Cystinosis

May 21, 2025

Lysosomal Storage Disorders, or LSDs, are rare genetic conditions that may not make headlines, but they affect thousands worldwide, often with life-changing consequences. LSDs happen when the body’s lysosomes, tiny cellular recycling centers, fail to break down waste materials due to missing or faulty enzymes. This...

Nov 26, 2021

Cystinosis is a genetic condition that causes the buildup of the amino acid "cystine" in the cells of the body is known as cystinosis. Due to the excessive accumulation of cystine in various cells, organs, tissues, including the kidneys, eyes, liver, thyroid, muscles, pancreas, brain, and white blood cells of the b...

May 07, 2020

Avrobio taps ADC of Magenta for enhancing gene therapy conditioning Avrobio is working to make conditioning safer. The company is collaborating with Magenta Therapeutics to check antibody-drug conjugate. Under the agreement, the partners will evaluate Magenta’s lead conditioning program, MGTA-117, with at lea...