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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · 17d

I’m honored to be speaking at the 14th WFH Global Forum in Montreal, Nov 13–14, 2025! 🌍 Join us for global insights on #BleedingDisorders care—covering #GeneTherapy, #VWD, safety, and more—all through the lens of equitable access.

Learn more 👉
wfh.org/global-forum/

WFH 2025 Global Forum

QUICK LINKS About the Global Forum Governmental Welcome Agenda Registration information Committee & Speakers Venue Travel Global Forum News Sponsors Advances and access to safe treatment products for ...

wfh.org

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jul 30

I don’t even know where to start.

Trump administration blocks NIH from awarding any research grants and contracts www.statnews.com/2025/07/29/t... via @statnews.com

Trump administration blocks NIH from awarding any research grants and contracts

The order applies to the entirety of new and, possibly, ongoing research grant dollars that go to universities and academic medical centers.

www.statnews.com

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jul 29

As #hemophilia B therapeutics become more sophisticated, there is an ⬆️ need to better understand genotype–phenotype relationships. Our in vitro studies are consistent with recent in silica approaches:

www.sciencedirect.com/science/arti...

“An integrated multitool analysis contributes elements to interpreting unclassified factor IX missense variants associated with hemophilia B”: comment from Lee et al.

www.sciencedirect.com

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 25

We have the best hematologists at @chopresearch.bsky.social

#ISTH2025 @hilarywhitworth.bsky.social

Black and white picture of smiling pediatric hematologists from the oldest pediatric hospital in the US.

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 24

Impaired fibrinolysis in APS, provides mechanism for elevated rate of CTEPH in APS.

#ISTH2025

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 24

OC 37.5

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 24

Brilliant visualization of extravascular FIX and very interesting (and surprising) differences in specific activity of extravascular FIX and FX in different tissues. 🤯

#ISTH2025 #hemesky

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 22

Cirrhosis seems like another reason to use apixaban.

SSC 10 at #ISTH2025

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 22

😳

Thrilled we finally have a apixaban pediatric FDA approval to implement these changes.

Saskia Middeldorp @middeldorps.bsky.social · Jun 22

This is what we were waiting for. A direct comparison between apixaban and rivaroxaban for the treatment of acute VTE. Apixaban reduces bleeding risk in the first 3 months by >50%!

Practice changing investigator-initiated RCT.

#ISTH2025

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 22

Potential emerging signal difference in ADAs in next-generation FVIII(a)-mimetics. 0/61 for Mim8 but 2/30 for NXT007. Surveillance may not be the same.

#ISTH2025 #hemophilia #hemesky

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 22

#Hemophilia A patient treated with Hympavzi developed spontaneous DVT 3 years into treatment.

Factor V Leiden heterozygous and sedentary lifestyle identified as risk factor.

#ISTH2025

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 22

Though pediatric #thrombosis is also distinct from adults

Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 22

Pediatric #thrombosis guidelines, guidance, and clinical trials need to distinguish between older and younger children.

#ISTH2025 #hemesky

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 21

“Transformative” is a great phrasing! Thanks.

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 21

Sabatino from @chopresearch.bsky.social giving a comprehensive overview of genotoxicity after AAV #genetherapy.

Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 21

Rate of immunosuppression after #genetherapy for #hemophilia B with #Hemgenix in the real-world seems higher than the ~20% rate reported in clinical trial. Though small numbers here, also consistent with anecdotal results.

We will need more rigorous real-world data.

#ISTH2025 #hemesky

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Reposted by Ben Samelson-Jones, MD, PhD

Scary Lannert @klannert.bsky.social · Jun 21

Compelling evidence presented at #isth2025 by Sebastian Leyes Porello from @samelsonjones.bsky.social’s group showing mim8 can enhance activity in hemophilia B variants in vitro, above enhancement this group saw with emicizumab. Curious about in vivo data teased during the Q&A 👀
#hemesky @isth.org

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 21

Nuanced points about the use the term “Cure” when discussing #genetherapy for #hemophilia at #ISTH2025.

I agree about avoiding “cure” in clinical setting but really liked that this might be different in discussions about Allocations or Research Funding.

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 21

IMO biggest update at Guideline presentation at #ISTH2025 is this recommendation for primary #VTE prophylaxis in children receiving long-term TPN.

I wish there was better GUIDANCE on intensity and type of anticoagulation for this recommendation.

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 21

Also on Wednesday:
SOA 22.2 - Use of bispecific antibodies for select #hemophilia B mutations

OC 69.3 - Safe multi-year bleeding reduction by AAV gene therapy in neonatal and juvenile #Hemophilia B dogs

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 21

Start off today with
OC 03.1 - Mim8 Enhances Procoagulant Activity of Select #Hemophilia B-causing Factor IX Variants

And finish on Wednesday with
OC 69.5 - Six-year outcomes following fidanacogene elaparvovec in adults with #hemophilia B

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 21

Can't wait for #ISTH2025 to start! Our group from
@chopresearch.bsky.social is presenting the first abstract and the last, and a few in between, all about #hemophilia B.

#Hemesky

Action shots of people enjoying ISTH events with a badge in the middle saying FOLLOW ME FOR UPDATES FROM #ISTH2025

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · Jun 11

Looking forward to #ISTH2025 and especially this session with Dom.

#hemophila #genetherapy

International Society on Thrombosis and Haemostasis @isth.org · Jun 11

[Supported Post] Join CSL Behring on June 22nd at 1:45pm for an insightful review of the value of Factor IX replacement in hemophilia B management. #ISTH2025 medicalaffairs.cslbehring.com/diseases-and...

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · May 22

I discuss our #asgct2025 abstract highlighting our multidisciplinary team approach to care for #genetherapy recipients.

Stay tuned, lots more to come.

www.cgtlive.com/view/samelso...

Ben Samelson-Jones, MD, PhD, on Conservative Management Following Treatment With DMD Gene Therapy Elevidys

The associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia discussed a case he presented at the American Society of Gene and Cell Therapy’s 2025 meeting.

www.cgtlive.com

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Ben Samelson-Jones, MD, PhD @samelsonjones.bsky.social · May 20

Nice article about ongoing work to bring #genetherapy to pet dogs with #hemophilia. Includes the importance of the research colonies in developing new therapies, both for humans and 🐶. Cute pictures!

Veterinarian aims to bring hemophilia treatment to pet dogs - News news.vin.com/default.aspx...

Veterinarian aims to bring hemophilia treatment to pet dogs

Effort to fund new gene therapy study spotlights rare disease with elusive remedy

news.vin.com

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