UNIVERSITY OF FLORIDA

NC State University’s CERSA held two workshops, attracting 13 participants from five countries to learn about bioregulation and its impacts on agriculture. The program, aimed at senior biotechnology regulators and policymakers, provided comprehensive insights into agricultural bioregulation through tours, lectures, and discussions with experts. Additionally, CERSA hosted a program for seven Cochran Fellows from Thailand focused on climate-smart agricultural technology, equipping them with knowledge to address climate change impacts on agriculture.

The Biotechnology Risk Assessment Grants (BRAG) program, co-administered by USDA’s NIFA and ARS, supports research aiding federal agencies in making decisions about genetically engineered organisms. In 2024, BRAG awarded eight research and three conference grants to institutions including Clemson University, Infinite Eversole, North Carolina State University, Oregon State University, Society for In Vitro Biology Incorporated, University of California, San Diego, University of Florida, University of Maryland, and University of Nebraska-Lincoln.

Solid Biosciences' SGT-001, an AAV vector-based gene therapy for DMD, is evaluated in the phase 1/2 IGNITE-DMD trial. The trial, initiated in 2017, initially included a control arm but was amended to remove it after 4 patients were dosed. The study, conducted at UCLA and UF, reached primary completion in 2024 with long-term follow-up ongoing until 2026. SGT-001 uses an AAV9 vector with a muscle-specific promoter and microdystrophin, delivered via IV infusion. Primary efficacy and safety endpoints are assessed at 12 months post-treatment. The trial is open to boys aged 4-17 with DMD and specific eligibility criteria. Long-term follow-up results from 7 patients showed preserved cardiac function. Solid Biosciences has deprioritized SGT-001 development in favor of SGT-003, currently in the phase 1/2 INSPIRE-DUCHENNE trial.

Elevidys, an AAV-based gene therapy for Duchenne muscular dystrophy, received FDA approval in 2023 for ambulatory patients aged 4-5 with specific DMD gene mutations, and expanded approval in 2024 for broader patient groups. While not curative, it offers a functional gene version, but faces challenges like insurance coverage and long-term data needs. Experts highlight the importance of patient education and choice amidst expanding treatment options.

UF scientists developed a gene therapy restoring vision in LCA1 patients, with a 10,000-fold light sensitivity improvement and successful navigation in a maze. The treatment showed a clean safety profile, paving the way for phase 3 trials and potential commercialization.

Atsena Therapeutics announced 12-month safety and efficacy data from its Phase I/II trial of ATSN-101, a gene therapy for Leber congenital amaurosis caused by biallelic mutations in GUCY2D, published in The Lancet. ATSN-101 demonstrated durable, clinically significant improvements in vision at the high dose and was well-tolerated. This marks the first gene therapy treatment for LCA1, with potential for a Phase III trial.