prionalliance

Today we celebrate the life of Byron Caughey, a beloved friend and generous mentor who made enormous contributions to the fundamental biological understanding, diagnosis, and potential for treatment of prion disease. Blog post: cureffi.org/2026/02/18/cel…

"the longer we spend inside any human system, the more its workings seem inevitable... But it is our job – all of our job – to notice the mirage of inevitability, and call it to account" - Sonia Vallabh prionalliance.org/2025/12/09/the…

Here is Sonia with our semi-annual newsletter prionalliance.org/2025/06/09/all… Donations will be matched 1:1 through end of June!

Today we introduce a new drug candidate for prion disease: a PrP-lowering divalent siRNA. A 4-year collaboration with @RTI_UMassChan enabled by @NINDSfunding IGNITE & URGenT mechanisms. biorxiv.org/content/10.110… Our hope is to advance this to the clinic, wish us luck!

Sonia with our end-of-year update on developing a therapy for prion disease. Two new efforts from our lab: CHARM and divalent siRNA, and an update on the Ionis clinical trial. Support our work with a 1:1 matched donation through end of 2024. prionalliance.org/2024/12/02/whe…

Our mid-year newsletter. The ASO is in the clinic and patients are really showing up. Sonia's TED talk, released today, pleads the case for prevention. Our most ambitious donation matching campaign yet. Let's do this! prionalliance.org/2024/05/30/mid…

We long assumed increases in NfL always meant neuronal damage, and decreases in NfL meant amelioration thereof. The data we report today have now made us think there are exceptions. @medrxivpreprint: medrxiv.org/content/10.110… Blog post: cureffi.org/2024/05/06/wha… 🧵👇

Beautiful, inspiring journalism by @meredithwadman science.org/content/articl… -- the Hoffmans of Reed City, MI are our new heroes and we are so happy for their ray of sunshine!

We've just updated our FAQ, which for years has said there are no ongoing trials in prion disease, to reflect that the first trial of a PrP-lowering ASO is now recruiting prionalliance.org/faq/ Trial posting: clinicaltrials.gov/study/NCT06153… Ionis statement: prionalliance.org/media/2023/10/…

Sonia with our semiannual newsletter. Human trials are finally around the corner. The outcome could be anything. It is the long view that gives us hope. A good time to remember the Stockdale Paradox. prionalliance.org/2023/12/05/end…

I first got to know Sonia Vallabh last year and am honored to call her a friend today. Her story has taught me so much about how to live with extreme uncertainty and still find equanimity. So thankful she’s sharing it with us all today. ❤️: apple.co/46i98VS

As promised, here is a new blog post unpacking what we know so far about the design of Ionis's upcoming PrProfile trial of ION717 in prion disease: cureffi.org/2023/12/04/fir…

Congratulations to Sonia M. Vallabh, Ph.D., and Erik Minkel, Ph.D., the winners of the OTS Paper of the Year Award - Basic Research Category! #ots23 #ots #oligomeeting #oligotherapeutics #biotech #biotechnology #science #research #barcelona @cureffi

Happy June! Here is our mid-year newsletter prionalliance.org/2023/06/01/mid… touching on the scientific progress from all corners that keeps us optimistic.

Prion2023: registrations are open at prion2023.org #Prion2023 @Prion2023 @adpdnet @UAlg @MBExC_de

ASO activity at single cell resolution - now published as a @NAR_Open Breakthrough article: academic.oup.com/nar/advance-ar… Blog post on implications for prion prevention: cureffi.org/2023/05/16/aso… Tweetstorm: twitter.com/cureffi/status… Thank you @OnoPharmaFound for funding this 🙏

If an ASO lowers target RNA 50% in bulk brain tissue, is it 50% in all cells or 100% in half? Are cell types all impacted equally? Does CSF reflect neuronal target engagement? We did single-cell seq in ASO-treated mice & NHPs to find out doi.org/10.1101/2023.0… Thread 👇

Our end-of-year newsletter: prionalliance.org/2022/11/29/end… "We must be constantly vigilant, vocal, and feisty; we must be, to a judicious degree and at the right moments, trouble."

In Part 3 of @NPRShortWave's series on prions, we describe our scientific strategy and the road to a cure npr.org/2022/11/14/113… — thanks @gabrielspitzer and @travlinscientst for working with us to tell our story!

We are honored to tell our story in this episode of @NPRShortWave from @gabrielspitzer & @emilykwong1234 npr.org/2022/11/14/113… - part of a fantastic series they are doing on prions. 2nd half to come next week!