Cell/Gene Tx channel feed

Steven Kelly, Carisma Therapeutics CEO

Con­tin­u­ing push be­yond Covid-19 vac­cines, Mod­er­na part­ners with 'CAR-M' biotech on in vi­vo can­cer cell ther­a­pies

Since coming into a massive windfall with its Covid-19 vaccines, Moderna has been looking for ways to expand its mRNA technologies into other promising areas. On Monday, the biotech took its next step toward that goal.

Moderna is partnering with Carisma Therapeutics on a huge slate of “CAR-M” programs to treat cancer, the companies announced Monday morning, promising $45 million upfront and $35 million in equity. Though the amount of milestones was not specified, the biotechs noted Moderna has the option to nominate up to 12 targets for development.

David Chang, Allogene CEO (Jeff Rumans)

Al­lo­gene wins a ma­jor re­prieve for its lead­ing off-the-shelf cell ther­a­py pipeline

Allogene is rolling into JP Morgan week with a big win for its development campaign for off-the-shelf CAR-Ts. Late last week the FDA agreed to lift the regulatory blockade that had been dropped on their full set of clinical trials after evidence of chromosomal changes in one of the patients triggered a safety alert.

In an advance of the announcement Monday morning, Allogene $ALLO CEO David Chang said in an interview that the company worked through the last 3 months with regulators to zero in on the exact trigger of the chromosomal mutations. Regulators, he said, agreed that the drug wasn’t involved and released the clinical hold.

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Jason Coloma, Maze Therapeutics CEO

Maze Ther­a­peu­tics re­fu­els with an­oth­er $190M to prove its ge­net­ic mod­i­fiers ap­proach holds up in the clin­ic

Jason Coloma remembers when Maze Therapeutics was nothing more than a bunch of PowerPoint slides. Several months after revealing exactly what his team of scientists have been up to, the chief executive is now gunning for the clinic with another $190 million in tow — and he says an IPO isn’t entirely out of the question.

“I mean, the good thing is with the financial flexibility that we now have, we’ve done a nice job,” Coloma said when asked about a public debut. “We’ll look at different options, but I think that would even include even staying private or looking at different business development transactions that might be helpful for us to kind of think of different ways to advance our pipeline.”

Hank Fuchs (BioMarin)

How durable is durable? Bio­Marin gears up to bring he­mo­phil­ia A gene ther­a­py to FDA again with 2-year da­ta

A year and a half after a stunning rejection, BioMarin says it has the durability data the FDA previously requested, paving the way for a new BLA in the second quarter of this year.

Touting “stable and durable” bleed control in what it calls the largest ever global study of a gene therapy involving patients with severe hemophilia A, BioMarin notes its drug, valoctocogene roxaparvovec (better known as valrox or Roctavian), met all primary and secondary efficacy endpoints at the two-year analysis.

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John Evans, Beam CEO

Pfiz­er, the new mas­ter of mR­NA de­liv­ery, teams up with Beam to ship base ed­i­tors straight to tar­get tis­sues

Awash in cash from its Covid-19 vaccine and leaner than ever, Pfizer has pick of the litter in biotech for deals set to shape its portfolio for the next 10 to 15 years. Gene editing has a ton of momentum right now, and Pfizer is biting in a big way on a buzzy upstart working on “CRISPR 2.0.”

Pfizer will pay $300 million upfront and up to $1.05 billion in additional downstream milestones to Beam Therapeutics as part of a four-year research collaboration to develop base editors for a trio of therapeutic targets including the liver, CNS and muscle, the partners said Monday.

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Mammoth chief business officer and head of therapeutics Peter Nell

Bay­er jumps on board Mam­moth­'s ul­tra-small CRISPR tech with sights set first on the liv­er

German drug giant Bayer has looked to reinvent itself in recent years, moving on from its past as a primarily consumer health brand into one built around next-gen therapies. Now, Bayer is taking a flyer on one of the children of CRISPR maven Jennifer Doudna’s hallowed lab working on tiny versions of the gene editing tech.

Bayer will pay $40 million upfront and more than $1 billion in potential downstream milestones for up to five in vivo gene editing candidates from Mammoth Biosciences, a Doudna lab spinout developing ultra-small enzymes for easier packaging and delivery into cells, the partners said Monday.

Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA beefs up its mR­NA work with a $780M buy­out of a boom­ing play­er

Bursting at the seams and executing plans for swift expansion to support its manufacturing work for the mRNA vaccine out of Pfizer/BioNTech, Indianapolis-based Exelead has now been scooped up in a $780 million cash buyout deal.

Germany’s Merck KGaA, which bought out another mRNA manufacturer, AmpTec, early last year, has been beefing up its ops around lipids, which, in mRNA vaccines, play a key role in turning human cells into a mini vaccine factories.

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UP­DAT­ED: You know AAV and LNP. What about PLV? Eli Lil­ly lines up new ve­hi­cle to de­liv­er its ge­net­ic med­i­cines

Eli Lilly is beefing up its fleet of vehicles being deployed to carry drugs to the brain.

Enlisting Canada’s Entos Pharmaceuticals, Eli Lilly has grabbed rights to a suite of proteo-lipid vehicles (PLVs) as part of a research collaboration that spans multiple programs focused on diseases of the central and peripheral nervous system. Entos will receive an upfront of $50 million, part of it as an equity investment, to start developing PLVs for Lilly’s selection.

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Rick Modi, Affinia Therapeutics CEO (Affinia)

Gene ther­a­py play­er Affinia aims to go pub­lic, hop­ing to raise big mon­ey in ef­fort to re­make AAVs

Nine months after Affinia Therapeutics raised $110 million in a Series B, the biotech has set its eyes on the Nasdaq.

The MA-based preclinical gene therapy biotech, which got $60 million in startup capital from New Enterprise Associates, F-Prime and Atlas in 2019, filed an S-1 with the SEC on Tuesday, announcing initial plans to go public and penciling in a $100 million raise. Affinia plans to list under the ticker $AFTX.

Ram Aiyar, Korro CEO

Can RNA edit­ing take At­las start­up where Ver­tex could­n't go? Ko­r­ro lands $116M to find out

As the CEO of RNA editing startup Korro Bio, Ram Aiyar often gets the same question.

“I get always asked, you know, if you can fix DNA, why bother with RNA?” he told Endpoints News. “And it’s like asking, which child do you prefer — your older one or your younger one?”

But investors are appreciating the difference. More than a year after closing its Series A just shy of the megaround mark, Korro Bio is back in the spotlight with $116 million in fresh cash and a lead candidate — targeting alpha-1 antitrypsin deficiency, or AATD.