Gene Therapies for Neurological Diseases Will Be Focus of New Shape Therapeutics, Roche Collaboration
The 2 companies will focus their strategic partnership on the development of gene therapies for rare diseases, as well.
The Future of Gene Therapies in Inherited Retinal Diseases
Mariya Moosajee, MBBS, BSc, PhD, FRCOphth, discussed the next generation of gene therapy for inherited retinal diseases.
Tisagenlecleucel Does Not Significantly Improve EF Survival Over SOC in Non-Hodgkin Lymphoma
The CAR T therapy tisagenlecleucel missed its EFS endpoint in the phase 3 BELINDA trial for B-cell non-Hodgkin lymphoma.
Investigating the Role of HTRA1 in Developing AMD: Brandi L. Williams, PhD
The research director at the Moran Eye Center discussed the genetics of developing age-related macular degeneration.
Gene Therapies for Hemophilia Have Positive Real-World Impact, Studies Show
Presentations at ISTH 2021 revealed attributes most important to patients when considering gene therapies and where further research is needed.
Treatment Options After CAR T Therapy for Large B-Cell Lymphoma
The professor of medicine from Duke University School of Medicine discussed treatment approaches for after relapse on CAR T cell therapy or for patients who are ineligible for CAR T therapy.
Using CAR T Therapy and Other Novel Treatments for Non-Hodgkin Lymphoma
Stephen M. Ansell, MD, PhD, discussed the potential of using non–CAR T-based novel approaches in the non-Hodgkin lymphoma treatment paradigm.
Tackling Dravet Syndrome With Gene Therapy
Salvador Rico, MD, PhD, chief medical officer, Encoded Therapeutics, discussed the different initiatives the company is conducting in Dravet syndrome.
iPSC-Derived NK Cell Agents Show Benefit in B-Cell Malignancies
FT516 and FT595 are both currently being evaluated in phase 1 trials for B-cell malignancies.
IND Cleared for New CAR T-Cell Therapy in Large B-Cell Lymphoma
KITE-363 is set to be investigated in a phase 1 clinical trial at the end of 2021.
Pfizer-BioNTech COVID-19 Vaccine Receives FDA Approval
The 2-dose vaccine has been approved for use 9 months after its Emergency Use Authorization (EUA).
Treating Endothelial Disease With One Cornea Donor
Edward Holland, MD, chief medical advisor, discussed an injectable technique where one cornea donor could potentially supply hundreds of patients with treatment for endothelial disease.
Later-Generation Cell Therapies Increase Utility in Hematologic Malignancies
CAR T-cell therapy use will likely expand to a wider array of hematologic malignancies.
Using CAR T Therapy in R/R Multiple Myeloma
Nina Shah, MD, and other experts discuss the role of CAR T-cell therapy in relapsed/refractory multiple myeloma.
A Review of Autologous Stem Cell Transplantation in Amyloidosis
Iuliana Vaxman, MD, and Angela Dispenzieri, MD, review eligibility criteria for ASCT in AL amyloidosis, conditioning dosing, efficacy in terms of hematologic and organ response, and future areas of research.
Ide-Cel Approved in EU for Relapsed/Refractory Multiple Myeloma
The FDA previously approved ide-cel as the first BCMA-directed CAR T-cell therapy for patients with relapsed/refractory multiple myeloma following 4 or more prior lines of therapy.
Recognizing and Managing AEs in CAR T-Cell Therapy
Kelly Garvin, BSN, RN, OCN, discussed methods to communicate the benefits and risks of CAR T-cell therapy to patients and recognize AEs.
Selecting AAV Manufacturing Platforms
The chief scientific officer and chief medical officer of Ultragenyx Gene Therapy discussed the company’s future research in gene therapies.
Mustang Bio, Mayo Clinic Collaborate on New CAR T Platform
Mustang Bio plans to file an IND and start a phase 1 clinical trial as soon as a lead construct is identified.
JQ1, a BET Inhibitor, Increases CAR T Efficacy and Durability
Investigators sought to understand the mechanisms of transferred T cell proliferation and expression.
Developing Oncolytic Cell Therapies With VISION
Jennifer Buell, PhD, president and chief operating officer of Agenus, discussed the VISION platform the company uses to develop therapies.
Immunotherapy as Single Agent, Combination Therapy for Multiple Myeloma
Krina K. Patel, MD, MSc, discussed moving away from chemotherapy and toward immunotherapy.
Bringing Gene Therapies to Market With the Help of MEAs
With a new paradigm of treating previously incurable diseases comes a new pay paradigm for million-dollar therapies.
Future Research in Gene and Cell Therapy Conditioning
Kevin Heller, MD, executive vice president of research, Jasper Therapeutics, discussed future research with JSP191.
Ongoing Research Supports CAR T Therapy's Use in Non-Hodgkin Lymphoma
Julie Vose, MD, professor of internal medicine and division chief, Division of Oncology and Hematology, University of Nebraska Medical Center, discussed the use of CAR T-cell therapy in non-Hodgkin lymphoma.
CAR T Therapy ALLO-715 Receives Orphan Drug Designation for Multiple Myeloma
The ongoing phase 1 UNIVERSAL trial has demonstrated an overall response rate of 60% among 10 patients.
FDA Lifts Hold on Danon Disease Gene Therapy
Rocket Pharmaceuticals plans to quickly resume the phase 1 trial and commence dosing in their new, low-dose, pediatric cohort in Q3 2021.
Immunotherapy and CAR T-Cell Therapy: Stephen M. Ansell, MD, PhD
The chair of the Lymphoma Group at Mayo Clinic discussed incorporating immunotherapies beyond CAR T-cell therapy in non-Hodgkin lymphoma.
The Potential of CRISPR Therapies in Retinal Disorders
Targeted CRISPR therapy has potential in treating inherited retinal diseases.
Challenges With CAR T Therapy in Follicular Lymphoma: Sattva S. Neelapu, MD
The professor from the University of Texas Anderson Cancer Center discussed challenges surrounding CAR T-cell therapy for the treatment of follicular lymphoma and other lymphomas.
