First Patient to Receive Gene Therapy in a Phase 1/2 Study of ACTUS-101 in Patients with Pompe Disease

Actus Therapeutics, a privately held portfolio company of AskBio, has recently announced through PRWeb, the initiation of patient dosing in a clinical trial testing its investigational gene therapy ACTUS-101.

About Pompe Disease

Pompe disease, an inherited disorder, is caused by the buildup of glycogen (a complex sugar) in the body’s cells. When glycogen is accumulated in organs and tissues, particularly in muscles, it affects their normal function. The disease causes various symptoms, often resulting in premature death.

According to the National Institutes of Health, researchers have described three types of Pompe disease: infantile-onset (begins within a few months of birth), non-classic infantile-onset (appears before age one), and late-onset that may appear in childhood, adolescence, or adulthood and is caused by a deficiency of acid-alpha-glucosidase (GAA).

Sheila Mikhail, J.D., the CEO and Co-Founder of AskBio calls it an “unforgiving disease” that is showing up in an increasing number of patients.

About the ACTUS-101 Study

ACTUS-101 was recently granted Fast Track designation for the treatment of patients with Pompe disease. Recruitment began in November 2018 for the Phase 1/2 clinical study.

The trial has been designed to evaluate the safety, efficacy and immune responses after administering one infusion of ACTUS-101, which is designed as a gene therapy for the disease, that “converts” the liver, allowing the continuous production of acid-alpha-glucosidase (GAA).

Mikhail suggests that “ACTUS-101 could possibly replace enzyme replacement therapy (ERT).” ERT is a useful method of treatment for Pompe disease.  However, some patients have experienced persistent muscle weakness, while other patients eventually produce antibodies that stop the treatment from working. Currently there is no therapy available to counteract this condition.

The study participants will continue their ERT with alglucosidase alfa while receiving ACTUS-101 during this open-label trial. Then, as the trial continues, the participants who meet the criteria for safety, transgene expression, and motor function will be able to suspend their ERT treatment.

About Ask Bio

Asklepios BioPharmaceutical, Inc. (AskBio),is a company focused on gene therapies and is specializing in rare genetic disorders. The company is known for its unique approach for the manufacture of cell lines. A follow-on program to the Actus-101 trial is in late-stage preclinical development.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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