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UCL Honors Dr. Victor Hernandez, Whose Bardet-Biedl Gene Therapy Our Donors Helped Fund

UCL Honors Dr. Victor Hernandez, Whose Bardet-Biedl Gene Therapy Our Donors Helped Fund

University College London has named Dr. Victor Hernandez, co-founder and Chief Scientific Officer of Axovia Therapeutics, one of its 200 Faces, the bicentenary campaign spotlighting people whose work defines what UCL stands for. His profile, Reaching for a cure: harnessing gene therapy to treat disease, traces the path from a UCL lab bench in 2008 to a gene therapy for Bardet-Biedl syndrome (BBS) that is now moving into clinical trials.

On October 22, 2026, Dr. Hernandez shared the honor in a LinkedIn post and thanked the colleagues, mentors and funders behind the work. Among them he singled out A Race Against Blindness for its support of Axovia and its mission over the years.

We are sharing this because it is the clearest picture yet of what your support has built. The $5.1 million in research grants our donors funded for Axovia's AXV-101 program went to the therapy this scientist spent nearly two decades developing. UCL's recognition of him is, in a real sense, recognition of every person who entered a fundraiser or made a donation along the way.

Who is Dr. Victor Hernandez?

Dr. Victor Hernandez is a molecular biologist, co-founder and Chief Scientific Officer of Axovia Therapeutics, and leader of a research group at Brunel University of London. He developed the gene therapies for Bardet-Biedl syndrome that Axovia is now taking into clinical trials.

Born and educated in Barcelona, he earned degrees in biology and a PhD in molecular biology at the Universitat de Barcelona before joining the UCL Institute of Child Health as a postdoctoral researcher in 2008. Over the following decade he helped work out the biology of ciliopathies, the family of rare genetic disorders that includes BBS and can cause blindness, obesity, kidney disease and neurological problems.

That basic science turned into treatment design. Working with colleagues at UCL's Institute of Child Health and Institute of Ophthalmology, he built gene therapies for BBS and showed in preclinical studies that they could restore eye, brain and metabolic function in animal models. The resulting patents and investment launched Axovia Therapeutics in 2019, which he co-founded with his mentor Professor Philip Beales, Axovia's CEO. He continues to collaborate with UCL's Genetics and Genomic Medicine department.

What is UCL's 200 Faces campaign?

200 Faces is part of UCL200, University College London's celebration of its 200th anniversary in 2026. The campaign profiles 200 people, past and present, whose work and lives reflect the university's purpose, from founders and Nobel laureates to current researchers, students and alumni.

Being chosen is a peer recognition. Dr. Hernandez noted in his post that colleagues at UCL proposed him for the initiative, and his profile sits alongside the people the university considers most representative of two centuries of work. For a rare-disease researcher whose field is small and whose patients number in the thousands, not millions, that is an unusual spotlight.

From a UCL lab to a clinical trial: the Bardet-Biedl gene therapy

Bardet-Biedl syndrome is a rare, inherited ciliopathy. Faulty genes disrupt the cilia, the tiny antenna-like structures on cells, and the damage shows up across the body: progressive loss of the retina's light-sensing cells, obesity, extra fingers or toes, kidney problems and learning difficulties. The vision loss usually begins in childhood with night blindness, narrows to tunnel vision, and most people with BBS are legally blind by their teens or twenties. More than 20 genes can cause it; BBS1 is the most common.

There is no approved treatment that slows the retinal degeneration. That is the gap Dr. Hernandez's work targets. Gene therapy replaces a faulty gene with a working copy inside the patient's own cells, and in BBS animal models his team's approach restored function in the eye, brain and metabolism.

Axovia's lead program, AXV-101, is an investigational gene therapy for the vision loss caused by BBS1. It received Clinical Trial Application clearance in the third quarter of 2025 and is designed as a first-in-human, open-label, dose-escalation study. The UCL profile describes the program as now moving into clinical trials, and Dr. Hernandez calls the journey from gene discovery to a human trial his proudest achievement.

