MISSISSAUGA, Ontario — Alexion, AstraZeneca Rare Disease’s Koselugo (selumetinib), an oral, selective MEK inhibitor, has been approved in Canada for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).1 The approval by Health Canada was based on positive results from KOMET, the largest and only...
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REDWOOD CITY, Calif. & RESEARCH TRIANGLE PARK, N.C. – Kriya Therapeutics, Inc., a fully integrated company pioneering novel technologies and therapeutics in gene therapy, announced today that it has acquired Warden Bio, a company developing novel AAV-mediated gene therapies for glycogen storage disorders (GSDs). Under the agreement, Kriya has obtained...
PITTSBURGH, PA — Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced an update on development plans for KB707, the Company’s redosable immunotherapy designed to drive sustained, localized expression of interleukin-2 (IL-2) and interleukin-12 (IL-12) in the tumor microenvironment and promote systemic immune-mediated tumor clearance following inhaled...
PITTSBURGH, PA — Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that on July 24, 2025, Japan’s Ministry of Health, Labour and Welfare (MHLW) granted marketing authorization to VYJUVEK® (beremagene geperpavec-svdt) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB), starting from birth. VYJUVEK is the first genetic medicine approved in Japan for...
VYJUVEK approved for the treatment of DEB from birth in Europe Approval allows for dosing at home or in a healthcare setting, as well as patient or caregiver administration if deemed appropriate by a healthcare professional PITTSBURGH, PA — Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that on...
PITTSBURGH, PA — Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that the U.S. Food and Drug Administration (FDA) approved a label update for VYJUVEK® (beremagene geperpavec-svdt) that expands the VYJUVEK eligible patient population to include dystrophic epidermolysis bullosa (DEB) patients from birth and provides patients full flexibility with respect...
PITTSBURGH, PA — Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that the first patient has been dosed in its Phase 1/2 clinical trial (EMERALD-1), a 2:1 randomized, double-masked, multicenter, placebo-controlled study evaluating KB801 for the treatment of neurotrophic keratitis (NK). “Our ophthalmology pipeline is well under way with...
PITTSBURGH, PA — Krystal Biotech. Inc (NASDAQ: KRYS), a commercial-stage biotechnology company, announced data on the compassionate use of beremagene geperpavec (B-VEC), administered as an eyedrop to treat a patient with dystrophic epidermolysis bullosa (DEB) with cicatrizing conjunctivitis has been published in the New England Journal of Medicine (NEJM). The...
PITTSBURGH, PA — Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that the United States Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to KB707, the Company’s redosable immunotherapy designed to drive sustained, localized expression of interleukin-2 and interleukin-12 in the tumor microenvironment, for the treatment of advanced...
Intra-patient, double-blind, multicenter, placebo-controlled study with crossover design Repeat administration under compassionate use was previously shown to be well tolerated and associated with full corneal healing by 3 months as well as significant visual acuity improvement from hand motion to 20/25 at 8 months PITTSBURGH, PA — Krystal Biotech, Inc. (the...