Achieving Deep Responses With CART-ddBCMA in Relapsed/Refractory Multiple Myeloma
Matthew J. Frigault, MD, discussed safety and efficacy findings on CART-ddBCMA.
Long-Term RELIANCE Trial Data Shows Durability With Relma-Cel in LBCL
At a median follow-up of 24 months, the best ORR achieved was 77.6% among 58 evaluable patients with large B-cell lymphoma treated with relma-cel.
Resurrecting ALS Mesenchymal Cell Therapy for Regulatory Approval
Merit Cudkowicz, MD, discussed corrected data from a phase 3 trial of NurOwn that BrainStorm believes supports a BLA submission.
Verismo Looks to Assess KIR-CAR Cell Therapy in Solid Tumors
If approved, the company will initiate the first-in-human STAR-101 phase 1 clinical trial.
Irene Agodoa, MD, on Assessing Orca-T for Safer Cell Therapy in Hematologic Malignancies
The senior vice president and head of medical affairs at Orca Bio discussed the initiation of the Precision-T phase 3 trial.
Dystrophic Epidermolysis Bullosa Gene Therapy BLA Accepted With Priority Review
The BLA was supported by data from 2 intrapatient, placebo-controlled clinical trials.
CGTLive’s Weekly Rewind – August 19, 2022
Review top news and interview highlights from the week ending August 19, 2022.
Genprex Patents REQORSA Immunogene Therapy for NSCLC
The company recently announced a dose escalation in the ACCLAIM-1 study.
Alexis Thompson, MD, MPH, on Beti-Cel's Approval in β-Thalassemia
The primary investigator and chief of hematology at Children’s Hospital of Philadelphia discussed beti-cel's approval.
Homology Trades in Gene Delivery for Gene Editing to Treat Phenylketonuria
The pheEDIT clinical trial initiated in June 2022.
Around the Helix: Cell and Gene Therapy Company Updates – August 17, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
FDA Approves Beti-Cel Gene Therapy for β-Thalassemia
ZYNTEGLO is now the world's most expensive gene therapy at a $2.8 million price tag.
First Patient With Classical Hodgkin Lymphoma Dosed With CAR T/Nivolumab Combo
Tessa Therapeutics presented positive data from the phase 2 CHARIOT study of TT11 in December 2021.
Regen Files for Patent on CAR T-Cell Exhaustion Prevention Technology
Multiple cell and gene therapy companies have been granted patents for novel technology in 2022.
ProQR Pivots From Ophthalmic to CNS, Liver RNA Therapies
The move comes after the EMA recommended an additional clinical trial of sepofarsen before applying for marketing authorization.
Merit Cudkowicz, MD, on Evaluating Mesenchymal Cell Therapy for ALS
BrainStorm Cell Therapeutics has decided to submit a BLA for NurOwn in ALS following an erratum to a phase 3 trial.
Genprex to Evaluate Higher Dose of REQORSA in NSCLC
A safety review committee approved the dose-escalation after reviewing the first cohort of patients.
Pompe Disease mRNA Therapy Receives FDA Orphan Drug Designation
Preclinical data showed a reduction of Gys1 mRNA and protein of more than 80% in multiple skeletal tissues.
Bristol Myers to Fund Inflammatory Bowel Disease Treg Therapies
GentiBio will use their proprietary regulatory T cell platform to develop the engineered therapies against multiple targets.
Shannon L. Maude, MD, PhD, on Favorable Safety of Tisagenlecleucel in Pediatric B-ALL
The oncologist from Children's Hospital of Philadelphia discussed long-term safety data with tisagenlecleucel in pediatric patients with acute lymphoblastic leukemia.
CGTLive’s Weekly Rewind – August 12, 2022
Review top news and interview highlights from the week ending August 12, 2022.
Zolgensma Gene Therapy Linked to 2 Deaths in SMA Patients, Novartis Reports
The 2 deaths, due to acute liver failure, occurred in patients treated in Kazakhstan and Russia.
ALS Cell Therapy Receives US Patent
AstroRx previously demonstrated clinically meaningful reductions in the rate of disease progression.
Frontotemporal Dementia Gene Therapy Trial Initiates Dosing
PBFT02, developed by Passage Bio, is delivered via intra cisterna magna administration to the brain.
CRISPR Therapeutic Ready to Dose in Duchenne Muscular Dystrophy
A single patient will be dosed with CRD-THM-001 in an upcoming trial.
NK Cell Therapy Trial Doses First Patient With Lymphoma
The FDA recently accepted the company’s BLA for omidubicel for priority review.
Around the Helix: Cell and Gene Therapy Company Updates – August 10, 2022
Ide-Cel Shows Benefit in Earlier Use in R/R Multiple Myeloma
A recent comparison of health-related quality of life scores also favored ide-cel over belantamab mafodotin treatment.
IND Cleared for Non-Hodgkin Lymphoma T-Cell Therapy
Marker will initiate a company-sponsored study after an initial trial at Baylor College of Medicine showed positive safety data.
Monitoring and Support for Patients Receiving CAR T-Cell Therapy
Experts discuss providing supportive care to patients receiving CAR T-cell therapy.
