Ben Samelson-Jones, MD, PhD
@samelsonjones.bsky.social
Pediatric Hematologist 🧪🩸| Gene Therapist | Biochemist | 👩🦱👨👩👦🐕 Dad | Husband | Williams Syndrome Advocate
#Medsky #Hemesky
#Medsky #Hemesky
#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...
Gene therapy changed the life of a South Jersey man with hemophilia | PhillyVoice www.phillyvoice.com/gene-therapy...
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.
www.phillyvoice.com
October 17, 2025 at 10:12 PM
#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...
Gene therapy changed the life of a South Jersey man with hemophilia | PhillyVoice www.phillyvoice.com/gene-therapy...
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/
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
September 22, 2025 at 9:41 PM
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/
Learn more 👉
wfh.org/global-forum/
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 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
July 30, 2025 at 2:01 AM
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 www.statnews.com/2025/07/29/t... via @statnews.com
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...
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
July 29, 2025 at 11:08 PM
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...
www.sciencedirect.com/science/arti...
June 25, 2025 at 2:42 AM
June 24, 2025 at 9:28 PM
😳
Thrilled we finally have a apixaban pediatric FDA approval to implement these changes.
Thrilled we finally have a apixaban pediatric FDA approval to implement these changes.
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
Practice changing investigator-initiated RCT.
#ISTH2025
June 22, 2025 at 9:21 PM
😳
Thrilled we finally have a apixaban pediatric FDA approval to implement these changes.
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
#ISTH2025 #hemophilia #hemesky
June 22, 2025 at 8:42 PM
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
#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
Factor V Leiden heterozygous and sedentary lifestyle identified as risk factor.
#ISTH2025
June 22, 2025 at 7:32 PM
#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
Factor V Leiden heterozygous and sedentary lifestyle identified as risk factor.
#ISTH2025
Pediatric #thrombosis guidelines, guidance, and clinical trials need to distinguish between older and younger children.
#ISTH2025 #hemesky
#ISTH2025 #hemesky
June 22, 2025 at 12:28 PM
Pediatric #thrombosis guidelines, guidance, and clinical trials need to distinguish between older and younger children.
#ISTH2025 #hemesky
#ISTH2025 #hemesky
Sabatino from @chopresearch.bsky.social giving a comprehensive overview of genotoxicity after AAV #genetherapy.
June 21, 2025 at 8:01 PM
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
We will need more rigorous real-world data.
#ISTH2025 #hemesky
June 21, 2025 at 6:59 PM
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
We will need more rigorous real-world data.
#ISTH2025 #hemesky
Reposted by Ben Samelson-Jones, MD, PhD
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
#hemesky @isth.org
June 21, 2025 at 5:28 PM
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
#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.
I agree about avoiding “cure” in clinical setting but really liked that this might be different in discussions about Allocations or Research Funding.
June 21, 2025 at 4:26 PM
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.
I agree about avoiding “cure” in clinical setting but really liked that this might be different in discussions about Allocations or Research Funding.
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
@chopresearch.bsky.social is presenting the first abstract and the last, and a few in between, all about #hemophilia B.
#Hemesky
June 21, 2025 at 11:57 AM
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
@chopresearch.bsky.social is presenting the first abstract and the last, and a few in between, all about #hemophilia B.
#Hemesky
[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...
June 11, 2025 at 12:16 PM
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...
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
May 22, 2025 at 11:14 AM
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...
Stay tuned, lots more to come.
www.cgtlive.com/view/samelso...
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 - 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
May 20, 2025 at 6:51 PM
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 - News news.vin.com/default.aspx...
#williams syndrome is a rare genetic disorder with a fascinating but challenging neurocognitive profile.
www.bbc.com/future/artic...
www.bbc.com/future/artic...
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.
www.bbc.com
May 15, 2025 at 9:48 PM
#williams syndrome is a rare genetic disorder with a fascinating but challenging neurocognitive profile.
www.bbc.com/future/artic...
www.bbc.com/future/artic...
Reposted by Ben Samelson-Jones, MD, PhD
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
Read more @jthjournal.bsky.social: www.jthjournal.org/article/S1538-7836(25)00213-2/abstract
May 14, 2025 at 5:57 PM
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
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
authors.elsevier.com/a/1k-re5TuFx...
w/ @rkaczmarekdr.bsky.social
authors.elsevier.com
April 30, 2025 at 5:41 PM
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
authors.elsevier.com/a/1k-re5TuFx...
w/ @rkaczmarekdr.bsky.social
In addition to demonstrating long-term durability of rAAV #genetherapy, here we show:
1) Hemostatic potential of FIX Padua with multiple major trauma not needing additional treatments.
2) Importance of limiting hepatotoxic exposures with binge drinking associated with irreversible FIX loss.
1) Hemostatic potential of FIX Padua with multiple major trauma not needing additional treatments.
2) Importance of limiting hepatotoxic exposures with binge drinking associated with irreversible FIX loss.
A paper in @NEJM describes 14 patients with #hemophilia B who had the Pfizer gene therapy. Their achieved FIX levels remained stable over a median of 5.5 years. Confirmation that durability following hemophilia B gene therapy is very different to that in hemophilia A. tinyurl.com/2b2zzc37
April 18, 2025 at 12:29 PM
In addition to demonstrating long-term durability of rAAV #genetherapy, here we show:
1) Hemostatic potential of FIX Padua with multiple major trauma not needing additional treatments.
2) Importance of limiting hepatotoxic exposures with binge drinking associated with irreversible FIX loss.
1) Hemostatic potential of FIX Padua with multiple major trauma not needing additional treatments.
2) Importance of limiting hepatotoxic exposures with binge drinking associated with irreversible FIX loss.
Thrilled to mark World #Hemophilia Day with this paper showing long-term benefits after #AAV gene therapy for #hemophiliaB
prn.to/4iraiEH
Paper at: www.nejm.org/doi/10.1056/...
prn.to/4iraiEH
Paper at: www.nejm.org/doi/10.1056/...
Children's Hospital of Philadelphia Researchers Find Gene Therapy Shows Long-Term Benefits for Hemophilia B Patients
/PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) in collaboration with researchers at Royal Prince Alfred Hospital in Sydney,...
prn.to
April 17, 2025 at 6:21 PM
Thrilled to mark World #Hemophilia Day with this paper showing long-term benefits after #AAV gene therapy for #hemophiliaB
prn.to/4iraiEH
Paper at: www.nejm.org/doi/10.1056/...
prn.to/4iraiEH
Paper at: www.nejm.org/doi/10.1056/...