Netherton syndrome is a rare disease caused by loss of activity of the lympho-epithelial Kazal-type-related inhibitor (LEKTI) protein, which in turn is caused by mutations in its encoding gene, SPINK5. This deficiency leads to the triggering of the kallikrein (KLK) signaling cascade resulting in skin barrier dysfunction, inflammation and atopy. At the recent Society for Investigative Dermatology meeting, Biocryst Pharmaceuticals Inc. presented early data on BCX-17725, a KLK5/KLK14 inhibitor fusion protein developed to restore LEKTI functioning in patients with Netherton syndrome.

Scientists at the La Jolla Institute for Immunology have identified and characterized human antibodies that neutralize the measles virus by blocking its entry into the cell. This is the first time that antibodies have been shown to bind effectively to two essential viral proteins, creating a dual blockade that prevents infection. Unlike the current vaccine, which is based on an attenuated virus and is not recommended for immunocompromised individuals, these monoclonal antibodies could be used both as a new vaccine approach and as a treatment for the entire population.

Avalo Therapeutics Inc. may have designed in lead asset abdakibart, a drug for hidradenitis suppurativa (HS) that offers a novel mechanism of action, wide dosing interval, and more-than-satisfying outcomes on the Hidradenitis Suppurativa Clinical Response 75 endpoint, just what the doctor – and Wall Street – ordered.

Evotec SE has announced the nomination of a small-molecule preclinical development candidate from its multi-target drug discovery alliance in medical dermatology with Almirall SA. The program is aimed at developing novel treatments for immune-mediated inflammatory skin diseases with high unmet medical need. The collaboration, established in 2022, leverages Evotec’s fully integrated, AI/machine learning enhanced discovery and preclinical development platforms.

BLR Bio LLC has presented new data highlighting the potential of BLR-200 for the treatment of systemic sclerosis (scleroderma). The company also presented data on its research on CCN proteins as potential noninvasive biomarkers for systemic sclerosis.

Dermatology specialist Leo Pharma A/S is moving into gene therapy with the $50 million acquisition of Replay Holdings LLC, a seed-stage company that is developing high capacity herpes simplex virus vectors to treat rare inherited diseases.

In its largest acquisition to date, Chiesi Group is buying Kalvista Pharmaceuticals Inc. for $27 per share, a roughly 40% premium to the previous day’s closing price, putting the total deal value at about $1.9 billion. In return, the Italian pharma firm adds to its rare disease franchise Kalvista’s recently approved hereditary angioedema (HAE) drug, plasma kallikrein inhibitor Ekterly (sebetralstat), which has gotten off to a strong commercial launch as the only orally available, on-demand treatment for HAE.