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.

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.

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.

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...

Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

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

Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

In his resignation letter, Marks cited disagreement with HHS Secretary Robert F. Kennedy Jr., who he said pushed “misinformation and lies.”

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...

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.

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.

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.

This series from BioPharma Dive examines new areas of biotech investment and startup activity, like radiopharmaceuticals, obesity drugs and CAR-T for lupus.

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.

The Trump administration’s plans to reduce NIH grant funding could have long-term consequences for the U.S. drug industry, investors said.

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.

Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for the bleeding condition.

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.

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.

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.

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.

NIH Funding Opportunities and Notices in the NIH Guide for Grants and Contracts: Supplemental Guidance to the 2024 NIH Grants Policy Statement: Indirect Cost Rates NOT-OD-25-068. OD

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.

Louisiana Sen. Bill Cassidy joined his Republican colleagues in voting to advance Kennedy's nomination to a vote by the full Senate.

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?

The offerings reflect an increase in IPO activity that could continue, as four other biotechs recently outlined plans to go public.

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.

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In an interview, the CEO discussed trying to gain support from investors hyper-focused on M&A, and competing with Pfizer and Alnylam in a closely watched commercial battle.

Announcement of the 15 medicines chosen for the second round of negotiations is one of the Biden administration’s final health policy acts.

Pharma dealmaking for drugs invented in China is putting pressure on U.S. biotechs to compete harder, according to investors and executives interviewed by BioPharma Dive at the J.P. Morgan Healthcare ...

AbbVie's CEO hinted his company may be less willing to invest in psychiatry after a costly setback, while Lilly explained what it got wrong forecasting GLP-1 drug demand.

Executives at Amgen made the case for MariTide's differentiation, while Merck's CEO contended that Keytruda's coming loss of exclusivity can be managed.

Young drugmakers confront several harsh realities this year, among them a contracting sector and competition from China.

Security was tight for the first day of the J.P. Morgan Healthcare Conference, which featured major M&A as well as presentations from several large pharma companies.

Intra-Cellular has had success selling Caplyta, a brain-rebalancing drug that's approved to treat schizophrenia and bipolar depression.

The pharma will spend up to $2.5 billion to acquire an experimental PI3K inhibitor from Scorpion, which will spin out a new company holding its employees and other assets.

The biotech will stop developing two drugs and lay off nearly three-quarters of its staff after its lead therapy wasn’t as potent in early testing as the company expected.

“Practice of medicine could fundamentally change with these types of therapies,” said CEO Jaideep Dudani, of T cell engagers now being tested in inflammatory conditions.

Political and economic uncertainty hangs over the industry, while major bets on metabolic and immune disease drug research are set to play out.

By the end of March, the regulator could clear a highly anticipated pain drug from Vertex and a heart drug that’s critical to Alnylam’s future.

Expected readouts in diabetes, cancer and depression headline a series of study results that could help the biotechnology sector regain its footing after a shaky year.

Novo shares lost nearly a quarter of their value after an experimental combination treatment called cagrisema fell short of the bar set by executives.

Mixed results from a study focused on lower back pain left analysts wanting as well as confused about Vertex's plans to forge ahead in the indication.

Led by former Seagen CEO David Epstein, the startup claims it has a drug that could stand out from other PD1- and VEGF-blocking cancer treatments.