Generation Bio Co.

Debra Klatte

Phillip Samayoa

Nathaniel Silver

Douglas Anthony Kerr

Viktoriya Syrovatkina

Brandon Michael Johnston

Lalita Oonthonpan

Salma Merchant

Anthony Joseph Perniciaro

The present disclosure provides bispecific stealth lipid nanoparticle (LNP) compositions engineered to target specific tissues or cell-types, e.g., hematopoietic stem cells, to modify the cells with therapeutic nucleic acid encapsulated in the LNP. The present disclosure also provides compositions and methods of making the LNPs and treatment using the same.

Bispecific stealth lipid nanoparticle compositions for cell targeting

The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a transgene encoding FVIII protein. Some ceDNA vectors further comprise cis-regulatory elements, including regulatory switches. Further provided herein are methods and cell lines for reliable gene expression of FVIII protein in vitro, ex vivo and in vivo using the ceDNA vectors. Provided herein are method and compositions comprising ceDNA vectors useful for the expression of FVIII protein in a cell, tissue or subject, and methods of treatment of diseases with said ceDNA vectors expressing FVIII protein. Such FVIII protein can be expressed for treating disease, e.g., hemophilia A.

Non-viral DNA vectors and uses thereof for expressing FVIII therapeutics

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Cambridge, MA, United States of America

Debra Klatte