Beacon launched in June 2023 with $120 million in funding, when it acquired UF startup and UF Innovate | Accelerate graduate AGTC, located in Alachua. The sale included AGTC’s lead clinical asset AGTC-501, which was in Phase II clinical trials at the time. UF’s Dr. Arun Srivastava from the Department of Pediatrics developed the AAV vectors used by AGTC.

In this round of funding, Forbion led the financing, which included existing investors Syncona Limited, Oxford Science Enterprises and the University of Oxford, as well as initial investments from TCGX and Advent Life Sciences. The funds will be used to support the continued clinical development of Beacon’s lead asset, AGTC-501 for X-Linked Retinitis Pigmentosa (XLRP) and generate Phase 1/2 clinical trial data for the company’s Dry Age-related Macular Degeneration (dAMD) program.

Ascend Advanced Therapies, a gene therapy development partner, has purchased Beacon Therapeutics’ contract, manufacturing, and controls team—along with the company’s site, previously owned by UF Innovate | Accelerate graduate AGTC, located in Alachua, FL—a town considered to be a “biotech hub” in the state. The acquisition introduces a good manufacturing practice (GMP) plant into the fold, along with process and analytical development capabilities.

Per the deal, Ascend also has a long-term agreement with Beacon which includes the production of its products for both clinical and commercial use, which allows for the ophthalmic gene therapy company to prioritize clinical development.

“Ascend was founded with a unique science and technology-driven strategy. As we continue building the most flexible adeno-associated virus (AAV) platform across serotypes on the market, we also understand the critical value of our highly experienced employees,” said Mike Stella, Ascend’s CEO. “This acquisition allows us to now support customers from research through to commercialization and adds to one of the most experienced gene therapy teams in the market. Every day we are working together to grow the company with a long-term mindset of quality first to appropriately balance quality and yield for the life of each product.”

There were robust improvements in visual sensitivity, the trial’s primary efficacy endpoint, in multiple patients three months after dosing, with a 62.5% response rate in dose group B and a 25% response rate in dose group A. This is well above the statistically significant 50% response rate the Vista Phase 2/3 trial for XLRP is powered to detect. The Company considers a responder to be a patient who has an improvement in visual sensitivity as assessed by microperimetry of at least 7 decibels (dB) in at least 5 loci. Importantly, for responders in this trial, the number of loci that improved by at least 7 dB was between 9 and 17. It is also notable that the mean visual sensitivity of the entire macula increased and the area of the macula with visual sensitivity also increased in the patients who were responders. Although these results exceeded the current standard set by the United States Food and Drug Administration (FDA) that at least 5 loci increase by at least 7 dB, the improved loci were not pre-specified, as also required in the FDA standard.

There was a significant difference in visual sensitivity as assessed by MAIA microperimetry in the treated eyes compared with the untreated eyes of all Skyline responders at 3 months. In the Phase 1/2 trial, patients who were responders at 3 months remained responders at 12 months. Based on the durability of the Phase 1/2 improvements in visual sensitivity, we believe the results observed at 3 months in the Skyline trial will be consistent at 12 months. Additional data related to the Skyline trial is available on the AGTC website or by clicking here.

At Blackstone Elementary in Roxbury, some students took a quick test outside the classroom, including Heaven, a fourth grader who sometimes has trouble seeing the board.

“My eyes — they are used to being shut like this — not open,” she said.

“Eighty percent of learning is visual, and 20% of students have a visual deficiency where they can’t see the board, and a simple pair of glasses can fix that,” said Alexis McLaughlin, CEO of 20/20 Onsite.

20/20 Onsite brings eye exams right to companies and schools with their mobile office. The organization attempts to provide easy access to care and facilitate clinical research. AGTC, a biotechnology company that incubated at UF Innovate | Accelerate at Sid Martin Biotech, decided to honor participants in their clinical trials by donating eye exams through Greater Gift.

