N. Nora Bennani, MD, on Diving Deeper Into T-Cell Lymphomas
The assistant professor at Mayo Clinic School of Medicine shared her outlook and predictions on research with T-cell lymphomas.
Safe Handling Guidelines Needed for Gene Therapies in the Clinic
Current handling guidelines do not align between the laboratory and clinical settings.
Anjali Pradhan, MS, on Using Single Cell Sequencing for Gene Editing Quality Control
The chief product officer at Mission Bio discussed the company’s Genome Editing Solution.
Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic Leukodystrophy
The deputy director, clinical research, San Raffaele Telethon Institute for gene therapy, discussed long-term follow-up data of up to 12 years.
LCA2 Gene Therapy Shows Visual, Retinal Improvements at Low Doses
No serious adverse events or significant retinal atrophy occurred.
Brian Kim, MBA, on the Importance of Single Cell Analysis to Cell and Gene Therapy
The CEO of Mission Bio discussed the company’s Tapestri platform for single cell sequencing.
Ethical Framework Needed for Gene Therapy Clinical Trials
An ASGCT poster focused on special ethical considerations of gene therapy human research and how to address them.
Xandra Breakefield, PhD, on Trying New Approaches to AAV Therapy for Glioblastoma
The investigator of neurology at Mass General Research Institute discussed research from her grad student, Lisa Nieland, presented at the ASGCT 2024 meeting.
Nathan Yozwiak, PhD, on Researching AAV Gene Therapy Delivery to the Brain
The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed research from the center presented at ASGCT 2024.
Cabaletta Bio’s CAR-T CABA-201 Shows Safety for First Patients Dosed in Myositis and Lupus Trials
The data comes from the phase 1/2 RESET-Myositis clinical trial (NCT06154252) and the phase 1/2 RESET-SLE clinical trial (NCT06121297).
Janice Chen, PhD, on Novel Approaches to Gene Editing
The cofounder and chief technology officer of Mammoth Biosciences discussed the importance of diverse approaches to gene editing to address a variety of indications.
Manipulation of the FOXO1 Gene Holds Potential to Enhance CAR T-Cell Fitness
Evan Weber, PhD, an assistant professor of pediatrics at Children's Hospital of Philadelphia, discussed his work on the role of the FOXO1 gene in T-cell persistence and exhaustion.
Maria Escolar, MD, on the Importance of Newborn Screening for Treating Krabbe Disease
The chief medical officer of Forge Biologics discussed how expanded newborn screening practices may help the company’s gene therapy for Krabbe disease, FBX-101, reach more patients.
Leigh Ramos-Platt, MD, on Allowing Access and Ensuring Preparation for Gene Therapies
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC discussed her talk on preparing for gene therapy administration.
John Murphy, PhD, on Using Gene Editing to Tackle Primary Hyperoxaluria Type 1
The chief scientific officer of Arbor Biotechnologies discussed the company’s preclinical candidate ABO-101.
Erika Fullwood Augustine, MD, MS, on Optimizing End Points for Rare Disease Clinical Trials
The associate chief science officer of the Kennedy Krieger Institute discussed an aspect of clinical trial design highly relevant to gene therapy development for rare diseases.
Maria Escolar, MD, on Continued Positive Results With Gene Therapy and UCBT in Krabbe Disease
The chief medical officer of Forge Biologics discussed updated data from the REKLAIM clinical trial evaluating FBX-101.
End-to-End Thinking is Key to Overcoming Friction in Gene Therapy Development
Abhishek Gupta, BS, the senior vice president of genetic medicines at Syneos Health, discussed common setbacks in gene therapy trials and how to overcome them.
Casey Maguire, PhD, on Potential Advantages of Encapsulated AAVs
The associate professor and associate investigator of neurology at Harvard Medical School discussed research confirming proof-of-concept with EV-AAVs.
Faraz Ali, MBA, on Trends in Precision Medicine for Cardiovascular Disease
The chief executive officer of Tenaya Therapeutics discussed the growing interest in genomic medicines in cardiology.
Cell and Gene Therapy Trials Face Disproportionate Amount of Clinical Holds
Oncological trials of biologics also face particularly higher rates of clinical holds.
Otoferlin-Related Hearing Loss Gene Therapy May Open the Door to Gene Therapies for Other Types of Hearing Loss
Lawrence R. Lustig, MD, discussed promising early results from the phase 1/2 CHORD trial evaluating Decibel Therapeutics and Regenerons’ DB-OTO.
Faraz Ali, MBA, on Adding Gene Editing Programs to Tenaya’s Cardiovascular Disease Pipeline
The chief executive officer of Tenaya Therapeutics discussed the company’s decision to supplement the gene transfer programs in its pipeline with gene editing programs.
Considerations for Gene Therapy Logistics and Implementation
Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.
Evan Weber, PhD, on Improving CAR T-Cell Fitness
The assistant professor of pediatrics at Children's Hospital of Philadelphia discussed the role of the FOXO1 gene in T-cell persistence and exhaustion.
Faraz Ali, MBA, on Continued Innovation With Cardiovascular-Targeted AAV Gene Therapy
The chief executive officer of Tenaya Therapeutics discussed the company’s research on capsids, promoters, and manufacturing improvements.
Multicharacteristic Opsin Gene Therapy Improves BCVA, MLSDT in Retinitis Pigmentosa
High dose MCO-010 yielded statistically significant improvements in BCVA and MLSDT at week 52.
Shankar Ramaswamy, MD, on Bringing Gene Therapy to Common Diseases
The cofounder, chairman, and CEO of Kriya Therapeutics discussed the company’s goal of bringing gene therapy to a much broader population of patients.
Kevin Campbell, PhD, on the Challenges of Gene Therapy Approaches in Advanced Muscular Dystrophy
The Howard Hughes Investigator at the University of Iowa discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.
Orchard Therapeutics’ Arsa-Cel Continues to Show Safety in Late Juvenile MLD
Updated data including a sixth patient and longer follow-up was presented at ASGCT’s 2024 Meeting.
