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.
Patient Considerations in R/R Mantle Cell Lymphoma Treatment: Anita Kumar, MD
The Regional Care Network Medical Site Director at Memorial Sloan Kettering Cancer Center Basking Ridge discussed patient factors to consider when selecting treatment in R/R MCL.
First Study of CAR T in R/R Thyroid Cancer Underway
The non-randomized, open-label trial of AIC100 is currently recruiting patients at the Weill Cornell Medical College in in New York.
CAR T-Cell Therapy and Others Refresh Acute Myeloid Leukemia Treatment Landscape
CAR T-cell therapy showed promising results in acute myeloid leukemia at ASCO 2021.
Novel Treatments for Multiple Myeloma: C. Ola Landgren, MD, PhD
The inaugural leader of the Experimental Therapeutics Program at Sylvester Comprehensive Cancer Center, University of Miami discussed exciting novel therapeutic approaches in multiple myeloma.
Allogeneic HSCT Yields Favorable Outcomes in Myelofibrosis
Jan Philipp Bewersdorf, MD, hospital resident at Yale University School of Medicine discussed the safety and efficacy of allogeneic hematopoietic stem cell transplant in myelofibrosis.
Improving AAV Production With HeLa Platform
Sam Wadsworth, PhD, chief scientific officer of Ultragenyx Pharmaceuticals, discussed the company’s platform for efficiently producing AAV vectors.
Fidanacogene Elaparvovec Well-Tolerated in Hemophilia B
No concerning effects to liver health were observed in the phase 1/2a study.
Targeting Relapsing/Refractory Multiple Myeloma With Cell Therapy
The president and chief operating officer of Agenus discussed the cell therapies the company is developing for the treatment of multiple myeloma.
Emerging Car T Therapies in Hematologic Malignancies: Jae H. Park, MD
The hematologic oncologist from Memorial Sloan Kettering Cancer Center discussed emerging CAR T-cell therapies in hematologic malignancies.
AGTC-401, AGTC-402 Gene Therapies Well-Tolerated in Achromatopsia
Clinical trials for both AGTC-401 and AGTC-402 show promising results.
FDA Lifts Clinical Hold on Novartis SMA Gene Therapy
The company announced plans to initiate the STEER study of intrathecal OAV-101 for older patients with SMA type 2.
Hemophilia B Gene Therapy AMT-060 Shows Durable Effect
Phase 3 studies are continuing in the enhanced Padua FIX variant AMT-061 for hemophilia B.
Investigating JSP191 for More Tolerable Gene and Cell Therapy Conditioning
The executive vice president of research and development at Jasper Therapeutics discussed positive data from trials of JSP191 in MDS and AML.
Advantages of CG0070 Over Standard Care for Non-Muscle Invasive Bladder Cancer
The chief executive officer of CG Oncology discussed the role of CG0070 in patients who are unresponsive and responsive to Bacillus Calmette-Guerin immunotherapy.
Gene Therapies for Batten Disease, ALS Receive FDA Designations
The designations allow for expedited drug development and review of the investigational gene therapies for CLN2 Batten disease and SOD1 ALS.
CAR T-Cell Therapy for Mantle Cell Lymphoma: Tycel Jovelle Phillips, MD
The clinical associate professor from the Rogel Cancer Center at Michigan Medicine discussed the future of CAR T-cell therapy in mantle cell lymphoma patients.
