CRISPR/Cas9 Therapy Shows Some Visual Improvements in LCA10
MT-ND4 LHON Gene Therapy Continues to Yield Vision Improvements
The safety profile of lenadogene nolparvovec at 5-years post treatment was similar to 2-year data.
LCA1 Gene Therapy Improves Sensitivity, BCVA at Highest Dose
No serious TEAEs have been related to ATSN-101 with data up to 6 months after treatment.
Cbl-b-Targeted Cell Therapy Controls Disease in 2 Patients With Solid Tumors
Overall, 6 patients were treated in a feasibility study for a disease control rate of 33.3%.
PD-L1 CAR T Therapy May Suppress the Gastric Cancer Tumor Microenvironment
Vax-CAR-T cells significantly suppressed tumor growth in mouse models in both smaller and larger tumors.
Afami-cel Prompts Antitumor Immune Response in Synovial Sarcoma, Myxoid/Round Cell Liposarcoma
Recent data supported the TCR therapy’s pending BLA submission.
Innate Cell Engager Therapy Shows Some Promise in R/R Peripheral T-Cell Lymphoma
Next steps may include assessing AFM13 in combination with natural killer cells.
Allogeneic CAR T Therapy Controls CD70+ Clear Cell Renal Cell Carcinoma
The data warrant further evaluation of ALLO-316 in patients with confirmed CD70+ tumors.
Gene-Agnostic Gene Therapy Shows Efficacy in NR2E3- and RHO- Associated Retinitis Pigmentosa
All treated eyes had stable or improved MLMT scores at 6 or 9 months of follow-up.
Here to Stay: Progress Solidifies the Future of Cell Therapy
CGTLive takes a look at recent progress in cell therapy development targeting solid tumors, featuring expert commentary on these advances.
Achieving Regeneration in Lumbar Degenerative Disc Disease
Matthew Gornet, MD, spine surgeon from The Orthopedic Center of St. Louis discussed data seen with IDCT cell therapy.
Cell Therapy Combination Shows Antitumor Response in Ewing Sarcoma
Vigil cell therapy previously showed some efficacy as a monotherapy in patients with ovarian cancer.
Allogeneic CAR T Fast Tracked for R/R Multiple Myeloma
The CaMMouflage trial dosed its first participant in March 2023.
Immatics’ Solid Tumor Cell Therapy Pipeline Evolves
Immatics is prioritizing its TCR-T and TCER cell therapy programs and leaving behind its first-generation programs.
Freeline Goes All in on Gaucher Disease
The company’s pipeline is down to 1 remaining clinical-stage program that is enrolling in its first-in-human trial.
Expanded Markets, Accelerated Approvals to Bring Gene Therapy into the Mainstream
Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, FDA, discussed his keynote address at the 2023 MDA Conference. c
Allogeneic Cell Therapy Shows Early Efficacy in Acute Myeloid Leukemia
Although the TCB-202-001 trial ended prematurely due to the COVID-19 pandemic, promising results warrant a phase 2 trial.
Astrocyte Cell Therapy Temporarily Slows ALS Decline
No treatment-emergent adverse events experienced were deemed related to AstroRx, although 9 serious TEAEs did occur.
HuidaGene’s LCA2 Gene Therapy Gets Orphan Drug Designation
The clinical trial of HG004 for RPE65-LCA2 dosed its first patient in February 2023.
Athersys Modifies Endpoints in Multistem Trial for Ischemic Stroke
The company also recently announced updates in the MATRICS-1 clinical trial for hemorrhagic trauma.
Multi-Characteristic Opsin Optogenetic Therapy Meets Phase 2 Endpoint in Retinitis Pigmentosa
Most patients with RP treated with MCO-010 had clinically meaningful improvements in vision.
First Patient With HPV16+ Solid Tumors in SQZ-AAC-HPV Trial Achieves CR
The Study Safety Committee has recommended that the trial proceed to enroll patients in the highest-dose cohort.
Lovo-cel BLA Submission Likely Delayed for Sickle Cell
The gene-edited cell therapy had previously been under a partial clinical hold restricting pediatric enrollment to the clinical trial.
RMAT Designation Granted to Hereditary Angioedema CRISPR Therapy
Intellia Therapeutics received clearance to enroll patients from the US in the global phase 2 portion of its clinical trial earlier this month.
Gene Therapy to be Assessed in Pediatric Patients With LCA, Retinitis Pigmentosa
OCU400 is also being evaluated in other retinal degeneration indications.
Ball in FDA’s Court for Lifileucel BLA in Advanced Melanoma
The FDA will determine the acceptability of the BLA within the next 60 days.
FDA Finally to Discuss NurOwn MSC-NTF Cell Therapy for ALS
The company's persistence in bringing NurOwn to the market amidst negative regulatory feedback has garnered an AdComm meeting.
Death Prompts CAR-NKT Cell Therapy Hold On Neuroblastoma Trial
The patient was treated at the new fifth dose level and had concomitant metapneumovirus infection.
CAP-1002 Yields Significant Upper Limb Improvements in Non-Ambulatory Patients With Later Stage DMD
Data from the HOPE-2-OLE were presented in a late-breaking session at the 2023 MDA Conference.
Zolgensma Yields 100% Bulbar Function Response in Presymptomatic SMA
All children included in the analysis achieved swallowing, oral nutrition, and airway protection outcomes.
