Fabry Gene Therapy Program Discontinued Following Transient Engraftment Issues
AVRIOBIO announced that it is shifting priorities to focus on their gene therapy programs in Gaucher disease.
Angelman Syndrome Gene Therapy Well Tolerated
The DSMB has recommended that enrollment expand and doses be escalated from 3.3 to 5 mg in 1 cohort of the KIK-AS study.
Gene Therapy Yields Complete Wound Healing in Dystrophic Epidermolysis Bullosa
The phase 3 GEM-3 study met both its primary and secondary end points compared with placebo in dEB.
Poseida’s CAR T-Cell Therapy IND Accepted for Solid Tumors
Poseida’s announcement follows their presentation of positive data in metastatic castrate-resistant prostate cancer in September 2021.
Eli-Cel Gene Therapy for CALD Is Granted Priority Review
The program is still on clinical hold following SUSARs of myelodysplastic syndrome in treated participants.
Anti-Thrombin siRNA Reduces Bleeding in Severe Hemophilia Without Inhibitors
Annualized bleeding rate, spontaneous bleeding, and joint bleeding rates were all reduced in patients dosed with fitusiran compared to those receiving on-demand treatment.
Anti-Thrombin siRNA Therapeutic Reduces Bleeding in Hemophilia
Over half of patients in the fitusiran arm of the ATLAS-INH study had 0 treated bleeding events.
Axi-Cel Superior to SoC in Second-Line Large B-Cell Lymphoma Treatment
Patients treated with the CAR T-cell therapy had an over 4-fold increase in event-free survival.
Gene Edited Cell Therapy Improves Fetal Hemoglobin Expression in Patients With Sickle Cell Disease
All evaluable treated participants experienced a reduction in vaso-occlusive events after treatment with ARU-1801.
Hemophilia B Gene Therapy Meets Primary End Points in HOPE-B Trial
CSL Behring plans to submit regulatory applications in the US and EU in the first half of 2022 for the AAV gene therapy etranacogene dezaparvovec.
Improving Personalized Cell Therapies for Solid Tumors
Cedrik Britten, MD, chief medical officer, Immatics discussed data on IMA203 presented at SITC 2021.
CAR T-Cell Therapy Shows Efficacy and Safety in B-Cell Malignancies
Adicet Bio reported positive interim data from the phase 1 GLEAN study.
American Gene Technology’s Cell Therapy Continues to Show Safety in HIV
An independent Data Safety and Monitoring Board has recommended that the phase 1 RePAIR trial continue at an accelerated pace.
IND Accepted for Sickle Cell Disease Cell Therapy
Beam Therapeutics will assess the safety and efficacy of BEAM-101 for SCD in the phase 1/2 BEACON-101 study.
IND Accepted for HER2+ Gastric and Gastroesophageal Junction Cancer Cell Therapy
CYNK-101 is set to be evaluated in combination with standard chemotherapy, trastuzumab and pembrolizumab in a phase 1/2a study.
CRISPR/Cas9-Edited CAR and NK Cell Therapies Show Preclinical Efficacy
CRISPR Therapeutics presented positive data on 2 preclinical programs at the 2021 SITC meeting.
Hemophilia A Gene Therapy Placed on Clinical Hold
Pfizer and Sangamo voluntarily paused the trial after participants experienced FVIII activity levels of over 150%.
FDA Accepts Beti-Cel BLA for β-thalassemia
The investigational gene therapy, which will receive priority review, has a PDUFA date of May 20, 2022.
Hemophilia A Gene Therapy Shows Sustained Factor VIII Expression
A 1-stage factor VIII assay revealed no apparent decrease in factor VIII activity over time.
Gene-Edited, Autologous HSP Cells Correct Metabolic Activity in Hurler Syndrome
The gene-edited approach hopes to overcome with the shortcomings of autologous hematopoietic stem-cell transplantation.
Danon Disease Gene Therapy Yields Sustained Clinical Benefit
Following updated safety data, Rocket Pharmaceuticals has decided to discontinue the high-dose cohort.
Charcot-Marie-Tooth Gene Therapy Yields Improvements in Disability and Muscle Loss
The phase 1 trial is the first in-human trial to assess a gene therapy for CMT.
OpRegen Shows Durable Efficacy in Dry AMD Geographic Atrophy
The Doheny Image Reading and Research Lab is leading a sub study to analyze OCT images with their proprietary 3D-OCTOR software.
CYNK-101 Demonstrates Anti-Tumor Activity in HNSCC Cells
The NK cell therapy is also being evaluated in a phase 1 study for acute myeloid leukemia.
Dendritic Cell Vaccine Boosts Survival in Glioblastoma
Overall survival was 72% at 12 months but dropped to 54% at 14.6 months.
FDA Manufacturing Data Request Delays Omidubicel BLA Submission
The request aims to ensure the comparability of omidubicel manufactured at different sites.
CAR Macrophages Well-Tolerated, May Reprogram Tumor Microenvironments
The first in-human study of CAR macrophages has dosed 2 participants so far.
Tmod CAR T-Cell Therapy Offers Precise Targeting of Solid Tumors
The BASECAMP-1 study is identifying patients with human leukocyte antigen (HLA) loss of heterozygosity (LOH) for future use of these therapies.
CAR T-Cell Therapy Plus Amplifying Vaccine Shows Initial Safety, Efficacy in Solid Tumors
Three patients dosed had tumors shrinkages of 18%, 21%, and 27%.
TCR T-Cell Therapy Shows Efficacy Across Solid Tumor Types
Data from the IMA203 trial were presented at the SITC 2021 annual meeting.