GeneDoctor

Vinay Prasad Is Out at the FDA—Again wsj.com/opinion/fda-ma…

“The FDA official responded, “You all know that as background, I’m a professor and I’m a practicing [hematology-oncology] doctor. I do miss my clinic and I miss teaching, and I miss being able to run my podcast and speak freely.” statnews.com/2026/03/06/fda…

BRAVO!

For Alzheimer's disease, it's no longer a debate of amyloid (Aβ) versus tau. They are interactive drivers and this new, optimistic review maps out a path towards disease-halting therapies @CellCellPress cell.com/cell/fulltext/…

Super cool @zhangf!

I’m the former head of Pfizer R&D. I’m very worried about biopharma’s future statnews.com/2025/12/19/pha… via @statnews

Good to see these type of combos to build critical mass in areas like rare disease!

“For decades, the U.S. has been the pinnacle of global science. Now experts fear it may have lost some of its luster, and that budding international researchers won’t… take their next career step in the U.S.” Not MAGA or MAHA.

A discovery of a gene underpinning the perception of pain with implications for treating the chronic condition that affects 600 million people globally economist.com/science-and-te… nature.com/articles/s4158…

@adamfeuerstein @Jasonmmast It turns out that the “arduous and treacherous” path to re-shipping the drug took 7 days! No new trial and no new safety data required. Seems the FDA source or reporting is off here. #FDA $SRPT

@adamfeuerstein @Jasonmmast FDA calls the pathway for reviewing a safety signal and death "arduous and treacherous." Odd choice of words for a scientific process - why the drama? #FDA $SRPT

EXCLUSIVE: Sarepta Therapeutics’ $SRPT Duchenne therapy faces ‘arduous’ path back to market, senior FDA official says statnews.com/2025/07/22/fda… via @Jasonmmast and me.

#Starlink down right now GLOBALLY! Wow! That’s a quick way to clear the rest of my zoom calendar for the day!

#SpaceX #Starship major explosion here in the Ragged Islands Bahamas. Taking cover.

@adamfeuerstein Most likely path forward is that the FDA label safety section is updated by end-Aug, possible REMS… then dosing continues in ambulatory DMD. No reason for another clinical trial when you have substantial real world data in-hand.

This week's Biotech Scorecard digs deeper into the Sarepta $SRPT #Duchenne gene therapy debacle. 1. Sarepta may not survive. If Elevidys cannot return to the market (and STAT's reporting this week suggests it will not) the company is facing a severe cash flow and debt crisis. I dig deeper into the numbers. 2. Putting business and stock prices aside, this week has been wrenching for Duchenne families. On this week's Readout LOUD podcast, we spoke with the mother of a boy who was treated with Elevidys. She wants Sarepta and the FDA to reach an agreement that returns Elevidys to the market. Listen when the episode drops tonight. 3. What happens to Doug Ingram? His future as CEO hinges on two of the eight Sarepta board members, including investor Rick Barry. Remember him? Yes, he's the $SAVA guy, too. I take a closer look at some weird Sarepta board dynamics. Want to read more? The web edition of this week's newsletter can be read at the link below.

@adamfeuerstein @Jasonmmast After-all, this was a voluntary halt. $SRPT could still restart with a robust internal PV / safety plan (e.g. sirolimus) in ambulatory. FDA did not require an aligned REMS (yet?)?

@adamfeuerstein @Jasonmmast A death "lost in the bureaucracy"? And an internal FDA plan to halt the drug with further signal? Why hide this from the sponsor? These leaks from Snr FDA officials don't help the agency image here.

Substantial in vivo platform derisking here. Why not reflected in the share price?

Scratching my head at $BEAM premarket movement. Hit 38 a share, now trading down premarket on positive results?

@adamfeuerstein @BertrandBio Nice to see some positive #crispr news!

$BEAM - Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (AATD), Demonstrating First Ever Clinical Genetic Correction of a Disease-causing Mutation investors.beamtx.com/news-releases/… Also, raised $500M via an over-the-wall PIPE

A major milestone for genetic medicines! First-ever in vivo genetic correction of a human disease! Amazing result for science, medicine, and patients! So proud of the ⁦Beam team! globenewswire.com/news-release/2…

@paras_biotech Now add Top 10 Data/RegA declines and Top 10 Bio Marketcap Losses??? Is it >$92B???

From Leerink: $92 Billion of shareholder value has been created by clinical, regulatory, commercial and M&A success on 1 day mkt cap gains basis with a caveat that some of those gains have not been preserved