Aegle Wins Debra’s Partners in Progress Award, Plans to Soon Open Clinical Trial in DEB Patients

Aegle Wins Debra’s Partners in Progress Award, Plans to Soon Open Clinical Trial in DEB Patients

With its lead therapy candidate AGLE-103 set to begin clinical testing in the coming months, Aegle Therapeutics is being honored with a Partners in Progress Award by the Dystrophic Epidermolysis Bullosa Research Association of America (Debra of America).

Aegle is among three companies being recognized for its efforts to treat dystrophic epidermolysis bullosa (DEB) at the 21st Annual Debra of America Benefit set for Oct. 21 in New York City. The others are Amryt Pharma and Lenus Therapeutics.

The company announced plans to initiate a Phase 1/2 clinical trial evaluating the safety of AGLE-103 at escalating doses in DEB patients at sites in the U.S. by the close of this year, following approval of its request for this study by the U.S. Food and Drug Administration (FDA).

DEB is one of the major forms of epidermolysis bullosa (EB), a genetic disorder in which the patients’ skin blisters and tears easily, causing painful wounds and scarring. Its three main subtypes  are caused by mutations in the COL7A1 gene, which provides instructions to make a protein called type 7 collagen (COL7) that is essential to maintain healthy skin.

AGLE-103 is part of the company’s proprietary extracellular vesicle (EV) therapy, which is based on the small vesicles produced by mesenchymal stem cells (MSCs) that are thought to possess wound-healing properties. (MSCs are cells that can be isolated from several tissues, and can grow into different cell types when cultured in a lab dish.)

Importantly, these small vesicles can be used as “vehicles” to transport and deliver functional versions of COL7 — either directly as a protein, or as messenger RNA (mRNA, the molecule that serves as a template for the production of a protein) — to skin cells of DEB patients that are unable to produce it.

“We are excited to be developing a multifaceted approach to DEB. In addition to carrying the regenerative wound healing messages of mesenchymal stem cells, Aegle’s EVs also deliver COL7 and COL7A1 mRNA to diseased cells, inducing recessive DEB cells to produce their own COL7 protein,” Evangelos Badiavas, MD, PhD, founder and chief scientific officer of Aegle, said in a press release.

“We believe this product has the potential to address patients’ COL7 deficiency as well as accelerate wound healing,” Badiavas added.

The company isolates extracellular vesicles released by allogeneic bone marrow-derived mesenchymal stem cells, but unlike those cells, EVs do not “migrate, differentiate, proliferate or engraft, addressing some of the more complex issues surrounding stem cell therapy,” Aegle reports on its website.

“We are excited to be the first company to advance extracellular vesicle therapy into the clinic. Our primary focus is moving AGL-103 into patients,” said Shelley Hartman, chief executive officer of Aegle. “We are only at the brink of understanding the potential of extracellular vesicles and we are excited to be pioneers in EV therapy.”

Joana holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. She is currently finishing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Joana holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. She is currently finishing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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