Tessera unveils promising in vivo gene editing treatment for sickle cell in an increasingly competitive field: #ASGCT24

Tessera Ther­a­peu­tics, a well-fund­ed start­up based out­side of Boston, an­nounced Fri­day that it de­vel­oped a lipid nanopar­ti­cle that shut­tles a gene edit­ing ther­a­py di­rect­ly to the bone mar­row of mice.

One or two in­fu­sions of the treat­ment cor­rect­ed enough of their bro­ken he­mo­glo­bin genes to be po­ten­tial­ly cu­ra­tive.

If the treat­ment works in peo­ple, it could pro­vide a sim­pler and po­ten­tial­ly more af­ford­able al­ter­na­tive to two re­cent­ly ap­proved ther­a­pies for sick­le cell dis­ease: Cas­gevy from CRISPR Ther­a­peu­tics and Ver­tex Phar­ma­ceu­ti­cals, and Lyf­ge­nia from blue­bird bio. Those treat­ments are med­ical mile­stones, but they are al­so ex­pen­sive and cum­ber­some.

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