Gene and Cell Therapies in Focus at ASGCT 2025: Tackling Rare Diseases, Autoimmune Conditions, and More

This year’s American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, held May 13–17 in New Orleans, is buzzing with new breakthroughs from the gene and cell therapy world. Biotech companies like Immusoft, Myrtelle, and Sail Biomedicines are presenting data that could reshape how we treat a range of tough-to-tackle diseases—from rare inherited disorders to autoimmune conditions. With new approaches like engineered B cells, tunable gene therapies, and RNA-based treatments, there’s a lot of momentum behind these next-gen innovations. Here’s a look at how these companies are aiming to change the game.

Innovative Therapies for Rare Genetic Disorders: Immusoft’s Pioneering B Cell Therapy for MPS I

Immusoft, a California-based cell therapy innovator, is presenting the world’s first clinical trial data for engineered B cells targeting mucopolysaccharidosis type I (MPS I), a rare genetic disorder caused by a deficiency in the enzyme iduronidase. This condition triggers a harmful buildup of substances, leading to severe developmental and physical challenges. At ASGCT 2025, Paul J. Orchard, MD, from the University of Minnesota, will share results from a first-in-human trial, highlighting the safety and early efficacy of autologous B cells genetically modified to produce iduronidase. This novel approach could transform therapeutic protein delivery and offers a promising path for MPS I patients.

Myrtelle’s Gene Therapy for Canavan Disease

In a related area of research, Myrtelle is working on a potential treatment for Canavan disease—a rare and fatal genetic disorder that appears in early childhood. The disease is caused by mutations in the ASPA gene, which prevents the brain from developing properly and disrupts the formation of myelin, the protective coating around nerve fibers. Without enough myelin, the nervous system cannot function correctly. Myrtelle’s experimental gene therapy, called rAAV-Olig001-ASPA, uses a specially designed viral vector to deliver a healthy version of the ASPA gene directly to brain cells, with the goal of restoring normal function. At ASGCT, the company will also highlight its participation in the FDA’s START Pilot Program, which offers faster and more flexible regulatory guidance for developing new treatments. CEO Adrian Stecyk emphasized how this program is helping move their therapy forward more quickly for families affected by this devastating condition. With Orphan Drug and Fast Track designations—intended to support treatments for serious and rare diseases—this approach could help address a significant gap in available options for Canavan disease.

Revolutionizing Autoimmune Disease Treatment—Sail Biomedicines’ In Vivo CAR-T Therapy

Sail Biomedicines is presenting preclinical data on SAIL-0804, a groundbreaking in vivo CAR-T therapy for autoimmune diseases. CAR-T therapy, traditionally used in cancer treatment, involves harvesting a patient’s T cells, genetically modifying them, and reintroducing them to target specific cells in the body. However, this process is complex, requiring cell harvesting and laboratory-based modifications. In contrast, SAIL-0804 uses targeted lipid nanoparticles to deliver circular RNA (eRNA) directly into the body, which temporarily reprograms T cells to deplete B cells. B cells are a type of immune cell that can contribute to autoimmune diseases, where the body’s immune system mistakenly attacks its own tissues.

In humanized mouse models, SAIL-0804 successfully achieved complete depletion of B cells across various sites, including the blood, spleen, lymph nodes, and bone marrow. B cells that repopulated after treatment displayed an immature phenotype, suggesting the immune system resets to a more balanced, less harmful state. This is important because it could potentially help in conditions where the immune system is overactive, such as lupus or rheumatoid arthritis.

SAIL-0804 simplifies treatment compared to traditional CAR-T therapies, avoiding complex cell-based procedures and lymphodepletion, and enabling outpatient use. This makes it a more accessible and cost-effective option for patients. Sail is now advancing SAIL-0804 into IND-enabling studies, the final step before clinical trials, which could bring this innovative therapy closer to helping patients with autoimmune diseases.

Changing the Game in Rare and Autoimmune Disease Treatments with Gene and Cell Therapies

The landscape of gene and cell therapies is rapidly evolving, with significant strides made in treating rare genetic disorders, autoimmune diseases, and neurological conditions. At the ASGCT 2025 Annual Meeting, companies like Immusoft, Myrtelle, and Sail Biomedicines are presenting groundbreaking approaches, such as engineered B cell therapies, tunable gene therapies, and RNA-based treatments. These innovations hold promise for transforming the treatment paradigms of challenging diseases.

The momentum in the field is reflected in recent industry developments. In 2024, the FDA approved several new gene and cell therapies, including Tecelra, the first T cell receptor gene therapy for synovial sarcoma, and afamitresgene autoleucel, a TCR gene therapy for solid tumors. Additionally, the gene therapy pipeline continues to expand, with over 500 therapies in development and expectations for 10–20 approvals annually by 2025. 

Looking ahead, the integration of artificial intelligence in gene therapy development is anticipated to enhance decision-making processes, potentially accelerating the approval of treatments for inherited disorders . As these therapies progress from the lab to clinical settings, they offer renewed hope for patients and families affected by previously untreatable conditions.

The advancements presented at ASGCT 2025 underscore a pivotal moment in the field of gene and cell therapies, signaling a future where personalized, effective treatments for complex diseases are increasingly within reach.

Author

Bernice Lottering