Brexu-Cel Shows Efficacy in Mantle Cell Lymphoma in Real-World Setting
High response rates were seen in patients that would not meet ZUMA-2 eligibility criteria.
CGTLive’s Weekly Rewind – June 3, 2022
Review top news and interview highlights from the week ending June 3, 2022.
Allogeneic CAR T-Cell Therapy Well-Tolerated and Effective Across B-Cell Malignancies
A recent meta-analysis reviewed data across 146 patients in 9 clinical trials.
CAR T-Cell Therapy Yields High Response Rates in B-NHL
Relma-cel was previously approved in China in September 2021 for the treatment of B-cell non-Hodgkin lymphoma.
ICER Finds Beti-Cel Superior to Standard-of-Care for β-Thalassemia
Beti-cel was rated a B+ for lingering questions about durability and unknown risks.
NK Cell Therapy Avelumab Combo Well Tolerated With Some Clinical Activity in Sarcoma
A phase 1 trial evaluated the combination in patients regardless of PD-L1 status and no differences were seen between those negative or positive.
Around the Helix: Cell and Gene Therapy Company Updates – June 1, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Val-Rox BLA for Hemophilia A Delayed Again
BioMarin now expects to file in September 2022 instead of June.
Liso-Cel Yields High Response Rates and Positive PROs in LBCL
Treated patients had significant improvements in fatigue and lymphoma symptoms.
Lifileucel Shows Promising Data in Advanced Melanoma
A lower objective response rate in cohort 4 reflected a higher disease burden with a greater number of tumors.
Lentiviral Cell Therapy Normalizes Hemoglobin in Pyruvate Kinase Deficiency
RP-L301 also improved hemolysis and eliminated the need for red blood cell transfusions for up to 1year after therapy.
Developing Safer Viral Vectors for Gene Therapy
The chief executive officer of Ring Therapeutics, Tuyen Ong, MD, discussed the potential of anellovirus vectors for use in gene therapy.
Around the Helix: Cell and Gene Therapy Company Updates – May 25, 2022
Gene Therapy May Eliminate Need for ERT in Late-Onset Pompe Disease
All study participants met criteria to remain off enzyme replacement therapy through 104 weeks after treatment.
Gene Therapy for Knee Cartilage Lesions Receives RMAT Designation
NeoCart was originally developed by the company Histogenics.
EtranaDez Accepted for Priority Review in Hemophilia B
A final analysis of the pivotal HOPE-B study was presented at ASGCT 2022.
Cell Therapy Modulates Tumor Microenvironment in Glioblastoma
The cell therapy was tolerated although there was a SUSAR of GGT elevations.
Gene Therapy for AADC Deficiency Receives Positive CHMP Opinion
The positive opinion was based on findings from studies conducted in Taiwan.
DMD Gene Therapy Shows Continued Microdystrophin Expression
Motor and pulmonary function have stabilized or improved in participants treated with SGT-001.
Homology-Directed Repair-Developed TCR Therapy Shows Potential in Type 1 Diabetes
Investigators from Seattle Children’s Hospital found that engineered T-cell regulatory products demonstrated significant suppression of effector T cells.
Analysis Elucidates Lentiviral Vector-Mediated Myelodysplastic Syndrome Cases
Multiple insertions were observed in affected participants, including insertions in the MECOM and PRDM16 genes.
Adenovirus/Chemotherapy Combination Well-Tolerated, Surprising OS Benefit Observed in mPC
One patient with metastatic pancreatic cancer remains alive on maintenance chemotherapy 3 years after treatment.
Autoantigen CAR T Cell Therapy Well-Tolerated in Mucosal-Dominant Pemphigus Vulgaris
Investigators found a dose-dependent increase in CAART cell persistence.
At-Risk Genotypes for DMD Gene Therapy Identified
Trial sponsors Pfizer, Sarepta, Genethon, and Solid Biosciences organized a group of experts to investigate shared serious AEs.
X-Linked Retinitis Pigmentosa Gene Therapy Improves BCVA
Interim data from the phase 2 Skyline trial demonstrated a 62.5% response rate.
Third Generation CAR T Product Shows Efficacy in Follicular Lymphoma
Further data from Mustang Bio’s phase 1/2 trial will be presented at the EHA 2022 Congress.
GD2-CAR T-Cell Therapy Yields Clinical Benefit in Glioma
While the lower IV dose was generally well-tolerated, the higher dose yielded cases of serious cytokine release syndrome.
Around the Helix: Cell and Gene Therapy Company Updates – April 13, 2022
Allogeneic CAR T-Cell Therapy Shows Promise in R/R B-Cell Acute Lymphoblastic Leukemia
The phase 1, investigator-initiated trial continues to recruit participants with B-ALL.
Cord Blood Combined Natural Killer Cell Engager Therapy Shows Efficacy in Lymphoma
AFM13 is currently also being evaluated on its own in lymphoma in a phase 2 registrational study.