The global race to create the first approved vaccine for COVID-19 has a new entry, a collaboration by two Harvard-affiliated teaching hospitals that has received a $1 million donation from Boston Celtics co-owner Wyc Grousbeck.
Researchers at Massachusetts Eye and Ear and Massachusetts General Hospital are working on a vaccine that uses a harmless virus as a Trojan horse to deliver the genetic sequence of the novel coronavirus into human cells to produce an immune response that would protect people from the disease.
They hope to begin testing it in healthy volunteers by the end of the year, as researchers around the world rush to create potential vaccines to end a pandemic that has killed more than 250,000 people.
Since 2017, drug companies have used the so-called adeno-associated virus, or AAV, as a way to deliver DNA in two gene therapies approved to treat two rare inherited diseases. But it has never been used successfully to make a vaccine.
The two hospitals teamed up about eight weeks ago to try, according to Luk Vandenberghe, who developed the potential vaccine as director of the Grousbeck Gene Therapy Center at Mass. Eye and Ear. The center is named for the Celtics co-owner, who chairs the board of the hospital.
“This is a major priority of the institution,” said Vandenberghe, whose hospital, like MGH, is overseen by Mass. General Brigham, the state’s largest health care system. “We’re trying to move as fast as possible.”
At least 111 COVID-19 vaccines are in development worldwide, according to the Milken Institute, an economic think tank based in Santa Monica, Calif. Drug companies, academic laboratories, and governments have scrambled to find a winning formula, recognizing that the vast majority of experimental vaccines and drugs for diseases never get approved by federal regulators.
Most health experts say that even if a coronavirus vaccine works, it would take at least 12 to 18 months before it could be deployed, and that would be remarkably fast. Nonetheless, President Trump said Sunday during a Fox News virtual town hall that his administration was confident the United States will have a vaccine by the end of the year.
Several COVID-19 vaccine candidates have roots in Massachusetts, including a collaboration between Beth Israel Deaconess Medical Center — another Harvard-affiliated teaching hospital ― and the drug-making arm of Johnson & Johnson. That collaboration has received more than $1 billion in federal and private funding.
Moderna, a biotech based in Cambridge, has another closely watched experimental vaccine that uses custom-built messenger RNA ― the genetic material that directs cells to do something ― to trigger an immune response. That vaccine entered clinical trials in Seattle in mid-March.
The leaders of the new Mass. General Brigham venture are employing another approach. They are using a harmless virus as a cloak for the genetic sequence of the coronavirus. The experimental vaccine, which is called AAVCOVID, has produced a robust immune response in mice, they said. They plan to test it in monkeys soon and, if it works, in healthy human volunteers by the end of the year.
“We’re not rooting against anybody,” said Dr. Mason Freeman, director and founder of the Mass. General Translational Research Center, who is overseeing the design of clinical studies to test the safety and effectiveness of the experimental vaccine. “The timeline we’re competing against is the potential of this virus to recycle and cause another catastrophe in the fall. That’s who we’re competing against.”
The effort involves about 55 researchers and has raised several million dollars from philanthropists besides Grousbeck and his wife, Emilia Fazzalari, chief executive of Cinco Spirits Group, according to Vandenberghe.
The leaders of the effort have also approached the foundation of Microsoft founder Bill Gates, the federal Biomedical Advanced Research and Development Authority, and the Coalition for Epidemic Preparedness Innovations, an Oslo-based foundation, about providing far greater sums to manufacture the vaccine, if it proves successful in trials.
“These are billion-dollar plays to go all the way,” Vandenberghe said.
The experimental vaccine employs the same delivery system used in two approved gene therapies: Luxturna, a treatment developed by Philadelphia-based Spark Therapeutics for a rare inherited form of vision loss, and Zolgensma, a drug marketed by a subsidiary of the Swiss drug giant Novartis for spinal muscular atrophy, which kills more infants than any other inherited disorder.
Like those cutting-edge medicines, the experimental vaccine uses a harmless virus called adeno-associated virus to deliver DNA into the body. In the case of the drugs, the DNA corrects genetic diseases at their source. In the case of the vaccine, the DNA from the spike protein portion of the coronavirus stimulates the immune system to make antibodies that would attack the actual virus if someone were exposed to it.
The vaccine would be administered with a single injection in the upper arm, said Vandenberghe. He said the project has commitments from three entities that he declined to identify to help test and make the vaccine.
Vandenberghe is an expert on gene therapies who previously helped found Akouos, a Boston startup that’s trying to develop the first gene therapy to treat hearing loss. Freeman is a venture partner at 5AM Ventures, a venture capital firm in Boston and San Francisco that has raised money for Akouos.
The effort to develop the vaccine has received support from the Mass. General Brigham Innovation Fund, which advances new science and technology based on the intellectual capital of the hospital system.
“The unprecedented coronavirus pandemic has challenged us all, and overcoming it demands the best thinking and the most creative ideas from our scientific and clinical teams supported and strengthened by our philanthropic communities,” said Mass. General president Peter Slavin.
Grousbeck has closely followed research at Mass. Eye and Ear for years, in part because his son, Campbell, is blind. When he learned that Vandenberghe had developed a potential vaccine for the coronavirus, he and his family were eager to donate money.
“My wife and I wanted to do something to fight COVID-19, and we have total faith in Luk [Vandenberghe] and the quality of his team,” Grousbeck said. “We will need several vaccines to succeed to cure the world, and we’re trying to cure the world here.”
Jonathan Saltzman can be reached at firstname.lastname@example.org.