HEИндивидуална стипендия2025–2027

NanoNAD · Nanoparticle-mediated ocular delivery of novel NAD-boosting small molecules for ophthalmic disease.

„Хоризонт Европа“ — Действия „Мария Склодовска-Кюри“

Период: 2025-04-01 → 2027-03-31
Финансиране от ЕС: 252 180 €
Участници: 1
Схема: HORIZON-TMA-MSCA-PF-EF

Линиите свързват координатора с партньорите.

Накратко на български

Наночастици с вещества за повишаване на NAD се тестват за защита на ретиналните ганглионови клетки при глаукома. Това може да подобри терапията при слепота и да помогне при други невродегенеративни заболявания като Алцхаймер и Паркинсон.

Този кратък обзор е генериран от изкуствен интелект

Кратко обяснение, генерирано от езиков модел по текста на CORDIS. Оригиналът е по-долу.

Резултати накратко

Nanoparticle-mediated ocular delivery of novel NAD-boosting small molecules for ophthalmic disease.

Glaucoma is a common neurodegenerative disease affecting ~80 million people worldwide and is the leading cause of irreversible blindness. It is characterized by the progressive degeneration and loss of retinal ganglion cells (RGCs), the output neurons of the retina. Age, genetics, and high intraocular pressure (IOP) are major risk factors. Current treatment strategies only target IOP lowering. However, 42% of treated patients go blind in at least one eye, demonstrating the need for alternative neuroprotective therapies. Several studies including ours show that NAD decline is one of the earliest changes occurring in glaucoma patients and animal models, both systemically and within the retina. Restoring NAD levels through supplementation have been found to be neuroprotective in both animals and humans. In NanoNAD, we aim to develop an ocular delivery of nano-formulations containing novel neuron-specific NAD-boosting small molecules that can protect the RGCs. Such nano-formulations would also allow for a sustained release of these compounds enabling fewer doses as opposed to multiple daily doses that current treatments require. Finally, success of NanoNAD in glaucoma would have therapeutic implications for other more common neurodegenerative diseases like Alzheimer’s and Parkinson’s diseases, due to their shared disease mechanisms.

Текст от CORDIS, на английски · Данни: CORDIS, © Европейски съюз

Цел на проекта

Metabolic decline may be a critical, and treatable, pathogenic component of glaucoma. Glaucoma is one of the most prevalent neurodegenerations worldwide, affecting 80 million patients worldwide. There are no neuroprotective therapies.We have discovered metabolic and mitochondrial dysfunction in the retina and optic nerve which occur prior to neurodegeneration in glaucoma. One of these early changes is a decline in the important metabolite nicotinamide adenine dinucleotide (NAD), which critically regulates neuronal survival in glaucoma. Preventing NAD decline pharmacologically or through targeted gene therapies robustly prevents neurodegeneration. We are now actively generating novel NAD-generating compounds based on our initial studies. Neuronal NAD production is an ideal target for neuroprotective drug discovery for neuroprotection in glaucoma and other axon degenerations.The goal of this research project is to develop clinically translatable nano-enabled neuroprotective strategies for glaucoma. I will focus on these novel strategies involving compounds that increase NAD metabolism developed by the Williams lab and formulated in nanocarriers by the Sotiriou lab. These will be translated into practical, effective agents that provide long-term effects in the retina. The current challenges we face are: (1) ocular delivery without toxicity, (2) high pay-load delivery, (3) delivery with sustained release. To overcome these, the Williams and Sotiriou labs will collaborate, where I will: (1) rationally design biocompatible nanocarriers by flame spray pyrolysis, (2) load these nanocarriers with novel NAD-generating drugs, (3) evaluate their NAD-generating capacity in vivo and the resulting neuroprotective effects. With this project, I will combine the expertise from the two laboratories to facilitate the integration of visual neuroscience, pharmacology, and nanoscale drug delivery systems to develop clinically translatable therapies for ophthalmic disease.

Оригинален текст от CORDIS (на английски).

Участници

KAROLINSKA INSTITUTET · STOCKHOLMКоординаторШвеция