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.
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.
