Sarep­ta ex­pands gene ther­a­py toolk­it, bets $72.5M to hitch a ride on Co­di­ak's ex­o­some tech

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.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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ProFound Therapeutics founding team

Flag­ship's lat­est biotech could turn some of the thou­sands of new pro­teins it dis­cov­ered in­to ther­a­pies — and it has $75M to start

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.

Richard Silverman, Akava Therapeutics founder and Northwestern professor

This time around, Lyri­ca's in­ven­tor is de­vel­op­ing his North­west­ern dis­cov­er­ies at his own biotech

Richard Silverman was left in the dark for the last five years of clinical development of the drug he discovered. The Northwestern University professor found out about the first approval of Lyrica, in the last few days of 2004, like most other people: in the newspaper.

What became one of Pfizer’s top-selling meds, at $5 billion in 2017 global sales before losing patent protection in 2019, started slipping out of his hands when Northwestern licensed it out to Parke-Davis, one of two biotechs that showed interest in developing the drug in the pre-email days, when the university’s two-person tech transfer team had to ship out letters to garner industry appetite.

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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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Up­dat­ed: US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

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.

Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

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.

Franz-Werner Haas, CureVac CEO (Christoph Schmidt/picture-alliance/dpa/AP Images)

Tak­ing an­oth­er shot at mR­NA glo­ry, Cure­Vac inks on­col­o­gy pact while keep­ing up with Covid work

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.

Pfizer CEO Albert Bourla at the World Economic Forum (Gian Ehrenzeller/Keystone via AP Images)

All about ac­cess: Pfiz­er moves to a non-prof­it mod­el for drug sales in 45 low­er-in­come coun­tries

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.