Latest Conference Coverage

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.

The associate professor and associate investigator of neurology at Harvard Medical School discussed research confirming proof-of-concept with EV-AAVs.

The chief executive officer of Tenaya Therapeutics discussed the growing interest in genomic medicines in cardiology.

Oncological trials of biologics also face particularly higher rates of clinical holds.

Lawrence R. Lustig, MD, discussed promising early results from the phase 1/2 CHORD trial evaluating Decibel Therapeutics and Regenerons’ DB-OTO.

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.

Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.

The assistant professor of pediatrics at Children's Hospital of Philadelphia discussed the role of the FOXO1 gene in T-cell persistence and exhaustion.

The chief executive officer of Tenaya Therapeutics discussed the company’s research on capsids, promoters, and manufacturing improvements.

The cofounder, chairman, and CEO of Kriya Therapeutics discussed the company’s goal of bringing gene therapy to a much broader population of patients.

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.

Updated data including a sixth patient and longer follow-up was presented at ASGCT’s 2024 Meeting.

The research trainee at Brigham and Women’s Hospital discussed developing RNA constructs to aid with RNA interference.

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.

All 139 tumors screened had enhanced expression of CCNG1, a seldom-recorded gene on NGS.

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.

The gene therapy is up for priority review with a PDUFA date of June 21, 2024.

In terms of safety, NGGT001 was deemed well-tolerated.

The medical director of clinical development at AskBio discussed safety and efficacy data from a phase 1 trial of AB-1002.

The DSMB recommended proceeding to dose the highest dose cohort in the phase 1/2 PRODYGY study.

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.

The associate investigator of neurology at Massachusetts General Hospital discussed research on extracellular vesicle-associated AAVs presented at ASGCT.

MELPIDA was well-tolerated in patients and nerve conduction was stable or improved after treatment.

The research scientist at Seattle Children's Research Institute discussed mouse model research he presented at ASGCT’s 2024 Meeting.

KL003 favorably compares to platelet and neutrophil recovery rates of approved gene therapies.

The senior research scientist at RTI International discussed how patient preference studies can help patients with rare diseases have their voices heard.

The professor of medicine at Baylor College of Medicine discussed research with NK-T cells and alternatives to αβ T-cells.

The pediatric neurologist at Nemours Children’s Health offered advice and discussed his experiences delivering pediatric gene therapy.