Gene-Independent Therapeutic Strategies for Retinal Degeneration
Researchers are exploring gene-independent therapeutic strategies to circumvent the challenges of gene-replacement therapies.
Expanding the Use of CAR T Therapy With shRNA
The chief executive and chief medical officers of Celyad Oncology discussed the company’s future research and plans.
Astellas Pauses Gene Therapy Trial for X-Linked Myotubular Myopathy
Following a voluntary pause, a patient experiencing a liver AE has died and the trial is now on clinical hold.
T Cell Therapy Trial for High-Risk Patients With COVID-19 Now Enrolling
TVGN-489 is an investigational allogeneic cytotoxic CD8+ T lymphocyte therapy that may help prevent breakthrough COVID infection.
Optimizing Manufacturing of NK Cell and CAR T Therapies for Cancer Treatment
Jan Davidson, MD, PhD, chief medical officer, Wugen, discussed the company’s cell therapy platforms.
New Collaboration to Develop Gene Therapy for Chronic Retinal Diseases
RGX-314 is being developed to target wet AMD, diabetic retinopathy, and other chronic retinal diseases.
Integrating CAR T Therapy in Multiple Myeloma: Krina K. Patel, MD, MSc
The associate professor from The University of Texas MD Anderson Cancer Center discussed the integration of CAR T-cell therapy into the treatment paradigm of R/R MM.
Bone Therapeutics Shifts Focus to Cell Therapies Following Phase 3 Fail in Knee Osteoarthritis
The company is currently evaluating ALLOB, its investigational cell therapy product for bone regeneration.
CAR T-Cell Utility in Hematologic Cancers: Alex Herrera, MD
The hematologist and oncologist from City of Hope discussed the utility of CAR T cells in patients with hematologic cancers.
TAC T-Cell Therapy vs CAR T-Cell Therapy
Paul Lammers, MD, MSc, president and chief executive officer, Triumvira Immunologics, discussed the company’s T-cell antigen coupler technology.
TIL Adoptive Cell Therapy for the Treatment of mNSC Lung Cancer
A new TIL therapy seeks to improve on current treatments for metastatic non-small cell lung cancer such as PD-1 inhibitors.
Addressing Exhausted T-Cells in CAR T Therapy: Joseph A. Fraietta, PhD
The director of the Solid Tumor Immunotherapy Lab at the University of Pennsylvania discussed the issue of exhausted T-cells.
AAV-Based Gene Therapy for Autoimmune Uveitis
A single-dose gene therapy for uveitis could fill an important treatment gap for patients.
Developing Natural Killer Cell Therapies for Hematologic Malignancies and Solid Tumors
The chief executive and medical officers of Cytovia Therapeutics discussed their natural killer cell therapy technology.
Gene Therapy for Congenital Hearing Loss Granted Orphan Drug, Rare Pediatric Disease Designations
Decibel Therapeutics previously presented favorable preclinical data at ASGCT 2021.
RNA-Modified vs DNA-Modified CAR T Products
Michael Singer, MD, PhD, chief scientific officer, Cartesian Therapeutics, discussed the company’s RNA Armory technology.
Future Research With CAR T Therapy in Multiple Myeloma: Frits van Rhee, MD, PhD
The professor and director of the myeloma center at the University of Arkansas for Medical Sciences discussed future research with CAR T in multiple myeloma.
Reduced Response to Adoptive Transfer of TIL Therapy in Metastatic Melanoma With Prior Treatment
Adoptive transfer of TILs elicited an ORR of 56% in patients with metastatic melanoma who were naïve to PD-1 inhibitors compared with 24% in patients refractory to PD-1 inhibitors.
Elucidating the Role of HTRA1 in Developing AMD: Gregory S. Hageman, PhD; Brandi L. Williams, PhD
Directors from the Moran Eye Center discussed their research on the genetics of age-related macular degeneration.
Addressing High Relapse Rates in CAR T-Cell Therapy
Paul Rennert, president and chief scientific officer of Aleta BioTherapeutics, discussed the company’s CAR T Engager protein technology.
PKU Gene Therapy Trial on Clinical Hold Following Tumor Development in Mice
Six of 7 mice treated with a new, higher dose of BMN-307 showed tumors in liver necropsy at 52 weeks post-treatment.
New Gene Therapy Collaboration Targets Huntington Disease
NeuExcell and Spark Therapeutics each add to their gene therapy pipelines with the collaborative program.
Rationale and Future of Ide-Cel: Larry D. Anderson, Jr, MD, PhD
The hematologist/oncologist at the Harold C. Simmons Comprehensive Cancer Center discussed the rationale behind using CAR T-cell therapy in relapsed/refractory multiple myeloma and data from the KarMMa trial.
Gene Therapy's Potential in Retinal Disease
A new animal model will facilitate the study and development of gene therapies in retinal diseases.
Emerging Therapeutics Optimize Hematologic Malignancy Treatment
Experts discussed novel options such as CAR T-cell therapy for optimizing treatment in MCL, myelofibrosis, and ALL.
CARTITUDE Trials in Multiple Myeloma
Experts discussed safety and efficacy data of cilta-cel for R/R MM from the CARTITUDE-1 and CARTITUDE-2 studies.
Gene Therapy Partially Restored Vision in Patient with Retinitis Pigmentosa
Addressing Unmet Needs With Gene Therapy
Eric Crombez, PhD, senior vice president and chief medical officer of Ultragenyx Gene Therapy, discussed indications the company is targeting.
Gavo-Cel Granted Orphan Drug Designation for Cholangiocarcinoma
The investigational cell therapy consists of T-cell receptor (TCR) fusion construct T cells (TRuC-T cells).
Gene Therapy for Retinal and Neurological Diseases
Magali Taiel, MD, chief medical officer, GenSight Biologics, discussed future research the company plans to pursue.
