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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
T-Cell Lymphoma CAR T Trial Placed on Hold
Only 1 patient has been dosed in the trial so far, which initiated in October 2021.
Gene Therapy for Friedreich Ataxia Cardiomyopathy Set to Move Forward
The FDA has approved LEXEO Therapeutics’ investigational new drug application.
Improving CAR T-Cell Therapies for Multiple Myeloma: Thomas G. Martin, MD
The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed CAR T-cell therapies and safety signals of cilta-cel.
NK Cell Therapy CYNK-101 Wins Orphan Drug Designation for HER2+ Gastric/GEJ Cancers
This designation follows a fast track designation and IND application approval for the agent.
Lysogene Gene Therapies Show Promise in GM1 Gangliosidosis and MPSIIIA
Updated data were presented on LYS-SAF302 and LYS-GM101 at WORLDSymposium.
The Need for Analysis in Lentiviral Cell Therapies: Luca Biasco, PhD
The director of research and development at AVROBIO discussed the analytical pipeline the company has developed for molecular follow-up of cell therapies.
Stem Cell-Derived Retinal Implants Demonstrate Long-Term Viability
Investigators did not find any clinical signs of rejection of the mismatched donor cells.
First In-Human Trial to Assess Krabbe Disease Gene Therapy
Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.
Achieving Remission in Chronic Lymphocytic Leukemia With CD4+ CAR T-Cells: Jan Joseph Melenhorst, PhD
The translational immunologist and research professor at Perelman School of Medicine, University of Pennsylvania, discussed persistence of CD4+ CAR T-cells in CLL.
Gene Therapy Well-Tolerated in Late-Onset Pompe Disease
Interim safety data from the FORTIS study were presented at WORLDSympsoium.
Latest Updates on Geographic Atrophy Gene Therapy FOCUS Trial: Nadia K. Waheed, MD
The associate professor from Tufts University School of Medicine discussed the latest updates on the FOCUS trial.
GeneTherapyLive’s Weekly Rewind – February 11, 2022
Review top news and interview highlights from the week ending February 11, 2022.
Molecular Analysis Elucidates Cell Therapy Destiny and Distribution
AVROBIO presented data on their analytical pipeline and exploratory studies of molecular follow-up from their lentiviral cell therapy products.
Gene Therapy Stabilizes Disease Progression in GM1 Gangliosidosis
Positive results from a phase 1/2 study of AXO-AAV-GM1 were presented at WORLDSymposium.
Fabry Disease Gene Therapy Shows Encouraging Effect on Cardiac End Points
The dual-mechanism of the therapy helps address both systemic and organ-specific deficits.
CNGB3 Achromatopsia Gene Therapy Improves Visual Sensitivity
Clinical development will be stopped on the CNGA3 achromatopsia program as no clinical improvements were seen.
