Acuitas: (noun) insight, perception, sharpness.

Frequently Asked Questions

Acuitas Therapeutics

How do vaccines work?

Vaccines train our body’s immune system to recognize and destroy a particular virus. This prevents the virus from being able to infect our body and cause disease.

How does our immune system “recognize” a particular virus?

In most cases, our immune system recognizes a specific part that is on the outside of the virus and that is often needed by the virus to infect cells in our body.

What are vaccines made of?

In many cases, conventional vaccines consist of the actual virus – but in a form that cannot cause disease (as it’s been killed or attenuated).

What is different about the vaccines that Acuitas Therapeutics is helping to develop?

The vaccines that Acuitas is helping to develop use a new technology called messenger RNA (or mRNA) technology. This allows vaccines to be developed much more quickly than ever before. Rather than injecting the entire virus as a vaccine, mRNA tells our body to make the component of the virus that our immune system recognizes.

Why is the technology called messenger RNA?

All living organisms, including humans and viruses, make proteins. Proteins are the workhorses that carry out all of the tasks needed by that organism. The “blueprints” for these proteins are in our genes (DNA), but this information needs to be converted into a message that tells the body to make a particular protein. This message is called messenger RNA (mRNA). An mRNA for a virus will tell the body to make a viral protein.

Scientists can make a synthetic mRNA that provides the body with the information to make any protein that is needed.

What technology does Acuitas provide that allows mRNA vaccines to work?

If we want to use a synthetic mRNA as a vaccine (or a drug), there are two challenges. First, the mRNA is very fragile and if injected into the body, it would break down very quickly. Second, it is a very large biological molecule and it cannot get into our cells – and it needs to get into the cells in our body to work. Acuitas provides tiny delivery vehicles (lipid nanoparticles) that protect the mRNA after it is injected and then the lipid nanoparticles carry the mRNA into our cells, allowing it to do its important job.

Can you provide an analogy as to how the Acuitas delivery technology works?

Imagine that you want to buy an exceptionally fragile glass ornament online and you would like to have it delivered to your home. If you used the equivalent of the Acuitas delivery technology, we would pack the ornament inside our carrier to shield it. No matter how bumpy or rough the journey was, our delivery technology would make sure that the ornament was protected. Our carrier would find your house, open the front door by itself, let itself in, unwrap your glass ornament, and leave it in the front hallway for you to pick up.

How does the Acuitas team work with partners in developing a vaccine? What is this process?

The first step is to identify and select the viral part that our immune system will be best able to recognize. Our partner synthesizes the mRNA. This mRNA is then provided to Acuitas and we put it inside our lipid nanoparticles (LNP). We then send the mRNA, packaged inside our LNP, back to our partner to test. Once these studies have been completed – and submitted to the regulatory authorities for review – clinical trials can begin.

Do all vaccines need a technology similar to what Acuitas has developed?

No, older vaccines are often just the virus itself in a form that does not cause disease. It is only new vaccine technology, such as mRNA, that needs LNP delivery technology.

How is Acuitas technology developed in a unique manner for each virus?

Acuitas has devoted many years of research to create an LNP carrier that works really well for mRNA vaccines. An advantage we now have is that this same LNP carrier can be used for many different mRNA vaccines.

The components that make the LNP carrier are unique to Acuitas and because they are used in a pharmaceutical product, they need to be manufactured under very stringent conditions to ensure their purity and safety. Different mRNA vaccines can use these same LNP components which are now available from Acuitas. This greatly simplifies and streamlines the development of new mRNA vaccines.

It is crucial that, for each new mRNA vaccine, Acuitas ensures that the new mRNA can be packaged efficiently within the LNP. We want to make sure that we can produce the vaccine product with the highest efficiency.

What are the advantages of using an mRNA vaccine rather than a conventional vaccine to address a new virus such as COVID-19?

A major advantage of mRNA vaccines is that they can be developed much more rapidly in response to a new virus compared to conventional vaccines. This can be done as a result of advances in science that allow rapid sequencing of the genetic code of a new virus. For example, once COVID-19 was isolated, its genetic code was determined in just a few days. From this genetic code, we can then identify which virus part our immune system will be best able to recognize and can generate an mRNA to code this part within weeks. Then, taking a safe, well-characterized Acuitas LNP, with its proprietary components, a candidate vaccine can be rapidly taken into clinical testing. In addition, this approach permits a scaling up of the manufacturing of mRNA vaccines, allowing for the production of hundreds of millions of doses in a relatively straightforward manner.

Were all of the required safety and efficacy studies completed before the BioNTech/Pfizer vaccine was approved?

BioNTech and Pfizer met or exceeded the safety standards, as set out by regulatory agencies around the world. Further, BioNTech and Pfizer have united with seven other companies that are developing COVID-19 vaccines to confirm their commitment to the safety and well-being of vaccinated individuals, to adhering to high scientific and ethical standards in the conduct of clinical trials and vaccine manufacturing, to only seeking regulatory approval or Emergency Use Authorization after demonstrating the safety and efficacy of the vaccine in a Phase 3 study designed and conducted in accordance with the requirements of expert regulatory authorities such as the FDA, and to providing sufficient supplies and vaccine options to address global access.

