FDA Accepts Mesoblast’s BLA for Remestemcel-L for Steroid-Refractory GvHD
After 3 tries, Mesoblast finally has a PDUFA date for its MSC therapy.
Caribou Biosciences chRDNA Technology Provides a Unique Approach to CRISPR-Based Gene Editing
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences, discussed the company’s platform for genome editing.
Stephanie Tagliatela on Researching AAV for Lennox-Gastaut, Alzheimer Disease, SCN9a Pain
The cofounder and chief scientific officer at Encoded Therapeutics shared preclinical research with the company’s AAV and miRNA platforms.
Stanford Study Finds Low Risk of Secondary Blood Cancers After CAR-T
Of 724 patients treated at the center, the cumulative incidence of secondary hematologic malignancy at 3 years posttreatment was 6.5%.
BlueRock Therapeutics Assessing the Potential of Cell Therapy for Parkinson Disease With Phase 1 Trial for Bemdaneprocel
In honor of World Brain Day, observed annually on July 22 by patient and clinician communities, CGTLive is taking a closer look at this ongoing study.
Miloš Miljković, MD, on mRNA-CAR-T Descartes-08's Potential for Treating Myasthenia Gravis
The chief medical officer of Cartesian Therapeutics discussed data presented at ASGCT 2024 from a phase 2a study.
Holistic Approaches to Treating Parkinson Disease May Be More Effective
Paul Y. Song, MD, the chairman and chief executive officer of NKGen, discussed the potential of SNK01, the company’s autologous natural killer cell therapy, in treating PD.
Ixo-vec Yields Potentially Best-in-Class VEGF Injection Reduction for Wet AMD
The full 52-week analysis of the phase 2 LUNA trial are expected in the first half of 2025.
Passage Bio Gets Positive Feedback From FDA on Plans to Test Gene Therapy PBFT02 in New Frontotemporal Dementia Indication
Passage Bio received the feedback on a proposal to amend the upliFT-D protocol in the context of a Type C meeting process with the FDA.
CGTLive®’s Weekly Rewind – July 19, 2024
Review top news and interview highlights from the week ending July 19, 2024.
Sarcoma Awareness Month 2024: Looking Back at a Year of Progress for Cell and Gene Therapy
In observance of Sarcoma Awareness Month, held annually in July, we took a look back at the past year's news in cell/gene therapy for this cancer.
Manali Kamdar, MD, on Liso-Cel's Ongoing Benefit in the Treatment Lanscape for LBCL
The associate professor of medicine at University of Colorado discussed data presented at ASCO 2024.
4D Molecular Therapeutics’ Wet AMD Gene Therapy 4D-150 Reduces Need for antiVEGF Injections
The gene therapy has continued to demonstrate efficacy in updated data from the phase 2 PRISM trial.
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.