What Dr. Hernandez said about A Race Against Blindness

In his post, Dr. Hernandez thanked the research funders behind the science (Great Ormond Street Hospital Charity, Fight for Sight and Apollo Therapeutics) and then thanked Axovia's backers, naming ALSA Ventures and singling out A Race Against Blindness in particular. He wrote that belief in gene therapy for BBS and related ciliopathies helps turn scientific discoveries into potential treatments for the children and families who urgently need them.

Here is what that support actually looks like. Since 2024, A Race Against Blindness has awarded Axovia Therapeutics $5.1 million in research grants to carry AXV-101 into its first human trial: $3 million in 2024, $1 million in September 2025, and $1.1 million in January 2026 that closed the final funding gap so a trial already cleared by regulators could proceed. Every dollar has been paid out, not merely pledged. It is the largest share of the $6.1 million in research grants our community has funded to date.

Our support is research grant funding from a nonprofit, paid for by people who enter our fundraisers and donate. A Race Against Blindness holds no stake in Axovia and receives nothing back if the therapy succeeds. The grants are milestone-driven and aimed at one failure point we have seen too often in rare disease: promising science that stalls at the threshold of human trials for lack of money. Dr. Hernandez's therapy reached that threshold, and our donors made sure it did not stop there.

Why this matters for families

For a parent whose child has just been diagnosed with BBS, the research timeline is the whole story. A gene discovered in a lab does nothing for a seven-year-old losing night vision unless someone carries it through animal studies, manufacturing, regulatory review and a human trial. Dr. Hernandez has now done the first three and reached the fourth, and he credits the families he has worked with as some of the best teachers of how the disease behaves.

UCL's profile closes on the moment he meets a family and knows what their child is likely to face. That is also where A Race Against Blindness started: a family, a diagnosis of inherited retinal disease, and a decision to fund the research rather than wait for it. Seeing the scientist on the other end of those grants honored by his own university is a reminder that the money went to people, and that those people are still pushing.

A necessary caution: AXV-101 is investigational. A first-in-human trial exists to test safety and find the right dose, not to deliver a cure, and results will take time. We will report what the trial finds as Axovia makes it public, whatever it shows.

Frequently asked questions

Who is Victor Hernandez? Dr. Victor Hernandez is a Barcelona-born molecular biologist, co-founder and Chief Scientific Officer of Axovia Therapeutics, and a research group leader at Brunel University of London. He developed Axovia's gene therapies for Bardet-Biedl syndrome during a decade at the UCL Institute of Child Health.

What is UCL 200 Faces? A campaign for University College London's 200th anniversary in 2026 that profiles 200 people whose work represents the university. Dr. Hernandez's profile is titled "Reaching for a cure: harnessing gene therapy to treat disease."

What is Axovia Therapeutics? A UCL spinout founded in 2019 by Professor Philip Beales and Dr. Victor Hernandez to develop gene therapies for Bardet-Biedl syndrome and other ciliopathies. Its lead program is AXV-101.

What is AXV-101? An investigational gene therapy for the vision loss caused by Bardet-Biedl syndrome type 1 (BBS1). It received Clinical Trial Application clearance in the third quarter of 2025 and is designed as a first-in-human, open-label, dose-escalation trial.

How is A Race Against Blindness connected to Axovia? A Race Against Blindness has awarded Axovia $5.1 million in research grants since 2024 ($3 million in 2024, $1 million in 2025, $1.1 million in January 2026) to bring AXV-101 into its first clinical trial. All of it has been paid out. A Race Against Blindness is a grant funder, not an investor, and holds no ownership stake in the company.

Is Bardet-Biedl syndrome treatable? There is no approved treatment for the retinal degeneration in BBS today. Care focuses on managing symptoms, including vision support, weight, kidney function and learning needs. Gene therapy is the first approach aimed at the underlying cause of the vision loss.

Can my child join the AXV-101 trial? Enrollment decisions belong to Axovia and the trial site, not to A Race Against Blindness. Families should speak with their retina specialist or genetic counselor, who can check eligibility as details are published.

How you can help

The grants that carried AXV-101 to the clinic were funded by people entering our fundraisers and by direct donations. Both still work the same way.

Congratulations, Dr. Hernandez. Keep reaching.

Sources

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