Six disruptive gene and cell therapy (GCT) advancements from Mass General Brigham have been awarded Innovation Discovery Grants (IDG) in the 2021 round of awards in the highly competitive IDG program. Each of the potential patient care and healthcare delivery breakthroughs in GCT will receive $100,000 toward ongoing development and future commercialization, based on the potential to improve health outcomes, meet articulated milestones, and attract follow-on investment as assessed by independent industry experts.
With its GCT focus, this round of the IDG considered discoveries in all clinical areas that treat a genetic disorder by influencing the structure, expression or inhibition of a gene and gene product(s). In addition to the financial grants, recipients will have access to a new mentoring program provided by senior business development and research executives from Bayer, which is actively pursuing different kinds of partnerships with academia, start-ups and biotechs and considers those as innovation drivers in the pharmaceutical industry.
The Mass General Brigham Harvard faculty receiving this year’s IDG awards are:
In a new aspect of IDG, experts from Bayer will provide mentoring to the six IDG awardees, covering scientific, technological, strategic and commercial aspects of innovation from proof of concept to market. The awardees will benefit from Bayer’s long-standing history of drug discovery and development, as well as the company’s track-record in successfully bringing innovative treatments to patients. Bayer will also offer a more comprehensive mentoring program for select awardees, which will enable mentees to participate in Bayer’s Open Innovation Initiatives such as the Center for Regulatory Excellence, a mentoring session with an expanded stakeholder group from research, business development, drug discovery and other related disciplines, and CEO roundtables.
“Advancements in gene and cell therapies are likely to redefine the future of medicine, enabling new treatment alternatives, and, potentially, curative options,” said Ravi Thadhani, MD, Mass General Brigham Chief Academic Officer. “These translational projects represent a significant opportunity to improve outcomes for patients faced with challenging diseases, both widespread and rare, in some of the most promising areas of medical research.”
IDG supports translation of breakthrough research from the lab to real-world products. Jürgen Eckhardt, head of Leaps by Bayer, noted that “Advances in life sciences will continue, and being able to find and fund entrepreneurs and new biotechnologies will be key to overcome some of today’s challenges in healthcare. Leaps by Bayer’s mission is to invest in breakthrough technologies that could transform the lives of millions of patients for the better. Supporting scientific advancements at the cutting edge of GCT fits precisely with this ambition.”
Following are descriptions of each of the 2021 IDG awards:
Generating Superior 'Killers' for Adoptive Cell Therapy in Cancer, Lydia Lynch, PhD, Brigham and Women's Hospital. There is an unmet need for developing cell therapy that will work in solid tumors, which account for the majority of the 1.7 million cancer diagnoses every year. New cell therapies may offer the ability to reach and penetrate these tumors. The project aims to use 'innate T cells' for adoptive cell therapy for solid tumors, capitalizing on their innate homing abilities, use of donor blood products instead of patient blood, and their metabolic fitness to survive in the tumor.
Novel Strategies to Enhance Tfr Treatment of Autoimmunity, Peter Sage, PhD, Brigham and Women's Hospital. It’s estimated that 50 million people are living with autoimmune diseases in the U.S. alone, creating a large need for therapeutic strategies to limit a host of potentially debilitating, and in some cases, life threatening diseases. The project centers on cell therapy that deploys Follicular Regulatory T (Tfr) cells as a much more specific and potent way to limit B cell mediated autoimmunity in diseases such as multiple sclerosis, as well as for chronic rejection after kidney transplantation. It uses an in vivo CRISPR screen to identify new pathways that can form the basis for novel therapeutics.
Long-Lasting mRNA Therapy for Genetic Disorders, Jinjun Shi, PhD, Brigham and Women’s Hospital. The use of synthetic mRNA nanotherapeutics has attracted significant attention given the recent FDA emergency-use authorization of mRNA COVID-19 vaccines. This IDG grant supports research that aims to develop a clinically translatable lipid nanoparticle (LNP) platform technology enabling long-lasting mRNA therapy for genetic disorders. Goals are to achieve a prolonged duration of mRNA-mediated protein expression in mice for at least 30 days after a single injection; and validate the efficacy and safety of the new mRNA LNPs for a particular severe genetic bleeding disorder, hemophilia A. This long-lasting mRNA LNP technology could be readily expanded to other genetic diseases that require restoration of normal protein functions, and to other biomedical applications such as cancer and metabolic diseases.
