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The company has begun enrolling participants in a new cohort for younger patients in the phase 1/2 AFFINITY DUCHENNE clinical trial.

Three participants with ATTR amyloidosis have received follow-on doses of NTLA-2001 with no serious complications for up to 3 years of follow-up.

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

The chief scientific officer of Precision for Medicine discussed unique difficulties of designing clinical trials for PD gene therapy candidates.

All 3 participants who have follow-up of at least 1 year have met the endpoint so far.

The company has dosed the sixth patient in the study, and expects to move onto the third cohort pending continued safety at 1 month posttreatment.

One patient is considered disease-free at 1 year posttreatment, but another patient’s disease relapsed at 5 months.

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.

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.