Eric Minikel

Cambridge

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Articles

One woman's hard pivot after receiving a devastating diagnosis

Oct 18, 2024 | wglt.org | Sonia Vallabh |Eric Minikel

Part 2 of the TED Radio Hour episode Approaches to life: Improvise, pivot or planWhen Sonia Vallabh learned she has the genetic mutation for prion disease, she and her husband dropped everything to change careers. Today, they lead a Harvard/MIT lab searching for a cure. About Sonia VallabhSonia Vallabh runs a prion research laboratory at the Broad Institute of MIT and Harvard alongside her husband, Eric Vallabh Minikel.
A big step forward for brain delivery of gene therapies

May 22, 2024 | cureffi.org | Eric Minikel

For years, Sonia and I have been getting the question in Q&A at our talks, “Why isn’t this a solved problem thanks to CRISPR?”. In 2019 I wrote a blog post about what gene therapy can & can’t do to try to sum up why it’s not so simple. There were/are a tremendous number of ideas (CRISPR is one of them) about what a gene therapy could do for prion disease conditioned on getting into the right cells.
New gene delivery vehicle shows promise for human brain gene therapy

May 16, 2024 | einpresswire.com | Eric Minikel

In an important step toward more effective gene therapies for brain diseases, researchers from the Broad Institute of MIT and Harvard have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein. Because the vehicle binds to a well-studied protein in the blood-brain barrier, the scientists say it has a good chance at working in patients.
End of the beginning

Mar 21, 2024 | cureffi.org | Eric Minikel

It’s now been 3 months since the first site began recruiting for Ionis’s ION717 ASO trial in prion disease (NCT06153966). A total of 9 trial sites are now recruiting worldwide, and today I was brought to tears by the description in this news article of some of the first brave people who’ve volunteered for the trial. I want to take this moment to share some reflections on what this milestone means and what lies ahead.
First ION717 trial site launches

Dec 21, 2023 | cureffi.org | Eric Minikel

This morning, Ionis Pharmaceuticals posted a small but important update to NCT06153966: the first trial site in Cleveland, OH is now recruiting:For those just tuning in now, Sonia and I are not affiliated with Ionis. We are scientists at the Broad Institute working to develop a drug for prion disease, for which Sonia is at risk.
First details of Ionis trial disclosed

Dec 4, 2023 | cureffi.org | Eric Minikel

Two months ago, Ionis Pharmaceuticals announced that it planned to launch a first-in-human trial nicknamed PrProfile (pronounced “profile”) of a PrP-lowering antisense oligonucloetide (ASO) by the end of the year. Last week, it posted the trial to ClinicalTrials.gov as A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717 (NCT06153966). (The contents at that link will change over time, so here is a snapshot of what it looked like today).
Hope for prion diseases

Nov 29, 2023 | drugdiscoverynews.com | Hannah Thomasy |Sonia Vallabh |Eric Minikel

Today, biologist Sonia Vallabh is an influential figure in the field of prion research. She and her husband, Eric Minikel, founded the Prion Alliance at the Broad Institute of MIT and Harvard to spearhead efforts to develop therapies for prion disease. Neither Vallabh nor Minikel began their careers planning to study prion disease or even planning to work in biology. In 2010, the couple were recently married.
Ionis re-commits to new timeline for prion disease ASO trials

Oct 4, 2023 | cureffi.org | Eric Minikel

Today Ionis Pharmaceuticals doubled down on its commitment to prion disease and announced a new timeline for a first-in-human clinical trial. Details are still limited but this blog post will unpack what we know and what we can expect. For those just tuning in now: Sonia and I are not affiliated with Ionis Pharmaceuticals. We are patient-scientists trying to cure Sonia’s disease from our laboratory at the Broad Institute of MIT and Harvard.
‘wildDISCO’ whole-mouse mapping; high-resolution imaging of living brain tissue

Jul 16, 2023 | spectrumnews.org | Michael Fergenson |Eric Minikel

Illustration by Laurène Boglio Ali Ertürk of Ludwig-Maximilians-Universität München detailed his team’s study, “Whole-body cellular mapping in mouse using standard IgG antibodies,” published in Nature Biotechnology 10 July. John-Paul Fuller-Jackson of the University of Melbourne and Kateryna Shkarina of the Institute of Innate Immunity were among the many who responded to Ertürk. The Allen Institute shared a link to its new resource, the Allen Brain Cell Atlas.
Duration of prodrome and of disease

May 17, 2023 | cureffi.org | Eric Minikel

Late last year, I blogged about a new pre-print from the London study of pre-symptomatic people at risk for genetic prion disease. That study was just officially published [Mok 2023].