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Massachusetts Eye and Ear

AAVCOVID Vaccine Program Summary

The spread and severity of the disease of COVID-19 has led to remarkable activity and ingenuity in the healthcare and biopharmaceutical world in order to provide medical solutions to this global crisis. Many researchers and companies are stepping up to work on medicines to treat and vaccines to prevent COVID-19, each with a different technical approach. This diversity of technologies is essential, as it increases the likelihood of finding one or more approaches that work.

Luk Vandenberghe, PhD, director of the Grousbeck Gene Therapy Center at Mass. Eye and Ear, saw a way to step up to this challenge as well. Dr. Vandenberghe and his team applied their knowledge and expertise in AAV viruses and gene transfer in a new way: to attempt to create a vaccine to prevent COVID-19.

This work – the AAVCOVID Vaccine Program – is different from the approaches being taken by others, and there is a strong rationale to rapidly advance its development.

The AAVCOVID Vaccine Program started in January – as soon as the genetic code of the SARS-nCov-2019 virus was published. That genetic code was the starting point. Since then, Dr. Vandenberghe and his team have designed and synthesized the vaccine candidates, begun testing them in animals, assembled a team of expert advisors, drafted a vaccine development plan, and crafted a regulatory strategy to discuss with the FDA in the short run.

The path forward is to complete the preclinical studies, to manufacture high quality (GMP) material to test in people, and to initiate the first phase of clinical trials. The team’s ambition is to start clinical trials this summer. To do this work, the AAVCOVID Vaccine Program needs to raise $15 million. In order to move rapidly to key development stages, our immediate philanthropic goal is $7M to be supplemented with grants, governmental and industry support in short succession. 

With these funds, the team can:

  • Develop and characterize vaccine candidates in the Grousbeck Center at Mass. Eye and Ear, directed by Luk H. Vandenberghe (ongoing)
  • Progress two vaccine candidates to manufacturing under Good Manufacturing Practice (GMP) through two external supplies for redundancy (initiation in April)
  • Bring to clinical studies one or two vaccine candidates, likely within one of the Partners hospitals and possibly within its own healthcare provider workforce on an unprecedented and accelerated timeline (pending discussions with the Institutional Review Boards and FDA).
  • Seek a partner with large scale capabilities for a seamless handoff after the initial Phase 1/2 human trials in the eventuality of success for broader deployment.

So far, Mass. Eye and Ear has raised $2.5 million and is actively working to raise an additional $4.5 million to fund the vaccine development effort for the next two months.

How are vaccines designed and how does the AAVCOVID vaccine work?

Generally speaking, a vaccine presents a part of the pathogen (virus or bacteria) to the body to forewarn and train the body’s immune system. That training leads to immunological memory, so that the body is prepared to react more quickly and forcibly to an infection as compared to a non-vaccinated state.

Traditional vaccines are composed of actual pieces (antigens) of the pathogen (in this case the Coronavirus).

The experimental AAVCOVID Vaccine, however, is a genetic vaccine. A genetic vaccine is not composed of actual pieces of a pathogen but rather the genetic code (DNA or RNA) for one of these pieces. This class of vaccines seeks to deliver the genetic code to the body in a way that allows it to produce the antigen from said code. A major advantage of genetic vaccines is that they can be more rapidly developed and thus are more adaptable to an emerging crisis.

The AAVCOVID Vaccine Program is based on a distinct gene delivery platform called adeno-associated viral vectors (AAV). AAV is a potent and well-established gene delivery system with extensive laboratory and clinical use as well as industry adoption. In fact, the past several years have seen 3 AAV-based drug approvals (one of which was first ever administered as an approved drug at Mass. Eye and Ear). The AAVCOVID experimental vaccine builds on the overall safety profile of AAV platform and the potency of the AAV system to express SARS-nCoV-2 Spike antigens for the induction of anti-COVID immunity.

How is the AAVCOVID Vaccine approach distinct?

The AAVCOVID vaccine approach has certain biological and feasibility advantages in comparison to each of the other approaches that compel us to progress this.

  • Relevant clinical experience. AAV technology has been taken to the clinic in gene therapy (not vaccines) for 25 years in thousands of patients with overall remarkable safety record.
  • Potentially favorable dosing. AAV has been safely used in gene therapy at doses that are many-fold higher than the dose expected to be needed for the AAVCOVID vaccine. This may support a good safety margin in clinical testing.
  • Industry infrastructure for AAV. AAV is an established industry with global manufacturing expertise based on the utility of the technology for gene therapy
  • How the immune system responds. The AAVCOVID technology is built on a specific AAV vector that was selected because it drives an immune response that has the potential to lead to SARS-nCoV-2 protection. This is based on both the quantitative and qualitative nature of the immune response this AAV elicits, specifically:
    • Immunity skewed toward antibodies, rather than T-cells.
    • Strong human and animal data indicate the technology AAVCOVID is built on works in intramuscular vaccination consistently in large populations.
    • AAV as a class is highly efficient in delivering its genetic program and driving its execution following an intramuscular injection.

AAVCOVID Vaccine Program Specific Goals, Milestones

  • SARS-nCoV-2019 Genetic vaccine, Spike protein (or subdomain thereof) antigen, AAV delivery
  • Progress 2 vaccine candidates at risk to GMP manufacturing in April (DNA, AAV)
    • GMP manufacturing redundancy with 2 providers, 2 processes
  • Data package to include animal safety and immunogenicity in two species
  • FDA Investigational New Drug Application (timing dependent on GMP manufacturing)
  • Phase 1/2 clinical study initiation (stretch goal: Summer 2020)
    • 1 or 2 vaccine candidates (depending on resources/preclinical data)
    • in high exposure risk populations, healthy young adult subjects
  • Partner with large scale capabilities lined up for seamless hand-off following Phase 1/2

AAVCOVID Vaccine Program Infrastructure, Academia-Industry Consortium

  • Institutional Priority at Mass. Eye and Ear and Partners Healthcare
  • Clinical Infrastructure: MGH Translational Medicine Group (Mason Freeman, MD)
  • Engagement of Partners risk assessment team, MGH Infectious Disease Specialists, Partners Innovation (partnering, contracting and legal)
  • Research infrastructure through Mass Consortium on Pathogen Readiness (Harvard Medical School, Public Health, +), collaborators at UPenn and University of Laval, QC
  • Senior external industry advisors on vaccines, AAV regulatory, biologics, AAV manufacturing, partnering

If you are interested in helping with the AAVCOVID Vaccine Program, please contact Melissa Paul, Chief Development Officer, at 617-573-4168 or .