Several clinical trials in 2025 are pushing the boundaries of gene and cell therapy, aiming to address a range of complex diseases. Below is an overview of five major trials that could provide significant breakthroughs for patients suffering from genetic disorders, cancer, and autoimmune conditions.
Intellia Therapeutics’ Gene-Editing Therapy for Hereditary Angioedema
Hereditary angioedema (HAE) is a rare genetic disorder that causes episodes of severe swelling, often in the face, hands, or airway, and affects about 1 in 50,000 people globally. Intellia Therapeutics is advancing its CRISPR/Cas9-based gene-editing therapy, NTLA-2002, in a phase 3 clinical trial, HAELO. The trial tests if a single-dose treatment can reduce HAE attacks and eliminate the need for long-term medications. Early results from phase 1/2 trials have shown promise, with significant reductions in attack frequency. Updated data from the ongoing studies are expected later this year.
EsoBiotec’s In Vivo CAR-T Therapy for Multiple Myeloma
Multiple myeloma is cancer of the plasma cells in bone marrow, affecting about 176,000 people worldwide. EsoBiotec is testing ESO-T01, an in vivo CAR-T therapy for relapsed/refractory multiple myeloma, in a phase 1 trial. The therapy involves reprogramming T-cells within the patient’s body using a lentiviral vector platform, eliminating the need for apheresis and lymphodepletion. ESO-T01 is administered as a single intravenous dose. Initial data from the trial are expected in late 2025.
Tenaya Therapeutics’ Gene Therapy for ARVC
Arrhythmogenic right ventricular cardiomyopathy (ARVC) is a genetic heart disease that causes the replacement of heart muscle with scar tissue, affecting about 1 in 1,000 to 5,000 individuals globally. Tenaya Therapeutics is advancing TN-401, an AAV-based gene therapy targeting the PKP2 gene in ARVC patients, in its phase 1b RIDGE-1 trial. The trial is testing the therapy’s safety and efficacy in correcting the underlying genetic defect. Early preclinical data has shown that the gene therapy can restore heart function and reduce scarring. Results from the trial are expected in 2025.
Adicet Bio’s T-Cell Therapy for Autoimmune Diseases
Autoimmune diseases, which occur when the immune system attacks the body’s tissues, affect more than 50 million people in the United States. Adicet Bio is testing ADI-100, an allogeneic gamma delta T-cell therapy, in a phase 1 trial for patients with lupus nephritis, a severe complication of lupus. The therapy uses engineered T-cells to modulate the immune response, potentially restoring balance to the immune system. The trial will also include patients with systemic lupus erythematosus and other rare autoimmune diseases. Initial results are expected in early 2025.
Sangamo Therapeutics’ Gene Therapy for Fabry Disease
Fabry disease is a rare genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A, leading to the accumulation of harmful substances in the body. It affects about 30,000 people globally, with males being more severely affected. Sangamo Therapeutics is advancing isaralgagene civaparvove (ST-920), an AAV-based gene therapy for Fabry disease, in clinical trials. The therapy aims to restore the missing enzyme with a single dose, improving kidney and heart function. Data from the trials are expected in the first half of 2025, with plans for FDA submission by mid-year.
These clinical trials represent key advancements in gene and cell therapy, targeting diseases that have long lacked effective treatments. Results from these studies will provide crucial information for determining the viability of new therapies and their potential to improve patient outcomes. The next few years will see major progress in treating these conditions, with significant benefits expected for the patients involved.
Created: Feb 1st, 2025
Citations:
CGTLive Staff. (2025, January 27). Five clinical trial readouts to watch in 2025. CGTLive. https://www.cgtlive.com/news/five-clinical-trial-readouts-to-watch-in-2025
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