09:28 AM EDT, 05/10/2024 (MT Newswires) -- Rocket Pharmaceuticals ( RCKT ) said Friday that longer-term data on its experimental gene therapies to treat patients with rare blood disorders showed sustained safety and efficacy.

Kresladi, a treatment for severe leukocyte adhesion deficiency, showed 100% stem cell transplantation-free survival with significant reductions in infection-related hospitalizations following engraftment, the company said.

Data from an early-to-mid-stage study of the company's other therapy, RP-L301, to treat patients with Fanconi Anemia demonstrated sustained and progressively increasing genetic correction in eight of 12 patients.

The company said an early-stage study of its gene therapy for patients with severe pyruvate kinase deficiency demonstrated clinically meaningful haemoglobin improvement in all patients.