Cardior plans to build on early clinical data for its antisense oligonucleotide in heart failure patients by moving the RNA therapy into niche indications where it believes the product can help dysfunctional...

DISEASE CATEGORY: Musculoskeletal; ophthalmic disease INDICATION: Myotonic dystrophy type 1 (DM1); ophthalmic A Scripps Research team led by Expansion Therapeutics Inc. co-founder Matthew Disney has designed an RNA-targeting small molecule that could treat DM1 and Fuchs...

This year’s ASH meeting provides an opportunity to take stock of clinical progress in hot target, modality and disease areas: CD47 inhibitors, allogeneic CAR T cells and sickle cell disease. Abstracts...

The move toward sickle cell disease therapies that address the pathology’s root causes is reflected in promising early-stage readouts at ASH, which include both disease event rates and changes in red blood cell biology. The...

DISEASE CATEGORY: Neurology INDICATION: Neurology Antisense oligonucleotides that induce exon skipping in lysosomal membrane protein CLN3 could treat CLN3 Batten disease, a neurodegenerative lysosomal...

Five years after reaping a strong return from cholesterol-lowering molecule obicetrapib, Forbion is again investing in the program based on a belief it can serve some dyslipidemia patients where others in its class fell short....

The U.S. government is taking non-conventional paths to point-of-care and pooled technologies in its push to bulk up and speed up the country’s strained COVID-19 testing capacity. In HHS’s case, the department is scrambling to...

Two days after raising $100 million in a series D round heavy with crossover investors, Annexon has filed to go public on NASDAQ. The filing adds to a burgeoning IPO queue and caps another busy...

The all-hands-on-deck COVID-19 response provides a glimpse of what’s possible when drug developers converge around solving a problem. One of the most important developments might be in preclinical research where consensus on harmonized models and...

Autobahn, which launched with a $76 million series B Tuesday, joins at least six other companies with disclosed remyelination programs for multiple sclerosis. But the biotech thinks its prodrug, slated to enter the clinic next...