- Data include evaluation of investigational B-VEC in a murine corneal wound model
- U.S. FDA has recently approved a compassionate use request for topical B-VEC application to one eye in a RDEB patient
PITTSBURGH, Sept. 09, 2021 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced that it will present data on its lead product candidate beremagene geperpavec (B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB) at the upcoming DEBRA International Conference 2021. Data to be presented includes preclinical evidence of safety and collagen VII expression after topical B-VEC application to induced corneal wounds. This preclinical study was conducted in response to a compassionate use request from a physician for the use of topical (eye drop) B-VEC in the eye of a single recessive DEB patient after undergoing surgical removal of the scared layer of the cornea. The U.S. FDA recently approved the physician’s request.
In the eye, persistent blistering due to mutations in the COL7A1 gene can lead to a buildup of fibrotic scar tissue in the cornea, resulting in a loss of visual acuity and potentially leading to blindness. There are currently no approved treatments for DEB-associated corneal lesions.
DEBRA International Presentation Details
Oral Abstract Title: Topical Application of Beremagene Geperpavec, an Engineered Herpes Simplex Virus Type I-Based Gene Therapy Vector Expressing Type VII Collagen, is Safe and Efficacious in a Murine Corneal Wound Model
Session date/time: Thursday September 16, 18:10-18:20 (Moscow time)
Session Title: Short Presentations
Abstract Number: 1
Oral Abstract Title: Technology Platform: Engineered HSV-1 Based Technology Enables Redosable Gene Delivery to Skin Cells Where One and Done Therapy is Not Applicable
Session Date/time: Friday September 17, 16:10-16:20 (Moscow time)
Session Title: Clinical Trials 2
Abstract Number: 5
e-Poster Title: Burden of Illness in Patients with Dystrophic Epidermolysis Bullosa in the US
Poster Number: 18
e-Poster Title: Understanding the Socioeconomic Costs of Dystrophic Epidermolysis Bullosa in Europe
Poster Number: 31
The oral and poster presentations will be available to conference attendees. To register for the conference, please visit https://debracongress2021.ru/register.
About Krystal Biotech
Krystal Biotech, Inc. (NASDAQ:KRYS) is a pivotal-stage gene therapy company leveraging its novel, redosable gene therapy platform and in-house manufacturing capabilities to develop therapies to treat serious rare diseases. For more information, please visit http://www.krystalbio.com.
Any statements in this press release about future expectations, plans and prospects for Krystal Biotech, Inc., including but not limited to statements about the development of Krystal’s product candidates, such as plans for the design, conduct and timelines of ongoing pre-clinical and clinical trials of beremagene geperpavec (“B-VEC”), and Krystal’s plans for filing of regulatory approvals and efforts to bring B-VEC to market; the market opportunity for and the potential market acceptance of B-VEC; plans to pursue research and development of other product candidates; the sufficiency of Krystal’s existing cash resources; the unanticipated impact of COVID-19 on Krystal’s business operations, pre-clinical activities and clinical trials; and other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “likely,” “will,” “would,” “could,” “should,” “continue,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and conduct of clinical trials, availability and timing of data from clinical trials, whether results of early clinical trials or trials will be indicative of the results of ongoing or future trials, uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of product candidates including B-VEC the sufficiency of cash resources and need for additional financing and such other important factors as are set forth under the caption “Risk Factors” in Krystal’s annual and quarterly reports on file with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Krystal’s views as of the date of this release. Krystal anticipates that subsequent events and developments will cause its views to change. However, while Krystal may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Krystal’s views as of any date subsequent to the date of this release.
Source: Krystal Biotech, Inc.