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-...

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-...

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...

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...

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...

“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...

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...

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'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 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...

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 gains interim coverage from UnitedHealth's Optum: www.biopharmadive.com/news/vertex-...

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...

“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...

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...

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-...

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.

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...

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...

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.

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...

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...

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...

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...

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-...

If you want to learn more about science and money, subscribe so I can make a 25k Bluesky post 🥹 link.biopharmadive.com/click/383693...

💊 BioPharma Dive’s Emerging Biotech Weekly newsletter hit 20,000 subscribers this week! This started 2.5 years ago when @benthefidler.bsky.social and Ned Pagliarulo hired me to cover the growth of biotech and VC. So proud to see a fun experiment (not the typical #biosky kind!) blossom.

In the present discussions about NIH and the need for reform, two recurring points I am seeing are: (1) NIH doesn't fund truly high impact research (2) NIH needs to experiment with alternative approaches to peer review, particularly for high risk-high impact research 1/n

New: a conversation with BridgeBio Pharma CEO Neil Kumar on trying to gain support from investors hyper-focused on M&A, and competing with Pfizer and Alnylam in a closely watched commercial battle. #biotech #biosky $BBIO $ALNY www.biopharmadive.com/news/bridgeb...

New: The Biden Administration, in one of its final health policy acts, just announced the 15 medicines chosen for the second round of pricing negotiations under the IRA. More here from Ned Pagliarulo: #biosky www.biopharmadive.com/news/medicar...

New: A sudden burst of dealmaking for drugs invented in China is pressuring US #biotech companies. Here’s what’s going on and what it could mean for the industry going forward. #biosky #JPM25 www.biopharmadive.com/news/biotech...

Latest #JPM25 report from @gwendolynawu.bsky.social, Ned Pagliarulo and Jon Gardner includes a look at how executives are thinking about the turnover at the FDA and when generalist investors may return to #biotech. #biosky www.biopharmadive.com/news/jpm25-t...

Latest from #JPM25: Executives at Amgen made the case for their obesity drug, while Merck’s CEO contended that Keytruda’s coming loss of exclusivity can be managed. #biosky #biotech www.biopharmadive.com/news/jpm-25-...

New: How will China licensing deals affect U.S. biotech? What might rekindle the cell and gene therapy field? We explored those and a few other questions facing #biotech #startups in 2025 below.  www.biopharmadive.com/news/biotech...

Dispatch from #JPM25 with @gwendolynawu.bsky.social and Delilah Alvarado: #biosky #biotech www.biopharmadive.com/news/jpm-25-...

New: a flurry of deals to kick off #JPM25 today, including this one from $LLY #biosky www.biopharmadive.com/news/lilly-p...

New: The latest #biotech #startup launching with a drug licensed from China is entering a very competitive race to develop so-called T cell engagers for autoimmune disease. #biosky www.biopharmadive.com/news/ouro-bi...

New: What regulatory impact will the Trump administration have? Will drug company stocks continue to underperform? Members of the BioPharma Dive team dug into these and a few other questions facing pharmaceutical companies in 2025. #biotech #biosky www.biopharmadive.com/news/pharma-...

New: here are 5 FDA approval decisions to keep an eye on over the next three months, including a highly anticipated pain drug from Vertex and a heart medicine that’s critical to Alnylam’s future. #biosky #biotech $ALNY $VRTX $SLNO www.biopharmadive.com/news/fda-app...

Some me-toos and some long shots, but two stand out: - $NVS $IONS Pelacarsen cardiovascular outcomes trial. Lp(a) has been associated with CV disease since the 1970s. First of a highly anticipated class of medicines - MORF-057 Ph2b in UC. Is $LLY on a roll also in BD and can orals replace biologics

New: Here's our latest biannual primer on some of the top #biotech trials to watch over the next six months, among them big readouts in Alzheimer's, cancer and heart disease. #biosky www.biopharmadive.com/news/biotech...

A new type of obesity drug being developed by Novo Nordisk has fallen short of expectations set by the company in a closely watched Phase 3 trial, wiping out billions of dollars in market value and lifting rival Eli Lilly. More below: #biosky #biotech www.biopharmadive.com/news/novo-no...

Vertex lost more than $15 billion in value Thursday as new data on its closely watched non-opioid pain drug disappointed Wall Street. Jacob Bell has more here: www.biopharmadive.com/news/vertex-...

New: A $140M round for Ottimo Pharma, led by former Seagen CEO David Epstein, is the latest example of a surge of investments in a drug class many now believe have the potential to supplant standard-of-care cancer immunotherapies. #biosky www.biopharmadive.com/news/ottimo-...

A startup working on a non-addictive alternative to opioids - and which has a complex relationship with Vertex - just got a big influx of investment after surviving for years on small grant funds. More here from Jacob Bell: www.biopharmadive.com/news/siteone...

Merck's search for a successor to its ultra-lucrative cancer immunotherapy Keytruda has hit another setback in a finding that could have implications for multiple other drugmakers. Jon Gardner has more below. #biosky www.biopharmadive.com/news/merck-d...

New: Results from a trial testing an inflammatory bowel disease drug from Teva and Sanofi surprised analysts and suggested it could be the most potent drug among a group of similar medicines in advanced testing. #biosky #biotech $SNY $TEVA www.biopharmadive.com/news/sanofi-...