13% success rates for FY2025. NIH finally releases some FY2025 success rate data drugmonkey.wordpress.com/202… via @drugmonkeyblog

Some context for the UniQure $QURE post below: Earlier today, the FDA held a media call with the same "senior FDA official" who spoke to me (and other reporters) individually earlier in the week. The call was set up obstensibly so that this senior FDA official could rail against UniQure without going on the record. He did exactly this for 30 minutes, repeating the same criticisms, claiming Uniqure's Huntington’s therapy is a failure, the sham surgery would be blinded even if just small "nicks" in the skin were made in a 30-minute procedure, dismissing the idea of formally reviewing the therapy, disregarding outside scientific experts, and then accusing the media of not adequately covering what the FDA (and this official) have been doing. Reporters from Bloomberg, STAT, NY Times, WSJ, Politico and other media organizations were on the call.

Useful summary of how much has changed this year with NIH/NSF funding. nytimes.com/interactive/2025…

“I had hemophilia for nearly five decades. I went to Philadelphia and had a 45-minute infusion, and my hemophilia was gone.” Genes-> Medicine @NatureMedicine nature.com/articles/s41591-0…

Efficient Scaling up EV‐AAVs Production via Cellular Nanoporation for Familial Hypercholesterolaemia Therapy - Yan - 2025 - Journal of Extracellular Vesicles - Wiley Online Library isevjournals.onlinelibrary.w…

Extracellular Vesicles-Associated AAVs for the treatment of Machado-Joseph Disease dlvr.it/TNcSNm #genetherapy #celltherapy

In a new study, researchers from @CGM_MGH and colleagues found that a customized gene-editing technology shows potential to treat lethal pediatric disease. The study was published in @natBME. Read more: massgeneralbrigham.org/en/ab… @BKleinstiver @MGBInnovation

Congratulations to all co-authors! And here is a free link to access the manuscript: rdcu.be/eFAEz

Our manuscript describing the optimization of a bespoke base editor to treat a severe pediatric vascular disease is now published in @natBME 🫀🧬 See my previous Xtorial below for details about this massive collaborative effort nature.com/articles/s41551-0…

Next Generation AAV-F Capsid gene therapy rescues disease pathology in a model of Pyruvate Dehydrogenase Complex Deficiency biorxiv.org/content/10.1101/…

– Pardoned January 6 rioters who violently assaulted police officers – Deputized Elon Musk to dig through your personal health and financial information – Froze funding for cancer research, Meals on Wheels, and disaster relief – Canceled contracts that American farmers were relying on – Shut down the agency that goes after scammers and predatory lenders – Shut down the agency that provides food and medicine to sick kids – Shut down Social Security offices across the country – Fired the experts in charge of overseeing the U.S. nuclear stockpile – Fired the top ethics official in charge of preventing conflicts of interest in government – Fired the Inspectors General who root out fraud and corruption in the government – Refused to fire advisors who disclosed classified military information in a group chat – Refused to fire advisor who said don't complain if your Social Security check doesn't come – Refused to fire advisor who said Social Security is the biggest Ponzi scheme of all time – Halted enforcement of the law to prevent bribery of government officials – Pocketed millions of dollars from pumping his own personal meme coin – Defied orders from federal judges and the Supreme Court – Crashed the stock market – Raised prices on groceries, clothing, and cars – Backed a bill to slash Medicaid – Deported a U.S. citizen with stage 4 cancer, without his medication. He's 4-years-old.

RT @mehdirhasan: Who voted for the cutting of funds to cancer research? Can someone show me those voters?