Working to Enable Gene Therapy for Cardiovascular Disease
Jonathan W. Weinsaft, MD, chief of cardiology and professor of medicine at Weill Cornell Medical College, discussed research the center is doing in the space.
Cell and Gene Therapy Trials Face Disproportionate Amount of Clinical Holds
Oncological trials of biologics also face particularly higher rates of clinical holds.
Considerations for Gene Therapy Logistics and Implementation
Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.
Liso-Cel/Breyanzi Gets Accelerated Approval for Relapsed/Refractory Follicular Lymphoma
The CAR T-cell therapy was also recently approved for treating chronic lymphocytic leukemia or small lymphocytic lymphoma.
AADC Deficiency Gene Therapy up For FDA Priority Review
Eladocagene exuparvovec is approved in the United Kingdom and European Union under the name Upstaza.
Multicharacteristic Opsin Gene Therapy Improves BCVA, MLSDT in Retinitis Pigmentosa
High dose MCO-010 yielded statistically significant improvements in BCVA and MLSDT at week 52.
ALS REGALS Trial Continues to Evaluate Cellenkos’ CK0803 Treg Therapy
The trial is set to dose its second cohort following a positive DSMB recommendation in March.
Data Roundup: April 2024 Features Updates from AACR, AAN, in Muscular Dystrophy and Cancer
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
Tumor Agnostic Deltarex-G+ Gene Therapy Demonstrates Activity in Breast, Pancreatic, Sarcoma Cancers
All 139 tumors screened had enhanced expression of CCNG1, a seldom-recorded gene on NGS.
Elevidys Shows Slight Statistical Improvements on Time to Rise, 10-Meter Walk/Run Despite NSAA Fail
The gene therapy is up for priority review with a PDUFA date of June 21, 2024.
Rod-Cone Dystrophy Gene Therapy Demonstrates Acceptable Safety, Study to Continue
The DSMB recommended proceeding to dose the highest dose cohort in the phase 1/2 PRODYGY study.
Casey Maguire, PhD, on Combining Viral-Like Particles and AAV
The associate professor and associate investigator of neurology at Harvard Medical School discussed research on extracellular vesicle-associated AAVs presented at ASGCT.
SPG50 Gene Therapy Warrants Further Study
MELPIDA was well-tolerated in patients and nerve conduction was stable or improved after treatment.
Patient With DMD Dies in Pfizer’s Phase 2 Gene Therapy Trial
In response to the death, Pfizer is pausing dosing associated with the crossover portion of the phase 3 CIFFREO trial.
TDT/SCD Lentiviral Therapy Boasts Quick, Well-Tolerated Engraftment in Patients
KL003 favorably compares to platelet and neutrophil recovery rates of approved gene therapies.
First Patient Set to Receive bluebird’s Approved Sickle Cell Gene Therapy, Lyfgenia
Lovo-cel was approved as Lyfgenia in December 2023.
ODAC Supports MRD Endpoint in Accelerated Approval for Multiple Myeloma
The ODAC unanimously voted in favor of the use of minimal residual disease negativity.
NIH Strategizes to Streamline Gene Therapy Development
PJ Brooks, PhD, deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed ongoing initiatives.
Parkinson Gene Therapy Shows Stability, Some Improvements in ON/OFF Time Disease Measures
Based on these data, the phase 2 Regenerate PD trial of AB-1005 will begin enrolling later this year.
Looking Forward to A Potential First Cell Therapy Approval for Sarcomas
John A. Charlson, MD, associate professor of medicine, Medical College of Wisconsin, discussed advantages of afami-cel for synovial sarcoma and other solid tumors.
ASO Fast Tracked for Myotonic Dystrophy
PGN-EDODM1 is being evaluated in the US and Canada in a phase 1 trial.
TMZ γδ T-Cell Regimen May Have Potential in Treating Glioblastoma
A phase 1 trial evaluating INB-200 has shown a PFS benefit in treated participants.
Taysha Transfers Deprioritized Gene Therapy Programs Back to Partners
The company’s TSHA-201 AAV gene therapy for Rett syndrome is its current priority.
Taking Lessons Learned With CAR-T in Oncology to the Autoimmune Space
David Porter, MD, director of Cell Therapy and Transplant, Penn Medicine discussed how experience in oncology enabled the logical expansion.
First Patient With Diabetic Foot Ulcers Screened for SkinTE Pivotal Trial
Another recent program in the investigational landscape was Helixmith’s deprioritized Engensis diabetic neuropathy gene therapy.
iNKT Cells Safe, Shows Anti-Inflammatory Response in Patients With SARS-CoV-2
The cells may have potential to modulate disease in other cases of respiratory disease and critical illness.
Lineage Cell to Evaluate Novel Delivery of OPC1 Cell Therapy for Spinal Cord Injury
The company expects to open the first clinical site in the DOSED study in the second quarter of 2024.
First Patient With Gastric Cancer Dosed in iNKT/CTLA-4 Inhibitor/Anti-PD-L1 Trial
A case study previously described successful immune modulation with AgenT-797 in a patient with high-risk gastric cancer leading to a partial response.
One Big Step Away From BLA Submission for Sangamo’s Fabry Gene Therapy
The FDA is allowing a small, single-arm study to support a BLA submission of ST-290, if Sangamo can find help to continue developing the therapy.
Improving Quality of Life in Patients With Fabry With Gene Therapy
Robert Hopkin, MD, Cincinnati Children’s Hospital Medical Center, discussed unmet needs and patient-reported outcomes from the STAAR trial.