15 Years of HSCI

2004

HSCI launches

HSCI is co-founded by Douglas Melton, Ph.D. and David Scadden, M.D., and launches with 7 Harvard schools, 7 teaching hospitals, 25 principal investigators, and ~100 scientists.

2005

Making stem cells

Cowan, Melton, and Eggan labs fuse adult skin cells with embryonic stem cells to reset adult cells to an embryonic form.

2006

Understanding stem cells

Orkin lab identifies a protein network in embryonic stem cells, improving understanding of how to reprogram cells.

Neuron development

Macklis lab finds that the prion protein plays an important role in neuron development and differentiation.

2007

ALS research accelerates

Eggan lab develops the first mouse stem cell lines carrying human genes for Amyotrophic Lateral Sclerosis (ALS), making it possible to study potential treatments in a lab dish.

Boosting blood stem cells

Zon lab identifies a hormone in zebrafish that expands blood stem cell numbers; this will lead to a clinical trial about four years later.

Ipieirian and Fate Therapeutics launch

2008

A new way to study disease

Daley, Cowan, and Hochedlinger labs create 20 disease-specific stem cell lines.

Stem cell line from a patient

Eggan lab creates the first patient-specific induced pluripotent stem (iPS) cells, marking the first time scientists ever produced a human stem cell line from adult patients with a genetic disease (ALS).

Stem cells to treat muscular dystrophy

Wagers lab treats muscular dystrophy in mice with muscle stem cell transplants.

Epizyme and Sentien Biotechnologies launch

2009

New facility

HSCI establishes the iPS Core at Massachusetts General Hospital, which can provide cells for the entire Harvard stem cell community. The facility later moves
to the Harvard campus.

2010

New way to deliver therapeutics

Rossi lab identifies a safer way to reprogram cells using modified messenger RNA, which also has applications for delivering therapeutics.

Youth factors in blood

Wagers lab finds factors in the blood of young mice that make blood stem cells in old mice act like those in young mice.

Moderna launches

2011

Repairing brain injury

Macklis lab shows that neuronal transplants can repair brain circuitry and normalize function in mice with brain disorders.

Heart repair

Lee lab identifies a specific cell population that can stimulate heart cells to repair damaged tissue.

Melanoma target

Zon lab finds a drug target for melanoma tumors.

GenSight Biologics launches

2012

Toward cystic fibrosis treatment

Rajagopal lab grows lung-surface tissue from stem cells.

ProteoThera and Scholar Rock launch

2013

Clinical trial for transplants

Zon lab publishes initial results of a clinical trial for a treatment that enhances the engraftment of umbilical-cord-blood stem cells for adult transplantation.

The therapeutic is licensed to Fate Therapeutics.

Aging and heart failure

Wagers and Lee labs identify a protein in mouse and human blood that may be the first effective treatment for age-related heart failure.

CRISPR Therapeutics, IVIVA Medical, and Tissium launch

2014

Diabetes game changer

Melton lab uses human stem cells to create functional, insulin-producing beta cells in the lab.

ALS breakthrough

Eggan and Woolf labs discover that stem cell-derived motor neurons from ALS patients point to a common problem among different forms of the disease. An FDA-approved epilepsy drug addresses the problem in a dish, leading directly to a successful clinical trial (2015–2018).

Pain in a dish

Woolf lab creates pain-sensing neurons in the lab, opening doors to studying the biology of pain and developing new treatments.

Audion Therapeutics, Intellia Therapeutics, Q-State Biosciences, Riparian Pharmaceuticals, and Yumanity Therapeutics launch

2015

Parkinson’s disease advance

Isacson lab finds that dopamine-producing neurons, derived from the skin cells of primates, reduce symptoms of the disease.

Slowing vision loss

Cepko lab develops a gene therapy that slows vision loss in mouse models of retinal degeneration.

Lab-grown kidneys

Morizane and Bonventre labs create 3D human mini-kidneys in a lab dish, using them to model human kidney development and genetic kidney disease, and to test for drug toxicity.

