2-year Data for Duchenne Muscular Dystrophy Gene Therapy Shows Durable Improvements
Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.
EMBARK Study Evaluates DMD Gene Therapy
Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.
Presymptomatic SMA Treatment With Zolgensma Associated With Normal Development
New data presented at MDA 2022 showcased positive motor and bulbar function data.
Assessing Lentiviral Cell Therapy for Cystinosis: Stephanie Cherqui, PhD
The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD
Improving Access to CAR T-Cell Therapies: Thomas G. Martin, MD
The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed potential effects of cilta-cel's approval.
Performing In-Depth Analysis of Lentiviral Cell Therapies: Luca Biasco, PhD
The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.
Cautious Optimism for GM1 Gangliosidosis Gene Therapy: David Weinstein, MD
The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.
Improving Rare Disease Awareness: Bruce Dezube, MD
The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.
A Positive Outlook for Rare Disease Research: Michael Singer, MD, PhD
The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.
Improving Rare Disease Awareness: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.
Advancing Treatments for Rare Diseases Requires a Team Approach
Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.
Developing Therapies for Rare Diseases: Kinnari Patel, PharmD
The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day.
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.
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.
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.
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.
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.
The Potential Impact of Gene and Cell Therapy in Cystinosis: Stephanie Cherqui, PhD
The associate professor from UC San Diego discussed the curative potential of gene therapy in cystinosis.
First In-Human Trial to Assess Krabbe Disease Gene Therapy
Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.
Gene Therapy Well-Tolerated in Late-Onset Pompe Disease
Interim safety data from the FORTIS study were presented at WORLDSympsoium.
Achieving Developmental Milestones With GM1 Gangliosidosis Gene Therapy: David Weinstein, MD
The senior vice president of clinical development at Passage Bio discussed new data from the IMAGINE-1 trial presented at WORLDSymposium.
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.
Around the Helix: Cell and Gene Therapy Company Updates - February 9, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Gene Therapies for Mucopolysaccharidosis Show Safety, Efficacy
Interim data from trials of RGX-121 and RGX-111 were presented at WORLDSymposium 2022.
Treating Solid Tumors With Personalized Immunotherapy: Julian Molina, MD, PhD
The hematologist/oncologist from Mayo Clinic discussed targeting HLA loss in solid tumors.
Study to Evaluate Gene-Edited Cell Therapy for MPSII
The LV-HSCGT therapy has demonstrated efficacy in preclinical studies in mouse models.