Patients with X-linked RP, an inherited retinal disease that begins in childhood, are characterized by night blindness, progressive loss of peripheral vision, and eventual loss of central vision. Affected patients have access to only 1 approved treatment, voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics) for RPE65, which represents about 1% of RP.

According to the company, a total of 14 patients have been enrolled across the high and low dose groups, exceeding the planned target enrollment of 12.

Sue Washer, president and CEO of AGTC, a graduate company of UF Innovate | Accelerate at Sid Martin Biotech, said in a news release that the company is pleased to have reached this milestone in the clinical development of AGTC-501, which positions the company to announce interim three-month trial results in the second quarter of 2022.

Gainesville, FL – On a sunny and fresh fall afternoon, UF Innovate | Tech Licensing hosted its annual event to recognize top innovations stemming out of the University of Florida. More than 150 innovators and entrepreneurs attended the in-person event, and around 40 joined the livestream to celebrate their achievements in fiscal year 2021.

The event started inside UF Innovate | Accelerate @ The Hub, where a new illustration, 42 feet by 10 feet, replaced the mural original to the building. The mural depicts technologies and startups birthed at the University of Florida. (Details on the mural unveiling here.)

After the mural’s unveiling, guests were invited to walk next door to Midpoint Park & Eatery, a food truck park located at the heart of Gainesville’s Innovation District. The modern, outdoor space was the perfect scene to announce the innovator and innovations of the year.

Dr. David Norton, vice president for UF Research, introduced the Innovator of the Year, Dr. William Hauswirth, a UF researcher and pioneer of AAV gene therapy.

“Today I have the pleasure to introduce one of our superstars here at the University of Florida,” said Dr. Norton while he introduced Dr. Hauswirth to the stage. “His work at UF’s Department of Ophthalmology in the College of Medicine resulted in the first FDA-approved gene therapy that cures blindness.”

Dr. Hauswirth has been a UF innovator since 1975. He is an inventor in more than 300 patent application filings around the world. He also co-founded UF startup and Sid Martin Biotech graduate AGTC, a company focusing on using gene therapy for ocular disease, neurodegenerative diseases and otology. The company, located in Alachua, is conducting clinical trials.

“In other words, his body of work is enormous, and the result of that work touches the lives of people around the world,” concluded Dr. Norton.

In addition to the Innovator of the Year, Jim O’Connell, assistant vice president of commercialization at UF and director of UF Innovate | Tech Licensing, introduced seven innovations of the year selected by the licensing teams out of 285 inventions disclosed in fiscal year 2021.

“These seven inventions and their innovators will impact the world in 20 years,” said O’Connell, as he began to introduce the awardees.

• Intratumoral Type I Interferons Sensitize Response to Checkpoint Inhibitors

Dr. Elias Sayour, a pediatric oncologist at UF’s College of Medicine and UF Health, developed an immunotherapy that uses the immune system to fight cancer. The immune system educates against the child’s cancer, and it can remember that cancer for life, providing continual protection. His team includes Drs. Hector Mendez-Gomez, Sadeem Qdaisat, Brandon Wummer, and Duane Mitchell.

• In-Line Ticketing

Computer and Information Sciences and Engineering Department Chair Dr. Juan Gilbert was concerned about people standing in line waiting to vote on the 2020 elections in the middle of the COVID-19 pandemic. This inspired him to create a technology that could mitigate the situation and make it safer with a ticketing system that would tell an individual when to return to vote instead of waiting in line.

• Gatorbulins – Novel Microtubule Destabilizing Agents

Hendrik Luesch, professor and chair for UF’s department of medicinal chemistry in the College of Pharmacy, discovered a marine organism that produces a compound that can fight cancer. This technology could be a crucial anti-cancer drug candidate in the near future.

• Stable Magnetic Cryopreservation Agents and Long Circulating Blood Pool Imaging Agents

Dr. Carlos Rinaldi-Ramos, chair in the chemical engineering department, and Dr. Andreina Chiu-Lam created a technology that could cryopreserve an entire organ. They modified magnetic nanoparticle coatings to improve stability in harsh cryopreservation environments where particle aggregation can prevent penetration deep into the organ for subsequent uniform rewarming.

