Following FDA’s first authorization of a COVID-19 vaccine, controversy over the Trump administration’s management of pandemic countermeasures will shift from FDA’s reviews to Operation Warp Speed’s procurement and deployment strategies. One of the most consequential...

DISEASE CATEGORY: Musculoskeletal INDICATION: Osteoarthritis Inhibiting the tyrosine protein kinase receptor ROR2 could treat osteoarthritis. Expression levels of the ROR2 ligand WNT5A were higher in human osteoarthritic cartilage than in healthy cartilage. In a mouse fibroblast...

In a move that raises concerns about the accuracy of tests, as well as liability protections for laboratories, FDA announced Wednesday that it will no longer review emergency use authorization requests for laboratory-developed tests to...

DISEASE CATEGORY: Cardiovascular INDICATION: Myocardial infarction (MI) Cilengitide, an integrin αvβ3 and integrin αvβ5 inhibitor tool compound, could treat post-myocardial infarction injury driven by COL5 deficiency. In mouse cardiac fibroblasts with COL5A1 knockout, cilengitide reduced the...

DISEASE CATEGORY: Renal INDICATION: Renal Exon skipping therapy with an antisense oligonucleotide (ASO) targeting a truncating variant in exon 21 of COL4A5 could treat severe X-linked Alport syndrome. The ASO led to complete exon 21...

DISEASE CATEGORY: Dermatology INDICATION: Blistering disorder Skin grafts based on CRISPR-edited induced pluripotent stem (iPS) cells could treat recessive dystrophic epidermolysis bullosa, a blistering disorder caused by mutations in COL7A1. Generation of the full-thickness grafts...

DISEASE CATEGORY: Autoimmune disease INDICATION: Osteoarthritis Combined TNKS and TNKS2 inhibition could treat osteoarthritis, which features progressive cartilage degeneration, and enhance the efficacy of stem cell-based therapies for cartilage regeneration. In osteoarthritis patients, TNKS and...

Non-invasive biomarkers in NASH are beginning to show where and how they can upstage liver biopsies, with the first impact appearing in patient recruitment. The biggest value will come when they can serve as regulatory...