September 28th 2023
Around half of patients with R/R large B-cell lymphoma are considered ineligible for autologous stem cell transplant and are thus ineligible for the approved indication of Yescarta.
September 27th 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
September 22nd 2023
The FDA issued a CRL for the therapy, to be marketed as Ryoncil, in August 2023.
September 21st 2023
Cells from patients with ALL treated in the CARPALL study were analyzed and sequenced.
September 20th 2023
Artiva NK Cell Therapy AlloNK/ Affimed Innate Cell Engager Combo Gets Fast Track Designation
The companies plan to evaluate the combination therapy in patients with HL in the phase 2 LuminICE-203 clinical trial.
Hemogenyx Clears a Path for AML CAR-T Therapy
The FDA has accepted the company’s plan to address its current IND clinical hold on HEMO-CAR-T.
CGTLive’s Weekly Rewind – September 15, 2023
Review top news and interview highlights from the week ending September 15, 2023.
Addition of Quizartinib FLT3 Inhibitor to Cell Transplant Improves Survival in AML
Patients were newly diagnosed and had acute myeloid leukemia positive for FLT3-iTD mutations.
MRD Negativity Predicts DOR With Cilta-Cel in Patients With Multiple Myeloma
A final analysis of data from CARTITUDE-1 was also presented at SOHO 2023, which revealed a PFS of 24.9 months in treated patients.
Around the Helix: Cell and Gene Therapy Company Updates – September 13, 2023
Multi-Base–Edited CAR T-Cell Therapy Trial Doses First Patient With R/R T-ALL/T-LL
Beam Therapeutics also expects to announce initial data from its BEACON trial in sickle cell in 2024.
RNAi Therapeutic Shows Dose-Dependent Reductions in Systolic Blood Pressure
Zilebesiran is being evaluated as a monotherapy in the KARDIA-1 study and as a combination therapy in KARDIA-2, which will report topline data in early 2024.
Axi-Cel Expands LBCL Indication in China
The CAR T-cell therapy, marketed as Yikaida in China, has been approved for second-line treatment for patients with refractory/relapsed large B-cell lymphoma.
Around the Helix: Cell and Gene Therapy Company Updates – September 6, 2023
CGTLive’s Weekly Rewind – September 1, 2023
Review top news and interview highlights from the week ending September 1, 2023.
CRISPR-Cas9 Edited Therapy OTQ923 Displays Early Promise for Sickle Cell Disease
In a small sample of patients, the CRISPR–Cas9–edited CD34+ hematopoietic stem- and progenitor-cell therapy showed expected safety while inducing red cell fetal hemoglobin.
Abeona’s Sending Off RDEB Cell Therapy For BLA Submission
In recent data from the phase 3 VITAL trial, EB-101 decreased pain and improved wound healing.
Around the Helix: Cell and Gene Therapy Company Updates – August 30, 2023
FDA Approves Bristol Myers Squibb’s Reblozyl for Expanded Indication in Myelodysplastic Syndromes
The FDA’s decision was based on data from the randomized, open-label phase 3 COMMANDS clinical trial.
Poseida's Dual-Targeted Allogeneic CAR T Trial Cleared for B-Cell Malignancies
The company also recently entered into a collaboration with Roche and was invested in by Astellas.
CGTLive’s Weekly Rewind – August 25, 2023
Review top news and interview highlights from the week ending August 25, 2023.
BCMA-Directed CAR-T NXC-201 Gets FDA Orphan Drug Designation in Multiple Myeloma
Because of the lack of neurotoxicity seen in patients treated thus far, Nexcella believes NXC-201 may have potential to serve as an outpatient treatment.
Around the Helix: Cell and Gene Therapy Company Updates – August 23, 2023
Wugen’s NK Cell Therapy Trial Doses First Patient With AML
The company also reported that WU-NK-101 has received orphan drug designation for treating acute myeloid leukemia from the FDA.
Hemophilia Gene Therapy Shared Decision Making Tool Needed, Providers Say
Surveyed clinicians and nurses from Rady Children's Hospital San Diego identified important factors and considerations needed in a potential SMD tool.
CGTLive’s Weekly Rewind – August 18, 2023
Review top news and interview highlights from the week ending August 18, 2023.
No AdComm Meeting for Sickle Cell Therapy Lovo-Cel
Bluebird's gene-edited cell therapy has a PDUFA date of December 20, 2023.
Around the Helix: Cell and Gene Therapy Company Updates – August 16, 2023
MB-106 Shows Potential in Indolent Lymphoma
Mustang reported new data and responses from a multicenter phase 1 trial evaluating its CAR T-cell therapy in patients with NHL, FL, and WM.
FDA Lifts Partial Clinical Hold on Arcellx’s Trial for Multiple Myeloma CAR-T CART-ddBCMA
The partial clinical hold was originally placed in June 2023 in relation to the death of a patient participating in the trial.
FDA Places Formal Clinical Hold On 2seventy bio's Trial for Acute Myeloid Leukemia CAR-T In Relation to Patient Death
The formal hold comes approximately 2 months after Seattle Children’s paused the trial in accordance with the clinical study protocol stopping rules.
Kedar Kirtane, MD, on CAR T-Cell Agent A2B530 and Its Unique Approach to Solid Tumors
The assistant member in the Department of Head and Neck-Endocrine Oncology at Moffitt Cancer Center spoke about A2B530's mechanism of action and how it could represent a leap forward in the application of CAR T-cell agents for patients with solid tumors.
HaemaLogiX and Peter MacCallum Cancer Centre to Collaborate on Kappa-Type Multiple Myeloma CAR-T Trial
KMA.CAR-T is directed at Kappa Myeloma Antigen, a receptor exclusively appearing on myeloma cells in kappa-type MM.
Allogeneic CAR-T Shows Durable Responses in LBCL
The phase 2 ALPHA2 trial of ALLO-501A should complete enrollment in 2024.
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