Noah Stansfield

Ntrust-2 will assess NKX019 in systemic sclerosis, idiopathic inflammatory myopathy, and ANCA-associated vasculitis.

The phase 1/2 GALILEO-1 trial has completed enrollment and a phase 3 trial is planned for 2025.

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed unique difficulties of designing clinical trials for PD gene therapy candidates.

Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed trends in precision medicine for cardiovascular indications.

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

Cartesian also announced that the first patient has been dosed in a separate clinical trial evaluating the CAR-T therapy in systematic lupus erythematosus.

The placebo-controlled multidose portion of the SELECT-HD trial included 23 patients with Huntington Disease in total.

Various improvements were seen in 2 adult patients and 2 pediatric patients treated in separate clinical trials.

Faraz Ali, MBA, 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.

Notably, 1 patient treated in the trial died of respiratory failure, but it is not clear whether the death was related to LYL797.

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

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

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.

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.

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.

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.

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

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.

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

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

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

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.

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

There was a 98% (2.7% SD) mean reduction in monthly HAE attack rate through the patients’ most recent assessment.

The subgroup analysis also looked at safety outcomes.