Skin probiotics to treat netherton syndrome

Ended Project Grant Awarded Last action

Award ID
R44AR082240
Recipient
Awarding agency
Obligated
$2,033,762

Award facts

Completion date
Place of performance
Berkeley, CA 94704, USA

Award details come from USAspending.gov.

Description

Abstract
Netherton syndrome (NS) is a genetic disorder of significant severity impacting the skin, hair, and immune system. The condition arises from mutations within the SPINK5 gene, responsible for encoding lekti (lympho-epithelial kazal-type inhibitor), a serine protease inhibitor. Ordinarily, the skin's exfoliation process is modulated by the balance between these serine proteases and their inhibitors. Mutations in lekti lead to elevated serine protease activity, leading to the persistent skin condition associated with NS. Unfortunately, an FDA-approved therapy remains absent for this condition, with management of symptoms primarily relying on antibacterials, emollients, and corticosteroids. Most therapeutic efforts are to develop small molecule inhibitors, but systemic absorption of these long- lasting molecule often results in toxicity. We propose to genetically engineer a harmless bacterium to temporarily populate the skin and deliver lekti fragments, that normally have a short half-life, continuously. In our phase i sbir, we established the safety and feasibility of our topical application. In this phase II application, we fully develop our final cell therapy and show efficacy in diseased in vitro and murine models. This data will provide the crux of our pre-IND (investigational new drug) meeting with the FDA. Our platform for continuous production of peptide drugs on the skin surface has the potential to revolutionize the treatment of NS.

Transactions

Action Obligated Date
Amendment $703,229
Original award $1,330,533

Quick answers

Who received award R44AR082240?

R44AR082240 was awarded to Resvita Bio Inc. by National Insititue of Arthritis and Musculoskeletal and Skin Diseases (National Institutes of Health) on September 15, 2022.

How much is award R44AR082240 worth?

$2,033,762 has been obligated on R44AR082240.

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