Disc Medicine Inc. found itself after an end-of-phase II meeting with the U.S. FDA in what Wainwright analyst Douglas Tsao called a “best-case scenario” regarding the path forward for bitopertin in erythropoietic protoporphyria (EPP).
Some rare skin diseases not only reduce the quality of life of patients, but also can be devastating conditions, leading to amputations or death. At the 31st annual congress of the European Society of Gene and Cell Therapy (ESGCT), held last week in Rome, different laboratories showcased their approaches to editing mutations related to this group of diseases.
Continuing its streak of promising early clinical data, Jasper Therapeutics Inc.’s briquilimab impressed in a preliminary readout from a phase Ib/IIa study in chronic inducible urticaria (CIndU), showing a clinical response of 93%. CIndU, an inflammatory skin condition causing hives that is often induced by physical or environmental stimuli, is commonly treated with antihistamines, though some patients are refractory. Beyond antihistamines, there is no treatment available globally, explained Edwin Tucker, Jasper’s chief medical officer, so briquilimab has the potential to be “a new treatment paradigm for patients,” both in reducing disease burden and in improving quality of life.
Phosphodiesterase 4 (PDE4) is an enzyme known to play a role in inflammatory responses, and it has been pinpointed as an interesting therapeutic target for diseases like atopic dermatitis (AD), but limited efficacy and side effects have prevented the approval of oral formulations.
Darier disease (DD) is a rare cutaneous disease caused by heterozygous mutations in the ATP2A2 gene, which encodes sarco/endoplasmic reticulum calcium ATPase isoform 2 (SERCA2).
Artelo Biosciences Inc. has presented data on its fatty acid-binding protein 5 (FABP5) inhibitor ART-26.12 from testing of its efficacy in preclinical models of psoriasis.
Researchers from Teva Pharmaceutical Industries Ltd. have presented data from preclinical studies evaluating the novel humanized human-proteinase‑activated receptor 2 (PAR2)-specific monoclonal antibody, TEV-56192, as potential treatment of skin inflammatory conditions.
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