Kidney disease
Also lined up are target action dates for Sanofi’s Gaucher disease therapy, GSK’s lung cancer medicine and BridgeBio’s muscular dystrophy drug.
In this episode of Denatured, you’ll hear from Bruce Culleton, CEO at ProKidney and Demetri Maxim, CEO at Nephrogen. We discuss why kidney disease has long been a hard target for cell and gene therapies, and what new delivery strategies, therapeutic approaches and regulatory clarity mean for commercially viable products.
Estuary Biotherapeutics is seeking to address the root cause of autosomal dominant polycystic kidney disease with two gene therapy programs based on science from Yale.
A late-stage test of Roche and Ionis Pharmaceuticals’ sefaxersen showed that the drug reduced protein in the urine, which suggests the RNA-targeting therapy could improve kidney function and delay transplant.
While Vertex is laser-focused on an upcoming FDA decision for povetacicept in immunoglobulin A nephropathy, the APOL1 inhibitor inaxaplin has demonstrated potential “best-in-class” data in a different kidney disease.
Vera Therapeutics’ atacicept, to be marketed as Trutakna, will go up against Novartis, Otsuka and possibly Vertex in the kidney disease primary IgA nephropathy after receiving an accelerated FDA approval.
AstraZeneca and CSPC Pharmaceutical Group have already inked two other agreements this year, including an obesity-focused deal in January and one focused on chronic diseases in June.
Travere Therapeutics will gain an exclusive license to the oral BTK inhibitor civorebrutinib, which analysts at Guggenheim Securities said could be “complementary” to the biotech’s IgA nephropathy drug Filspari.
For an investment of up to $1.9 billion, Eli Lilly will be able to use Ascidian Therapeutics’ platform that removes mutated exons from mRNA molecules, avoiding the expression of disease-causing proteins.
The FDA has greenlit Travere Therapeutics’ Filspari as the only available treatment for focal segmental glomerulosclerosis despite the drug’s loss to Sanofi’s Avapro in a Phase 3 study.
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