Krystal Biotech Stock Price, News & Analysis

Krystal Biotech announced the completion of the 26-week dosing period and safety follow-up for its pivotal Phase 3 GEM-3 clinical trial evaluating beremagene geperpavec (B-VEC) as a treatment for dystrophic epidermolysis bullosa (DEB). A total of 31 patients participated, with 29 completing the study without missing visits. Topline results are expected in Q4 2021, with potential FDA and EMA regulatory filings planned if the data is positive. The trial aims to provide a topical, genetically corrective therapy for DEB, a severe genetic skin condition.

Krystal Biotech (NASDAQ: KRYS) announced the presentation of pre-clinical data on KB407, a redosable gene therapy for cystic fibrosis, at the North American Cystic Fibrosis Conference. The data highlighted results from an IND-enabling GLP toxicology study. The company received approval for a Phase 1 clinical trial of inhaled KB407 in Australia and plans to initiate a clinical trial in the U.S. in 2022. KB407 targets the underlying genetic cause of cystic fibrosis, addressing a significant unmet medical need for patients not responsive to current therapies.

Krystal Biotech and GeneDx have launched the Krystal Decode DEB™ program, providing no-charge genetic testing for Epidermolysis Bullosa (EB), specifically the dystrophic form known as DEB. This collaboration aims to accelerate diagnosis, allowing quicker access to optimal care. The program is designed for US residents presenting EB symptoms who haven't undergone genetic testing. It utilizes a comprehensive gene panel to aid in accurate diagnosis, addressing the significant diagnostic delays often faced by patients. This initiative has the potential to improve patient outcomes significantly.

Krystal Biotech (NASDAQ: KRYS) announced its participation in the Chardan Virtual 5th Annual Genetic Medicines Conference on October 4-5, 2021. CEO Krish Krishnan will engage in a fireside chat, while SVP Whitney Ijem will join a panel on non-traditional viral vectors on October 5. The fireside chat is scheduled for 11:00 AM ET, following the panel at 9:30 AM ET. Interested parties can access live webcasts through the provided links, with replays available for 30 days post-event. Krystal Biotech focuses on developing therapies for rare diseases using its innovative gene therapy platform.

Krystal Biotech has received approval from the Bellberry Human Research Ethics Committee in Australia for a Phase 1 clinical trial of its investigational inhaled gene therapy, KB407, aimed at treating cystic fibrosis (CF). This therapy delivers the CFTR gene to lung cells via nebulization. Enrollment for the trial is expected to start soon, following additional institutional approvals. The study will assess safety and tolerability, focusing on lung function improvements over three dosing cohorts. The company's efforts follow COVID-related delays, with plans to initiate a US trial in 2022.

Krystal Biotech (NASDAQ: KRYS) has appointed Laurent Goux as General Manager of Europe, bringing over 20 years of experience in biotechnology. Previously a key player at Galderma, Goux has successfully launched multiple dermatologic medicines and will enhance Krystal’s commercial capabilities in Europe. This leadership change comes as the company advances its lead investigational program, B-VEC for DEB, and aims to build a fully integrated biotechnology company. Goux expressed enthusiasm for contributing to Krystal's mission of developing treatments for rare diseases.

Krystal Biotech, a leader in redosable gene therapies, announced it will present data on its product candidate, beremagene geperpavec (B-VEC), for treating dystrophic epidermolysis bullosa (DEB) at the DEBRA International Conference 2021. The data includes findings from a murine model demonstrating safety and collagen VII expression after B-VEC application. The U.S. FDA approved a compassionate use request for topical B-VEC in a DEB patient's eye following surgery. DEB currently lacks approved treatments for corneal lesions.

Krystal Biotech (NASDAQ: KRYS) will present at the H.C. Wainwright 23rd Annual Global Investment Virtual Conference, occurring from September 13-15, 2021. The company specializes in redosable gene therapies for rare diseases.

The pre-recorded presentation will be available on-demand starting September 13 at 7:00 A.M. (ET). For attendees, the presentation will be accessible for 90 days via the Krystal Biotech website. Learn more about their innovative therapies and ongoing developments at their official site.