TCR T-Cell Therapy to be Evaluated in Advanced Esophageal or EGJ Cancers
The SURPASS-2 trial will run parallel to the ongoing SURPASS trial.
HLA LOH Prevalent in GI Cancers, Identifies Future Study Participants
The BASECAMP-1 study is identifying patients for future use of Tmod CAR T-cell therapies.
Omidubicel BLA Submission for Hematologic Malignancies Expected First Half of 2022
The FDA and Gamida Cell have reached a consensus on the comparability of omidubicel manufactured at different sites.
Novel Chemo/NK Cell Therapy Combination Shows Efficacy in Pancreatic Cancer
Data from the phase 2 QUILT-88 study were presented at the 2022 ASCO GI symposium.
First In-Human Study Initiated for CAR Macrophage Cell Therapy for Solid Tumors
Carisma Therapeutics and Moderna are also collaborating to develop CAR-M cell therapies.
First Patient Dosed in CAR T-Cell Therapy/Pembrolizumab Trial for Colorectal Cancer
Further details on the KEYNOTE-B79 trial were presented at the 2022 ASCO GI Symposium.
Around the Helix: Gene and Cell Therapy Company Updates - January 19, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in gene and cell therapies.
Trial Initiated for Pediatric Liver Cancer Cell Therapy
The phase 1/2 ARYA-2 trial will be run in parallel with the similar phase 1/2 ARYA-1 and ARYA-3 trials.
FDA Extends BLA Review for bluebird bio’s β-Thalassemia and CALD Gene Therapies
Eli-cel remains on clinical hold in cerebral adrenoleukodystrophy after a report of persistent anemia.
Fabry Disease Gene Therapy Shows Efficacy in First 2 Patients
The first patient with Fabry disease dosed with FLT190 2 years ago continues to have elevated, sustained α-Gal A.
Improving Outcomes in Solid Tumors With Personalized Cell Therapies
Cedrik Britten, MD, chief medical officer, Immatics, discussed the advantages of IMA203 over other cell therapies.
Sickle Cell Disease Cell Therapy Clinical Trial Enrolls First Patient
Details on the phase 1/2 CEDAR trial were presented at the 2021 ASH meeting.
The State of Gene and Cell Therapy for Sickle Cell Disease
Exciting new therapies for sickle cell disease are on the horizon. We explored some of the most promising investigational therapies in recognition of National Blood Donor Month.
Gene Therapy Relieves Radiation-Induced Xerostomia
Six of 7 patients reported improvements in dry mouth after treatment with AAV-hAQP1.
Around the Helix: Gene and Cell Therapy Company Updates
Many cell and gene therapy companies are initiating new programs and collaborations with the start of the new year.
Gene Therapy Continues to Show Efficacy in Duchenne Muscular Dystrophy
The placebo crossover cohort experienced statistically significant improvements in NSAA score over external control.
Hemophilia A Cell Therapy Stalls; Sigilon to Shift Focus to MPS and Diabetes Programs
The reprioritization comes as the SIG-001 program remains on clinical hold after scarred and inviable cell spheres were observed in a treated patient.
Hemophilia A Gene Therapy Yields Sustained Bleeding Reductions in Severe Cases
Reductions in annualized bleeding rate remained significant at 2 years in the phase 3 study although recently presented phase 2 data showed significant FVIII reductions at 5 years.
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.