A bespoke CRISPR therapy suggests a blueprint for treating ‘N-of-1’ diseases A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design and test a new CRISPR medicine in just a few months.

New: A #CRISPR gene editing drug custom-made for a critically ill baby in just a few months appeared to be safe and working as intended, according to results newly published in the New England Journal of Medicine. Details below:
#biotech #biosky #ASGCT2025
www.biopharmadive.com/news/crispr-...

Prasad’s FDA appointment pressures cell and gene therapy stocks A vocal opponent of his predecessor Peter Marks, Vinay Prasad will now lead the office tasked with reviewing some genetic medicines, adding more uncertainty to an already struggling field of research.

New: Shares in more than a dozen gene and cell therapy developers tumbled by double digits after Vinay Prasad, a vocal opponent of predecessor Peter Marks, was named head of the FDA office that regulates their products. #biotech #biosky #genetherapy

www.biopharmadive.com/news/prasad-...

Facing ‘uncertainty on steroids,’ biotech dealmakers tread more cautiously Experts say market turmoil has made it harder for buyers and sellers to agree on price, which can be an especially imposing obstacle to large acquisitions.

New: market turmoil has made it harder for buyers and sellers to agree on price, slowing down #biotech M&A. More from Jacob Bell below:

www.biopharmadive.com/news/deals-p...

Biotech ‘megarounds’ hold steady as startups, VCs wait on IPOs More than three-quarters of private biotech investment tracked by BioPharma Dive between January and March was distributed via rounds worth at least $100 million, which experts believe reflects a stra...

New: @gwendolynawu.bsky.social looks back at the first quarter in #biotech venture funding, which was again highlighted by financings of $100 million or more. Here's what's driving that trend, and the trade-offs investors are making as a result. #biosky

www.biopharmadive.com/news/venture...

Cell, gene therapy makers lose a champion at FDA with exit of Peter Marks Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

lots happening in the chaos with CDC, FDA, HHS firings this week - but one #biosky takeaway many are still reeling from is the departure of Peter Marks, a long-time champion for cell and gene therapy. from @benthefidler.bsky.social for @biopharmadive.com www.biopharmadive.com/news/peter-m...

HHS begins layoffs in chaotic fashion Reduction-in-force notices went out Tuesday morning to employees across HHS. At FDA, OND director Peter Stein is departing as cuts hit CDER.

“The loss of many support and review functions across OND that are critical to the work we do is going to have a deep impact on our efficiency and our capabilities,” departing office of new drugs director Peter Stein told @nedpagliarulo.bsky.social: #biosky

www.biopharmadive.com/news/hhs-lay...

Cell, gene therapy makers lose a champion at FDA with exit of Peter Marks Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

New: The resignation of Peter Marks leaves the cell and gene therapy field without a regulator many view as “integral” to its progress over the last decade. #biosky #biotech #genetherapy

www.biopharmadive.com/news/peter-m...

Peter Marks, FDA’s top vaccine official, resigns In his resignation letter, Marks cited disagreement with HHS Secretary Robert F. Kennedy Jr., who he said pushed “misinformation and lies.”

New, from @nedpagliarulo.bsky.social: Peter Marks, the FDA's top vaccine official, has resigned following disagreement with HHS Secretary Robert F. Kennedy Jr., who he said pushed “misinformation and lies.” #biosky

www.biopharmadive.com/news/peter-m...

Alnylam prices heart drug at premium to rivals Initially, Amvuttra’s annual list price will be nearly double the yearly cost of rival medicines from Pfizer and BridgeBio. But executives argue its “compelling and highly differentiated value” justif...

Alnylam's new heart drug Amvuttra is nearly twice the price of its pharmaceutical rivals'. Here's why, from @benthefidler.bsky.social www.biopharmadive.com/news/alnylam...

Alnylam drug gets long-awaited FDA approval in deadly heart disease Amvuttra’s clearance in a rare form of cardiomyopathy could help Alnylam turn a regular profit — if it can wrest control of a competitive and fast-changing market.

Alnylam gets expanded FDA approval for Amvuttra, which is now cleared to treat transthyretin amyloidosis with cardiomyopathy.

The new indication is seen as vital for Alnylam's growth, as @benthefidler.bsky.social reports:

www.biopharmadive.com/news/alnylam...

Duchenne patient dies after receiving Sarepta gene therapy A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the treatment in the future, some analysts wrote.

New: A young man treated with Sarepta's #Duchenne gene therapy Elevidys, which has long faced questions about its benefits, died of acute liver failure. The case may give doctors pause before prescribing the treatment in the future:

#biosky #genetherapy

www.biopharmadive.com/news/sarepta...

Vertex’s new pain drug gets first coverage nod from major insurer Optum, the pharmacy benefit manager owned by UnitedHealth Group, placed Journavx on some of its commercial formularies in an interim decision while a formal review continues.

Vertex’s new pain drug gains interim coverage from UnitedHealth's Optum:

www.biopharmadive.com/news/vertex-...

State of Play: A closer look at hotspots of emerging biotech research This series from BioPharma Dive examines new areas of biotech investment and startup activity, like radiopharmaceuticals, obesity drugs and CAR-T for lupus.

This is the latest edition in our ‘State of Play’ series examining hotspots of biotech research. You can read some of the others - on RNA editing, autoimmune cell therapy and more - here:

www.biopharmadive.com/news/emergin...

Blocking PD-1 and VEGF: The bispecific cancer drugs that could best Keytruda Striking study results last year indicated a new type of medicine may improve on Merck’s dominant immunotherapy, spurring a wave of research practically overnight.

