Share this page!

Enter URL or ID to Unroll

You can paste full URL like: https://x.com/threadreaderapp/status/1644127596119195649
or just the ID like: 1644127596119195649

How to get URL link on X (Twitter) App

  1. On the Twitter thread, click on or icon on the bottom
  2. Click again on or Share Via icon
  3. Click on Copy Link to Tweet
  4. Paste it above and click "Unroll Thread"!
  5. More info at Twitter Help
Ben Samelson-Jones, MD, PhD Profile picture
Ben Samelson-Jones, MD, PhD
@DrSamelsonJones
Pediatric Hematologist @childrensphila | Gene Therapist | Husband | 👩👦👧👶 🐕 Dad | #WilliamsSyndrome advocate | Views my own
Jul 22, 2022 • 7 tweets • 5 min read
@chowdarypm, @ProfMakris, and others report another successful #genetherapy for #hemophilia B clinical trial using the hyperactive FIX variant Padua.
tinyurl.com/346ty35v

🧵 on the clinical development of FIX Padua in gene therapy:

1/7 This is now the 4th successful Phase1/2 #genetherapy study using #AAV vectors using FIX Padua. The initial demonstration of the benefit of FIX Padua in #genetherapy for #hemophilia B was in a study led by @LindseyAGeorge.

nejm.org/doi/full/10.10…

2/7
Jul 12, 2022 • 4 tweets • 2 min read
Thrombin generation assays doing a lot of work at non factor therapy for #hemophilia session at #Isth2022 demonstrating

1/4 🧵

1️⃣ Stability of antithrombin knockdown in subjects receiving fitusiran Image 2️⃣ Increased potency of Mim8 compared to emicizumab in phase 1 subjects Image