Review top news and interview highlights from the week ending May 17, 2024.

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

The invitation-only annual scientific summit, held on March 29, in New York City, brought together top scientists in cancer cell and gene therapy.

Verismo intends to go forward with plans for the phase 1 CELESTIAL-301 clinical trial, which the company expects to initiate in the second half of this year.

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 CAR T-cell therapy was also recently approved for treating chronic lymphocytic leukemia or small lymphocytic lymphoma.

Eladocagene exuparvovec is approved in the United Kingdom and European Union under the name Upstaza.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and 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 invitation-only annual scientific summit, held on March 29, in New York City, brought together top scientists in cancer cell and gene therapy.

VERVE-102 is intended to reduce LDL-C in patients with heterozygous familial hypercholesterolemia or premature coronary artery disease.

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

High dose MCO-010 yielded statistically significant improvements in BCVA and MLSDT at week 52.

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 trial is set to dose its second cohort following a positive DSMB recommendation in March.

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.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

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.

Review top news and interview highlights from the week ending May 10, 2024.

In observance of World Lupus Day, held annually on May 10, we took a look back at a year of progress in bringing CAR-T to this autoimmune disease.