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.
Gene Therapy for Sickle Cell Disease
Experts discuss the safety profile and cost of gene therapy for the management of sickle cell disease.
Developing Cell Therapies for Solid Tumors and Hematologic Malignancies
Sonny Hsiao, PhD, chief executive officer, president and cofounder, Acepodia, discussed the company’s cell therapy technologies.
Gene Therapy for Pompe Disease: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed gene therapy programs being conducted for Pompe disease.
Nadofaragene Firadenovec Demonstrates Durable Efficacy in Advanced NMIBC
In 2 year follow-up data, 20 patients (19.4%) remained free from high-grade recurrence.
Challenges With CAR T Therapy in Multiple Myeloma: Larry D. Anderson Jr, MD, PhD
The associate professor from the UT Southwestern Medical Center Harold C. Simmons Comprehensive Cancer Center discussed the challenges faced with CAR T-cell therapy in patients with multiple myeloma.
GeneTherapyLive’s Weekly Rewind – September 24, 2021
Review top news and interview highlights from the week ending September 24, 2021.
Addressing Adverse Events in Gene Therapy
The recent FDA Advisory Committee meeting follows a turbulent year for gene therapy studies.
Mitigating Cytokine Release Syndrome in CAR T for Multiple Myeloma: Krina K. Patel, MD, MSc
The associate professor from The University of Texas MD Anderson Cancer Center discussed mitigating CAR T-cell therapy–related cytokine release syndrome in multiple myeloma.
Reducing Off-Target Effects of CAR T-Cell Therapies
Eric von Hofe, PhD, president and chief operating officer of AffyImmune, discussed advantages of the Affinity tuning platform.
OpRegen's Continued Efficacy in Dry AMD Geographic Atrophy
Brian Culley, chief executive officer, Lineage Cell Therapeutics, discussed updated data from the phase 1/2 study of OpRegen.
Targeting Cancers With CAR T and NK Cells
Jan Davidson, MD, PhD, chief medical officer, Wugen, discussed the company’s future plans and research.
beti-cel BLA Submitted for β-Thalassemia
The news comes after another of bluebird bio’s programs, eli-cel, was placed on clinical hold.
