“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...

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...

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):

"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 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...

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...

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...

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

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 endpts.com/regeneron-sh...

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!