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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Bob Duggan, Summit Therapeutics co-CEO

Bounc­ing from ma­jor set­back, Sum­mit hands out $500M cash for can­cer drug — thanks to a loan from bil­lion­aire CEO

After hitting a dead end with Summit Therapeutics’ lead program, Bob Duggan has found the drug that he believes will usher into a compelling second act. So compelling, in fact, that it involves $500 million cash — and he’s taking money out of his own pocket to fund the deal.

Striking a partnership with Akeso Therapeutics out of China, Summit is bringing in a bispecific antibody that blocks both PD-1 and VEGF called ivonescimab. Akeso, which has a PD-1/CTLA-4 bispecific approved in China, has already taken ivonescimab into multiple clinical trials, including a Phase III in lung cancer.

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Eu­ro­pean Com­mis­sion lays ground­work to un­wind Il­lu­mi­na's $7B+ Grail merg­er

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.

US sup­ports ex­ten­sion for Covid-19 IP waiv­er de­ci­sion

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.

Mar­ket­ingRx roundup: Phar­mas lay off Twit­ter ads for an­oth­er week; WPP un­cov­ers LGBTQ+ mar­ket­ing find­ings

When Twitter’s new owner Elon Musk tweeted this weekend, “Just a note to thank advertisers for returning to Twitter,” he likely wasn’t talking about big pharma companies. The vast majority of the top spending pharma advertisers had not returned last week, according to updated tracking data Pathmatic for Endpoints News.

Only three pharma advertisers spent any money at all, which is about the same as the past several weeks. AstraZeneca rejoined the active advertiser list, although at $700 spent hardly worth a personal Musk expression of gratitude. GSK remained active with $3,500 spent ad much lower than its previous spending, according to the Pathmatics data. Only Bayer spent any significant amount in advertising, with $244,000 spent last week, but that’s a considerable drop from almost $500,000 spent on OTC, prescription and corporate Twitter ads in each of the previous two weeks.

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Jonathan Montagu, HotSpot Therapeutics CEO

Ab­b­Vie puts up $40M to li­cense a treat­ment from HotSpot Ther­a­peu­tics

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.