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.

The European Commission has recommended steps that — though not yet final — would require Illumina to “swiftly” unwind its controversial $7.1 billion Grail buyout.

The Commission delivered a “statement of objections” on Monday, detailing the process Illumina would need to take in divesting Grail, its blood testing spinout launched in 2016. Illumina re-acquired Grail back in August, despite criticism from both the FTC and EU.

After much debate, the US government is now calling for a deadline extension to discuss a controversial potential IP waiver for Covid-19 diagnostics and therapeutics.

Over the last five months, the Office of the United States Trade Representative said it has consulted with members of Congress, public health advocates, organized labor groups, academics, think tanks, companies and trade associations on the WTO’s recent TRIPS agreement, which established a 5-year waiver of certain patent requirements on Covid-19 vaccines.

HotSpot Therapeutics has managed to gain some steam financially in the past few years, as the company wrangled several multi-million dollar raises. But its latest deal not only puts more cash into its pockets, it also connects with a major name in pharma.

On Tuesday, AbbVie and HotSpot announced they have entered an “exclusive” global collaboration, with the option to license HotSpot’s IRF5 program, which is designed to treat autoimmune diseases. The deal will see AbbVie hand HotSpot $40 million upfront, with the biotech eligible to receive $295 million in “option fees” and R&D milestones.