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...
01.07.2025 15:52 — 👍 1 🔁 0 💬 0 📌 0
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):
01.04.2025 15:08 — 👍 824 🔁 332 💬 42 📌 33
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.
05.03.2025 00:33 — 👍 49 🔁 30 💬 1 📌 2
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.
12.02.2025 16:45 — 👍 386 🔁 159 💬 17 📌 19
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!
16.01.2025 20:18 — 👍 7 🔁 1 💬 0 📌 0
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