AAV-Based Gene Replacement Therapy Improves Targeting and Clinical Outcomes in a Childhood CNS Disorder, Yulia Grishchuk, PhD, Massachusetts General Hospital. Mucolipidosis IV (MLIV) is a highly debilitating central nervous system disorder resulting in resulting in motor and cognitive deficits and vision loss among children. There is currently no therapy for this disease. The project is developing and testing adeno-associated virus (AAV) gene replacement therapy with improved biodistribution and tissue targeting to address complex pathology involving CNS dysfunction and vision loss. The research may also pave the way for expanding use of this vector for other diseases.
Towards a Permanent Genetic Cure for Spinal Muscular Atrophy, Benjamin Kleinstiver, PhD, Massachusetts General Hospital, Center for Genomic Medicine. Spinal Muscular Atrophy (SMA) is one of the leading causes of infantile death worldwide. While there are promising, FDA-approved therapies, these have potential limitations such as the need for repeated dosing or incomplete efficacy. The goal of this project is to develop a permanent genetic cure for SMA using novel genome editing technologies that target and permanently correct the disease-causing mutations of the SMN genes. This approach could establish a new paradigm for treating SMA, and, more broadly, other neurogenetic diseases.
Differentiation of Retinal Neurons for Cell Replacement in Glaucoma -- Petr Baranov, MD, PhD, Massachusetts Eye and Ear. An estimated three million people are affected by glaucoma in the U.S., and increasing lifespans exacerbate the disease’s socio-economic and quality of life impact. The human retina lacks the ability to regenerate, and glaucoma has historically led to irreversible vision loss. Currently no cell or other therapies are available to compensate for the lost function or to regenerate or replace retinal ganglion cells (RCGs). The long-term goal of the project is to develop cell replacement therapy for glaucoma with the potential to restore lost vision.
The IDG awards were announced as part of the World Medical Innovation Forum, which is focusing on gene and cell therapy and includes previews of other promising Mass General Brigham GCT technologies in its annual First Look program, and a new, virtual poster session.
Since IDG’s inception in 2014, 59 projects have been awarded a combined $3.6 million and together have raised more than $72 million to further their development. More information about the 2021 IDG program can be found on the IDG website.
Innovation is the 150-person business development unit of Mass General Brigham responsible for the worldwide commercial application of the unique capabilities and discoveries of Mass General Brigham's 74,000 employees. Innovation supports the research requirements of its 6,200 Harvard Medical School faculty and research hospitals. It has responsibility for industry collaborations, venture investing, international consulting, licensing, innovation management, company creation, technology marketing, open innovation alliances, and workforce development. Its annual World Medial Innovation Forum is underway virtually May 19-21.
The CAO oversees Mass General Brigham’s $1.9 billion research enterprise and furthers the healthcare system’s commitment to applied knowledge and academics for the advancement of patient care. Its core components include AI development, personalized medicine, research computing, human research affairs, graduate medical education and continuing professional development. The CAO serves as the Harvard Medical School Dean for Academic Programs at Mass General Brigham.
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to drive sustainable development and generate a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2020, the Group employed around 100,000 people and had sales of 41.4 billion euros. R&D expenses before special items amounted to 4.9 billion euros. For more information, go to www.bayer.com.
Mass General Brigham:
Director U.S. External Communications, Bayer
Mass General Brigham is an integrated academic health care system, uniting great minds to solve the hardest problems in medicine for our communities and the world. Mass General Brigham connects a full continuum of care across a system of academic medical centers, community and specialty hospitals, a health insurance plan, physician networks, community health centers, home care, and long-term care services. Mass General Brigham is a nonprofit organization committed to patient care, research, teaching, and service to the community. In addition, Mass General Brigham is one of the nation’s leading biomedical research organizations with several Harvard Medical School teaching hospitals. For more information, please visit massgeneralbrigham.org.