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Engineered Bacteria Studied for Rare Inherited Skin Disorder

f Staphylococcus epidermidis

Scanning electron micrograph image of Staphylococcus epidermidis bacteria (CDC, Pixnio)

1 August 2018. A biotechnology company and research lab are evaluating a modified skin bacteria as a treatment for Netherton syndrome, a rare inherited skin disease affecting mainly infants and young children. The collaboration between Azitra Inc. and Jackson Laboratory, both in Farmington, Connecticut, is funded by a $225,000 Small Business Innovation Research grant from National Institute of Arthritis and Musculoskeletal and Skin Diseases, part of National Institutes of Health.

Netherton syndrome is an inherited condition affecting some 1 in 200,000 individuals, where the skin of newborns lacks the ability to protect their bodies, resulting in dehydration and infections on the skin surface, which can spread throughout the body. The skin of infants with Netherton syndrome appears red and scaly, causing itchiness, which is often exacerbated leading to lesions and more infections.  The disease can affect the hair, and people with the condition also experience immune-system problems such as food allergies, asthma, or eczema, another skin disorder. There are currently no treatments for Netherton syndrome.

Azitra develops therapies for skin diseases based on common benign bacteria known as Staphylococcus epidermidis, or S. epidermidis found naturally on human skin. The company genetically alters the bacteria for the microbe to secrete therapeutic proteins directly to the skin surface. In this case, the engineered bacteria, code-named AZT-02, secretes the LEKT1 protein, usually found in healthy skin, but missing in people with Netherton syndrome. LEKT1 proteins help provide the skin’s protective barrier and aids in the growth of hair and development of lymphocytes, white blood cells in the immune system.

The project, led by Azitra founder and chief scientist Travis Whitfill and Jackson Lab microbiome researcher Julia Oh, is assessing the feasibility of engineered S. epidermidis in AZT-02 to secrete LEKT1 protein. The study is also expected to test AZT-02’s ability to reduce the severity of Netherton syndrome symptoms in preclinical lab cultures or animals. Today’s announcement by Azitra did not give a timetable for completing the study.

Azitra is a 4 year-old company, and a spin-off enterprise from Yale University in New Haven, Connecticut, where Whitfill is a research scientist in pediatrics at the university’s medical school. Azitra is also a portfolio company of Breakout Labs, started by Silicon Valley entrepreneur and investor Peter Thiel, co-founder of PayPal and early venture backer of Facebook. Thiel’s goal is to provide initial funding and support for new enterprises practicing what he calls “radical science” that are often overlooked or rejected by traditional funding agencies and venture financiers.

The award for the project was made under NIH’s Small Business Innovation Research program that sets aside funding for small, early-stage businesses in the health care and life science fields. In the current (2018) fiscal year, NIH expects to channel more than $1 billion into its small business set-aside programs.

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