• Clear Single-Use Passive Perfusion Enabled 3D Bio-Reactor Well Plate for Cell Culture and Experimentation

Mechanical engineer Dr. Greg Sawyer works with micro tumors created from real cancer material. He invented a 3D culture system that is accessible and easy to use, and it allows researchers to do the work in their own laboratory environments. His team included Drs. Ryan Smolchek, Juan Uruena Vargas, Jack Famiglietti, Duy Nguyen.

• Adaptive Olfactory Measure of Threshold (ArOMa-T)

Dr. Steven Munger, director of the Center for Smell and Taste at UF, found a better way to screen for smell loss by creating the ArOMa-T. This simple, inexpensive test allows a person to self-assess and identify dysfunction in the sense of smell.

• Precision Health Interventions Using Virtual Clinicians

UF’s College of Journalism and Communications professor, Dr. Janice Krieger, and a multidisciplinary group of UF researchers created ALEX, a virtual health assistant that delivers precision messages to the diverse population in Florida. The virtual clinicians replicate real-life movements of what will occur in face-to-face interactions.

The event, sponsored by Wolf Greenfield Intellectual Property Attorneys, concluded with a cocktail celebration and refreshments provided by Taste Elegant Catering and Midpoint Park & Eatery food trucks MexiCocina and Plantology.

About Standing InnOvation:

Standing InnOvation is an annual event to honor University of Florida innovators who have disclosed, licensed, or optioned a technology in the fiscal year just ended. The name of the event intentionally hints at giving a “standing ovation” to UF “innovators” for the work they have done. The event is also a reminder of the “standing invitation” inventors have to work with UF Innovate | Tech Licensing, which protects and licenses research discoveries.

About UF Innovate | Tech Licensing:

UF Innovate | Tech Licensing works with innovators, entrepreneurs, investors and industry partners to maximize the potential of University of Florida innovations through commercialization for the global good. In fiscal year 2020, Tech Licensing received 393 invention disclosures, executed 132 commercial transactions and launched 16 companies. In 2017, the Milken Institute ranked the University of Florida third in the nation for its tech commercialization efforts. In 2020, the George W. Bush Institute and the Opus Faveo Innovation Development consulting firm, named UF the most productive large university in the country at leveraging its research funding into new companies, new jobs and new ideas.

Media contact:
Sara Dagen
UF Innovate
Marketing & Communications Manager
saradagen@ufl.edu
(352) 294-0998

By Veronica Talavera, Public Relations Specialist, UF Innovate

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“We are thrilled to welcome Jonathan Lieber to the AGTC team,” said Sue Washer, President and CEO of AGTC. “He’s a seasoned financial and operations executive with valuable experience in the healthcare sector, making him a great addition to the AGTC team. We are excited to see his strategic perspective and apply it to our operational planning.”

Mr. Lieber brings 30 years of experience as a both CFO for emerging growth public and private life sciences companies and an investment banker. In his role as a CFO, Mr. Lieber has led numerous capital raises and had responsibility for financial reporting and regulatory compliance. He has developed relationships with investors and provided strategic counsel to C-suite executives and Boards of Directors to support strategic planning, business development, and investor relations activities.

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“The pandemic has shone a bright light on the need for clinical trial participation as never before in history,” said Lilly Skok Bunch, Executive Director of Greater Gift. “Greater Gift has been celebrating and acknowledging clinical trial participants for more than 10 years; this is the first time that a program will recognize trial participants in a way that also yields benefits for students with vision challenges.”

“AGTC is thrilled to partner with Greater Gift to honor clinical trial participants and their families for their contribution to the clinical research community,” said Halley Losekamp, Director of Patient Enrollment at AGTC. “Through this partnership, our goal is to enhance awareness within the community about the value of clinical trial participation in advancing the science and understanding of gene therapies in IRDs while underscoring AGTC’s commitment to supporting the underserved communities that have limited access to vision care services.”

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