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.
Future Research With CAR T in Lymphoma: Julie Vose, MD
The professor and chief, of oncology and hematology at University of Nebraska Medical Center discussed research with CAR T therapy in lymphoma.
Gene Therapy for Wilson Disease Receives FDA Fast Track Designation
A phase 1/2 study evaluating Vivet Therapeutic’s VTX-801 is set to initiate in August 2021.
Targeting LHON and Retinitis Pigmentosa With Gene Therapy
Magali Taiel, MD, chief medical officer, GenSight Biologics, discussed the company’s work in ophthalmic diseases.
Managing CAR T Therapy Toxicities With Standardized Care Guidelines
David G. Maloney, MD, PhD, covered the importance of standardized criteria in managing CRS and ICANS with CAR T therapy.
Allogeneic vs Autologous CAR T-Cell Therapies
The chief executive and chief medical officer of Celyad Oncology discussed the advantages of allogeneic CAR T therapies over autologous ones.
Understanding the Genetics of Age-Related Macular Degeneration: Gregory S. Hageman, PhD
The executive director of the Steele Center for Translational Medicine at the Moran Eye Center discussed the research his team has been conducting in AMD.
Hemophilia Gene Therapy Registry Launched to Monitor Long-Term Outcomes
The World Federation of Hemophilia’s registry will include long-term safety and efficacy data in people with hemophilia treated with gene therapies.
Weighing Benefits and Risks of Cell Therapy in Multiple Myeloma: Suzanne Lentzsch, MD
The professor of clinical medicine, director, Multiple Myeloma and Amyloidosis Program, New York-Presbyterian Hospital, discussed utility of cellular therapies in multiple myeloma.
Selecting CAR T-Cell Therapies for R/R DLBCL
Experts discuss the use and selection of CAR T therapies in relapsed/refractory diffuse large B-cell lymphoma.
Activating SCN1A to Treat Dravet Syndrome
Salvador Rico, MD, PhD, chief medical officer, Encoded Therapeutics, discussed the company’s gene therapy pipeline, including their lead program ETX101.
FDA Places Clinical Hold on Bluebird Bio’s eli-cel for CALD
Bluebird Bio’s latest clinical hold follows another report of MDS in a treated patient.
Gene Therapy for Diabetic Macular Edema Halted Following Serious Adverse Reaction
Despite setbacks in DME, Adverum Biotechnologies said it will continue to develop ADVM-022 for wet AMD.
Selecting CAR T-Cell Therapies for Mantle Cell Lymphoma: Jae Park, MD
The hematologic oncologist from Memorial Sloan Kettering Cancer Center discussed factors to consider in selecting CAR T-cell therapies.
Novel Agents for Multiple Myeloma
Noa Biran, MD, discusses exciting new agents in the field that may fulfill unmet patient needs.
