Krystal Biotech is opening its second gene therapy facility, called Astra, to support the production and potential commercialization of B-VEC, the company’s gene therapy for people with dystrophic epidermolysis bullosa (DEB).
The new cGMP (current good manufacturing practices) facility is based in Findlay Township, Pennsylvania. It will have the capacity to manufacture, test, and distribute commercial gene therapies to treat those with rare genetic disorders. The 100,000-square-foot facility will be built out and validated over the next 12–15 months.
“We are very excited to announce the creation of Astra which will be a global resource for production of gene therapies with the potential to bring new treatments to rare disease patients around the world,” Krish S. Krishnan, Krystal’s chairman and CEO, said in a press release. “The success with our first GMP facility, Ancoris, gives us the experience and confidence to have Astra be functionally ready in time for the anticipated launch of our lead therapeutic, B-VEC.”
B-VEC (bercolagene telserpavec), previously known as KB103, is a gene therapy designed to deliver a functional version of the COL7A1 gene directly to skin cells. Using a modified, harmless form of the herpes simplex virus, the therapy seeks to restore the production of type VII collagen, a crucial protein for skin health that is missing in DEB patients.
The therapy previously received PRIME (PRIority Medicines) designation from the European Medicines Agency and regenerative medicine advanced therapy from the U.S. Food and Drug Administration (FDA) for the treatment of DEB.
An ongoing Phase 1/2 trial called GEM-1 (NCT03536143) is assessing if topical treatment with B-VEC can safely and effectively promote skin healing in people with DEB. The latest data from this study showed that nine of the 10 skin wounds treated with B-VEC fully healed within an average of 17.4 days. The therapy was well-tolerated with repeated dosing, and did not cause inflammation or infections.
Based on these positive findings and pending clearance from the FDA, Krystal is planning to launch a pivotal trial of B-VEC to support its regulatory approval.
Astra will also allow Krystal to expand the production of other investigational and commercial products that are part of the company’s pipeline.
Several leaders from the local government and life sciences industry attended Astra’s opening ceremony on Jan. 24.
“We are delighted to see Krystal Biotech expand their headquarters in Pittsburgh,” Christopher Molineaux, president of Life Sciences Pennsylvania, said. “Pennsylvania is home to more than 2,800 life sciences establishments and Krystal Biotech has become a beacon of transformational science in that community that will ultimately serve patients around the world.”
Added Mark Thomas, president of the Pittsburgh Regional Alliance: “We congratulate Krystal Biotech on the success it has achieved in the Pittsburgh region since 2016 and its latest plans for expansion — the largest biotech investment in the region to date.”