New gene based vaccine procedure gets award from the Bill and Melinda Gates Foundation

Overview

• Post By : Kumar Jeetendra


• Source: Massachusetts Eye and Ear Infirmary


• Date: 29 Jan,2021

The AAVCOVID vaccine application, a novel gene-based vaccine plan that utilizes an adeno-associated virus (AAV) vector, was granted an award for up to $2.1 million from the Bill & Melinda Gates Foundation.

The grant will assist the effort to bring further preclinical validation into the AAV vaccine platform. An AAVCOVID vaccine candidate is set to enter clinical trials in 2021.

The AAVCOVID vaccine program was founded at Mass General Brigham at the beginning of the pandemic.

Preliminary tests of the vaccines’ stability and effectiveness at different temperatures revealed that AAVCOVID applicants remained powerful and effective when stored at room temperature for up to one month. This room temperature equilibrium and the fact AAVCOVID vaccines do not require cold-chain freezer , could enable this AAV-based platform to develop a vaccine for COVID-19 and other diseases for developing countries without the essential cooling infrastructure some vaccines require.

AAVCOVID is developed via an academia-industry consortium with groups within Mass Eye and Ear, and Massachusetts General Hospital, both member hospitals of Mass General Brigham at Boston, the University of Pennsylvania in Philadelphia and Novartis Gene Therapies.

Novartis Gene Therapies contributes to the consortium its own distinctive technology, experience and supply chain to manufacture at-scale AAVCOVID for clinical trial testing through an in-kind partnership.

The initial work was financed by Individual contributions such as a lead gift from Celtics co-owner and former Mass Eye and Ear Board Chairman Wyc Grousbeck and Emilia Fazzalari, research grants from the Harvard-led Massachusetts Consortium for Pathogen Readiness and FastGrant, and manufacturing ventures with Aldevron, Catalent and Viralgen.

Experimental vaccine designed to address dosing and cold-chain challenges
AAVCOVID aims to address two logistical and biological barriers to international distribution: the need for simplicity of this dosing regimen and restricted dependence on cold-chain infrastructure, or the demand for freezers for storage and transportation.

Many COVID-19 vaccines require two doses, which might raise adherence concerns since individuals require two different appointments weeks apart.

Doubling the dose needed also increases costs, need for logistical support and production needs. Testing thus far in both mouse and nonhuman primate models have shown a strong neutralizing antibody responses from just a single dose.

Another hurdle for some top vaccines is they need below-freezing temperatures for storage which require special freezers which aren’t widely available. AAVCOVID candidates have been stored at various temperatures and the investigators found they maintained stability and potency at up to room temperature for a month.

This room temperature stability is in line with other AAV-based medications already being used. Without needing freezing temperatures, Dr. Vandenberghe hopes such a vaccine if validated in human studies can be dispersed in regions of the world with poor health care infrastructure which lack adequate refrigeration capabilities.

Program seeks to leverage existing manufacturing capacity
The AAVCOVID vaccine program is a gene-based vaccine strategy that works to deliver genetic sequences of the SARS-CoV-2 with an AAV vector. Vaccination delivers genetic DNA fragments of SARS-CoV-2 which generate a coronavirus spike antigen protein that is designed to elicit an immune response to prevent infection.

Dr. Vandenberghe and his lab began work on the vaccine in mid-January 2020 following the Wuhan outbreak and the first publication of genetic sequences of the new coronavirus. AAVCOVID uses a particular AAV called rh32.33 with desirable inflammatory properties required for vaccines and lacks pre-existing immunity in humans.

AAVCOVID attempts to induce immunity to prevent disease and or disease in healthy populations, leveraging the present manufacturing capabilities of an established AAV gene therapy market. The app has a manufacturing agreement with different gene therapy industry partners led by Novartis Gene Therapies, to help reach the scalability goals in the event of approval. The company is among the first in the world to have successfully scaled up gene therapy manufacturing with over 1 million square feet of manufacturing capacity.