Gene Therapies for Mucopolysaccharidosis Show Safety, Efficacy
Interim data from trials of RGX-121 and RGX-111 were presented at WORLDSymposium 2022.
Study to Evaluate Gene-Edited Cell Therapy for MPSII
The LV-HSCGT therapy has demonstrated efficacy in preclinical studies in mouse models.
Beti-Cel Yields Durable Transfusion Independence in β-Thalassemia
The gene therapy is currently under FDA review for the treatment of β-thalassemia across genotypes.
Etranacogene Dezaparvovec Superior to FIX Prophylaxis in Hemophilia B
Results from the final analysis of the HOPE-B trial were presented at the 2022 EAHAD meeting.
COVID-19 Cell Therapy Trial Completes Dosing
No dose-limiting toxicities or treatment-emergent adverse events have been reported.
LogicBio’s Methylmalonic Acidemia Gene-Editing Therapy Program Placed on Clinical Hold
The hold was prompted by the second development of a serious AE of thrombotic microangiopathy in a patient in the younger age group.
Around the Helix: Gene and Cell Therapy Company Updates - February 2, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in gene and cell therapies.
IND Cleared for Adrenomyeloneuropathy Gene Therapy
SwanBio previously presented positive preclinical data at the ESGCT 2021 meeting.
Giant Axonal Neuropathy Gene Therapy Well-Tolerated in High-Dose Cohort
The positive news is countered by a report of a patient death in Taysha’s gene therapy program for GM2 gangliosidosis.
A Snapshot of Ophthalmologic Cell and Gene Therapies in Development
Exciting new therapies are on the horizon for inherited retinal disease. We’ve compiled a quick snapshot of promising investigative therapies.
X-Linked SCID Gene Transfer Therapy Placed on Clinical Hold
The latest hold on MB-207 follows a similar hold on another XSCID therapy, MB-107.
Around the Helix: Gene and Cell Therapy Company Updates - January 26, 2022
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
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.