AVROBIO has announced the first patient has been dosed in the company’s Phase 1/2 trial of AVR-RD-04, an investigational gene therapy for cystinosis. Find the full news release here.
AVROBIO has also provided a patient-friendly summary of this news. Please see below.
Key facts About the Phase 1/2 Trial:
- Academic collaborators at University of California, San Diego are conducting this trial under the name CTNS-RD-04.
- It will enroll up to six people with cystinosis (4 adults in the first two cohorts and potentially a third cohort of two more adults or adolescents 14 years or older). Both males and females are eligible.
- The primary goal is to evaluate safety and tolerability, which will be measured for up to two years after treatment.
- Its secondary endpoints will measure efficacy through:
- Cystine levels in white blood cells.
- Cystine levels in the blood, intestinal mucous membrane, and skin.
- Cystine crystal counts in the eye and skin.
- Clinical tests of kidney function, vision, muscle strength, lung function, and neurological and psychological function.
- Assessments of participants’ quality of life after treatment.
- The trial is funded by grants to UC San Diego from the California Institute for Regenerative Medicine (CIRM) as well as the Cystinosis Research Foundation.
- To learn more about the trial, including full eligibility criteria, visit ClinicalTrials.Gov identifier NCT03897361.
News Release Summary
AVROBIO announced that the first patient has been dosed in the company’s AVR-RD-04 investigational gene therapy program for cystinosis. This ongoing Phase 1/2 clinical trial (CTNS-RD-04) is sponsored by academic collaborators at the University of California San Diego.
- The trial will enroll up to six patients who meet edibility criteria.
- The primary goal is to evaluate safety and tolerability of AVR-RD-04.
- Secondary endpoints will evaluate efficacy through measures of cystine levels, clinical tests of organ function, and assessments of quality of life after treatment.
- To learn more about this trial, including full eligibility criteria, visit ClinicalTrials.Gov identifier NCT03897361.
AVR-RD-04 is a lentiviral-based gene therapy designed to potentially stop the progression of cystinosis with a single dose of the patient’s own hematopoietic stem cells. The stem cells are modified so they can produce functional cystinosin with the aim of substantially reducing levels of cystine in cells throughout the body. The buildup of cysteine in the body causes the serious and potentially fatal damage to multiple organs and tissues in people with cystinosis. Learn more about AVROBIO’s technology at avrobio.com/technology.