AMASA Technologies, Decibel Therapeutics, Frequency Therapeutics, and Semma Therapeutics launch

2016

Optic nerve regeneration

The He lab demonstrates that vision can be restored using an optic nerve regeneration approach that does not interfere with tumor suppressor genes.

Alzheimer plaque producer

Young-Pearse lab identifies neurons that secrete the substance responsible for plaques that build up in the brains of Alzheimer’s disease patients.

Working heart muscle grown in the lab

Ott lab grows first-of-its-kind functional heart muscle by seeding biological scaffolds with stem cells.

Alivio Therapeutics, Fulcrum Therapeutics, Goldfinch Bio, Magenta Therapeutics, QurAlis, and Twenty-eight Seven launch

2017

Anemia treatment

Daley and Zon labs use patient stem cells to identify a potential drug to treat Diamond-Blackfan anemia.

Better stem cell transplants

Scadden and Hoggatt labs uncover a novel drug combination that mobilizes stem cells within 15 minutes in a single injection in mice, offering hope for improved bone marrow transplants.

The treatment is under clinical development by Magenta Therapeutics.

Hearing loss

Karp and Edge labs develop a method to replace hair cells in both mouse and human ear tissue, an important step towards treating hearing loss.

The treatment is under clinical development by Frequency Therapeutics.

Akouos, Clear Creek Bio, and LifeVault Bio launch

2018

Exercise makes the heart younger

Lee and Rosenzweig labs find that exercising mice make over four times as many new heart muscle cells as their sedentary counterparts.

Cystic fibrosis breakthrough

Rajagopal lab identifies the specific cells responsible for making CFTR, a key protein in cystic fibrosis.

Turning cancer against itself

Shah lab engineers self-targeting cells that deliver therapeutic molecules straight to tumors.

Vision repair

Two separate clinical trials start recruiting patients to test stem cell therapies that regenerate damaged corneas. The Jurkunas lab uses the patient’s own cells, while the Frank labs use donor cells to stimulate repair.

The Frank labs initiate their clinical trial with Rheacell.

CAMP4 Therapeutics, Elevian, Enclear Therapies, and Odylia Therapeutics launch

2019

Editing stem cells where they live

Wagers lab creates technology that delivers gene-editing cargo directly into several different types of skin, blood, and muscle stem and progenitor cells in mice.

Gene therapy for heart arrhythmia

Pu and Parker labs bioengineer a heart tissue model of arrhythmia, use it to identify a potential gene target, then design a gene therapy and show that it suppresses the disease in an animal model.

Universal donor cell

Cowan lab develops method to make stem cells genetically engineered to hide from the immune system; the stem cells can be converted into any cell type and transplanted into a patient.

The technology is being further developed by Sana Biotechnology.

Immunitas Therapeutics, Nocion Therapeutics, Sana Biotechnology launch

Boston area becomes home to a new center for cell and gene therapies

A new center for advanced biological innovation and manufacturing in Massachusetts, launched in 2019 by Harvard, MIT, industry partners, hospitals, and state officials, is set to remove major bottlenecks in the development of cell and gene therapies.

An ongoing shortage of advanced biological materials has slowed down the process of turning new discoveries into therapies for patients. Because of high demand, researchers can wait up to 18 months for commercial manufacturers to produce the engineered cells and viral vectors needed for their work.

The 30,000-square-foot facility will ease that bottleneck for the project partners, including HSCI scientists. It will also provide the critical mass needed to develop and refine methods for DNA, RNA, peptide, and cellular therapies. Dedicated manufacturing spaces will provide the process control needed to manufacture materials for use in human trials, while innovation space will be dedicated to late-stage research from academic labs or start-ups.

Harvard and MIT are joined by industry partners Fujifilm, Alexandria Real Estate Equities, and GE Healthcare Life Sciences; Harvard-affiliated teaching hospitals Massachusetts General Hospital, Brigham and Women’s Hospital, Beth Israel Deaconess Medical Center, Boston Children’s Hospital, and the Dana-Farber Cancer Institute; and the Commonwealth of Massachusetts.