SpliSense’s IND of SPL84 has been cleared by the US FDA to commence the P-II clinical evaluation in CF due to its favorable safety profile supported by P-I data. Preclinical studies demonstrated its ability to fully restore CFTR protein & activity, based on clinically predictive CF models
Furthermore, the company has obtained funding from the CF Foundation & other investors that will be utilized for the development of SPL84’s P-II study & other pulmonary programs
SPL84, administered to the lungs through inhalation, works by binding with the mutated CFTR RNA in the targeted sequence and modulates it to produce fully functional CFTR proteins
Ref: PR Newswire | Image: SpliSense
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