Krystal Biotech reports news on its commercial genetic-medicines business, led by VYJUVEK, a redosable gene therapy approved in the United States, Europe, and Japan for dystrophic epidermolysis bullosa. Company updates commonly cover VYJUVEK revenue and global commercialization, manufacturing and platform developments, and regulatory actions tied to its HSV-1-based gene delivery technology.
Recurring clinical and scientific updates address investigational programs including KB407 for cystic fibrosis, KB707 for advanced or metastatic non-small cell lung cancer, KB111 for Hailey-Hailey disease, KB803 for corneal abrasions in DEB patients, and additional HSV-1-based vectors. News also includes quarterly operating results, conference presentations, FDA designations, and investor-event participation.
Krystal Biotech (NASDAQ: KRYS) announced the submission of a Biologics License Application (BLA) to the FDA for B-VEC, a topical gene therapy for dystrophic epidermolysis bullosa (DEB). This therapy aims to address the COL7A1 gene deficiency, crucial for skin integrity. The BLA is supported by two clinical trials, GEM-3 and GEM-1/2, demonstrating safety and efficacy with no serious adverse events. The company plans further applications in Europe and Japan, expecting EMA submission in 2H 2022.
Krystal Biotech (NASDAQ: KRYS) announced its participation in the Goldman Sachs 43rd Annual Global Healthcare Conference scheduled for June 15, 2022. The company will be represented by Chairman and CEO Krish Krishnan in a session at 10:40 am PDT. Investors can access a live webcast of the presentation on the company’s website. Krystal Biotech is focused on developing innovative redosable gene therapies for serious conditions, particularly in skin and lung diseases.
Krystal Biotech, Inc. (NASDAQ: KRYS) unveiled new data from the phase 3 GEM-3 clinical trial on the investigational gene therapy B-VEC for dystrophic epidermolysis bullosa (DEB) at the Society for Investigative Dermatology Annual Meeting. The therapy aims to treat DEB by delivering copies of the COL7A1 gene to enhance collagen production. B-VEC has received orphan drug designation and fast track status from the FDA, indicating its potential for significant market impact. The findings contribute to Krystal's ongoing efforts to innovate in gene therapy for rare skin diseases.
Krystal Biotech (NASDAQ: KRYS) reported key operational updates for Q1 2022, highlighting progress on the B-VEC application for Dystrophic Epidermolysis Bullosa, expected to be filed in the US in 2Q 2022 and in the EU in 2H 2022. The FDA has approved at-home dosing in the Open Label Extension Study of B-VEC. Positive Phase 1 results for KB301 were also reported. The company ended the quarter with a strong cash position of $468.0 million, although net losses widened to $50.0 million.
PITTSBURGH, May 3, 2022 - Krystal Biotech, Inc. (NASDAQ: KRYS), a leader in redosable gene therapy, announced that CEO Krish S. Krishnan will speak at the Bank of America Merrill Lynch Global Healthcare Conference on May 10, 2022, at 9:20 AM PDT. The event will be accessible via webcast at this link. A replay will be available on the company's website for 90 days after the event. Krystal Biotech focuses on developing gene therapies for serious conditions, particularly rare diseases.
Krystal Biotech (NASDAQ: KRYS) announced that it will allow patients with dystrophic epidermolysis bullosa (DEB) in the GEM-3 open-label extension study to receive treatment at home by a healthcare professional. This decision, based on FDA feedback, aims to ease patient burden and enhance compliance. The company plans to incorporate home dosing into its Biologics License Application (BLA) submission anticipated in Q2 2022. The GEM-3 study, which began in May 2021, aims to assess the long-term safety of the investigational gene therapy B-VEC.
Krystal Biotech, Inc. (NASDAQ: KRYS) recently announced promising results from the Phase 1 and 2 study of its investigational topical gene therapy, B-VEC, for treating dystrophic epidermolysis bullosa (DEB). The treatment showed robust functional COL7 expression and significantly improved durable wound closure compared to placebo, with minimal adverse events reported. This marks a potential breakthrough in corrective treatment options for DEB patients, previously limited to palliative care. The therapy has received various designations from the FDA and EMA, highlighting its potential impact on severe skin diseases.
Krystal Biotech (NASDAQ: KRYS) presented detailed results from the GEM-3 Phase 3 study of its investigational gene therapy, B-VEC, at the 2022 American Academy of Dermatology Annual Meeting. The study evaluated B-VEC's safety and efficacy for treating dystrophic epidermolysis bullosa (DEB) in 31 patients. Results showed a significant increase in complete wound healing compared to placebo at both 3 and 6 months (p 0.005). B-VEC is designed to deliver the gene non-invasively, addressing DEB's molecular mechanisms. An investor call will discuss these findings on March 28, 2022.
Krystal Biotech (NASDAQ: KRYS) announced that its CEO, Krish S. Krishnan, will participate in two upcoming conferences: H.C. Wainwright's Gene Therapy and Gene Editing Conference on March 30, 2022, at 7:00 a.m. EDT and Guggenheim's 3rd Annual Genomic Medicines and Rare Disease Day on March 31, 2022, from 2:00 to 2:45 p.m. EDT. Webcasts of the presentations will be available, with links to access them provided in the press release. Krystal Biotech focuses on redosable gene therapy for serious diseases, leveraging proprietary technology for potential life-changing treatments.
Krystal Biotech (NASDAQ: KRYS) will showcase late-breaking research on March 26, 2022, at the American Academy of Dermatology Annual Meeting in Boston. The presentation focuses on Phase 3 study results of beremagene geperpavec (B-VEC) for dystrophic epidermolysis bullosa (DEB), a rare and severe skin disease. B-VEC is an investigational topical gene therapy designed to address the genetic causes of DEB by delivering a functional COL7A1 gene. With orphan drug and fast track designations from the FDA, B-VEC aims to provide crucial treatment for DEB patients.