The AAVCOVID vaccine program, a new gene – based vaccination strategy using adeno – associated virus (AAV) vector, received an award of up to $ 2.1 million from the Bill & Melinda Gates Foundation.
The donation will help with the effort to bring more preclinical testing to the AAV vaccination platform. The AAVCOVID vaccine candidate is expected to enter clinical trials in 2021.
The AAVCOVID vaccination program was established at Brigham Mass General at the onset of the pandemic.
Initial tests on the stability and potency of vaccines at different temperatures showed that AAVCOVID candidates remained strong and effective when stored at room temperature for up to a month. The temperature stability of this room and the fact that AAVCOVID vaccines do not require cold chain freezer storage, could enable this platform based on developing AAV vaccine for COVID-19 and other diseases for developing countries without its essential cooling infrastructure that some vaccines require.
We are extremely grateful and humbled to be one of the COVID-19 vaccination efforts funded by the Bill & Melinda Gates Foundation. We share a desire to develop vaccines that will end the global threat of this deadly disease. We believe our data on single-dose immunogenicity and room temperature stability promise to overcome key supply challenges to an effective vaccination campaign in this global crisis, especially in countries with limited infrastructure. “
Luk H. Vandenberghe, PhD, Chief Investigator AAVCOVID and D.irector of Grousbeck Gene Medicine Center, Massachusetts Eye and Ear
Vandenberghe anslo an Associate Professor of Ophthalmology at Harvard Medical School.
AAVCOVID is developed through an academic-industry partnership with organizations within Mass Eye and East, and Massachusetts General Hospital, both member hospitals of Mass General Brigham in Boston, the University of Pennsylvania in Philadelphia and Novartis Gene Therapies .
Dr. Vandenberghe works in partnership with the program’s co-directors Mason Freeman, MD, who serves as director of the Interventional Medicine Group of the General Mass Center for Computational and Integrated Biology and the Advanced professor of medicine at Harvard Medical School, and pioneer of gene therapy James M. Wilson, MD, PhD, director of the Gene Gene Therapy program at the University of Pennsylvania. Novartis Gene Therapies is contributing to the consortium its unique technology, expertise and supply chain to manufacture at the scale of AAVCOVID for clinical trial testing through non-generic partnership.
The rapid development of AAVCOVID was first made possible through research support from Mass Eye and Ear with additional support from the Department of Ophthalmology, led by Joan W. Miller, MD, Head of Ophthalmology at Mass Eye and Ear, Massachusetts General Hospital, and Ophthalmologist as Head of Brigham and Women’s Hospital, and Chair of Ophthalmology and David Glendenning Cogan Professor of Ophthalmology at Harvard School of Medicine.
The first work was funded by individual donations including a keynote gift from Celtics co-owner and former Mass Eye and Ear Board Chair Wyc Grousbeck and Emilia Fazzalari, research grants from the Harvard-led Massachusetts Consortium for Readiness Pathogen and FastGrant, and manufacturing partnerships. with Aldevron, Catalent and Viralgen.
An experimental vaccine designed to treat cold chain doses and challenges
AAVCOVID aims to address two logical and biological barriers to global circulation: the need for simplicity of the dosing regimen and limited dependence on cold chain infrastructure, or the need for freezers for storage and transportation.
Many COVID-19 vaccines require two doses, which can raise maintenance concerns as people need two different positions weeks apart.
Doubling the required dose will also increase costs, logistical support and manufacturing needs. Tests to date in mouse and nonhuman prime models have shown that strong immune responses are neutral from just one dose.
Another hurdle for some major vaccines is that they require sub-freezing temperatures for storage that require special freezers that are not widely available. AAVCOVID candidates were stored at different temperatures and the researchers found that they maintained stability and strength at room temperature for one month.
The temperature stability of this room is in line with other AAV-based medications already in use. In the absence of the need for freezing temperatures, Dr. Vandenberghe hopes such a vaccine if proven in human studies will be distributed in parts of the world with a health care infrastructure that lacks adequate cooling capabilities.
The program seeks to accelerate existing manufacturing capacity
The AAVCOVID vaccine program is a gene-based vaccination strategy that attempts to deliver genetic sequences of the SARS-CoV-2 using an AAV vector. Vaccines deliver DNA genetic fragments of SARS-CoV-2 that generate coronavirus spike antigen proteins designed to elicit an immune response to prevent infection.
Dr. Vandenberghe and his laboratory began work on the vaccine in mid-January 2020 after the Wuhan revolution and the first publication of genetic sequences of the new coronavirus. AAVCOVID uses a specific AAV called rh32.33 with desirable inflammatory properties that are required for vaccines and have no previous immunity.
AAVCOVID seeks to stimulate immunity to prevent disease and or disease in healthy populations, taking advantage of the existing manufacturing capabilities of the established AAV gene industry. The program has a manufacturing agreement with several gene therapy industry partners led by Novartis Gene Therapies, to help achieve scalability goals if adopted. The company is one of the first in the world to have successfully manufactured gene therapy with over 1 million square feet of manufacturing capacity.
Source:
Massachusetts Eye and Ear Hospital