 

The BioNTech/Pfizer COVID-19 vaccine is based on mRNA technology, which codes for a protein made by the novel coronavirus. The vaccine works by allowing the human immune system to recognize this protein so that it can prevent the virus from infecting our cells and causing disease.

 

A major advantage of mRNA vaccines over conventional vaccines is that they can be developed more rapidly in response to a new virus.

 

You can see the BioNTech/Pfizer Phase 3 publication of results that was published in the New England Journal of Medicine.

Is the vaccine safe?

Vaccines that receive government approval for use have undergone several phases of clinical trials and that data is submitted to the government when requesting approval.

 

In speaking specifically about the BioNTech/Pfizer COVID-19 vaccine, which Acuitas was involved in the development of, it was shown to be safe in clinical trials conducted prior to seeking regulatory approval. As with all drugs and vaccines, safety will continue to be monitored – even after approval. Vaccines were first developed in 1798 and since then have provided the greatest benefit to human health and well-being of any other medical advancement. Diseases which were common prior to the development of vaccines – such as smallpox, polio, diphtheria, tetanus, typhoid fever and many others – killed hundreds of millions of people globally. Most of these diseases are now almost eliminated in the developed world. This has resulted in substantial reductions in child mortality and longer average lifespans. Most of us have not witnessed the horrors that these diseases caused and so we can find it harder to appreciate how important vaccines really are. Our experience with COVID-19 provides an example of how devastating such diseases can be and we should also recognize that other viruses can be even more dangerous.

During vaccine trials – do children participate as a part of the study?

While children may not be included in early clinical studies, if the vaccine is intended to provide a clinical benefit to children, they will typically be included once an appropriate safety database is available.

 

BioNTech/Pfizer received regulatory approval to test their COVID-19 vaccine in children 12 years of age and older, and safety data from approximately 100 children 12-15 years of age are included in the Emergency Use Authorization request submitted to the FDA on November 20, 2020.

Should I be vaccinated?

Vaccines can only be fully effective when the vast majority of the population is vaccinated. Vaccination is a social compact, where we jointly agree to protect each other and the most vulnerable among us by preventing transmission of the virus. We strongly encourage everyone to contribute to defeating COVID-19 by getting vaccinated.

What would you say to anti-vaxxers?

We are scientists. We work with facts and with validated scientific principles and data. People who make a personal choice not to get vaccinated put themselves and others at serious risk. Essentially, they are opting out of the social compact that we should all work together to protect the community and the most vulnerable within that community.

 

We recommend that anyone who has a concern about vaccination should speak with a doctor and get the facts. There is data to support the safety and efficacy of the COVID-19 vaccines. If there is a reason why certain individuals should not be vaccinated, your doctor will be able to advise you of this.

Should I wait until a later time to get vaccinated to see if it is really safe?

There is data to support the safety and efficacy of the BioNTech/Pfizer vaccine and any other vaccine on the market. An unnecessary delay in getting vaccinated would simply extend the duration of the pandemic. This, in turn, would place continuing pressure on our health care system and health care providers and would delay the recovery of our economy.

Will a vaccine protect me from COVID-19?

The short answer to this is yes. For example, the clinical data shows that the BioNTech/Pfizer vaccine is 95% effective in preventing COVID-19 infection. In addition, even those relatively few individuals who do contract COVID-19 after vaccination may be protected from experiencing the more serious symptoms and outcomes of the disease. During a press conference given by BioNTech on December 2, 2020, Dr. Ozlem Tureci (Chief Medical Officer) indicated that in their Phase 3 study, only one individual who received the vaccine developed serious disease. At this time, we do not know how long the vaccine will protect us against COVID-19; however, ongoing studies should provide an answer to this question. As with other vaccines, we may need our immune systems to be boosted at some point in the future.

If there is a vaccine, do we still need to keep washing our hands and using hand sanitizer?

It is important to continue with the personal hygiene practices that we have all incorporated into our lives since the pandemic started. Wash your hands often, use hand sanitizer if you cannot wash, and if you are sick – stay home. These are all sensible precautions that we can take to reduce the risk of any viral infection.

What about social distancing?

Once a vaccine has been administered to a majority of the population in your community or social circle, it is expected that we will be able to go back to hugging, indoor dining and other close-contact activities in public that we have had to avoid since the pandemic started.

Do I really need to have both injections of the vaccine?

The short answer to this is yes. The clinical studies conducted by BioNTech and Pfizer showed that the full protective effect of the vaccine requires two injections, approximately four weeks apart. A single injection may only provide partial protection.

What are the potential side effects?

Pfizer and BioNTech have indicated that their COVID-19 vaccine has not shown any serious safety concerns to date.

 

You can see the BioNTech/Pfizer Phase 3 publication of results that was published in the New England Journal of Medicine.