Adding an­oth­er type of fu­el to its gene ther­a­py en­gine, Sarep­ta has signed Co­di­ak Bio­sciences as a part­ner to ex­plore re-dos­ing of its sig­na­ture pre­ci­sion med­i­cines for neu­ro­mus­cu­lar dis­or­ders.

Doug Williams

For $72.5 mil­lion in up­front cash and near-term li­cense fees plus re­search fund­ing, Co­di­ak is com­mit­ting to in­ves­ti­gate up to five tar­gets on its ex­o­some en­gi­neer­ing plat­form in the com­ing two years. Once the projects get through the IND stage, Sarep­ta can opt to take over.

In the process, Sarep­ta is al­so build­ing out its con­stel­la­tion of biotech part­ners — many around the Cam­bridge, MA area — to ce­ment its rep­u­ta­tion as the lead­ing de­vel­op­er of ge­net­ic treat­ments for dis­eases like Duchenne mus­cu­lar dy­s­tro­phy and limb gir­dle mus­cu­lar dy­s­tro­phy. From start­up Dyno Ther­a­peu­tics to the more es­tab­lished Se­lec­ta, it’s stack­ing up on tech­nolo­gies for the tool­box.

Co­di­ak’s spe­cial­ty is co-opt­ing ex­o­somes, the body’s nat­ur­al in­ter­cel­lu­lar com­mu­ni­ca­tion sys­tem, to car­ry pay­loads wher­ev­er they want it — es­sen­tial­ly putting an ad­dress on it with mol­e­c­u­lar tech­niques to achieve tar­get­ed de­liv­ery.

Doug In­gram

They al­so have a cru­cial prop­er­ty that ade­no-as­so­ci­at­ed virus­es, the pri­ma­ry vec­tors for gene de­liv­ery, don’t. Co­di­ak CEO Doug Williams calls it “im­mune-cloaked.”

“Gene ther­a­py has al­ways been one and done on­ly be­cause it has to be,” Williams tells End­points News.

But AAV can get trapped in­to ex­o­somes — some­thing re­searchers ob­served when mak­ing AAV in mam­malian cells, and Co­di­ak has al­so seen when pu­ri­fy­ing ex­o­somes from virus pro­duc­er cells. And when the AAV rides an ex­o­some in­to cells, they be­come shield­ed from neu­tral­iz­ing an­ti­bod­ies, which had been a crit­i­cal bar­ri­er to re-dos­ing.

The next steps will be to re­fine the process for slip­ping AAV in­to ex­o­somes, ren­der­ing it more ef­fi­cient. It’s as much a man­u­fac­tur­ing prob­lem as it’s an R&D ques­tion, but that’s what Co­di­ak’s siz­able man­u­fac­tur­ing group — em­ploy­ing as many staffers as the R&D unit — was built for.

The col­lab­o­ra­tion al­so cov­ers oth­er tech­nolo­gies in Sarep­ta’s ar­se­nal such as gene edit­ing and RNA, CEO Doug In­gram adds.

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.