CRISPR/Cas9 gene editing therapies / Bayer, CRISPR Therap - LARVOL DELTA

[VIRTUAL] Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9–Modified CD34+ Hematopoietic Stem and Progenitor Cells (ASH 2020) - P1/2 | "Peripheral CD34+ HSPCs were collected by apheresis after mobilization with G-CSF (filgrastim) and plerixafor (for TDT) or plerixafor alone (SCD)...Prior to CTX001 infusion on Day +1, patients received myeloablation with 4 days of busulfan... These data demonstrate that CTX001, a first-in-human, CRISPR-Cas9–modified autologous HSPC product, has resulted in increases in HbF and total Hb in the first 7 patients infused. All patients infused with CTX001 demonstrated hematopoietic engraftment with a post-infusion safety profile generally consistent with myeloablation. All 5 patients with TDT have been transfusion-free since ~2 months after CTX001 infusion and the 2 patients with severe SCD have had no VOCs during follow-up after CTX001 infusion."

Clinical • Acute Respiratory Distress Syndrome • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Infectious Disease • Pain • Pneumonia • Respiratory Diseases • Sickle Cell Disease • CD34 • CSF3

[VIRTUAL] Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia (TDT) or Sickle Cell Disease (SCD): Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells (HSPCs) (TCT-ASTCT-CIBMTR 2021) - P1/2 | "We collected peripheral CD34+ HSPCs by apheresis after mobilization with G-CSF and plerixafor (TDT) or plerixafor alone (SCD)...In all 7 patients, the safety profile after CTX001 infusion was generally consistent with busulfan myeloablation... CTX001, a first-in-human, CRISPR-Cas9-modified autologous HSPC product, has led to increases in HbF and total Hb in the first 7 patients treated. Its post-infusion safety profile is generally consistent with myeloablation. All 5 TDT patients have been transfusion-free since ~2 months after CTX001 and the 2 SCD patients have had no VOCs after CTX001."

Clinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Pain • Sickle Cell Disease • CD34 • CSF3

[VIRTUAL] INITIAL SAFETY AND EFFICACY RESULTS WITH A SINGLE DOSE OF AUTOLOGOUS CRISPR-CAS9 MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS IN TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE (EHA 2020) - P1/2 | "Peripheral CD34+ HSPCs were collected by apheresis after mobilization with G-CSF (filgrastim) and plerixafor (TDT) or plerixafor alone (SCD)...Two serious AEs (SAEs) occurred, both considered unrelated to CTX001: veno-occlusive liver disease (related to busulfan) and pneumonia in the presence of neutropenia; both resolved...Data will be updated for the presentation. Submitted on behalf of the CLIMB THAL-111 and CLIMB SCD-121 Investigators."

Clinical • Gene Therapies • Genetic Disorders • Hematological Disorders • Hepatology • Infectious Disease • Neutropenia • Pain • Pneumonia • Respiratory Diseases • Septic Shock • Sickle Cell Disease • Transplantation • CD34 • CSF3

[VIRTUAL] Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9–Modified CD34+ Hematopoietic Stem and Progenitor Cells (ASH 2020) - P1/2 | "Peripheral CD34+ HSPCs were collected by apheresis after mobilization with G-CSF (filgrastim) and plerixafor (for TDT) or plerixafor alone (SCD)...Prior to CTX001 infusion on Day +1, patients received myeloablation with 4 days of busulfan... These data demonstrate that CTX001, a first-in-human, CRISPR-Cas9–modified autologous HSPC product, has resulted in increases in HbF and total Hb in the first 7 patients infused. All patients infused with CTX001 demonstrated hematopoietic engraftment with a post-infusion safety profile generally consistent with myeloablation. All 5 patients with TDT have been transfusion-free since ~2 months after CTX001 infusion and the 2 patients with severe SCD have had no VOCs during follow-up after CTX001 infusion."

Clinical • Acute Respiratory Distress Syndrome • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Infectious Disease • Pain • Pneumonia • Respiratory Diseases • Sickle Cell Disease • CD34 • CSF3

[VIRTUAL] EARLY SAFETY AND EFFICACY RESULTS WITH A SINGLE DOSE OF AUTOLOGOUS CRISPR-CAS9-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS IN TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE (EBMT 2021) - P1/2 | "We collected peripheral CD34+ HSPCs by apheresis after mobilization with G-CSF and plerixafor (TDT) or plerixafor alone (SCD)...Patients received myeloablative busulfan before CTX001 infusion... CTX001, a first-in-human, CRISPR-Cas9-modified autologous HSPC product, has led to increases in HbF and total Hb in the 10 treated patients. Its post-infusion safety profile is generally consistent with myeloablation. All 7 TDT patients have been transfusion-free since ~2 months after CTX001 and the 3 SCD patients have had no VOCs after CTX001."

Clinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Pain • Sickle Cell Disease • Transplantation • CD34 • CSF3 • HP

[VIRTUAL] EARLY SAFETY AND EFFICACY RESULTS WITH A SINGLE DOSE OF AUTOLOGOUS CRISPR-CAS9-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS IN TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE (EBMT 2021) - P1/2 | "We collected peripheral CD34+ HSPCs by apheresis after mobilization with G-CSF and plerixafor (TDT) or plerixafor alone (SCD)...Patients received myeloablative busulfan before CTX001 infusion... CTX001, a first-in-human, CRISPR-Cas9-modified autologous HSPC product, has led to increases in HbF and total Hb in the 10 treated patients. Its post-infusion safety profile is generally consistent with myeloablation. All 7 TDT patients have been transfusion-free since ~2 months after CTX001 and the 3 SCD patients have had no VOCs after CTX001."

Clinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Pain • Sickle Cell Disease • Transplantation • CD34 • CSF3 • HP

[VIRTUAL] EARLY SAFETY AND EFFICACY RESULTS WITH A SINGLE DOSE OF AUTOLOGOUS CRISPR-CAS9-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS IN TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE (EBMT 2021) - P1/2 | "We collected peripheral CD34+ HSPCs by apheresis after mobilization with G-CSF and plerixafor (TDT) or plerixafor alone (SCD)...Patients received myeloablative busulfan before CTX001 infusion... CTX001, a first-in-human, CRISPR-Cas9-modified autologous HSPC product, has led to increases in HbF and total Hb in the 10 treated patients. Its post-infusion safety profile is generally consistent with myeloablation. All 7 TDT patients have been transfusion-free since ~2 months after CTX001 and the 3 SCD patients have had no VOCs after CTX001."

Clinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Pain • Sickle Cell Disease • Transplantation • CD34 • CSF3 • HP

[VIRTUAL] EARLY SAFETY AND EFFICACY RESULTS WITH A SINGLE DOSE OF AUTOLOGOUS CRISPR-CAS9-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS IN TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE (EBMT 2021) - P1/2 | "We collected peripheral CD34+ HSPCs by apheresis after mobilization with G-CSF and plerixafor (TDT) or plerixafor alone (SCD)...Patients received myeloablative busulfan before CTX001 infusion... CTX001, a first-in-human, CRISPR-Cas9-modified autologous HSPC product, has led to increases in HbF and total Hb in the 10 treated patients. Its post-infusion safety profile is generally consistent with myeloablation. All 7 TDT patients have been transfusion-free since ~2 months after CTX001 and the 3 SCD patients have had no VOCs after CTX001."

Clinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Pain • Sickle Cell Disease • Transplantation • CD34 • CSF3 • HP

[VIRTUAL] EARLY SAFETY AND EFFICACY RESULTS WITH A SINGLE DOSE OF AUTOLOGOUS CRISPR-CAS9-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS IN TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE (EBMT 2021) - P1/2 | "We collected peripheral CD34+ HSPCs by apheresis after mobilization with G-CSF and plerixafor (TDT) or plerixafor alone (SCD)...Patients received myeloablative busulfan before CTX001 infusion... CTX001, a first-in-human, CRISPR-Cas9-modified autologous HSPC product, has led to increases in HbF and total Hb in the 10 treated patients. Its post-infusion safety profile is generally consistent with myeloablation. All 7 TDT patients have been transfusion-free since ~2 months after CTX001 and the 3 SCD patients have had no VOCs after CTX001."

Clinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Pain • Pediatrics • Sickle Cell Disease • Transplantation • CD34 • CSF3 • HP

[VIRTUAL] The Role of BAFF-R signaling in the growth of primary central nervous system lymphoma (SNO 2020) - "Here, we created a BAFF-R knockout lymphoma cell line (BAFF-R-KO) using CRISPR-Cas9...Additionally, median survival of BAFF-R-KO mice was significantly prolonged. Altogether, our results indicate a potential of BAFF-R as a novel treatment target for PCNSL."

Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor

New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001™ for Severe Hemoglobinopathies Accepted for Oral Presentation at the 25th European Hematology Association (EHA) Congress (GlobeNewswire, CRISPR Therapeutics AG) - "CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that new data from two ongoing Phase 1/2 clinical trials of the CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies have been accepted for an oral presentation at the EHA Congress, which will take place virtually from June 11-14, 2020. An abstract posted online today includes 12 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and 6 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Updated data will be presented at EHA, including longer duration follow-up data for the first two patients treated in these trials and initial data for the second patient treated in the CLIMB-111 trial."

Clinical • Clinical data • Enrollment status • Anemia • Beta-Thalassemia • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

[VIRTUAL] EFFICIENT DELETION OF CCR5 PROVIDES COMPLETE PROTECTION AGAINST HIV IN XENOGRAFT MICE (CROI 2020) - "These data demonstrate that high frequency CRISPR/Cas9-mediated editing of CCR5 in human HSPCs is achievable and is sufficient to prevent infection during multiple, high dose exposures to a highly pathogenic strain of HIV. These experiments provide the basis to explore the prevention of systemic HIV rebound in an autologous transplant setting to help guide future clinical approaches to achieve a functional cure."

Late-breaking abstract • Preclinical • CD34 • CXCR4

A Long-term Follow-up Study in Subjects Who Received CTX001 (clinicaltrials.gov) - P=N/A; N=90; Enrolling by invitation; Sponsor: Vertex Pharmaceuticals Incorporated; Not yet recruiting ➔ Enrolling by invitation

Clinical • Enrollment open