CAR T-Cell Therapy gets Orphan Drug Designation for Acute Lymphoblastic Leukemia
The cell therapy is approved for B-cell non-Hodgkin lymphoma and B-ALL in China.
Identifying Future Patients for Tmod Cell Therapy in BASECAMP-1: Julian Molina, MD, PhD
The hematologist/oncologist from Mayo Clinic discussed the BASECAMP study and how it will inform future studies assessing Tmod technology.
American Gene Technology’s Cell Therapy Continues to Show Safety in HIV
An independent Data Safety and Monitoring Board has recommended that the phase 1 RePAIR trial continue at an accelerated pace.
Mesenchymal Stem Cells and Other Upcoming Treatments for Progressive MS: Jeffrey Cohen, MD
The director of the Mellen Center for MS Treatment and Research at Cleveland Clinic discussed upcoming new therapies for relapsing MS.
New Treatment Post-Tisagenlecleucel Being Developed for Patients With LBC Lymphoma
Two sites in California and Missouri are actively recruiting patients with r/r LBCL who were treated with tisagenlecleucel to receive efineptakin alfa in the study.
IND Accepted for Sickle Cell Disease Cell Therapy
Beam Therapeutics will assess the safety and efficacy of BEAM-101 for SCD in the phase 1/2 BEACON-101 study.
Bone Marrow-Derived Mesenchymal Stem Cells Safe in ALS
ALSFRS-R and FVC values were stable 3 months after treatment but significantly decreased 6 months after treatment, warranting further investigation.
Advantages of T-Cell Immunotherapy Over CAR T-Cells
AJ Joshi, MD, chief medical officer, Atara Biotherapeutics, discussed the advantages of tab-cel over other T-cell therapies.
CAR T: A Novel Approach for Mantle Cell Lymphoma
New treatment options expand management of relapsed or refractory disease.
Advantages of Concurrently Investigating Gene Therapies in Dogs and Humans
Dan Oliver, cofounder and chief executive officer, Rejuvenate Bio, discussed the advantages of developing gene therapies for both dogs and humans.
IND Accepted for HER2+ Gastric and Gastroesophageal Junction Cancer Cell Therapy
CYNK-101 is set to be evaluated in combination with standard chemotherapy, trastuzumab and pembrolizumab in a phase 1/2a study.
CAR T and Other Emerging Therapies in R/R Multiple Myeloma: Kenneth H. Shain, MD, PhD
The hematologist/oncologist at Moffitt Cancer Center discussed emerging therapies for patients with relapsed/refractory multiple myeloma.
Study Aims to Gain Insights into Genetics of Glaucoma
The study is aiming to gain insight into glaucoma in African American populations.
CRISPR/Cas9-Edited CAR and NK Cell Therapies Show Preclinical Efficacy
CRISPR Therapeutics presented positive data on 2 preclinical programs at the 2021 SITC meeting.
Utility of CAR T-Cell Therapy in Relapsed Multiple Myeloma: Thomas G. Martin, MD
The co-leader of Cancer Immunology & Immunotherapy Program, UCSF Helen Diller Family Comprehensive Cancer Center discussed he utility of CAR T-cell therapy in late relapsed multiple myeloma.
GeneTherapyLive’s Weekly Rewind – November 26, 2021
Review top news and interview highlights from the week ending November 26, 2021.
Developing Gene Therapies for Indications With Unmet Needs
The chief executive and scientific officers of Lysogene discussed Lysogene’s patient-focused therapy development.
CD19+ B-Cell Malignancy Cell Therapy Receives RMAT Designation
In 26 patients with LBCL followed for at least 28 days, the objective response rate (ORR) was 58% with complete responses (CRs) in 38% of patients.
Advantages of Developing Platform-Agnostic Gene Therapies
The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s ex-vivo and in-vivo gene therapy platforms.
Hemophilia A Gene Therapy Placed on Clinical Hold
Pfizer and Sangamo voluntarily paused the trial after participants experienced FVIII activity levels of over 150%.
Targeting the Putamen in AADC Deficiency: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed the benefits of delivering gene therapy directly to the putamen in AADC deficiency.
FasTCAR Cell Therapy Granted Orphan Drug Designation for Multiple Myeloma
The designation follows durable responses seen in a phase 1 study of the agent.
Overcoming Challenges With CAR T Therapies in Solid Tumors: Julian Molina, MD, PhD
The hematologist/oncologist from Mayo Clinic discussed the role of HLA loss in a variety of cancers.
FDA Accepts Beti-Cel BLA for β-thalassemia
The investigational gene therapy, which will receive priority review, has a PDUFA date of May 20, 2022.
Gene Therapy for Parkinson Disease Demonstrates Safety, Improved Motor Function
From baseline, all participants showed improvements on CGI-Improvement scale scores while all but 1 patient demonstrated improvement on modified Hoehn and Yahr scores.
Cilta-Cel is Well-Tolerated with Durable Efficacy in R/R Multiple Myeloma
The recommended phase 2 dose of cilta-cel elicited deep and durable responses, along with a tolerable safety profile.
CAR T-Cell Therapy's Continued Benefit in B-Cell Non-Hodgkin Lymphoma
Positive data has been seen with CAR T-cell therapy in patients with indolent lymphoma.
Gene Therapy Optogenetics Combination Efficacious in Retinitis Pigmentosa: José-Alain Sahel, MD
The professor from University of Pittsburgh School of Medicine discussed the combination of an optogenetics device and gene therapy in treatment of RP.
CAR T or Transplant in DLBC Lymphoma: John M. Burke, MD; Marin F. Xavier, MD
Experts discussed using newer targeted treatment approaches as frontline therapy instead of transplant in patients with relapsed/refractory diffuse large B-cell lymphoma.
Hemophilia A Gene Therapy Shows Sustained Factor VIII Expression
A 1-stage factor VIII assay revealed no apparent decrease in factor VIII activity over time.
