Axi-Cel Shows Efficacy in Patients With R/R LBCL Ineligible for ASCT
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.
Abeona's RDEB Cell Therapy Up for Review
Abeona has submitted a BLA for its EB-101 cell therapy.
Patients Receiving Autologous Stem Cell Transplant After Chemical Burns Show Corneal Improvements
The phase 1/2 trial is opening its second phase of recruitment after being well-tolerated in the first 4 patients and demonstrating some vision improvements.
Optic Atrophy Gene Therapy Cleared for Trials in Australia
NFS-05 is an ophthalmic injection that uses an AAV vector to deliver the OPA1 gene to retinal ganglion cells.
Mesoblast Gears Up for Third Push for Remestemcel-L's Approval in GvHD
The FDA issued a CRL for the therapy, to be marketed as Ryoncil, in August 2023.
Research Identifies Cell Signatures of CAR T-Cells in Long Remission
Cells from patients with ALL treated in the CARPALL study were analyzed and sequenced.
First Patient Dosed With LCA5 Gene Therapy
Opus Genetics plans to add a pediatric cohort to the phase 1/2 trial once safety is established in adults.
Anixa Makes Progress in CER-T Trial for Ovarian Cancer
The trial has dosed its third patient, and, safety validation pending, plans to dose a second cohort beginning at the end of 2023.
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.
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.
First Patient Receives Gene Therapy for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
Adstiladrin was approved in December 2022 and is now available for select patients under an Early Experience Program.
First Patient With MSLN-Solid Tumors Receives SynKIR-110 CAR T-Cell Therapy
SynKIR-110 is based off of the KIR-CAR platform developed at the University of Pennsylvania.
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.
Nusinersen Show Benefits in Other SMA Populations
New data shows the ASO therapy’s benefit in older populations, following beneficial findings in patients previously treated with gene therapy.
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.
Kyverna Makes Progress Evaluating Lupus Nephritis Cell Therapy in US, Germany
Phase 1/2 trials have been cleared in Germany and dosed first patients in the US.
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.
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.
Arrowhead Seeks Clinical Trials for ALS, Muscular Dystrophy RNAi Therapies
Pending approval, ARO-DUX4 will be evaluated in patients with facioscapulohumeral muscular dystrophy and ARO-SOD1 will be evaluated in patients with amyotrophic lateral sclerosis.
CAR-M/Pembrolizumab Trial Doses First Patient With HER2-Overexpressing Solid Tumors
Initial data from the combination substudy are expected in the second half of 2023.
Gene Therapy Improves Grade 2/3 Xerostomia Symptoms
Participants treated both unilaterally or bilaterally reported clinically meaningful improvements on GRCQ and XQ scale scores.
Gaucher Disease Type 1 Gene Therapy Trial Proceeds to Dose Second Cohort
The company announced that it had completed dosing in the first cohort of the GALILEO-1 trial of FLT201.
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.
No AdComm Meeting for Sickle Cell Therapy Lovo-Cel
Bluebird's gene-edited cell therapy has a PDUFA date of December 20, 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.
Innovative Targets Enable Immunotherapy for Sarcomas
Both Adaptimmune and Replay have placed their bets on targeting cancer testis antigens for synovial sarcoma and myxoid/round cell liposarcoma cell therapy.
MUC16-Targeted Bispecific T-Cell Engager Cleared for Trial in Ovarian Cancer, Other Tumors
The first participant was dosed with LBL-033 in April 2023 in Nanjing Lead Biolabs's ongoing trial in China.
Lifileucel's Phase 3 TILVANCE-301 Trial Kicks Off in Patients With Melanoma
The TIL therapy has a PDUFA date of November 25, 2023.
Gene Therapy Shows Evidence of Dose-Dependent Effects on GM1 Gangliosidosis Biomarkers
At dose level 2, 3 of 4 participants had increases of β-Gal activity to normal levels for up to 12 months.
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