Regenerative Medicine Minnesota awards 13 research grants for 2024

Regenerative Medicine Minnesota awards 13 research grants for 2024

By
Susan Buckles

October 3, 2024

Regenerative Medicine Minnesota (RMM) has awarded 13 grants totalling $4.3 million to support discovery, development, translation and commercialization of regenerative medicine-based innovations to improve human health in Minnesota and beyond.

The award-winning projects and awardees for 2024 are:

Clinical Trial Award:

Phase 1 Study of Intravenous Administration of Adeno-Associated Virus Gene Therapy for Individuals with Propionic Acidemia

Michael Barry, Ph.D., Mayo Clinic

Propionic acidemia (PA) is a severe metabolic disorder caused by mutations in the PCCA gene. Traditional treatments, including stringent life-long protein restrictions and liver transplants, have limited success and significant risks. This trial will evaluate the safety and preliminary efficacy of a groundbreaking gene therapy in young children, aiming to reduce metabolic crises, improve quality of life, and preserve cognitive function. The successful execution of this project will represent a significant advancement in the treatment of PA, offering hope to patients and their families.

Infrastructure Award:

Expanding Anatomic Infrastructure for Scaling Manufacturing Operations

Patrick Walsh, Anatomic Incorporated

Anatomic, a University of Minnesota start-up company based in Minnesota, develops neural tissues used in basic science and drug development. Its current flagship product, RealDRG, can be used to support development of novel non-opioid pain medications to address the ongoing, major unmet clinical need given the substantial life loss due to the opioid epidemic. This grant will support expansion of Anatomic's facilities to produce additional neural tissues that will enable researchers to understand central mechanisms of pain and amyotrophic lateral sclerosis (ALS) resulting in new therapies for these diseases.

Discovery Science Awards:

Reactivating Netrin1/DCC Signaling to Promote Optic Nerve Regeneration

Zhe Chen, Ph.D., University of Minnesota

Vision impairment that results from optic nerve damage, such as from glaucoma, poses a significant challenge to public health. To date, there are no effective therapeutics to block or reverse optic nerve degeneration, leading to irreversible vision impairment, including blindness. This project aims to identify molecular mechanisms that could be targeted therapeutically to promote regeneration of the optic nerve.

Human Microglia Model of Spinocerebellar Ataxia Type 1 (SCA1)

Marija Cvetanovic, Ph.D., University of Minnesota

Spinocerebellar ataxia type 1 (SCA1) is a dominantly inherited neurodegenerative disease with no disease modifying treatments or therapies available. Patients with SCA1 have relentlessly progressive movement and cognitive deficits and die prematurely 10 to 20 years from the disease onset. The goal of this project is to develop a human microglia (the immune cells of the brain) model of SCA1 to better understand the disease development and how mutations in the ATXN1 gene contributes to disease progression. Successful completion of this project will pave the way for future studies to develop therapies that will target and modulate microglia resulting in better treatments for patients with SCA1.

Illuminating Elusive Proteins Associated with Age-Related Macular Degeneration

John Hulleman, Ph.D., University of Minnesota

Age-related macular degeneration (AMD) is the leading cause of blindness in the industrialized world. Although variants in two genes—ARMS2/HTRA1—are the largest genetic contributor to AMD, their role in disease pathology has been challenging to study with existing methods. The goal of this project is to develop a new approach to gain insight into the pathobiology of ARMS2 and its relevance to AMD. Researchers will develop a series of stem cells that will eventually be used to identify new treatments that promote retinal health and prevent AMD-associated blindness.

A Novel Combined Allogeneic and Autologous Cell Therapy for Meniscus Tissue Repair

Daniel Saris, M.D., Ph.D., Mayo Clinic

Despite advanced surgical techniques, meniscus repair remains a significant clinical challenge due to high failure rates and chronic disability among young patients. This project aims to address this issue through a novel, one-stage cell therapy combining allogeneic adipose-derived mesenchymal stem cells and autologous meniscus cells to promote regeneration. This therapy builds on the researchers' pioneering work with a similar approach for cartilage injuries, which showed promising results in clinical trials. The anticipated outcome is a paradigm shift in meniscus injury management, significantly reducing long-term disability and improving quality of life for young patients.

Translational Research Awards:

Feasibility of Reducing Inflammation and Lung Injury to Regenerate Lung Tissue Using an Extracellular Biotherapeutic Aerosol in a Model of Acute Respiratory Distress Syndrome (ARDS)

Bryce Beverlin II, Ph.D., Quench Medical

Acute respiratory distress syndrome (ARDS) is a severe form of lung injury and a common cause of respiratory failure. Effective drug therapies that address underlying mechanisms have not been realized and ARDS remains a leading cause of death in critically ill patients. Biologic therapeutics derived from mesenchymal stem cells and exosomes are currently being evaluated as a therapeutic strategy. However, their effectiveness is limited by the ability to reach the lungs. To overcome this limitation, Quench Medical is developing a minimally invasive inhaled biologic aerosol that delivers exosome-based therapeutics directly to lung tissues. This project aims to demonstrate the efficacy of this approach in reducing inflammation from ARDS and regenerating damaged lung tissue.

