The U.S. FDA issued a complete response letter (CRL) for the NDA to privately held Saol Therapeutics Inc.’s rare disease treatment, sodium dichloroacetate (SL-1009), for pyruvate dehydrogenase complex deficiency. The inhibitor of pyruvate dehydrogenase kinases is the only drug in development for treating the rare genetic disorder, according to Cortellis. There are no FDA-approved treatments for the disease.
Remedium Bio Inc. has entered into a multitarget research and development collaboration with Eli Lilly & Co. to advance gene therapies for type 2 diabetes and obesity using Remedium’s Prometheus dose-adjustable gene therapy platform.
Barth syndrome is a rare genetic disorder caused by mutations in the TAZ gene, which encodes an enzyme essential for remodeling cardiolipin, critical for mitochondrial function. A recent study published in Nature identified the enzyme ABHD18 as a candidate deacylase in the cardiolipin biosynthesis pathway and a potential therapeutic target for this syndrome.
A recent paper in the Journal of Clinical Medicine quantified some of the potential bodily ravages ahead for patients with hypoparathyroidism (hypoPT), specifically the loss of kidney function, with the risk of chronic kidney disease going up every year by 11%. A handful of drug developers continue to advance prospects in hypoPT, where Ascendis Pharma A/S’ hormone replacement therapy Yorvipath (palopegteriparatide) – the first and only treatment for adults with the rare endocrine disease – has set the bar for efficacy.
Congruence Therapeutics Inc. has closed a $32 million financing round to advance its pipeline of small-molecule correctors for diseases of protein misfolding.
Researchers from Mount Sinai Center for Translational Medicine and Pharmacology at Icahn School of Medicine at Mount Sinai and colleagues have developed a therapeutic humanized antibody that blocks the action of follicle-stimulating hormone (FSH), a pituitary hormone previously thought to only play a role in fertility.
Rallybio Corp. has received an equity milestone payment of $12.5 million from Recursion Pharmaceuticals Inc. triggered by the initiation of additional preclinical studies for REV-102, an investigational oral ENPP1 inhibitor.
Acute pancreatitis took center stage as Wall Street took heed of phase III data from Ionis Pharmaceuticals Inc. with olezarsen for severe hypertriglyceridemia (sHTG), while another player in the space, Arrowhead Pharmaceuticals Inc., signed a sizeable deal in a separate therapeutic area with Novartis AG.
Diabetic nephropathy (DN) is a complication of diabetes and a leading cause of end-stage renal disease globally, with a rate of about 40% in patients with diabetes and limited access to therapeutic options.
Editas Medicine Inc. has nominated a lead in vivo development candidate, EDIT-401, a potential one-time therapy designed to significantly reduce LDL cholesterol (LDL-C) levels. The in vivo gene editing medicine is designed to treat hyperlipidemia by directly editing the LDLR gene to increase LDLR protein expression and reduce LDL-C levels.
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