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

The funds will facilitate the clinical progression of ABO-101, the company’s lead therapeutic candidate targeting PH1.

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

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).

About Primary Hyperoxaluria Type 1 (PH1) PH1 is a potentially life-threatening progressive genetic disease characterized by the accumulation of oxalate in the kidney due to an enzyme deficiency in ...

Financing extends Arbor's cash runway into 2027 and supports the clinical development of lead program ABO-101 and continued advancement of a broader portfolio of CNS-targeted gene editing therapeutics ...

The firm will use the funds to develop its lead candidate in primary hyperoxaluria type 1 and other pipeline products in liver and CNS disorders.

Primary Hyperoxaluria (PH) is a rare autosomal recessive genetic disorder caused by enzyme deficiencies in hepatic oxalate metabolism, leading to excessive oxalate production and systemic oxalate ...

The YOLT-203 in-vivo gene editing therapy was developed to treat patients living with primary hyperoxaluria type 1 (PH1), a condition caused by a genetic deficiency in the liver enzyme alanine ...

Primary Hyperoxaluria (PH) is a rare autosomal recessive genetic disorder caused by enzyme deficiencies in hepatic oxalate metabolism, leading to excessive oxalate production and systemic oxalate ...

Tue, Feb 25, 2025, 3:15 AM 4 min read ...