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

The round will fund a “one-and-done” treatment for a rare kidney condition that its CEO says can address concerns patients have with therapies from Alnylam and Novo Nordisk.

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

Primary hyperoxaluria type 1 (PH1) is a metabolic stone disease that, if left undiagnosed, can have devastating consequences, including progressive kidney decline and end-stage kidney disease (ESKD).

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 ...

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 ...

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 ...

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 ...

Still, Novo is steadfast in its goal to eventually become a leader in the space, the company’s EVP of rare disease, Ludovic ...