Sarepta licenses next-gen AAV tech from Broad Institute in deal covering five indications — including Duchenne – Endpoints News

Sarep­ta’s re­search agree­ment with the Broad In­sti­tute is tak­ing the next step Mon­day.

The biotech will li­cense a new group of ade­no-as­so­ci­at­ed virus­es from the in­sti­tute for an undis­closed up­front pay­ment and mile­stone promis­es, the pair an­nounced Mon­day morn­ing. Un­der the agree­ment, Sarep­ta will have the rights to five neu­ro­mus­cu­lar and car­diac in­di­ca­tions, in­clud­ing Duchenne mus­cu­lar dy­s­tro­phy, where it’s al­ready well-versed.

Ac­cord­ing to Sarep­ta, the deal cov­ers the MyoAAV pro­gram that aims to de­liv­er more ef­fi­cient gene ther­a­pies us­ing mod­i­fied cap­sids. End­points News has al­so reached out to the Broad In­sti­tute and will up­date this sto­ry ac­cord­ing­ly.

The group of virus­es, per Sarep­ta:

• De­liv­ered 25-50 times greater gene ex­pres­sion in mul­ti­ple skele­tal mus­cles and 10-15 times greater gene ex­pres­sion in car­diac mus­cle;

• Demon­strat­ed re­duced de­liv­ery to the liv­er by 50 per­cent and showed low­er ac­cu­mu­la­tion in the liv­er;

• Can be used at up to a log low­er dose than tra­di­tion­al AAV vec­tors, due to in­creased ef­fi­cien­cy.

Louise Rodi­no-Kla­pac

Full re­search for MyoAAV in a Duchenne mouse mod­el was pub­lished in Cell in 2021, the pair added. Sarep­ta CEO Doug In­gram said in a state­ment that the virus­es could “sub­stan­tial­ly re­duce vi­ral load,” while CSO Louise Rodi­no-Kla­pac tout­ed the plat­form’s “broad ap­plic­a­bil­i­ty.”

That Sarep­ta says it’s plan­ning to con­tin­ue pur­su­ing next-gen­er­a­tion Duchenne ther­a­pies sug­gests the biotech has no in­ten­tion of rest­ing on its lau­rels. Ex­ecs shep­herd­ed the first Duchenne gene ther­a­py across the FDA fin­ish line back in 2016 and have since won two more ap­provals. They’re seek­ing a fourth lat­er this year.

But those green lights have not come with­out con­tro­ver­sy. Al­most five years be­fore the Bio­gen brouha­ha with Aduhelm, Sarep­ta won an ac­cel­er­at­ed OK de­spite out­side ex­perts rec­om­mend­ing against ap­proval due to a lack of da­ta clar­i­ty. Though the FDA is not bound by law to fol­low its pan­el’s ad­vice, they typ­i­cal­ly do so in about four out of every five cas­es.

Sarep­ta’s next two ap­provals al­so fol­lowed the ac­cel­er­at­ed path, and the up­com­ing ap­pli­ca­tion will al­so seek a quick OK. A key part of this path­way in­volves a study to con­firm a ther­a­py’s ben­e­fit in or­der to con­vert the ac­cel­er­at­ed ap­proval to a full en­dorse­ment, but Sarep­ta has still not com­plet­ed any of the three con­fir­ma­to­ry tri­als.

An­a­lysts ex­pect an­oth­er ad­comm for Sarep­ta’s newest pitch, and the pan­el is like­ly to dis­cuss heart in­flam­ma­tion as­so­ci­at­ed with the com­pa­ny’s lat­est tri­al. Though the ef­fi­ca­cy da­ta proved large­ly pos­i­tive, a new oc­cur­rence of my­ocardi­tis in a sep­a­rate co­hort of old­er pa­tients (and clas­si­fied as a se­ri­ous ad­verse event) cloud­ed the read­out.