Trial Cleared to Start Dosing Pediatric Patients With Autism Due to SHANK3 Haploinsufficiency
Patient enrollment is anticipated to begin in the first quarter of 2025.
Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
The senior researcher at Seattle Children’s discussed the development and validation of A2-CAR-CISC EngTreg cells.
Dopaminergic Progenitor Cell Therapy Shows Some Symptom Improvement in Parkinson Disease
Data from the high-dose cohort of a phase 1/2 clinical trial in South Korea will be shared in Fall 2024.
Gene Therapy Improves Some Disease Biomarkers of Friedreich Ataxia Cardiomyopathy
Despite the improvements, Lexeo was unable to discern a benefit on cardiovascular fitness and Peak VO2 from LX2006 with current measurements.
Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart Failure
The medical director of clinical development at AskBio discussed setbacks in the phase 1 trial of AB-1002 gene therapy.
RPE Cell Therapy Shows Improvements in Early GA With AMD
RG6501/OpRegen was well-tolerated, with no serious treatment-related AEs reported in follow-up data from a phase 1/2 trial.
Steve Kanner, PhD, on Caribou’s chRDNA Technology
The chief scientific officer of Caribou Biosciences discussed the company’s platform for genome editing.
Genetic Forms of Parkinson Disease May Hold Most Potential for Targeted Treatment
Ignacio Mata, PhD, an associate professor of neurology at the Cleveland Clinic Lerner Institute, pointed out that genetic forms of PD are relatively well-understood compared to other types.
Interius BioTherapeutics In Vivo CAR Therapy INT2104 Cleared for Phase 1 Clinical Trial in B-cell Malignancies Australia
In light of the CTN clearance, Interius expects to initiate plans for a phase 1 clinical trial (INVISE) for INT2104 within the final quarter of 2024.
Low-Dose Gene Therapy Well-Tolerated, Shows Some Trends of Improvement for Cystic Fibrosis
1x1015 vg 4D-710 has been identified as the maximum tolerated dose in the AEROW trial.
CGTLive®’s Weekly Rewind – July 12, 2024
Review top news and interview highlights from the week ending July 12, 2024.
Second-Generation NK Cell Engager Heading to Trial for CD33+ Leukemia
GT Biopharma anticipates initiating the phase 1 clinical trial in the second half of 2024.
Paul Y. Song, MD, on Bringing SNK01 to Clinical Trials for Parkinson Disease
The chairman and chief executive officer of NKGen discussed the new clinical study design for evaluating the NK cell therapy in PD.
Tr1X Doses First Patient in Trial Evaluating Allogeneic Treg Therapy TRX103 for GvHD Prevention
Tr1X reported that the first patient has cleared the trial’s safety period successfully, having experienced no serious adverse events.
Lisa Nieland on Slowing Tumor Growth in Glioblastoma With Novel AAV Therapy
The PhD candidate from Lieden University Medical Center and Breakefield Lab at Mass Gen discussed her work presented at the ASGCT 2024 meeting.
First Patient in US Trial Receives Nexcella’s Light Chain Amyloidosis CAR-T NXC-201
The open-label, single-arm, multisite NEXICART-2 study will aim to enroll around 40 participants in total.
Manali Kamdar, MD, on Acclimating to Routine CAR T Practice in the Field
The associate professor of medicine at University of Colorado discussed how the field has shifted to support the growing use of CAR Ts and progress to come.
BrainStorm’s New Trial May Support ALS Cell Therapy BLA
The BCT-006-US trial is set to begin dosing this year.
Around the Helix: Cell and Gene Therapy Company Updates – July 10, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
High-Dose Gene Therapy Slows Huntington Disease Progression
Treated patients had a –0.2 mean change in cUHDRS compared with a –1.0 change in external control data.
Paul Y. Song, MD, on Using Autologous Natural Killer Cells to Treat Parkinson Disease
The chairman and chief executive officer of NKGen discussed the mechanism behind the company’s NK cell therapy SNK01 and promising early results in patients with PD.
Jennifer Taylor-Cousar, MD, MSCS, on Early Signals of Efficacy With Cystic Fibrosis Gene Therapy
The professor of internal medicine and pediatrics at National Jewish Health discussed updated data from the phase 1/2 AEROW trial of 4D-710.
Nkarta Launches Trial for CAR-NK Therapy NKX019 in Lupus and Gains Clearance for Separate Trial in Other Autoimmune Diseases
Ntrust-2 will assess NKX019 in systemic sclerosis, idiopathic inflammatory myopathy, and ANCA-associated vasculitis.
Data Roundup: June 2024 Features Updates from ASCO and in Diabetes, Rare Disease, and more.
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC also discussed current strategies with gene therapy administration.
Spur Therapeutics Continues to Make Progress in Evaluation of Gaucher Disease Gene Therapy FLT201
The phase 1/2 GALILEO-1 trial has completed enrollment and a phase 3 trial is planned for 2025.
SPEARHEAD-1 Supports Potential TCR-T Approval for Synovial Sarcoma
The Prescription Drug User Fee Act (PDUFA) target action date is August 4, 2024.
Designing Clinical Trials for Parkinson Disease Gene Therapy Presents Substantial Challenges
Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed unique difficulties of designing clinical trials for PD gene therapy candidates.
Paul Y. Song, MD, on Taking a Holistic Approach to Treating Parkinson Disease
The chairman and chief executive officer of NKGen discussed the potential of SNK01, the company’s autologous natural killer cell therapy, in treating PD.
Interest in Genomic Medicine Research for Heart Disease Continues to Grow
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed trends in precision medicine for cardiovascular indications.
