C07C323/26

IONIZABLE LIPIDS AND NANOPARTICLE COMPOSITIONS THEREOF

Provided herein are ionizable lipids represented by the Formula (I): or a pharmaceutically acceptable salt thereof, wherein R.sup.1, R.sup.2, R.sup.3, R.sup.4, R.sup.5, R.sup.6, R.sup.1′, R.sup.2′, R.sup.3′, R.sup.4′, R.sup.5′, R.sup.6′, m, and n are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising an ionizable lipid of the invention and a capsid-free, non-viral vector (e.g., ceDNA). These LNPs can be used to deliver a capsid-free, non-viral DNA vector to a target site of interest (e.g., cell, tissue, organ, and the like).

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IONIZABLE LIPIDS AND NANOPARTICLE COMPOSITIONS THEREOF

Provided herein are ionizable lipids represented by the Formula (I): or a pharmaceutically acceptable salt thereof, wherein R.sup.1, R.sup.2, R.sup.3, R.sup.4, R.sup.5, R.sup.6, R.sup.1′, R.sup.2′, R.sup.3′, R.sup.4′, R.sup.5′, R.sup.6′, m, and n are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising an ionizable lipid of the invention and a capsid-free, non-viral vector (e.g., ceDNA). These LNPs can be used to deliver a capsid-free, non-viral DNA vector to a target site of interest (e.g., cell, tissue, organ, and the like).

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ADO-resistant cysteamine analogs and uses thereof

The present disclosure is directed to methods for treating diseases for which cysteamine is indicated and compounds useful in such methods.

ADO-resistant cysteamine analogs and uses thereof

The present disclosure is directed to methods for treating diseases for which cysteamine is indicated and compounds useful in such methods.

ADO-RESISTANT CYSTEAMINE ANALOGS AND USES THEREOF

The present disclosure is directed to methods for treating diseases for which cysteamine is indicated and compounds useful in such methods.

ADO-RESISTANT CYSTEAMINE ANALOGS AND USES THEREOF

The present disclosure is directed to methods for treating diseases for which cysteamine is indicated and compounds useful in such methods.

ADO-resistant cysteamine analogs and uses thereof

The present disclosure is directed to methods for treating diseases for which cysteamine is indicated and compounds useful in such methods.

ADO-resistant cysteamine analogs and uses thereof

The present disclosure is directed to methods for treating diseases for which cysteamine is indicated and compounds useful in such methods.

ADO-RESISTANT CYSTEAMINE ANALOGS AND USES THEREOF

The present disclosure is directed to methods for treating diseases for which cysteamine is indicated and compounds useful in such methods.

ADO-RESISTANT CYSTEAMINE ANALOGS AND USES THEREOF

The present disclosure is directed to methods for treating diseases for which cysteamine is indicated and compounds useful in such methods.