New: an in-depth look at the fast-growing effort to determine whether an emerging class of multi-pronged, PD-1 x VEGF-targeting drugs can build upon Keytruda and other cancer immunotherapies like it. #biosky #biotech

www.biopharmadive.com/news/pd1-veg...

‘Fear and uncertainty’: Biotech investors warn of impact from NIH research cuts The Trump administration’s plans to reduce NIH grant funding could have long-term consequences for the U.S. drug industry, investors said.

“How are you going to get the next generation of therapies? There’s no biotech without venture, but there’s also no biotech without academic discovery.” I wrote about the proposed NIH funding cuts for @biopharmadive.bsky.social & how it'd affect #biosky #medsky www.biopharmadive.com/news/biotech...

‘Fear and uncertainty’: Biotech investors warn of impact from NIH research cuts The Trump administration’s plans to reduce National Institutes of Health grant funding could have long-term consequences for the U.S. drug industry, investors said.

New: @gwendolynawu.bsky.social looks at the long-term implications, for U.S. #biotech #startups, of a prolonged pullback in NIH grant funding. #biosky

www.biopharmadive.com/news/biotech...

Pfizer stops selling hemophilia gene therapy, citing weak demand Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for the bleeding condition.

New, from @nedpagliarulo.bsky.social: Pfizer has decided to stop selling a treatment called Beqvez in the latest sign of sparse patient interest in gene therapies for hemophilia. #biosky

www.biopharmadive.com/news/pfizer-...

Bluebird, at risk of default, agrees to take-private deal The pioneering gene therapy developer will be acquired by investment firms Carlyle Group and SK Capital for a fraction of what it was once worth years ago.

New: Bluebird Bio is being acquired in a deal that marks an ending of sorts for a company long at the forefront of gene therapy research and whose scientific achievements — and struggles — have been emblematic of the field’s ups and downs. More below: #biosky

www.biopharmadive.com/news/bluebir...

Termination notices going out this afternoon to more than 300 employees of the National Cancer Institute. Access likely ends today. Four weeks paid leave and that’s it.

Strong power move for Team Cancer.

‘It’s not for the faint of heart.’ How 3 CEOs took their biotechs public Speaking this week at BIO CEO, the heads of Septerna, Upstream Bio and Actuate Therapeutics shared lessons from going public in a challenging market for biotech stocks.

New: an inside look from Jacob Bell at the painstaking preparations a #biotech has to make to lay the foundation for a successful #IPO. #biosky

www.biopharmadive.com/news/biotech...

New study offers hints of GLP-1 drugs’ potential in curbing alcohol cravings A small semaglutide trial suggests the medicine's suppression of appetite may extend to alcohol, but larger tests are needed to establish a conclusive benefit.

Might popular weight loss drugs, already proven to have myriad other health benefits, also be helpful treating alcohol use disorder? Early study findings are pointing in that direction. More here from @byjongardner.bsky.social: #biosky

www.biopharmadive.com/news/ozempic...

Stand Up And Be Counted

Biopharma companies and CEOs are keeping their heads down at their own peril. They should speak up about what’s happening to the NIH and other science agencies before it’s too late.

Silence gives consent. And no one should consent to this.

Novartis pays $925M to reel in a startup it helped launch The acquisition of Anthos Therapeutics could be worth up to $3.1 billion and returns to Novartis a blood thinner it licensed to the Blackstone-backed startup six years ago.

New: Novartis on Tuesday acquired a startup it joined with Blackstone Life Sciences to form six years ago and, along with it, a blood thinner that originated from its own labs. More below: $NVS $REGN #biosky #biotech

www.biopharmadive.com/news/novarti...

1. Today the NIH director issued a new directive slashing overhead rates to 15%.

I want to provide some context on what that means and why it matters.

grants.nih.gov/grants/guide...

Here is how we know that vaccines do not cause autism Vaccines do not cause autism. You’ve almost certainly read that before — but often without a full explanation of how we know it to be true. Here's how we know.

VACCINES DO NOT CAUSE AUTISM.
(Yes, I'm typing in my loud voice.)

@matthewherper.bsky.social lays out the facts.

www.statnews.com/2025/02/03/v...

RFK Jr. clears key hurdle on path to become HHS secretary Louisiana Sen. Bill Cassidy joined his Republican colleagues in voting to advance Kennedy's nomination to a vote by the full Senate.

www.biopharmadive.com/news/rfk-ken...

A new, non-opioid pain drug is here. Getting it to patients could be agony. After decades of research, Vertex Pharmaceuticals now has an approved pain medication. Can one of the world’s most powerful biotechs contend with a healthcare system that’s long favored opioids?

New: Don’t miss this deep dive from Jacob Bell on the challenges Vertex faces introducing a first-of-its-kind, non-opioid painkiller to the U.S. healthcare system. #biosky #biotech $VRTX

www.biopharmadive.com/news/pain-dr...

Metsera, Maze secure combined $415 million in IPOs The offerings reflect an increase in IPO activity that could continue, as four other biotechs recently outlined plans to go public.

New: Metsera and Maze Therapeutics both priced IPOs on Thursday, adding to an uptick in activity some in the industry expect will continue throughout this year. From @gwendolynawu.bsky.social:

#biosky #biotech

www.biopharmadive.com/news/maze-me...

Vertex’s non-opioid pain drug gets FDA approval in milestone for company and research Despite high demand for an option like Journavx, doctors fear the drug’s price could be a major hangup for insurers, potentially limiting patients’ ability to access it.

New: The FDA has just approved a first of its kind, non-opioid painkiller from Vertex Pharmaceuticals. Jacob Bell has more here:

www.biopharmadive.com/news/vertex-...

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