresorbable 3-D printed personalized implant for cartilage regeneration in pediatric growth plate injuries.
Martin Zamora, Ph.D.: Clinical Development of Autologous CD117+ Progenitor Cell Therapy for Solid Organ Transplantation
Dr. Martin Zamora, now Professor Emeritus at CU Anschutz, joined the Division of Pulmonary Sciences and Critical Care in 1990 and served as directing pulmonologist for the Lung Transplant Program since it was founded in 1991. Dr. Zamora was awarded a Grubstake Award for clinical development of autologous CD117+ progenitor cell therapy for solid organ transplantation. His research aims to improve outcomes and quality of life following organ transplantation. Dr. Zamora, together with Robert Plenter, is in the process of preparing a pre-IND package for filing with the FDA for the use of the technology in preventing transplant rejection following solid organ transplant.
Jeffrey Olson, M.D.: Intraocular Device to Potentiate Retinal Stem Cell Transplantation in Macular Degeneration
Dr. Jeffrey Olson, an Associate Professor and Vitreoretinal Surgeon at CU Anschutz, was awarded a Grubstake Award to develop an intraocular medical device to protect retinal cells from damage and cell death for diseases with unmet medical needs such as dry Age-related Macular Degeneration. To continue development, Dr. Olson has established AmpVision, LLC, and is in the process of exclusively licensing this technology into AmpVision. Dr. Olson is actively working with the FDA to prepare for starting clinical trials.
Kenneth Liechty, M.D.: Conjugated Nanoceria to Treat Oxidative Stress and Prevent Chronic Inflammation
Dr. Kenneth Liechty is the Sandy Wolf Chair in Maternal Fetal Surgery and Director of the Colorado Fetal Care Center for Children’s Hospital at CU Anschutz. Dr. Liechty was awarded a Grubstake Award to develop a platform technology useful for treating a variety of diseases where inflammation and oxidative stress are pathognomonic in the progression of the disease. Dr. Liechty’s initial focus is treatment of diabetic ulcers, where compelling in vitro and animal model data exists showing that treatment results in closure rates of diabetic wounds equal to nondiabetic wounds. Dr. Liechty has already filed his pre-IND package with the FDA for this indication, and is currently working on his IND package. To continue development of this technology, Dr. Liechty has established Ceria Therapeutics, Inc., and is in the process of exclusively licensing this technology into Ceria.
David Wagner, Ph.D.: A 15-Amino Acid Peptide to Potentially Stop Demyelination and Restore Myelin Production in Multiple Sclerosis
Dr. David Wagner, an Associate Professor in the Webb-Waring Center at CU Anschutz, was awarded a Grubstake Award to develop a platform technology useful for treating a variety of autoimmune diseases including multiple sclerosis by blocking aberrant immune signaling. To continue development of this technology, Dr. Wagner established OpT, LLC, obtained two SBIR Phase II grants, and exclusively licensed this technology into OpT. Dr. Wagner has prepared and filed a pre-IND document with the FDA for the use of the proprietary peptide for the treatment of Type I diabetes, and is in the process of developing his IND package for filing with the FDA.
Ganna Bilousova, Ph.D., and Igor Kogut, Ph.D.: Induced Pluripotent Stem Cell Services as a Platform for Clinical Research
Dr. Ganna Bilousova and Dr. Igor Kogut, Assistant Professors at CU Anschutz, were awarded a Grubstake Award to develop a method to generate iPSCs in a clinically viable manner from a small number of cells, and without requiring genetic modifications. They have continued their work, developing a method of combined reprogramming and genetic correction currently being developed for use in the treatment of Epidermolysis bullosa (EB), a skin blistering disease without effective treatment. They are working with Dr. Dennis Roop, Director of the Gates Center for Regenerative Medicine, to prepare a pre-IND package to file with the FDA for the use of gene corrected keratinocytes for the treatment of EB. Dr. Bilousova and Dr. Kogut have also established the Stem Cell Biobank and Disease Modeling Core at CU Anschutz to provide access to their technology to scientists at CU Anschutz and worldwide.
Steven Dow, DVM, Ph.D., and Mary Ann DeGroote, M.D.: Antimicrobial Stem Cell Therapy for Infected Diabetic Foot Ulcers
Dr. Steven Dow, the Director, Center for Immune and Regenerative Medicine at CSU, and Dr. Mary Ann DeGroote, and Assistant Professor at CSU, were awarded a Grubstake Award to develop their activated mesenchymal stromal cell (MSC) product for use in antibiotic resistant non-healing wounds. Dr. Dow is in the process of preparing his pre-IND package to file with the FDA for the use of activated MSCs to treat antibiotic-resistant chronic orthopedic infections. To continue development, Dr. Dow has established Validus Cellular Therapeutics, Inc., and is in the process of exclusively licensing this technology into Validus.
Karin Payne, Ph.D., and Vikas Patel, M.D.: Structural Living Bone Allografts to Enhance Bone Formation in Orthopedic Surgery
Dr. Karin Payne, an Assistant Professor, and Dr. Patel, the Chief of Orthopedic Spine Surgery at CU Anschutz, were awarded a Grubstake Award to develop approaches to enhance bone formation in orthopedic surgery.
Xiao-Jing Wang, M.D., Ph.D.: Fusion Protein to Treat Chronic Wounds and Inflammatory Disorders
Dr. Xiao-Jing Wang is the John Gates Endowed Chair of Cancer Stem Cell Biology and Director of the Head & Neck Cancer Research Program CU Anschutz. Dr. Wang was awarded a Grubstake Award to develop Smad7-based fusion proteins for clinical use in several indications with unmet medical needs including oral mucositis, psoriasis, chronic wounds, scar prevention, and fibrotic diseases. To continue development of the technology, Dr. Wang founded Allander Biotechnologies, LLC and exclusively licensed this intellectual property portfolio. She has continued to develop the technology using funds from angel investors and two SBIR Phase II grants.
Antonio Jimeno, M.D., Ph.D.: Proprietary Humanized Mouse Model to Assess Cancer Treatments
Dr. Antonio Jimeno is the Director of the Developmental Therapeutics/ Pharmacodynamic Laboratory at CU Anschutz. Dr. Jimeno was awarded a Grubstake Award to develop humanized mouse models to identify improved cancer treatments.