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.

Flagship Pioneering, the incubator of Moderna and dozens of other biotechs, says it has landed upon tens of thousands of previously undiscovered human proteins. The VC shop wants to potentially turn them into therapeutics.

Like other drug developers that have turned proteins into therapeutics (think insulin for diabetes), Flagship’s latest creation, ProFound Therapeutics, wants to tap into this new trove of proteins as part of its mission to treat indications ranging from rare diseases to cancer to immunological diseases.

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

CureVac may have lost out on the initial mRNA race to bring a Covid-19 vaccine to the market, but it’s still eager to prove that it has what it takes to be a serious player in the field.

As it updates investors on its second-generation vaccine candidates for infectious diseases in Q1 results, the German biotech says it’s beefing up its oncology pipeline.

To that end, it has struck a new collaboration with Belgium’s myNEO, which boasts of a neoantigen discovery and selection platform, to identify new targets for mRNA immunotherapies.

Leading the way to increase access to cheaper drugs worldwide, Pfizer said Wednesday it will provide all current and future patent-protected medicines and vaccines available in the US or EU on a not-for-profit basis to about 1.2 billion people in 45 lower-income countries.

Rwanda, Ghana, Malawi, Senegal and Uganda are the first five countries to sign on to this accord, which will also seek to blaze new paths for quick and efficient regulatory and procurement processes to reduce the usual delays in making new medicines and vaccines available in these countries.