AavantiBio, Inc.

AavantiBio is a preclinical-stage biopharmaceutical company committed to developing and commercializing novel gene transfer and gene editing therapies for the treatment of rare genetic diseases. The company is rapidly building a diverse portfolio of gene transfer and gene editing therapies to target debilitating genetic diseases for which the unmet medical need is high and for which there are typically no approved therapies treating the underlying disease.

AavantiBio’s vision is to harness the transformative science of gene transfer therapy and gene editing technologies to improve the lives of people living with fatal genetic diseases.




Scientific Research & Development


Alachua County


3700440000% increase from
2017 – 2020


1300% increase from
2017 – 2020

Bo Cumbo

CEO & President

Can you tell us a bit about your background?

I’ve been in the industry for about 27 years, and I’ve been focused on diseases with high, unmet needs. I started to work in HIV in the 90s and then hepatitis C ,which then led me to rare diseases. The last disease I was in was Duchenne muscular dystrophy, which affected boys. It’s a fatal disease with a high unmet need.

Now I’m in a disease state called Friedreich’s Ataxia, or FA for short. Like the others, it has very high unmet needs and is a severe disease. My career has been focused on areas where people need help, trying to do the best I can.

That must be rewarding, yet hard at the same time?

The patients need something, and they need it quick, but the science doesn’t move as quickly as you would like it to. It’s very challenging because time is not on their side, and you need to make sure you’re doing the best possible work, so you don’t have to backtrack and do additional work. It’s challenging but rewarding.

Tell us more about AavantiBio.

At AavantiBio, we’re trying to bring life-saving therapies to patients who need treatments for the specific disease states that we’re focused on. Our lead drug for Friedreich’s Ataxia is called AVB 202, and that’s where the value of our company is. Our customers need that medication. 

What would you say is your competitive edge?

We have a very highly skilled team with a strong science background. We’re tied to the University of Florida, with the Powell Gene Therapy Center, Dr. Barry Byrne, and Dr. Manuela Corti. Our competitive edge is our deep bench in science, from our research and developers to our manufacturers.

Friedreich’s ataxia (FA) is a rare, inherited progressive genetic neuromuscular disease with high unmet medical need.

FA affects the nerves and spinal cord, causing loss of control of body movements (ataxia).

Frataxin, the primary cause of FA, is a triplet repeat mutation in the FXN gene, which codes for the frataxin protein.

Iron is critical for many essential cellular processes, including energy production. However, too little or too much iron can be harmful

The frataxin deficiency associated with FA disrupts iron regulation and mitochondrial function

As a result, cells in the heart and brain accumulate iron, which promotes the production of reactive oxygen species and leads to mitochondrial damage

What is your company culture like?

Our company culture is one that is very open, inviting, and hardworking, but also fun and relaxed. We’ve created an organization where you can speak your mind, provide ideas, and be listened to and appreciated. I come from the south, so I think a lot of our culture can be equated to Southern hospitality. Be polite and be nice to people. We’re a blue-collar team, meaning we roll up our sleeves and no job is too small. I don’t care what your title is; help your teammates and your peers when they need it.

Can you talk about some challenges you faced in growing your business?

I’m sure most businesses can relate; our challenges biggest challenges are capital and talent. I think those two are always at the forefront for startups building out and trying to grow. Of course, over the last year and a half, COVID has been another challenge for us. Starting the company in the middle of a pandemic presents its own difficulties, especially because the supply chain has been completely disrupted. With that, everyone’s schedules got pushed out. It’s been a challenge scheduling preclinical studies or manufacturing batches. Hiring people and raising funding is your top one and two, but overall, COVID has really changed the way we have to plan for scheduling and purchasing materials and supplies that are needed.

Since you started the business during COVID, was there anything you adopted mid-pandemic that stuck? 

I was a dinosaur prior to COVID. Ironically, I thought everything had to be in person. That’s how I grew up in the corporate world. We had a headquarters, and everyone was there working their nine to five. That’s clearly changed, and I realized that we’re not only just as productive but maybe even more productive doing some things virtual.

Everybody that’s in research and development, manufacturing, analytical development, or quality control must be here in person since they need to be in the labs. About 70% of our company falls into those categories. The other 30% are non-essential to be in person all the time. Essential to the company, but non-essential meaning outside the labs. 

What are some of the goals you have for your company?

Our main goal is to drive our lead program, AVB 202, which is for Friedreich’s Ataxia. We want to drive this to the clinic, meaning get this approved by the FDA for in-human studies. That should happen in the next year or two, and then we can start dosing patients. Hopefully, that will lead us to the path of FDA approval. That is our focus right now, but outside of that, it would be to continue to build out our pipeline of programs for other rare, severe diseases. We’d like to move other products into our clinical network to dose patients and hopefully lead other programs to the FDA approval. It all starts with our FA program and then building out our pipeline, moving these other programs forward, and hopefully reaching a broader number of patients that really need medications for severe or fatal disorders.

What’s the process like to get FDA approval?

On average, it takes at minimum 10-12 years or so for a drug to reach FDA approval. In the beginning, it takes at least three or four years just to do preclinical work. That’s done prior to any dosing in humans, and typically, in human trials, you have three phases. IND phase one, phase two, phase three, and then you try to get FDA approval after your phase three, or registrational trial, once you show clinical benefit. It is a long, time-consuming, and expensive process.

What’s your proudest moment so far as CEO?

I’m hopeful that I will continue to have additional moments, but right now, I think it’s really getting the company off the ground. Bringing in the great team, hiring in the people that we’ve hired, and becoming a family. We are all aligned, on the same page, and dedicated to moving the program forward. There’s not really one single moment; there’s a lot of little moments that build up to something big. But I think it all starts with the individuals that are working here.

What does it mean to you to be selected as a GrowFL Florida Companies to Watch Honoree?

It’s wonderful. I mean, Florida is one of the biggest states in the nation. There are thousands of companies in the state all doing amazing things. It’s an honor to be selected this year, not only for AavantiBio, but also for the University of Florida since we started there at the Powell Gene Therapy Center. We are deeply appreciative and thrilled to be named an Honoree considering there is so much competition in the state.

Is there anyone you’d like to thank?

The company was founded around Dr. Barry Byrne and Dr. Manuela Corti. They are the scientific founders and were the ones who developed the lead program. But it takes a village to move these programs forward, especially in the high regulatory environment that we are faced with today. You sure can’t do this alone. So, to the whole team, we couldn’t do it without you. I’d like to thank everyone along the way who has come together to turn a budding scientific program into reality.

My advice to aspiring entrepreneurs is…

"I think it's very simple, but not always easy, don't get down on yourself. Keep moving forward; even 1% of progress every day adds up over time. Success is not linear. You will have ups and downs, but if you keep fighting every day, success will happen. It's all about perseverance."

Celebrate with the AavantiBio, Inc. team!

Tickets are on sale now for the 11th Annual GrowFL Florida Companies to Watch Awards Celebration! Don't wait, they will sell out!