Krystal Biotech and CRISPR Therapeutics Announce Key Updates in Gene Therapy Programs
核心洞察
Krystal Biotech reported a 473% increase in full-year revenue, driven by VYJUVEK sales, and anticipates European and Japanese regulatory decisions in 2025.
Krystal Biotech's KB407 for cystic fibrosis (搜索) received full sanctioning for its Phase 1 CORAL-1 study, with interim molecular data expected mid-2025.
CRISPR Therapeutics reported positive momentum in the CASGEVY launch, with over 50 activated treatment centers and patients initiating cell collection globally.
Krystal Biotech and CRISPR Therapeutics have recently announced significant progress in their respective gene therapy programs, marking potentially transformative advancements in treating various diseases.
Krystal Biotech Reports Strong Financial Growth and Clinical Advancements
Krystal Biotech (NASDAQ: KRYS) reported its fourth quarter and full-year 2024 financial results, highlighting a substantial increase in revenue driven by its product VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for dystrophic epidermolysis bullosa (搜索) (DEB). The company's full-year revenues increased by 473% to $290.5 million compared to 2023. Krish S. Krishnan, Chairman and CEO of Krystal Biotech, stated that the company's focus in 2025 would be on the global launch of VYJUVEK and advancing its rare disease and oncology programs.
VYJUVEK, a non-invasive, topical, redosable gene therapy, recorded $91.1 million and $290.5 million in net product revenue for the fourth quarter and full year of 2024, respectively, with a gross margin of 95% for the fourth quarter. The company has secured over 510 reimbursement approvals for VYJUVEK in the U.S. and maintains strong access nationwide, including positive access determinations for 97% of lives covered under commercial and Medicaid plans. Krystal Biotech anticipates a Committee for Medicinal Products for Human Use opinion on its European Marketing Authorization Application in Q1 2025 and expects a decision on its Japan New Drug Application in 2H 2025.
Advancing Pipeline Programs
Krystal Biotech is also advancing its pipeline programs, including:
- KB803 for ocular complications of DEB: A Phase 3 study, IOLITE, is expected to commence in 1H 2025 to evaluate KB803's effect on corneal abrasions in DEB patients.
- KB407 for cystic fibrosis (CF): The Cystic Fibrosis Foundation (CFF) Therapeutic Development Network (TDN) Clinical Research Executive Committee granted full sanctioning of the Company’s KB407 Phase 1 CORAL-1 study protocol. Interim molecular data for Cohort 3 patients is expected in mid-2025. Single and repeat doses of KB407 were safe and well-tolerated in Cohorts 1 and 2.
- KB408 for alpha-1 antitrypsin deficiency (AATD) lung disease: Interim clinical update for Cohorts 1 and 2 of its ongoing KB408 Phase 1 SERPENTINE-1 study showed successful SERPINA1 gene delivery and functional alpha-1 antitrypsin expression reaching therapeutic levels. Results from Cohorts 2 and 3 are expected in 2H 2025.
- KB707 for solid tumors of the lung: Early clinical evidence of monotherapy activity was observed in heavily pre-treated patients with advanced non-small cell lung cancer treated with inhaled KB707, achieving an objective response rate of 27% and disease control rate of 73% as of data cut-off. Enrollment in KYANITE-1 is ongoing.
CRISPR Therapeutics Focuses on CASGEVY Launch and Pipeline Expansion
CRISPR Therapeutics (Nasdaq: CRSP) reported its fourth quarter and full-year 2024 financial results and outlined its strategic priorities for 2025, emphasizing the continued launch of CASGEVY (exagamglogene autotemcel [exa-cel]) and updates on key development programs. The company starts the year with approximately $1.9 billion in cash, cash equivalents, and marketable securities.
CASGEVY, a CRISPR/Cas9 gene-edited cell therapy, is approved in multiple regions, including the U.S., Great Britain, the EU, and the UAE, for treating sickle cell disease (搜索) (SCD) and transfusion-dependent beta thalassemia (搜索) (TDT). As of the end of 2024, more than 50 authorized treatment centers (ATCs) have been activated globally, and over 50 patients have initiated cell collection. The number of new patients initiating cell collection is expected to grow significantly throughout 2025.
Advancing Clinical Programs
CRISPR Therapeutics is also advancing its clinical programs, including:
- CTX112 for oncology and autoimmune diseases: Clinical trials are ongoing for CTX112, a next-generation CAR T product candidate targeting CD19. Updates for CTX112 in oncology and autoimmune diseases are expected in mid-2025. CTX112 was awarded Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma.
- CTX131 for solid tumors and hematologic malignancies: Clinical trials are ongoing for CTX131, a next-generation CAR T product candidate targeting CD70, with updates expected in 2025.
- CTX310 and CTX320 for cardiovascular disease: Clinical trials are ongoing for CTX310, targeting ANGPTL3, and CTX320, targeting LPA, with updates expected in the first half of 2025.
- CTX211 for Type 1 diabetes (T1D): Progress in regenerative medicine continues with the ongoing clinical trial for CTX211 in Type 1 diabetes (T1D), along with the development of next-generation programs. The Company expects to provide an update in 2025.
These advancements underscore the ongoing innovation and progress in the field of gene therapy, offering potential new treatments for a range of diseases with high unmet medical needs.