Cryopreservation of Pancreatic Islets to Achieve Diabetes Cure through Transplantation

Erik Finger, M.D., Ph.D., University of Minnesota

Diabetes is a debilitating disease that has a tremendous impact on a person's health and wellbeing. Pancreatic islet transplantation offers a potential cure, yet an insufficient quantity of high-quality islets currently limits the success of this treatment. A method for cryopreserving or “banking” of islets prior to transplant can achieve the high viability, function, and clinical scalability required for human transplantation. This study will validate the function of cryopreserved islets using the novel technology cryomesh vitrification-rewarming with the goal to launch a clinical trial of vitrified islet transplants to increase accessibility of pancreatic islet transplants to cure diabetes.

Rotator Cuff Regeneration Using BMP5

Scott Riester, M.D., Ph.D., Mayo Clinic

Rotator cuff disease causes severe shoulder pain and limited arm movement. Current treatments offer temporary relief but do not regenerate damaged tendons. Preliminary studies show that injections of the biologic agent BMP5 can improve tendon strength by 30 percent. This project aims to complete pre-clinical studies to evaluate BMP5’s safety for rotator cuff treatment and has the potential to provide a new regenerative treatment option and reduce the need for surgery.

Optimized Chimeric Antigen Receptor T Cell Therapy for Colorectal Cancer

Omar Gutierrez Ruiz, Ph.D., Mayo Clinic

Colorectal cancer (CRC) is the third most common cancer in Minnesota, yet current treatments for advanced CRC have low survival rates. This project aims to develop a targeted cell therapy that enhances the killing of cancer cells and disrupts the tumor microenvironment. The researchers will further optimize this therapy in preclinical models, paving the way for a Phase I clinical trial to improve outcomes for CRC patients.

Use of Human Lung Exosomes to Prevent Ischemia/Reperfusion Injury in Lung Transplantation

Sahar Saddoughi, M.D., Ph.D., Mayo Clinic

This study aims to mitigate ischemia reperfusion injury (IRI), a major cause of primary graft dysfunction in lung transplants, by using an FDA-approved lung spheroid cell exosome (LSC-Exo) therapy. Preliminary studies show that LSC-Exo improves lung function and reduces IRI. Researchers will test LSC-Exo in a lung transplant model and in human donor lungs rejected for transplant. Their goal is to enhance lung transplant outcomes, increase the number of usable donor lungs, and improve patient survival and quality of life. Successful completion of this study will pave the way for clinical trials.

Functional Correction of Mucopolysaccharidosis Type I (Hurler Syndrome) Using Genetically Modified Autologous Memory T Cells

Nicole Shirkey-Son, Ph.D., Kommodo Therapeutics

This study aims to develop safer and more effective therapies for patients with a class of genetic diseases, called enzymopathies resulting from missing or defective enzymes. Current therapeutic approaches fail to stop disease progression and adequately replace deficient enzymes. Researchers are developing a novel cell-based therapeutic approach using genetically modified autologous memory T cells that is capable of delivering sustained enzyme replacement. In this project researchers will complete studies on efficacy, safety, dosing, and manufacturing that are critical for translating their novel cell-based therapy for MPS I. Successful completion of this project will advance this therapeutic approach into a clinical trial to improve outcomes for patients with MPS I.

Androgen Receptor Blockade to Augment Liver Regeneration

Rory Smoot, M.D., Mayo Clinic

A liver resection, or hepatectomy, is a surgical procedure to remove part of the liver. Post-hepatectomy liver failure is a major complication, with no approved treatments currently available. This project focuses on repurposing an FDA-approved drug to enhance liver regeneration, addressing a significant unmet need for patients undergoing liver resection. Researchers will assess the efficacy and safety of this approach, ensuring it does not promote tumor growth. Successful completion of these studies will pave the way for clinical trials, potentially offering a new therapeutic option to prevent or treat post-hepatectomy liver failure and significantly improve patient outcomes.

2025 Request for Proposals

For 2025 awards, RMM is calling for transformative proposals aimed at overcoming barriers that currently prevent or hinder regenerative medicine therapies from getting into patients. Key barriers to translation in regenerative medicine include availability of preclinical models and human microphysiological systems, lengthy and expensive development timelines, scalability of manufacturing processes and product consistency. RMM aims to support projects that leverage strengths in Minnesota to overcome these barriers and position the state at the forefront of regenerative medicine. Letters of Intent are due Nov. 8. Learn more at www.regenmedmn.org/apply-grant

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About Regenerative Medicine Minnesota

Regenerative Medicine Minnesota was established in 2014 by the Minnesota State Legislature to improve the health of Minnesotans by advancing regenerative medicine. This state-wide initiative opens new economic opportunities through commercialization of technologies and leverages the strengths of Minnesota institutions to position the state at the forefront of regenerative medicine. The initiative distributes approximately $4 million in funding statewide every year for research, commercialization, and clinical translation initiatives that improve or increase access to scientifically proven regenerative medicine throughout the state. Learn more at www.regenmedmn.org.

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