Business
CRISPR Therapeutics Provides Business Update and Reports Second Quarter 2020 Financial Results
-Dosing re-initiated in clinical trials of CTX001™ for patients with severe hemoglobinopathies- - CTX001 has received orphan drug designation from the U.S.
About this update from Crispr Therapeutics Ag
[{"type":"text","content":"-Dosing re-initiated in clinical trials of CTX001™ for patients with severe hemoglobinopathies-\n - CTX001 has received orphan drug designation from the U.S. FDA for sickle cell disease (SCD)- -Expands regenerative medicine portfolio through a collaboration with University Health Network (UHN), gaining access to hepatocytes- ZUG, Switzerland and CAMBRIDGE, Mass., July 27, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the second quarter ended June 30, 2020. “We continue to make substantial progress driving our multiple, ongoing clinical development programs. Enrollment in our immuno-oncology trials is ongoing, and we’ve re-initiated dosing in our CTX001 trials,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “Further, we expect to report data from our CTX001 program targeting hemoglobinopathies and our CTX110 program later this year. Despite the challenges posed by COVID-19, we continue to execute on our programs and remain focused on our commitment to patients and their families.” Recent Highlights and Outlook Beta Thalassemia and Sickle Cell Disease CRISPR Therapeutics and its partner Vertex provided new clinical data at the European Hematology Association (EHA) Congress from the two ongoing Phase 1/2 studies of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with transfusion-dependent beta thalassemia (TDT) and in patients with severe sickle cell disease (SCD). Data from two TDT patients demonstrated clinical proof-of-concept for CTX001 in this disease, and longer duration data from one SCD patient showed durable effects on fetal hemoglobin (HbF) levels and the patient was free of vaso-occlusive crises. Screening, enrollment and mobilization in these studies are ongoing; conditioning and dosing have been resumed following temporary COVID-19-related pauses in both studies. CRISPR Therapeutics and Vertex expect to report data from additional patients in the second half of 2020. In May, CRISPR Therapeutics and its partner Vertex announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001, an investigational, autologous, gene-edited hematopoietic s...