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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.

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.

Cell and gene ther­a­py 2.0 dom­i­nates lat­est round of biotech IPOs in scram­ble for a $300M-plus Nas­daq wind­fall

Back at the start of January last year, Poseida Therapeutics thought it had an IPO-worthy game plan that would sell on Wall Street. Instead, with the market in something of a dyspeptic mood, the San Diego-based startup with its next-gen approach to cell and gene therapy switched to a Series C mega round and dropped the IPO.

Until now, while the market is running red hot for all things drug-development related. And on Friday Nasdaq loaded up a new round of biotech IPOs to sell to investors.

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As Bio­Marin’s lead ri­val wanes, Pfiz­er and Sang­amo post a promis­ing up­date for he­mo­phil­ia A gene ther­a­py

Pfizer and its hemophilia A gene therapy partners at Sangamo have posted the latest update on their Phase I/II trial for SB-525 — and it continues to look good as their rivals at BioMarin cruise to a likely approval with a not-so-perfect leader.

Median Factor VIII expression levels for their gene therapy are running at 64.2% for the 5 patients now taking the 3e13 vg/kg dose. That includes patients up to 61 weeks after dosing, putting the crew on the trial at potentially better than par with BioMarin’s valrox at a year in, which has attracted plenty of questions as FVIII levels declined over 4 years to a median of 16.4%, as we reported yesterday.

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Rick Klausner (file photo)

Two Bay Area up­starts out to de­liv­er on cell ther­a­py 2.0 join forces on a quest: pur­su­ing a Holy Grail in on­col­o­gy R&D

The first time Lyell CEO Rick Klausner looked at what PACT Pharma was trying to accomplish with neoantigens, non-viral T cell engineering and cancer, he felt they couldn’t get it done. But in the 3 years since they’ve launched, Klausner has become a believer.

Now, he’s a believer and a partner.

Early Thursday morning, Klausner and PACT CEO Alex Franzusoff announced a plan to jointly pursue one of the Holy Grails of oncology R&D. Blending their technologies and bringing a wide network of leading experts to the table, the two companies are working on a personalized T cell therapy for solid tumors. And an IND is in the offing.

The collaboration joins the Lyell team, which has been concentrating on overcoming the exhaustion that afflicts the first generation of cell therapies, with a PACT group that has developed tech to identify a patient’s unique signature of cancer mutations and use a non-viral method to engineer their T cells into cancer therapies.

I spent some time on Wednesday talking with Klausner and Franzusoff about the deal, which comes with an undisclosed set of financials as Lyell invests in the alliance.

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Bio­Marin spells out a trou­bling slide in Fac­tor VI­II lev­els for its big block­buster con­tender val­rox

We already knew from BioMarin’s statement a few days ago that their gene therapy for hemophilia A continued to protect patients against the bleeding episodes that can threaten their lives. Now, 4 years in, we’re getting the hard data on durability for valrox — and here the biotech faces considerable potential trouble.

As Stifel’s Paul Matteis noted recently, at the end of the first, second and third years, post-infusion, mean Factor VIII activity level of the high dose 6e13 vg/kg cohort was 64.3, 36.4 and 32.7 IU/dL respectively as measured by the CS assay.

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David Kirn, 4D Molecular Therapeutics CEO

Eye­ing a hot IPO mar­ket, 4DMT tops up its cash re­serves and preps a leap in­to the clin­ic with be­spoke gene ther­a­py vec­tors

It took 4D Molecular Therapeutics 7 years to build its gene therapy vector platform, line up a marquee industry deal and prep a string of INDs. And now it has the money to fund the next big stage as the team plans a near-term launch of their first clinical studies to test its custom-built gene therapies in humans.

The 80-person 4DMT team led by CEO David Kirn put out word Tuesday morning that they have landed a $75 million C round to keep the work on track.

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Nick Leschly, bluebird CEO (Jeff Rumans/JPM20)

Lenti­Glo­bin con­tin­ues to de­liv­er in lat­est sick­le cell up­date as blue­bird out­lines path to ac­cel­er­at­ed ap­proval, de­spite pa­tient death

Still reeling from the FDA’s stunning refusal to file its CAR-T therapy ide-cel, bluebird is eager to win back some credibility with a new slice of the LentiGlobin data they will be taking to the FDA for an accelerated approval.

To be sure, the BLA submission won’t happen until the second half of 2021 — giving bluebird plenty of time to figure out the manufacturing. But an update presented at the EHA virtual congress has apparently helped them reach “general agreement” with regulators and boosted execs’ confidence.

David Altshuler at Endpoints event in 2018 (Rob Tannenbaum for Endpoints News)

Ver­tex and CRISPR spot­light an­oth­er im­por­tant gene edit­ing ad­vance in a march to a hoped-for cure

Vertex and CRISPR Therapeutics have successfully navigated another stretch of the maiden clinical journey of the gene editing therapy CTX001 in a tiny group of patients suffering from beta thalassemia and sickle cell disease. And the researchers involved say they have good reason to believe that they are still on the right track to proving they are advancing a potentially curative approach with a shot at best-in-class status.

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Juno, WuXi JV in Chi­na score $100M in fresh fund­ing as it sets stage for first CAR-T ap­proval

China’s oncology-focused biotech hub has raised already millions in venture funding and lured powerful global biopharma partners to address the high rates of cancer in the country. In yet another example, the joint venture between Juno and to WuXi’s AppTec has raised a sizable $100 million in a fresh round of funding.

The Shanghai-based company, christened JW Therapeutics (an initial each from its root companies), was created to marry Juno’s CAR-T development experience and WuXi’s AppTec’s muscular manufacturing capacity. In 2018, it scored $90 million to take its lead experimental B cell malignancy-focused drug, JWCAR029, into the clinic and build a new manufacturing plant.

Sanofi sends a pair of gene ther­a­pies back to Ox­ford Bio­med­ica. Are they sal­vage­able?

The same day Oxford Biomedica signed a 5-year deal to make AstraZeneca’s Covid-19 vaccine, the UK gene therapy company announced they received word Sanofi was sending a couple of decade-old experimental drugs back to them.

In 2009, Sanofi and Biomedica slashed a co-development deal on TroVax, a cancer vaccine that had been one of the centerpieces of the then 14-year-old company. But the pair immediately replaced it with a deal, 8 years before the approval of Spark Therapeutics’ Luxturna, to develop gene therapies for two eye-related disorders.