Wired News – Krystal Biotech’s Lead Product Candidate, KB103, for Treatment of DEB Receives Orphan Medicinal Product Designation in Europe

Stock Monitor: Mersana Therapeutics Post Earnings Reporting

LONDON, UK / ACCESSWIRE / April 23, 2018 / Active-Investors.com has just released a free research report on Krystal Biotech, Inc. (NASDAQ: KRYS) (”Krystal”). If you want access to this report all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=KRYS as the Company’s latest news hit the wire. On April 19, 2018, the Company announced that the European Medicines Agency (EMA) granted the Orphan Medicinal Product Designation (OMPD) for its lead product KB103, a gene therapy candidate for the treatment of Dystrophic Epidermolysis Bullosa (DEB). Krystal’s KB103 has the distinction of being the first investigational HSV-1 (herpes simplex virus type-1) based gene therapy in DEB to receive this designation. Register today and get access to over 1,000 Free Research Reports by joining our site below:

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Active-Investors.com is currently working on the research report for Mersana Therapeutics, Inc. (NASDAQ: MRSN), which also belongs to the Healthcare sector as the Company Krystal Biotech. Do not miss out and become a member today for free to access this upcoming report at:

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Active-Investors.com is focused on giving you timely information and the inside line on companies that matter to you. This morning, Krystal Biotech most recent news is on our radar and our team decided to put out a fantastic report on the company that is now available for free below:

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Krystal’s KB103 was granted Orphan Drug Designation by the US Food & Drug Administration (FDA) in November 2017. At present there are no approved medical treatments for addressing the underlying cause of DEB and this is where Krystal’s KB103 has the potential to address this highly unmet medical need.

Benefits of getting OMPD status?

The EMA grants OMPD status to companies developing products for treating life-threatening or chronically debilitating conditions where patients with affected conditions in the European Union are not more than 5 in every 10,000 people. The OMPD is only granted if there are no available treatments which have been approved for the specific ailment. The benefits to Companies getting OMPD status include:

A 10-year market exclusivity for the product from the time of getting EMA approval.

Protocol assistance from the EMA at lower fees in the product development phase.

Access to centralized marketing authorization.

Companies developing drugs with orphan indications get major tax and other financial incentives.

Sharing her views on getting OMPD status for KB103, Suma Krishnan, Founder and COO of Krystal, said:

”Being granted Orphan Medicinal Product Designation in Europe represents another important global milestone for our KB103 program. Last month we filed an Investigational New Drug application for KB103 in the US and intend to advance the clinical development of KB103 in the US in 2018 and in EU in 2019.”

About KB103

Krystal’s KB103 is a lead product candidate which uses gene therapy to treat dystrophic epidermolysis bullosa (DEB). KB103 is currently in preclinical development stage. KB103 is a replication-defective, non-integrating viral vector that has been engineered employing Krystal’s STAR-D platform to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. HSV-1 is Krystal’s replication-deficient, non-integrating viral vector that can penetrate skin cells more efficiently than other viral vectors. Its high payload capacity allows it to accommodate large or multiple genes and its low immunogenicity makes it a suitable choice for direct and repeat delivery to the skin.

About DEB

DEB is a major form of epidermolysis bullosa and a rare and severe genetic disease which is caused by a lack of collagen protein in the skin. There is currently no approved treatment for DEB. Epidermolysis bullosa is a group of genetic conditions that cause the skin to be very fragile and to blister easily. Patients with DEB have very fragile skin and in mild cases just causes minor inconvenience, but in severe cases, it can affect the mouth, gullet, and eyes in addition to the skin.

About Krystal’s STAR-D Gene Therapy Platform

Krystal’s Skin TARgeted Delivery platform (STAR-D platform) is a proprietary gene therapy platform that the Company is using to develop off-the-shelf treatments for dermatological diseases for which there are no known effective treatments. The STAR-D platform includes an engineered viral vector and skin-optimized gene transfer technology. According to the Company, the STAR-D platform provides an optimal approach for treating dermatological conditions mainly due to the nature of the HSV-1 viral vector it has created. At the core of the STAR-D platform is the ability to combine the existing features of the HSV-1 virus, with the ability to strategically modify the virus to use it as a gene delivery tool. This is where the STAR-D platform has a distinct advantage over other viral vector platforms used in dermatological applications.

About Krystal Biotech Inc.

Pittsburgh, Pennsylvania-based Krystal is a gene therapy company dedicated to developing and commercializing novel treatments for patients with rare dermatological diseases. The Company is using its proprietary gene therapy platform – STAR-D platform for developing treatments for rare or orphan dermatological indications.

Stock Performance Snapshot

April 20, 2018 – At Friday’s closing bell, Krystal Biotech’s stock fell 2.98%, ending the trading session at $10.10.

Volume traded for the day: 8.19 thousand shares.

Stock performance in the previous three-month period – up 3.27%; and past six-month period – up 4.02%

After last Friday’s close, Krystal Biotech’s market cap was at $103.32 million.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry.

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