Celyad Pauses CAR T Cell Therapy Trial of CYAD-101 Following Patient Deaths
The phase 1b KEYNOTE-B79 trial is investigating the allogeneic cell therapy in participants with metastatic colorectal cancer.
Improving Rare Disease Awareness: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed the importance of Rare Disease Day.
Gene Therapy Updates in Neuromuscular Diseases
Crystal Proud, MD, outlines advances in technology and research in neuromuscular diseases.
Ongoing Gene Therapy Trials in Stargardt Disease : Prof. Carel Hoyng
The professor of ophthalmology from Radboud University, The Netherlands, discussed ongoing gene therapy trials in Stargardt disease.
CGTLive’s Weekly Rewind – February 25, 2022
Review top news and interview highlights from the week ending February 25, 2022.
Advanced Ovarian Cancer Immunotherapy Trial Continues
An interim safety review of 81 patients informed the DSMB's recommendation for the trial to continue.
Launching PDGENEration to Advance Genetic Testing in Parkinson Disease: James Beck, PhD
The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the organization’s initiative to advance genetic testing.
Exploring Cell Therapy for the Treatment of Drug-Resistant Epilepsy
Cory Nicholas, PhD, cofounder and chief executive officer, Neurona Therapeutics, discussed the company’s lead program in mesial temporal lobe epilepsy.
R/R Multiple Myeloma CAR T-Cell Therapy Receives Orphan Drug Designation
CT103A is co-developed by IASO Biotherapeutics and Innovent Biologics.
EtranaDez’s Efficacy in Hemophilia B: Wolfgang Miesbach, MD, PhD
The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed new efficacy findings from the HOPE-B study.
Around the Helix: Cell and Gene Therapy Company Updates - February 23, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Managing Safety in Fitusiran Treatments of Hemophilia: Guy Young, MD
The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed mitigation strategies in trials and clinic.
Patient-Informed Study to Evaluate Hunter Syndrome Gene Therapy
The trial design was informed by both patient and physician perspectives.
Unmet Needs in Hemophilia: Steven W. Pipe, MD
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed the advantages of AAV gene therapies.
Enrollment Pauses in Mesothelioma CAR T-Cell Therapy Trial
The pause follows a fatal serious adverse event in the first patient treated in the highest dose cohort.
CAR T-Cell Therapy Demonstrates Feasibility in Resistant Prostate Cancer
The trial, sponsored by Mustang Bio, will continue to enroll patients and doses will be escalated to 300 million cells.
Second Phenylketonuria Gene Therapy Study Put on Clinical Hold
The pheNIX clinical hold follows an announcement that BioMarin’s PKU hold may last several quarters.
Liso-Cel Gets Priority Review for Second-Line R/R Large B-Cell Lymphoma
The new application is based on data from the phase 3 TRANSFORM trial.
Gene Therapy for Diabetic Retinopathy Yields Continued Improvements
The therapy has been well-tolerated in the 15 patients treated so far.
Assessing Safety of Fibroblast Cell Therapy in Multiple Sclerosis: Hamid Khoja, PhD
The chief scientific officer of FibroBiologics discussed the benefits of fibroblast cell technology in MS.
Stem Cell Model Developed to Study Albinism-Related Eye Conditions
Researchers will use the model to study how lack of pigmentation affects RPE physiology and function.
Adrenomyeloneuropathy Gene Therapy Granted Fast Track Review
The FDA cleared SBT-101's investigational new drug application earlier in February 2022.
Risk Alleles in Age-Related Macular Degeneration: Steffen Schmitz-Valckenberg, MD
The ophthalmologist from John A. Moran Eye Center discussed the close association between disease progression and uncovered risk alleles.
CGTLive’s Weekly Rewind – February 18, 2022
Review top news and interview highlights from the week ending February 18, 2022.
BioMarin’s PKU Gene Therapy On Hold Pending New Studies
The company also announced that the phase 3 study of val-rox has completed enrollment.
Targeting Unmet Needs in Rare Dermatologic Diseases With Gene Therapy
Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed FCX-013 for the potential treatment of scleroderma.
Memory T Cell CAR T Therapy Showcases Anti-Tumor Activity in Resistant Prostate Cancer
Interim data from the SPOTLIGHT study were presented at the ASCO GU Symposium.
Gene Therapy Shows Potential in Canavan Disease
Myrtelle’s therapy targets oligodendrocytes to deliver a functional copy of the ASPA gene.
Elucidating Associations Between EBV and MS: Jakob Dupont, MD
The global head of research and development at Atara Biotherapeutics discussed the link between EBV and MS.
Around the Helix: Cell and Gene Therapy Company Updates - February 16, 2022
