Regulating Cell and Gene Therapy Activity
Paul Wotton, PhD, chief executive officer, Obsidian Therapeutics, discussed the company’s pipeline and technologies.
Gavo-Cel Efficacious in Refractory Mesothelin-Expressing Solid Tumors
Investigators have identified the maximum tolerated dose and continue to refine the recommended phase 2 dose in the phase 1 part of the study.
Adverse Events in CAR T-Cell Therapies: Joseph A. Fraietta, PhD
The director of the Solid Tumor Immunotherapy Lab at the University of Pennsylvania discussed safety concerns with CAR T-cell therapies.
SGT-001 Improves Pulmonary Function in Duchenne Muscular Dystrophy
Improvements in peak expiratory flow, forced expiratory volume, and forced vital capacity were observed in all evaluable treated patients in the IGNITE-DMD trial.
Challenges in Developing Gene Therapies for Gaucher Disease: Neil Weinreb, MD
The chair of the International Collaborative Gaucher Group Registry discussed the place of gene therapies in the Gaucher treatment landscape.
Ide-Cel Continues to Show Durable Efficacy in Multiple Myeloma
Overall response rate was 73% in all patients treated with ide-cel at a median follow-up of 24.8 months (range, 1.7-33.6).
Future Research With Stem Cell Therapies in Hematologic Malignancies
The chief executive and chief medical officers of Vor Biopharma discussed the company’s future research and plans.
Suprachoroidal Delivery of RGX-314 Well-Tolerated in Wet AMD
Patients treated with RGX-314 also had stable BCVA and central retinal thickness.
Brexucabtagene Autoleucel Approved for Refractory B-Cell Acute Lymphoblastic Leukemia
ZUMA-3 showed that a single infusion of the CAR T-cell therapy elicited a high and durable response rate in heavily pretreated patients with relapsed/refractory B-ALL.
ASC Transplant Eligibility in Multiple Myeloma: Elisabet E. Manasanch, MD
The associate professor from The University of Texas MD Anderson Cancer Center discussed eligibility for autologous stem cell transplant in patients with multiple myeloma.
Angelman Syndrome Gene Therapy Trial Resumes Following Clinical Hold
A clinical hold was previously placed after patients treated with the highest dose developed lower extremity weakness.
Exposing the Advantages of iPSC-Natural Killer Cell Therapies
Allen Feng, PhD, chief scientific officer, HebeCell, discussed advantages of induced pluripotent over hematopoietic stem cell therapies.
Gene Therapy for AADC Deficiency Yields Durable Developmental Improvements
Improvements persisted through up to 10 years of follow-up.
Cellular, Tissue, and Gene Therapies AdComm Meeting Takeaways: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed the recent meeting of the FDA Cellular, Tissue, and Gene Therapies Advisory Committee.
GeneTherapyLive’s Weekly Rewind – October 1, 2021
Review top news and interview highlights from the week ending October 1, 2021.
CAR T-Cell Therapy Axi-Cel Seeks Expanded Indication for Second-Line LBC Lymphoma
Treatment with the CAR T-cell therapy yielded a 60% reduction in the risk of EFS events compared with SOC with a median follow-up of 2 years.
Integrating TIL-Based Therapy into Melanoma Treatment: Omar Hamid, MD
The chief of translational Research and immunotherapy at The Angeles Clinic and Research Institute discussed the integration of tumor infiltrating lymphocyte–based therapy into the melanoma armamentarium.
High Dose Achromatopsia Gene Therapy Yields Severe Inflammation
The dose was previously evaluated in adult patients with no comparable inflammation.
Pfizer Restricts Enrollment in Duchenne Gene Therapy Trial Following Adverse Events
The decision comes after 3 participants experienced serious adverse events during the phase 3 trial.
KarMMA: Key Findings of Ide-Cel in Multiple Myeloma: Nina Shah, MD
The hematologist and oncologist from UCSF Helen Diller Family Comprehensive Cancer Center discussed key findings from the phase 2 KarMMA trial.
CRISPR Therapy Shows Some Benefit in Leber Congenital Amaurosis
A competing RNA-based therapy for LCA10 is currently being evaluated in a phase 2/3 trial.
Bolstering Stem Cells Against Off-Target Effects of Cancer Therapies
The chief executive and chief medical officers of Vor Biopharma discussed their focus on AML, CD33, and their lead program VOR33.
CAR-Macrophage Receives Fast Track Designation for Solid Tumors
CT-0508 is currently under investigation in a phase 1 clinical trial.
Developing Gene Therapies for AMD: Brandi L. Williams, PhD; Gregory S. Hageman, PhD
Directors from the Moran Eye Center discussed researching gene therapy approaches targeting HTRA1 in AMD.
Cardiac-Derived Cell Therapy Shows Efficacy in Duchenne Muscular Dystrophy
CAP-1002 met primary and secondary end points in the HOPE-2 trial.
Investigating BET Inhibitors to Improve CAR T Therapy: Joseph A. Fraietta, PhD
The director of the Solid Tumor Immunotherapy Lab at the University of Pennsylvania discussed JQ1 and other BET inhibitors he would like to study.
Gene Therapies for Liver Diseases the Focus of New Collaboration
Precision BioSciences is collaborating with iECURE, a new company focused on mutation-agnostic, liver disease gene therapies.
CAR T as Frontline Therapy for DLBC Lymphoma: Alex Herrera, MD
The hematologist/oncologist from City of Hope discussed the potential for CAR T-cell therapy in earlier lines of treatment for patients with diffuse large B-cell lymphoma.
SGT-001 Shows Efficacy in DMD in 1.5-Year Data
Patient-reported outcomes, 6-minute walk test, and NSAA scores all showed improvements over natural history data.
Cilta-Cel Shows Durability in Relapsed/Refractory Multiple Myeloma
Overall response rate was 95%, including 75% of patients who achieved a complete response (CR) or better, with a median follow-up of 5.8 months at data cutoff.
