Bellicum Walks Away From GoCAR-T Cell Clinical Trials in Prostate Cancer, Solid Tumors
The announcement comes in light of serious adverse events reported in the phase 1/2 clinical trials for BPX-601.
FDA Extends Val-Rox PDUFA Date in Hemophilia A
The extension follows the submission of additional phase 3 data from the GENEr8-1 clinical trial that constitutes a major amendment to the biologics license application. The new deadline for review is June 30, 2023.
Hematopoietic Cell-based Gene Therapy Shows Durable Response in Fanconi Anemia
Long-term hematologic stability has been observed in at least 6 out of 10 patients with at least 12 months of follow-up.
10 Cell and Gene Therapies to Watch in 2023
With confidence building, numerous cell and gene therapies will likely go before the FDA and other global regulatory agencies this year, in addition to key data readouts.
FDA Approves Ferring's Gene Therapy for Bladder Cancer
The approval follows a CRL that was issued in 2021 due to problems with chemistry, manufacturing, and controls.
bluebird bio's Lovo-Cel Has Durable Effect in Sickle Cell Disease
Additional data on 2 patients who developed persistent anemia suggests a genetic cause may be behind the serious adverse event.
BioMarin Prepares for PDUFA Delay for Val-Rox Hemophilia Gene Therapy
Recent communication with the FDA suggests that the agency's decision might be delayed past Q1 2023.
DMD Patient Dies in CRISPR Gene Therapy Trial Led By Nonprofit Biotech Cure Rare Disease
The trial was launched through a partnership between Cure Rare Disease and UMass Chan Medical School.
Bridging the Gap: Education, Awareness Key to Translating Medical Innovation to Community Health Care in Rare Diseases
With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.
Eli-Cel Approved for CALD, Marking Second Gene Therapy Win for bluebird bio
The approval follows unanimous support of eli-cel's benefits in CALD from an FDA AdComm meeting and an approval for beti-cel in beta-thalassemia.
Intellia Nets Positive Data Across In Vivo CRISPR Therapies for ATTR Amyloidosis Cardiomyopathy, Angioedema
Administration of NTLA-2001 led to rapid and deep reductions in serum TTR by day 28.
BioMarin to Investigate B-ALL Case in Val-Rox Gene Therapy Clinical Trial
The gene therapy received a conditional marketing approval in the European Union in August 2022.
UniQure Pauses High Dose of AMT-130 Huntington Disease Gene Therapy for Severe Adverse Events
No such adverse events have occurred in the lower-dose group.
COVID-19 Cell Therapy Trial Dosing Complete With Good Safety Profile
Elderly or high-risk patients with COVID-19 received 1 of 4 escalating dose levels.
Clinical Hold Lifted on Celyad's Phase 1b Trial in Refractory Metastatic Colorectal Cancer
The company voluntarily paused the trial in February 2022 to investigate 2 reported deaths in the trial.
Daniel Judge, MD, on Making Genetic Testing, Insights More Mainstream
MUSC recently launched In Your DNA SC, a 4-year study to collect and glean insights from genetic testing in the general population.
CD30-Targeted CAR T Cell Therapy Promising in Heavily Pretreated Hodgkin Lymphoma
A phase 2 clinical trial evaluating a T-cell memory enriched anti-CD30 CAR-T is now underway.
CD33 CAR NK Cell Therapy Shows Improved Targeting in Acute Myeloid Leukemia
As of the data cutoff, 3 of 5 treated participants remain in complete response.
Off the Shelf CD19 Cell Therapy for CAR T Relapse Patients Shows Promise
Data from a phase 1/2a study showed notable response rates in patients who had received a median of 5 prior lines of therapy.
Poverty, Opportunity May Impact CAR T-Cell Therapy Access, Outcomes
The findings suggest that less disadvantaged patients with high disease burden may have greater ability to advocate for CAR T-cell therapy.
Gene Therapy for LAD-I Posts Impressive Survival Data
In an interview with CGTLive, study primary investigator Donald Kohn, MD, discussed the safety and efficacy observed in the phase 2 trial.
Gene Therapy for Refractory Angina Promotes Increased Exercise Tolerance
Safety and efficacy data suggest this to be a feasible and clinically meaningful treatment for a patient population with high disease burden.
Improvements in Developmental Milestones Observed With GM1 Gangliosidosis Gene Therapy
Data from 2 patients dosed in the Imagine-1 clinical trial were presented in a late breaking session at ASGCT 2022.
Improved Function Observed Following Mini-Dystrophin Gene Therapy in DMD
Study sponsor Pfizer recently announced the launch of a phase 3 trial in patients with Duchenne muscular dystrophy.
Editas’ Gene-Edited Cell Therapy for Beta Thalassemia Wins Orphan Status
The investigational therapy is also currently being evaluated in a clinical trial for sickle cell disease.
Caribou’s CRISPR-Edited CAR T-Cell Therapy CB-010 Elicits Complete Response in R/R B-NHL
Caribou will present the initial data at the upcoming European Hematology Association (EHA) meeting in June.
RNA CAR T-Cell Therapy for Myasthenia Gravis Shows Benefits in Early Results
Late-breaking interim data were presented at the 14th Myasthenia Gravis Foundation of America (MGFA) International Conference on Myasthenia and Related Disorders.
Arcellx Doses First Patient in ARC-SparX CAR-T Program for Multiple Myeloma
The cell therapy is the first to come out of the company’s proprietary ARC-SparX platform.
REGENXBIO DMD Gene Therapy Trial Delayed Due to Manufacturing Issue
The trial was set to begin patient dosing in the first half of this year.
Real-World Luxturna Data Shows Good Efficacy, New Adverse Reaction in Retinal Dystrophy
Investigators presented up to 2 years of post-marketing data on the first-ever gene therapy.
