First Look | World Medical Innovation Forum - Gene and Cell Therapy

First Look

First Look

Realizing GCT Therapies

At the recent World Medical Innovation Forum, 16 Mass General Brigham investigators presented their research into high-potential new GCT technologies, from cellular therapeutics to replace immunosuppressive drugs in kidney transplants, to RNA-targeting therapies for brain malignancies, and gene therapies to restore hearing and vision. Leading clinicians and researchers from Harvard-affiliated hospitals shared the latest potential breakthroughs that could transform the field of gene and cell therapy in this First Look at what’s ahead.

These showcased technologies are designed to inform investors, entrepreneurs, investigators, donors and others who share a passion for accelerating the application of high impact technologies to the benefit of patients.

2022 Winners

The World Forum’s First Look sessions promote early stage innovations from across Mass General Brigham. Two of these presentations were selected with additional recognition for addressing unmet need, having clinical impact, scientific strength and collaboration opportunities. In 2022 the two First Look prizes were awarded to Anil Chandraker, MD, Medical Director of Kidney and Pancreas Transplantation, BWH; Associate Professor of Medicine, HMS and Anna Krichevsky, PhD, Associate Professor of Neurology, BWH, HMS.

A Novel Cellular Therapeutic Approach to Replacing Toxic Immunosuppressive Drugs in Kidney Transplantation
Anil Chandraker, MD

Medical Director of Kidney and Pancreas Transplantation, BWH; Associate Professor of Medicine, HMS

Development of RNA-Targeting Therapies
for Malignant Brain Tumors
Anna Krichevsky, PhD

Associate Professor of Neurology, BWH

2022 Presenters

Microbiota-Informed Strategies to Combat Disease: Leveraging Human-Microbiota Molecular Mechanisms of Action in
Therapeutic Applications
Lynn Bry, MD, PhD

Director, Massachusetts Host-Microbiome Center, BWH; Associate Professor of Pathology, HMS

A Novel Cellular Therapeutic Approach to Replacing Toxic Immunosuppressive Drugs in Kidney Transplantation
Anil Chandraker, MD

Medical Director of Kidney and Pancreas Transplantation, BWH; Associate Professor of Medicine, HMS

3D Human Blood-Brain-Barrier Organoids for Modeling Therapeutic Delivery to the CNS
Choi-Fong Cho, PhD

Assistant Professor of Neurosurgery, BWH

Restoration of Hearing and
Vision with Gene Therapy
David Corey, PhD

Bertarelli Professor of Translational Medical Science, Neurobiology, HMS

Genetic Studies of the NCLs: A Window into New Therapeutic Strategies for the NCLs and Related Neurodegenerative Disorders
Susan Cotman, PhD

Assistant Professor, Center for Genomic Medicine, MGH; Assistant Professor of Neurology, HMS

Overcoming Gene Therapy Development Barriers for Ultra-Rare Diseases
Yulia Grishchuk, PhD

Assistant Investigator, Center for Genomic Medicine, MGH; Assistant Professor of Neurology, HMS

Precision Medicine for Dementia: Patient-Derived Stem Cell Models & Targeted Protein Degradation
Stephen Haggarty, PhD

Director, MassGeneral Chemical Neurobiology Laboratory, Center for Genomic Medicine, MGH; Associate Professor of Neurology, HMS

cGMP Methodology and Clinical Trial Preparations for Cell Therapy using
Autologous iPSCs for Parkinson’s Disease
Ole Isacson, MD, PhD

Director, Neuroregeneration Research Institute, McLean; Professor of Neurology & Neuroscience, HMS

Programming Cell Therapies with Targeted Protein Degradation
Max Jan, MD, PhD

Assistant Professor, Center for Cancer Research, MGH

Development of RNA-Targeting Therapies
for Malignant Brain Tumors
Anna Krichevsky, PhD

Associate Professor of Neurology, BWH

Antisense Oligonucleotides for Cancer Immunotherapy
Marco Mineo, PhD

Instructor in Neurosurgery, BWH

yBOTs: Self-Tunable Engineered Yeast for Controlled Therapeutic Delivery
Francisco Quintana, PhD

Professor, Neurology, Ann Romney Center for Neurologic Diseases, BWH; Kuchroo Weiner Distinguished Professor of Neuroimmunology, HMS

Gene Therapy for Tuberous Sclerosis Complex
Vijaya Ramesh, PhD

Co-Director, Neuroscience, Associate Geneticist in Neurology, MGH; Professor of Neurology, HMS

Olfactory Ensheathing Glia: An Unconventional Cell for Cancer Gene/Cell Therapy
Bakhos Tannous, PhD

Director, Experimental Therapeutics Unit, Director, Viral Vector Core, MGH; Associate Professor of Neurology, HMS

A New Hydrogel for Ocular Delivery
Michael Young, PhD

Director, Minda de Gunzburg Center for Retinal Regeneration, Associate Scientist, Schepens Eye Research Institute, Mass Eye and Ear; Associate Professor of Ophthalmology, Co-Director, Ocular Regenerative Medicine Institute, HMS

An AAV-based Single Dose, Thermostable Vaccine Platform that Provides Durable Immunogenicity
Nerea Zabaleta, PhD

Principal Investigator, Grousbeck
Gene Therapy Center, Mass Eye and Ear;
Instructor in Ophthalmology, HMS