Krystal Biotech Receives $700K Equity-Based Award to Develop Gene Therapy for DEB

Inês Martins, PhD avatar

by Inês Martins, PhD |

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The gene therapy company Krystal Biotech has received a $700,000 equity-based award from the Epidermolysis Bullosa Research Partnership (EBRP) and the Epidermolysis Bullosa Medical Research Foundation (EBMRF) to continue developing a treatment for dystrophic epidermolysis bullosa (DEB).

EBRP and EBMRF are nonprofit organizations dedicated to funding medical research in epidermolysis bullosa (EB). To date, EBRP has raised over $15 million for research grants. EBMRF continues to raise awareness through special events, the media, and fundraising programs.

“We are looking forward to working with EBRP and EBMRF, two organizations spearheading the funding of cutting-edge research to treat and cure EB,” Krish S. Krishnan, chairman and chief executive officer of Krystal Biotech, said in a press release.

“Our lead product candidate KB103, based on our proprietary gene therapy platform, is designed to be an ‘off-the-shelf’ topical product to treat dystrophic epidermolysis bullosa (DEB). We currently expect to file an Investigational New Drug Application (IND) in the U.S. in the first quarter of 2018,” Krishnan added.

KB103 is Krystal’s lead product candidate, currently in preclinical development for the treatment of DEB. The gene therapy is based on a viral vector created using Krystal’s STAR-D platform to deliver functional human COL7A1 genes directly to a patient’s skin cells.

COL7 is a protein produced by skin cells called keratinocytes and fibroblasts. While both types of cells can secrete COL7, scientists believe keratinocytes are responsible for secreting COL7 at the dermal-epidermal junction, a skin layer that helps keep the upper epidermis attached to the lower dermis.

Mutations in the COL7A1 gene, which produces COL7 protein, are associated with one type of EB, recessive dystrophic epidermolysis bullosa (RDEB). RDEB is the most severe form of DEB.

Krystal is developing KB103 for the treatment of a broad DEB population, including both recessive and dominant forms of the disease (RDEB and DDEB).

Gene therapy involves delivering healthy copies of the COL7A1 gene to patients’ cells so that they can produce normal COL7 protein.

The two nonprofit organizations partnered to provide this funding after a competitive application and screening process. EBRP’s scientific advisory board selected the grant’s winner. Under the terms of the award, EBRP and EBMRF will receive 70,000 shares of Krystal’s common stock.

“The EB Medical Research Foundation is thrilled to collaborate with Krystal Biotech on KB103, which we believe is a groundbreaking approach to developing a gene therapy treatment for dystrophic EB,” said Paul Joseph, EBMRF’s chief financial officer. “As committed supporters dedicated to transforming the course of this disease, our goal is to collaborate with fellow foundations and private industry to treat the root cause of DEB by correcting the disease at the source.”