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The deep roots of gene therapy in Florida

Posted: 26 November 2024

Scientists have known about adeno-associated virus (AAV) for more than six decades. Researchers from around the world have been exploring our understanding of the structure, biology, and applications of AAV-based molecules during this time. Incidentally, many of those experts gravitated to the University of Florida (UF) in Gainesville, a legacy hub for AAV innovation. Flashing forward to today, Ascend has found a GMP manufacturing home located just outside of Gainesville, FL in Alachua. Not only does this place the team in one of the original birthplaces of AAV innovation, but Florida continues to gain more and more recognition as a continued focal point of activity in the AAV field and across biotech modalities.

Industry writer, Cynthia Challener, PhD, offers a look back at how it started and where we are today.

AAV was initially discovered approximately 60 years ago. 1, 2 After its structure, growth, and latency were understood, the first infectious clones were produced and sequenced. This information enabled construction of the first AAV vectors, which were of little interest to virologists, but attractive to biologists investigating the delivery of genetic material into human cells.

Leading researchers in the field of AAV from the earliest days included Ken Berns and Barrier Carter, both of whom initially worked at the National Institutes of Health. Berns in 1976 became the chair in Immunology and Medical Microbiology at the University of Florida College of Medicine in Gainesville. While there, he continued his AAV research and recruited several young faculty members. One of them, Nick Muzyczka, became involved in AAV research through the efforts of mutual graduate student Richard Jude Samulski. Berns’ student Bill Hauswirth eventually became a UF professor.

Berns is considered a pioneer in the AAV field. Muzyczka and Jürgen Kleinschmidt did groundbreaking work on basic AAV biology. Samulski cloned the first AAV vector. Hauswirth discovered the mechanism of AAV replication. Arun Srivastava identified ways to improve vector potency while reducing immunogenicity. Mavis Agbandje McKenna discovered the structure of AAV capsids and with Aravind Asokan founded StrideBio, a capsid development company acquired by Ginko Bioworks in 2023. Richard Snyder became CEO of FloridaBiologix, one of the first AAV manufacturers, which was acquired by Brammer Bio and is now part of Thermo Fisher Scientific.

In 1999 while still at UF, Samulski, Hauswirth, and Muzyczka, along with Barry Byrne (also a UF professor) and Terence Flotte (U Mass) founded Applied Genetic Technologies Corporation (AGTC) located in Alachua/Gainsville. Flotte is notable because he undertook the first AAV gene therapy trial in Cystic Fibrosis.

The pipeline of ophthalmic AAV gene therapy candidates being developed by AGCT attracted significant attention, and in late 2022 the company was acquired by British investment trust Syncona Limited for $23.5 million. 3 AGCT’s lead asset AGTC-501 was transferred to Beacon Therapeutics, an ophthalmic gene therapy company launched by Syncona in mid-2023.

When Beacon made the strategic decision to focus on clinical development activities, the company sold its Chemistry, Manufacturing and Controls (CMC) team and site in Alachua to Ascend in April 2024. The deal brought additional expertise and capabilities in AAV process development and manufacturing to Ascend and included a long-term agreement for continued supply of clinical and commercial material for Beacon’s AAV programs.

And, of course, the proximity to UF is a huge benefit for Ascend. The University is the world’s top-ranked institution for published works on AAV (over 600 articles from 1991 to 2022 - 10% of all published AAV research) and the only one with 3 current or former faculty members among the world's top 10 authors of gene therapy and AAV publications. 4 Research is conducted in multiple departments and colleges, and the university continues to actively recruit new faculty.

Being a part of this unique AAV research hub with access to leaders in the field ensures Ascend stays at the forefront of AAV science and technology. To learn more about our Alachua location, please visit this page or email business@ascend-adv.com.

References

  1. Barrie J. Carter, “Adeno-associated virus and the development of adeno-associated virus vectors: a historical perspective,” Mol Ther. 10(6): 981-989 (2004). DOI: 10.1016/j.ymthe.2004.09.011

  2. Eric Hastie and R. Jude Samulski, “Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective,” Hum Gene Ther. 26(5): 257–265 (2015). DOI: 10.1089/hum.2015.025

  3. Robert Barrie, “Beacon Therapeutics enters gene therapy arena with $120m launch,” Pharm Tech. June 143, 2023. https://www.pharmaceutical-technology.com/news/beacon-therapeutics-enters-gene-therapy-arena-with-120m-launch/

  4. "Study: UF is world's top-ranked institution for published gene therapy research." Contify Life Science News, 22 Dec. 2023, p. NA. Gale OneFile: Health and Medicine, link.gale.com/apps/doc/A776983237/HRCA?u=anon~1b197a50&sid=sitemap&xid=af85499f