Q&A with Nobel winner Fred Ramsdell on basic research in biotech, and the future of cell therapy Fred Ramsdell, who won the Nobel Prize in Physiology or Medicine with Mary Brunkow and Shimon Sakaguchi, spoke with Endpoints on Monday about the 'surreal' honor, cell therapy for autoimmune diseases,...

I spoke with Nobel prize winner Fred Ramsdell about conducting basic research in biotech, cell therapy for autoimmune disease, and more

(I also learned he was briefly a theater major)

Read our conversation in @endpts.com:
endpoints.news/qa-nobel-win...

Biopharma is wagering billions of dollars that CAR-T therapy can be easier. Will it work this time? Biopharma companies spent $4B+ in 2025 on in vivo CAR-T startups, including BMS's $1.5B Orbital deal, as first human data emerges from China via Esobiotec's multiple myeloma trial.

Biopharma is wagering billions of dollars that CAR-T therapy can be easier. Will it work this time?

endpoints.news/biopharma-is...

Advocates urge J&J to ‘put everything on the table’ for gene therapy despite failed trial Patients, researchers and advocates are imploring J&J to apply for approval of the X-linked retinitis pigmentosa gene therapy known as bota-vec despite a failed Phase 3 study

“If the second biggest [pharmaceutical] company in the world is reticent about trying to get approvals, what on earth does that mean for rare disease?”

Advocates urge J&J to apply for approval of gene therapy for a rare eye disease called XLRP despite a failed trial

endpoints.news/advocates-ur...

Verve unveils early data from second attempt at heart disease gene editing Verve Therapeutics is trying to swerve the gene editing industry downturn with new results on a potential one-time treatment to lower cholesterol. The experimental gene editing therapy cut bad cholest...

Some good news for the CRISPR field. Verve Tx unveils positive (but early) data from its 2nd attempt at heart disease, with strong cholesterol reduction and no safety concerns that hindered its 1st attempt. @leileiwu.bsky.social has the story for @endpts.com: endpts.com/verve-unveil...

Big Pharma Needs to Get Off the Sidelines The industry can’t continue to sit idly by while Robert F. Kennedy Jr. guts the nation’s public health agencies.

Noticeably silent amid the upheaval at HHS? Big pharma. Every new drug has NIH somewhere in its DNA--and NIH funding supports the training of their workforce. Now they're finding out FDA isn't safe, either. Wrote about how keeping quiet isn't working My latest @opinion.bloomberg.com (gift link):

Firings sweep across FDA as former leaders lament drug agency's gutting Mass firings and forced resignations swept through the FDA on Tuesday, as part of thousands of planned job cuts at federal health agencies being implemented by the Trump administration.

"FDA as we know it is finished," the agency's former commissioner lamented as the agency he once led is gutted by the new administration. Follow the developing story: endpts.com/firings-swee...

Servier pays $70M upfront for Black Diamond’s RAS- and RAF-targeted cancer drug Black Diamond Therapeutics has found a buyer for an early-stage targeted cancer drug, which was brushed to the side last year as it focused resources on developing a next-generation lung ...

Servier pays $70M upfront for Black Diamond’s phase 1 RAS- and RAF-targeted cancer drug, BDTX-4933, & up to $710M in milestones, plus tiered royalties. From @leileiwu.bsky.social @endpts.com
endpts.com/servier-pays...

Sixteen-year-old dies after receiving Sarepta’s Duchenne gene therapy A teenage boy who received Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys died from liver failure, the biotech said Tuesday morning.

A sixteen-year-old died after receiving Sarepta’s Duchenne gene therapy. This is the first death possibly related to Elevidys

endpts.com/sixteen-year...

RFK Jr. convened roundtable around ways to reduce stem cell regulation HHS Secretary Robert F. Kennedy Jr. and his staff last Wednesday hosted a regenerative medicine roundtable primarily to discuss how to lower regulatory barriers for stem cell treatments, three people ...

HHS Secretary Robert F. Kennedy Jr. and his staff last Wednesday hosted a regenerative medicine roundtable primarily to discuss how to lower regulatory barriers for stem cell treatments

My story in @endpts.com :

endpts.com/rfk-jr-conve...

Trump administration reverses lease termination for major FDA quality lab in St. Louis Two days after announcing plans to shut down an FDA lab devoted to ensuring the US drug supply remains safe and potent, the Trump administration has changed plans and will leave the lab open, Endpoint...

@zacharybrennan.bsky.social with more reporting on that FDA lab in St. Louis. After originally planning to cancel the lease, the Trump administration has reversed course, sources tell him. endpts.com/trump-admini...

I visited this lab when at FDA — lead for pharmaceutical quality, key for investigations of contaminants in drug supply. If this move interrupts agency function, it’s a big deal.

DOGE terminates lease on FDA quality lab in St. Louis, and 29 other sites DOGE says that it has terminated the leases of 30 FDA offices and buildings, including a 52,000 square-foot St. Louis-based lab that operates under the FDA's Office of Pharmaceutical Quality.

DOGE terminates lease on FDA quality lab in St. Louis, and 29 other sites

from @zacharybrennan.bsky.social
endpts.com/doge-shutter...

RFK Jr. moves to eliminate public comment in HHS decisions HHS Secretary Robert F. Kennedy Jr. moved to rescind the longtime practice of giving the public a chance to comment on the health agency’s plans.

Breaking: RFK Jr. moves to quietly eliminate public comment from HHS's decision making: www.statnews.com/2025/02/28/r...

After rejection, Regeneron seeks accelerated approval of bispecific for only one of two types of lymphoma Regeneron is now expecting an accelerated approval decision this summer on its T cell engager for one form of lymphoma, while it's giving up on accelerated approval for a different form of the disease...

After rejection, Regeneron seeks accelerated approval of bispecific for only one of two types of lymphoma

endpts.com/regeneron-sh...

Financial peril could doom a famed New York paleontological institute “Exceptional” fossil collections housed by the Paleontological Research Institute risk being orphaned

www.science.org/content/arti...

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.

Had the honor of interviewing two incredible women this week for @endpts.com. Thanks Janet Woodcock and @jenniferdoudna.bsky.social for joining me on stage!