Steve Kanner, PhD, on Evaluating chRDNA Technology for ATTR and Familial Hypercholesterolemia
The chief scientific officer of Caribou Biosciences discussed results from preclinical research evaluating the gene editing approach.
FDA Recognizes Lomecel-B Cell Therapy for Alzheimer Disease Ahead of New Data
Longeveron will present updated data from the phase 2a CLEAR MIND study at AAIC 2024 at the end of July.
David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases
The chief medical officer at Creyon Bio overviewed the development of an allele-selective TNPO2 protein for a single patient with a de novo pathology.
Biosyngen’s CAR-T Therapy BRG01 Cleared for Pivotal Trial in EBV-Positive Nasopharyngeal Carcinoma in China
BRG01 is engineered to target the EBV antigen, which is frequently expressed on nasopharyngeal carcinoma tumor cells.
Around the Helix: Cell and Gene Therapy Company Updates – July 17, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
CBER To Conduct Interactive CGT Site Tours to Improve Communication, Share Best Practices
The FDA announced its START Pilot Program in October 2023 with a similar goal.
Manali Kamdar, MD, on The Importance of Bringing Liso-Cel to Earlier Lines of Lymphoma Treatment
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
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.
