Immusoft Celebrates FDA's Orphan Drug Designation for ISP-002 in Treating MPS II

Immusoft Achieves FDA Orphan Drug Designation for ISP-002 in MPS II

On December 15, 2025, Immusoft, a California-based biotechnology company, made a significant announcement regarding its investigative therapy, ISP-002, now recognized with the Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). This designation pertains to ISP-002's application for treating mucopolysaccharidosis type II (MPS II), also famously known as Hunter syndrome, which is classified as a rare and life-threatening lysosomal storage disorder.

Understanding MPS II

MPS II is caused by a deficiency of the enzyme iduronate-2-sulfatase (IDS). This deficiency results in the buildup of glycosaminoglycans in various body tissues, leading to severe multi-system issues. Patients suffering from MPS II face a range of complications, including skeletal deformities, cardiopulmonary difficulties, and significantly shorter life expectancies. The existing treatments, predominantly enzyme replacement therapies, are burdensome. They require frequent infusions throughout the patient's life, leaving many needs unmet in terms of treatment persistence and disease management.

Innovative Approach of ISP-002

ISP-002 harnesses Immusoft's proprietary engineered B cell platform. This novel approach utilizes the patient's own B cells, reprogramming them to produce therapeutic enzymes persistently within the body. Through continuous enzyme generation, ISP-002 aims to revolutionize the treatment landscape in genetic disorders, navigating past the significant limitations of current therapeutic options and experimental gene therapies.

Commentary from Immusoft's CEO

Sean Ainsworth, Immusoft's CEO, expressed, "The FDA granting Orphan Drug Designation for ISP-002 marks an invaluable milestone for our MPS II venture, reinforcing the promise of our engineered B cell platform." He emphasized the company's dedication to developing sustainable therapies tailored to meet the long-standing requirements of those living with rare genetic afflictions.

Clinical Validation from MPS I Program

Significant headway in the realm of mucopolysaccharidosis type I (MPS I) laid a solid groundwork for this advancement into MPS II. Immusoft's lead therapy candidate, ISP-001, currently stands as the first engineered B cell treatment trialed in human subjects. At present, it is undergoing a Phase 1/2 clinical study. Initial results indicate a favorable safety profile, with successful re-administration of the therapy absent of lymphodepletion, immunosuppression, or the necessity for any pre-conditioning.

Expert Insights

Dr. Paul Harmatz, a leading clinician specializing in MPS and a critical investigator in the ISP-001 study, noted the potential impact of therapies capable of sustained endogenous enzyme synthesis, stating, "This could significantly alleviate the burdens of the disease and symbolize a promising leap forward for patients battling Hunter syndrome."

Support from CIRM

The development of Immusoft’s engineered B cell platform benefited from financial support by the California Institute for Regenerative Medicine (CIRM), an organization devoted to accelerating groundbreaking treatments for patients facing severe medical conditions. Lisa Kadyk, PhD, a CIRM Fellow, remarked, "CIRM is eager to be part of the innovative strides Immusoft is making in treating MPS I and II. Their approach has the potential to redefine treatment modalities for rare genetic conditions."

Future Steps for Immusoft

With the Orphan Drug Designation granted, Immusoft is geared to propel ISP-002 forward into clinical development, whilst also broadening its engineered B cell platform to address further lysosomal storage disorders and additional medical challenges. Immusoft is a fully owned subsidiary under Immusoft Corporation and is committed to producing pioneering therapies directed at rare illnesses through sustained delivery mechanisms from within patients’ own cells. Their unique platform, Immune System Programming (ISP™), enables profound modifications in B cells, urging them to yield gene-encoded therapeutic medicines, thus turning them into efficient protein therapeutic biofactories.

For further information about Immusoft and their pioneering work, please visit Immusoft's website.