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.
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.
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.
Debora Mazzetti, MS, on Multitargeting MicroRNA in Glioblastoma
The research trainee at Brigham and Women’s Hospital discussed developing RNA constructs to aid with RNA interference.
Abhishek Gupta, BS, on Addressing Friction Points in the Commercialization of Gene Therapies
The senior vice president of genetic medicines at Syneos Health discussed the importance of end-to-end thinking and collaboration when bringing gene therapies from development to the market.
Tumor Agnostic Deltarex-G+ Gene Therapy Demonstrates Activity in Breast, Pancreatic, Sarcoma Cancers
All 139 tumors screened had enhanced expression of CCNG1, a seldom-recorded gene on NGS.
Francesca Del Bufalo, MD, PhD, on Evaluating GD2-Directed CAR-T in Pediatric Neuroblastoma
The medical doctor and scientist at Bambino Gesù Chidren’s Hospital discussed phase 1/2 clinical trial data she presented at ASGCT’s 2024 Meeting.
Elevidys Shows Slight Statistical Improvements on Time to Rise, 10-Meter Walk/Run Despite NSAA Fail
The gene therapy is up for priority review with a PDUFA date of June 21, 2024.
NGGT Biotechnology’s Gene Therapy NGGT001 Improves Vision in Patients With Bietti’s Crystalline Dystrophy
In terms of safety, NGGT001 was deemed well-tolerated.
Luke Roberts, MBBS, PhD, on Early Clinical Data on Congestive Heart Failure Gene Therapy
The medical director of clinical development at AskBio discussed safety and efficacy data from a phase 1 trial of AB-1002.
Rod-Cone Dystrophy Gene Therapy Demonstrates Acceptable Safety, Study to Continue
The DSMB recommended proceeding to dose the highest dose cohort in the phase 1/2 PRODYGY study.
Lawrence R. Lustig, MD, on Evaluating Gene Therapy for the Treatment of Otoferlin-Related Hearing Loss
The chair of the Department of Otolaryngology—Head and Neck Surgery at Columbia University College of Physicians discussed the phase 1/2 CHORD clinical trial evaluating DB-OTO.
Casey Maguire, PhD, on Combining Viral-Like Particles and AAV
The associate investigator of neurology at Massachusetts General Hospital discussed research on extracellular vesicle-associated AAVs presented at ASGCT.
SPG50 Gene Therapy Warrants Further Study for Spastic Paraplegia
MELPIDA was well-tolerated in patients and nerve conduction was stable or improved after treatment.
Travis Drow, BS, on Evaluating Engineered Tregs for Potential in Treating Multiple Sclerosis
The research scientist at Seattle Children's Research Institute discussed mouse model research he presented at ASGCT’s 2024 Meeting.
TDT/SCD Lentiviral Therapy Boasts Quick, Well-Tolerated Engraftment in Patients
KL003 favorably compares to platelet and neutrophil recovery rates of approved gene therapies.
Advances in CRISPR/Cas9 Gene Editing Will Be Highly Represented in ASGCT Presentations This Year
Terence R. Flotte, MD, the vice president of ASGCT, discussed what attendees can expect at the ASGCT Annual Meeting this year.
