The round will fund a “one-and-done” treatment for a rare kidney condition that its CEO says can address concerns patients ...

The rate of PH1 progression is unpredictable, with kidney function potentially declining suddenly due to dehydration from acute illness or any other causes. As the kidneys become unable to excrete ...

As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead ...

The gene therapy world is in turmoil, but Arbor, armed with more than a billion dollars in partnerships and raises, is going forward.

Patients with PH1 produce far too much oxalate, a substance consumed in food and produced by the body. This combines with calcium to cause kidney stones and deposits in the kidney, which can lead ...

PH1 is a potentially life-threatening progressive genetic disease characterized by the accumulation of oxalate in the kidney due to an enzyme deficiency in the liver. Calcium oxalate crystals are ...

PH1, the most common subtype, is triggered by AGXT gene mutations and typically manifests in childhood. Patients often present with kidney stones, nephrocalcinosis, renal insufficiency ...

The condition typically manifests in childhood with symptoms such as kidney stones and blood in urine ... levels through to week 16, a marker of PH1. At the same time, there were no reported ...

YolTech Therapeutics, a clinical-stage in vivo gene editing company committed to pioneering the next generation of precision genetic medicines, reported positive results from the ongoing ...