Turning Back The Clock In Aging-related Diseases

FOR IMMEDIATE RELEASE

Lucina Biotherapeutics Announces Publication in Science Translational Medicine Showing Restoration of Visual Function in Experimental Dry AMD 

Breakthrough study demonstrates replenishment of essential VLC-PUFAs through a single intravitreal injection rejuvenates retinal structure and function in a naturally aged animal model. 

SANTA CRUZ, Calif. — September 24, 2025 — Lucina Biotherapeutics, a biotechnology company pioneering therapies that restore critical lipids lost with age and disease, today announced that its foundational research has been published in Science Translational Medicine. The study, led by Lucina’s scientific co-founder Dr. Dorota Skowronska-Krawczyk, establishes a direct link between age-related lipid decline in the retina and vision loss, while demonstrating that targeted replenishment of these lipids can restore retinal structure and function.

The publication highlights several key findings:

• Age-related lipid decline: Loss of very long-chain polyunsaturated fatty acids (VLC-PUFAs) in the retina is strongly associated with decline in retinal function. These essential membrane lipids are critical to the normal homeostasis of photoreceptors, retinal pigment epithelium (RPE) and their respective mitochondria
• Mechanistic target: Genetic and molecular data confirm that reduced activity of ELOVL2, a critical enzyme in the lipid elongation pathway, accelerates retinal aging and vision loss
• Therapeutic rescue: A single intravitreal dose of C24:5n-3, an essential VLC-PUFA precursor and sole product of ELOVL2 enzymatic activity, restored retinal structure and function in aged mice for at least 4 weeks post-injection. In contrast, other related PUFAs, such as EPA or DHA, that are upstream or downstream of C24:5n-3, provided no benefit
• Molecular rejuvenation: C24:5n-3 lipid treatment reduced complement activation, inflammation, and drusen-associated proteins in the outer retina, while shifting the retinal transcriptome toward a more youthful profile
• Human genetic validation: Analysis of patient data revealed two ELOVL2 variants are linked to faster progression of age-related macular degeneration (AMD), underscoring translational relevance of the study
  
  

“This linchpin publication is a convergence of independent data from multiple laboratories that validates our therapeutic hypothesis that replenishing lost lipids can restore function in aged retinal cells,” said Christopher Chavez, PhD, Co-Founder and Chief Executive Officer of Lucina Biotherapeutics. “With dry AMD representing a $40 billion global market opportunity, Lucina is uniquely positioned to deliver the first disease-modifying therapy in an indication with enormous market potential.”

“As a retina specialist, I see the urgent need for therapies that restore vision in dry AMD. Lucina’s research offers new hope by showing that targeted lipid replenishment can rejuvenate aging retinal tissue”, said Theodore Leng, MD, MS, FACS, Director of Clinical & Translational Research, Stanford Byers Eye Institute. “This breakthrough has the potential to change outcomes for millions of patients.”

Next Steps Toward the Clinic

Lucina is advancing LUC-101, its proprietary intravitreal formulation of C24:5n-3, through IND-enabling development. 

About Dry AMD

Dry AMD affects over 15 million people in the United States and more than 200 million globally. Current FDA-approved treatments, such as Syfovre™ and Izervay™, only slow lesion growth in late-stage disease (geographic atrophy) and do not markedly improve vision. Lucina’s approach targets the earlier stages of AMD by addressing a root biochemical deficit, with the goal of restoring vision and modifying disease progression.

About Lucina Biotherapeutics

Lucina Biotherapeutics is a biotechnology company developing first-in-class therapeutics to combat aging-related diseases by restoring critical lipids that decline over time. The company’s PUFActive™ platform leverages synthetic biology and lipid chemistry to produce and deliver very long-chain polyunsaturated fatty acids (VLC-PUFAs) for ocular and systemic indications. Lucina’s lead program, LUC-101, targets dry AMD with the potential to be the first therapy to restore vision in this condition.

https://www.science.org/doi/10.1126/scitranslmed.ads5769

For more information, visit www.lucina.bio

https://www.fiercebiotech.com/research/single-eye-injections-fatty-acid-restored-vision-elderly-mice

Injecting a fatty acid that is used to make very long fat molecules into the eyes of elderly mice improved their vision for up to a month, indicating that the natural compound could potentially be used to treat age-related vision conditions in humans.

The results, published in Science Translational Medicine on Sept. 24, “were shocking" to Dorota Skowronska-Krawczyk, Ph.D., a vision researcher at the University of California, Irvine and study lead. 

“We were thinking we will have to add something else, but the supplementation of only this one fatty acid was already giving the result," she told Fierce Biotech.

That one fatty acid has a name that hardly rolls off the tongue: 24:5n-3, or tetracosapentaenoic acid (TPA). It’s a key precursor to the extremely long fatty acid molecules that are vital for vision, but Skowronska-Krawczyk’s team found that as mice age, the gene that makes it becomes less and less expressed. This confirms a link between the TPA gene, called ELOVL2, and eye aging in mice that the researchers previously identified in 2020.

In the new study, the researchers also analyzed genetic data from patients with age-related macular degeneration (AMD) and found that certain forms of ELOVL2 were associated with an earlier onset of the disease.

With TPA proving capable of restoring vision in mice, Skowronska-Krawczyk is now keen to test the approach in nonhuman primates and is also trying to determine whether the fatty acid can be given orally rather than through an eye injection. Meanwhile, a company she co-founded called Lucina Biotherapeutics is formulating the molecule for investigational new drug application-enabling studies.

“In a sense, we are closer than many other drugs, because it's a natural product,” Skowronska-Krawczyk said. If oral delivery proves effective, she thinks that TPA could serve as both a low-dose supplement that people take to stave off vision loss and a high-dose, targeted treatment for eye diseases like AMD.

Fatty acids, especially omega-3s like those found in fish oil, have long been associated with eye health. “Maybe eat a lot of fish before we come with the pill,” Skowronska-Krawczyk joked.

Most TPA is made in the liver, but the eye also makes its own supply because TPA is needed to produce docosahexaenoic acid (DHA) and very long-chain polyunsaturated fatty acids (VLC-PUFAs), which the eye depends on. Fatty acids are used in every single cell membrane, but rod cells in the retina in particular need those fatty acids to be very long so that their cell membranes are flexible, Skowronska-Krawczyk explained. Rhodopsin, the light-sensing protein of rod cells, needs a flexible membrane in order to function properly.

“This part of the photoreceptors is used up every day a little bit, because of the light and light damage, but also because of the visual cycle itself,” Skowronska-Krawczyk said. In order to regenerate these lost cells, the eye needs a constant supply of DHA and VLC-PUFAs; if there isn’t enough TPA to make those long fatty acids, as can happen with aging, the eye will have no choice but to settle for shorter fatty acids instead, impairing the ability of the photoreceptors to respond to light.

The idea to inject TPA into the eyes as a way to restore vision came from years of basic research into how the visual system works, Skowronska-Krawczyk said. In the current tough funding environment, she hopes her team’s breakthrough underscores the importance of supporting basic academic research.

“This is not only for us to play in the laboratory, but actually to bring something real to patients,” she said.