Polycystic kidney: 103 million for a one-time gene therapy
Californian biotech Estuary comes out of stealth with 103 million dollars to treat the most common inherited kidney disease, which has only one dedicated drug.
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One person in 400 to 1,000 is thought to have autosomal dominant polycystic kidney disease, the most common inherited kidney disease. Only one drug is approved for it: tolvaptan.
Estuary Biotherapeutics, a Californian biotech founded in 2022 by two Yale professors and a former Lilly executive, came out of stealth on Friday with 103 million dollars: a 93-million Series A led by RA Capital, plus 10 million in seed money. Biogen is an investor, as is GV, Alphabet’s fund.
One dose, two genes
Its two candidates are gene therapies designed to be given once, locally. EST-201 delivers a full copy of the PKD2 gene, with first human trials planned for 2027. EST-101 delivers part of the product of the PKD1 gene, for 2028.
The field is not empty. Novartis is buying Regulus for 800 million dollars, and Sanofi failed in a key study. Renasant Bio has also emerged with 55 million in seed money. Estuary promises a cure in one go. It still has to show it in humans. Is one treatment worth a lifetime of pills, and who will set the price of that one time?
Based on Endpoints News and BioPharma Dive