Targeting Leptomeningeal Brain Tumors With CAR T Therapy
Manuel Litchman, MD, the president, chief executive officer, and director of Mustang Bio, discussed upcoming research on MB-101 cell therapy.
The Need for New Therapies in DLBC Lymphoma: Gilles Salles, MD
The lymphoma service chief at Memorial Sloan Kettering Cancer Center discussed the need for new therapies to treat diffuse large B-cell lymphoma.
CAR T and Other Approaches for Patients with R/R DLBC Lymphoma
Loretta Nastoupil, MD, and John Burke, MD, discussed treating relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL).
Changes in Photoreceptor Morphology Quantifiable Following Gene Therapy
Short-term morphological rescue was seen after voretigene neparvovec-rzyl treatment.
Improving Gene and Cell Therapy Conditioning Regimens With JSP191
The executive vice president of research and development at Jasper Therapeutics discussed how JSP191 is designed to be better-tolerated than current conditioning regimens.
MRI Data Supports Gene Therapy for Sanfilippo Syndrome Type A
Abeona Therapeutics is seeking a path towards BLA filing based on the promising clinical data.
NT-501 Cell Therapy Slows Degeneration in Macular Telangiectasia
NT-501 previously received orphan drug and fast track status from the FDA and Neurotech will pursue approval in late 2022.
BioNTech's Next mRNA Vaccine: Malaria
The company plans to use the mRNA platform it harnessed for its COVID-19 vaccine to develop the vaccine for malaria.
CG0070: A Genetically Modified Immunotherapy for Non-Muscle Invasive Bladder Cancer
The first patient in CG Oncology's phase 2 study of CG0070 was dosed in December 2020.
CaMKII Enzyme: A New Target for Vision Preservation?
Investigators found that reactivating the CaMKII enzyme could protect against further vision loss.
Skysona Gene Therapy Approved in EU for Early CALD
Bluebird Bio plans to submit its biologics license application in the US by mid 2021.
Benefits of CAR T Therapy in Follicular Lymphoma: Julie Vose, MD
The professor of internal medicine in the Division of Oncology and Hematology at the University of Nebraska Medical Center discussed the benefits of CAR T-cell for patients with follicular lymphoma.
Advantages of Manufacturing Cell Therapies In-House
The chief technology and chief executive officer of Mustang Bio discussed the company’s integrated approach to drug development.
DLBCL Second-Line Therapy Recommendations
Experts discussed their recommendations for second-line treatment options for patients with DLBCL.
Unique Challenges With Gene Therapy in Duchenne Muscular Dystrophy
The co-founder, president, and chief executive officer of Solid Biosciences, whose own son has DMD, discussed challenges in developing gene therapies for DMD.
Tisagenlecleucel Efficacious, Durable in Relapsed/Refractory Follicular Lymphoma
Stephen J. Schuster, MD, discussed tisagenlecleucel's efficacy and safety over other available treatments for relapsed/refractory FL.
Exploring Advanced Approaches for Pain Relief
The chief executive officer and co-founder of Flexion Therapeutics discussed the company’s pain-focused pipeline, which includes a gene therapy that targets inflammation.
Valoctocogene Roxaparvovec’s Treatment Durability in Hemophilia A in Question
A drop in endogenous Factor VIII expression was observed from treatment to 5-year follow-up despite continued demonstration of efficacy.
Novel Therapies in Development for Dravet Syndrome
Joseph Sullivan, MD, and Elaine C. Wirrell, MD, discuss new developments for the treatment of Dravet syndrome beyond seizure control including gene editing and therapy.
Improving Efficiency in CAR T Cell Therapy Production
The chief technology officer of Mustang Bio discussed the company’s approach and future plans in CAR T production.
HER2-Specific CAR T Therapy Shows Safety and Efficacy in Pediatric Tumors
Encouraging results from the phase 1 BrainChild-01 trial were recently published.
ctDNA Changes May Predict CD19-Targeted CAR T-cell Therapy Response in LBCL
Heterogeneity in the cellular and molecular features of CAR T-cell products contributes to variation in efficacy and toxicity follow treatment with axicabtagene ciloleucel.
HuCART19 Efficacious and Durable in B-cell Acute Lymphoblastic Leukemia
huCART19 is designed to yield longer remission rates for pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia.
Ide-Cel Shows Durable Response in R/R Multiple Myeloma: Larry D. Anderson, Jr, MD, PhD
The hematologist/oncologist from the Harold C. Simmons Comprehensive Cancer Center discussed the updated results of the KarMMa trial in relapsed/refractory multiple myeloma.
First Patient With Retinitis Pigmentosa Dosed in Trial of MCO-010
MCO-010 is designed to deliver multi-characteristic opsin to retinal cells.
CD20-Targeted Gene Therapy
Manuel Litchman, MD, the president, chief executive officer, and director of Mustang Bio discussed the company’s lead and second program.
Orca-T for Graft-Versus-Host Disease: Mehrdad Abedi, MD
The professor of medicine at the UC Davis Comprehensive Cancer Center discussed Orca-T, a high precision cell therapy, for patients with acute and chronic graft-vs-host disease.
OpRegen Shows Sustained Efficacy in Treating Dry AMD
Three patients continue to show improvements in BCVA 3 years post-treatment.
Challenges in Conducting Research in DMD
The co-founder, president, and chief executive officer of Solid Biosciences, whose own son has DMD, discussed some challenges the company is facing.
Novel Therapies in Heavily Pretreated Multiple Myeloma: Omar Nadeem, MD
The physician from the Dana-Farber Cancer Institute discussed the uptake of novel therapies in heavily pretreated multiple myeloma.
