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The professor from Cedars Sinai discussed further research being conducted in the field after lifileucel’s approval.

The CLARA trial has also completed enrollment of participants with corneal edema secondary to corneal endothelial disease.

The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed work the center is engaged in with cell and gene therapy.

With the company having recently announced that it intends to expand the clinical trial with a new cohort, CGTLive® has decided to take a closer look at this ongoing study.

AMT-130 has shown some evidence of dose-dependent clinical benefits in treated study participants.

Fractyl Health is conducting IND-enabling studies for Rejuva.

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

For the 3 patients who were treated at the higher dose, immunohistochemistry showed that a mean of 54% of muscle fibers were expressing microdystrophin.

The chief medical officer of Encoded Therapeutics overviews the company’s timeline and strategy for its gene regulation program for Dravet syndrome.

NRTX-1001 consists of human interneurons that provide long-term secretion of gamma-aminobutyric acid, an inhibitory neurotransmitter, which is expected to repair neural networks.

Review top news and interview highlights from the week ending June 21, 2024.

The company anticipates that it will be able to submit a BLA in late 2024 or early 2025.

KYV-101 racks up another potential indication in addition to its primary autoimmune investigations.

The gene therapy is now indicated for ambulatory patients aged 4 years and older, and has been granted accelerated approval for nonambulatory patients.

The data, from the first patients dosed in each trial, also continues to show safety.

The clinical professor of neurology and pediatrics at Keck School of Medicine of USC discussed her learning experience administering gene therapies for the first time.

Data from up to 5 and 6 years of follow-up were presented at the 2024 EHA Congress.

In observance of World Sickle Cell Day, CGTLive brought together a variety of expert insights on navigating the rapidly expanding landscape of care for this inherited blood disorder.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The Senior Director and Head of Technology Development at Epic Bio discussed potential applications of the new technology.

The case study was recently published in the Proceedings of the National Academy of Sciences.

An investigator noted challenging side effects related to immunosuppressant treatment.

The responses seen in the 9 patients included 3 complete responses (CRs), 2 very good partial responses, and 4 partial responses.

The medical director of clinical development at AskBio discussed the progress of an early clinical trial of AB-1002.

Treated participants have achieved some mild improvements on MDS-UPDRS Part III at 18 months posttreatment.

In terms of safety, SNK02 was characterized as “well-tolerated”.

The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed areas that the agency is targeting.

Erika Fullwood Augustine, MD, MS, 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.

Earlier and prophylactic interventions have enabled low rates and severity of AEs in recent years of administration.

On the other hand, enhanced lymphodepletion increased incidence and severity of CRS and infection.