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.
Assessing Fitusiran for Hemophilia in ATLAS-INH: Guy Young, MD
The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed the ATLAS-INH trial.
Challenges in Managing Hemophilia: Steven W. Pipe, MD
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed challenges in hemophilia A and B.
Ide-Cel Approved in Japan for R/R Multiple Myeloma
The approval was based on findings from the phase 2 KarMMa trial and the phase 1 Study CRB-401 trial.
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.
Axi-cel Label Update Approved to Include Use of Prophylactic Corticosteroids
The FDA has approved the prescribing label update based on data from a new safety management cohort of the phase 1/2 ZUMA-1 trial.
First Patient Dosed in Wet AMD Gene Therapy Trial
The clinical trial of 4D-150 expects to enroll around 60 participants with wet AMD.
Tumor-Infiltrating Lymphocyte Therapy After Immunotherapy in Advanced Melanoma: Omid Hamid, MD
The director of the Melanoma Program at Cedars-Sinai Medical Center discussed the efficacy of TIL therapy following immunotherapy in advanced melanoma.
First Patients Dosed in Gene Therapy Trial for Usher Syndrome, Retinitis Pigmentosa
Both the Sirius and Celeste phase 2/3 trials are evaluating QR-421a.
Brexucabtagene Autoleucel in R/R Mantle Cell Lymphoma
Experts discussed the ZUMA-2 clinical trial of brexucabtagene autoleucel.
GeneTherapyLive’s Weekly Rewind – January 28, 2022
Review top news and interview highlights from the week ending January 28, 2022.
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.
Evaluation Criteria for TIL Therapy in Melanoma: Omid Hamid, MD
The director of the Melanoma Program at Cedars-Sinai Medical Center discussed evaluation criteria for TIL therapy in melanoma.
Cilta-Cel in the Multiple Myeloma Treatment Landscape: Thomas G. Martin, MD
The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed data from both the CARTITUDE-1 and CARTITUDE-2 studies.
Around the Helix: Gene and Cell Therapy Company Updates - January 26, 2022
Expanding Study Populations of Cell Therapy in Sickle Cell Disease: Michael Grimley, MD
The medical director of Bone Marrow Transplantation and Immune Deficiency at Cincinnati Children's discussed data on ARU-1801 presented at ASH 2021.
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.
Challenges of Cell Therapy in Solid Tumors: Julian Molina, MD, PhD
The hematologist/oncologist from Mayo Clinic discussed advantages of Tmod cell therapy in solid tumors.
Stem Cell Gene Therapy Efficacious in Metachromatic Leukodystrophy
Investigators from IRCCS San Raffaele analyzed results from a phase 1/2 study on atidarsagene autotemcel.
Selecting Targets in Hematologic Malignancies
Dolores Schendel, PhD, chief executive and chief scientific officer, Medigene, discussed MDG1011 and Medigene’s future research and targets.
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.
T-Cell Therapy Gets Orphan Drug Designation for Pancreatic Cancer
The T-cell therapy targets 6 tumor-associated antigens that are highly expressed in pancreatic cancer.
Evaluating MB-106 in B-NH Lymphoma and CL Leukemia: Mazyar Shadman, MD
The associate professor from Fred Hutchinson Cancer Research Center discussed the methods utilized in an ongoing phase 1/2 trial evaluating MB-106.
DLBC Lymphoma CAR T Therapy Granted RMAT and Fast Track Designations
Positive data from a phase 1 trial were presented at the 2021 ASCO meeting.
Improving Outcomes in Hemophilia With Fitusiran: Guy Young, MD
The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed advantages of the siRNA therapeutic.
GeneTherapyLive’s Weekly Rewind – January 21, 2022
Review top news and interview highlights from the week ending January 21, 2022.
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.
