AavantiBio Launches With $107M to Advance Gene Therapy for FA

The gene therapy[1] startup AavantiBio[2] has launched with $107 million in funding to advance its lead clinical program for the treatment of Friedreich’s ataxia[3] (FA).

Early this year, the Boston-based company received[4] a $1 million grant from the Muscular Dystrophy Association[5] (MDA)’s Venture Philanthropy Fund[6] to start producing its gene therapy candidate, expected to be tested in a Phase 2 clinical trial in the near future.

AavantiBio’s funding was a result of series A financing[7] featuring the biotech company Sarepta Therapeutics[8] (which provided a $15 million equity investment), hedge fund sponsor Perceptive Advisors[9], and investment managers RA Capital Management[10] and Bain Capital Life Sciences[11].

“AavantiBio has a unique opportunity to change the lives of those living with FA and other rare diseases,” Alexander “Bo” Cumbo, AavantiBio’s CEO and president, and former chief commercial officer and executive vice president at Sarepta, said in a press release[12].

“Central to our mission is building on the advancements in gene transfer therapies and harnessing these revolutionary technologies to realize their potential as life-altering medicines,” Cumbo said.

The investor group noted its investment “represents a truly unique opportunity to thoughtfully advance a differentiated pipeline of innovative gene transfer therapies in areas of high unmet medical need.”

“We are excited to collaborate with Bo Cumbo, an accomplished executive leader, and to provide the necessary resources to enable the company to build a best-in-class gene therapy platform that brings transformative products to patients and families impacted by these devastating diseases,” the group stated.

AavantiBio’s lead clinical program is in Friedreich’s Ataxia[13], a rare disease caused by abnormally low levels of a protein called frataxin due to mutations in the FXN[14] gene.

Since frataxin is essential for the normal functioning of mitochondria (the cell’s powerhouses), its deficiency leads to mitochondrial malfunction, impaired energy production, and progressive neuromuscular and cardiac damage.

Contrary to current symptom-managing treatments for Friedreich’s ataxia, the company’s gene therapy candidate is meant to target the underlying cause of the disease. It does so by delivering a healthy copy of the FXN gene to cells through a modified and harmless adeno-associated virus[15] (AAV).

Experimental gene therapies for Friedreich’s ataxia have shown promising results[16] in animal models of the disease.

AavantiBio has said[17] it intends to test the therapy in both pediatric and adult patients in clinical trials.

The company’s research expands on foundational gene therapy studies conducted by its co-founders, University of Florida[18] (UF) researchers Barry Byrne, MD, PhD, and Manuela Corti, PhD. Byrne is an associate chair of pediatrics and the director of UF’s Powell Gene Therapy Center[19]; Corti is an assistant professor of pediatrics at UF.

Since 2015, Corti has received several awards[20] to advance the development of a gene therapy for Friedreich’s ataxia and assess which is its best route of administration in mouse models of the disease. She also is working[21] on the best combination of immunosuppressive medications to prevent immune reactions against the virus.

AavantiBio will benefit from strategic partnerships with the Powell Gene Therapy Center and the MDA Care Center at UF Health, where the two renowned gene therapy researchers maintain their research and clinical practices.

Doug Ingram, Sarepta’s president and CEO, said its equity participation in AavantiBio serves the strategy to build a “gene therapy engine through targeted investment in potentially life-enhancing therapies as well as partnering with renowned genetic medicine pioneers such as Drs. Byrne and Corti.”

“We look forward to continuing to work with Bo as he builds a strong AavantiBio team and advances therapies to treat FA and other rare diseases,” Ingram added.

“I’m excited to build a company focused on deep science, innovation, and collaboration, and grateful for the partnership with three of the biotechnology industry’s most respected investors,” Cumbo said, adding that he is “thrilled to continue to collaborate with the talented team at Sarepta.”

Cumbo, Byrne, and Corti will be part of AavantiBio’s board of directors, alongside two independent directors and representatives of the investor group. Louise Rodino-Klapac, PhD, senior vice president of Sarepta’s gene therapy, will serve as a board observer.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência.
Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.


Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.


  1. ^ gene therapy (medlineplus.gov)
  2. ^ AavantiBio (www.aavantibio.com)
  3. ^ Friedreich’s ataxia (friedreichsataxianews.com)
  4. ^ received (friedreichsataxianews.com)
  5. ^ Muscular Dystrophy Association (www.mda.org)
  6. ^ Venture Philanthropy Fund (www.mda.org)
  7. ^ series A financing (corporatefinanceinstitute.com)
  8. ^ Sarepta Therapeutics (www.sarepta.com)
  9. ^ Perceptive Advisors (www.perceptivelife.com)
  10. ^ RA Capital Management (www.racap.com)
  11. ^ Bain Capital Life Sciences (www.baincapital.com)
  12. ^ press release (investorrelations.sarepta.com)
  13. ^ Friedreich’s Ataxia (friedreichsataxianews.com)
  14. ^ FXN (medlineplus.gov)
  15. ^ adeno-associated virus (www.ncbi.nlm.nih.gov)
  16. ^ promising results (friedreichsataxianews.com)
  17. ^ said (pharmaphorum.com)
  18. ^ University of Florida (www.ufl.edu)
  19. ^ Powell Gene Therapy Center (powellcenter.med.ufl.edu)
  20. ^ several awards (pediatrics.med.ufl.edu)
  21. ^ also is working (strongly.mda.org)

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