Ben Samelson-Jones, MD, PhD

Receiving gene therapy for hemophilia changed this South Jersey man's life The novel treatment, partly developed by CHOP, eliminates the need for regular infusions while preventing the life-threatening bleeds caused by the condition.

#genetherapy for #hemophilia can be life changing. Proud to offer this transformative treatment at @childrensphila.bsky.social

Gene therapy changed the life of a South Jersey man with hemophilia | PhillyVoice www.phillyvoice.com/gene-therapy...

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 ...

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/

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.

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

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

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...

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

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

#ISTH2025 @hilarywhitworth.bsky.social

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

#ISTH2025

OC 37.5

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

#ISTH2025 #hemesky

Cirrhosis seems like another reason to use apixaban.

SSC 10 at #ISTH2025

😳

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

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

#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

Though pediatric #thrombosis is also distinct from adults

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

#ISTH2025 #hemesky

“Transformative” is a great phrasing! Thanks.

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

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

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

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.

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.

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

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

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

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

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

#hemophila #genetherapy

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.

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...

Veterinarian aims to bring hemophilia treatment to pet dogs Effort to fund new gene therapy study spotlights rare disease with elusive remedy

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...

“Hi” was my daughter’s first word.

Williams Syndrome: The people who are too friendly People with Williams Syndrome treat strangers as their new best friends. Now the condition is revealing clues to our evolutionary past – and what makes us human.

#williams syndrome is a rare genetic disorder with a fascinating but challenging neurocognitive profile.

www.bbc.com/future/artic...

For the first time in 16 years, ISTH is returning to the U.S. for an in-person Congress. As we prepare to welcome the world to Washington, D.C., we’re guided by our mission & appreciation for our community🌍

Read more @jthjournal.bsky.social: www.jthjournal.org/article/S1538-7836(25)00213-2/abstract

It is essential for #genetherapy progress for unsuccessful clinicals trials to be published. We discuss recent study showing no benefit for prophylactic steroids after #AAV for #hemophilia.

authors.elsevier.com/a/1k-re5TuFx...

w/ @rkaczmarekdr.bsky.social