Etranacogene Dezaparvovec May Yield Long-Term Factor IX Expression Improvement in Hemophilia B Researchers determined 1 infusion of etranacogene dezaparvovec may lead to long-term sustained endogenous factor IX expression and low ABRs in hemophilia B.

One infusion of etranacogene dezaparvovec may lead to long-term sustained endogenous factor IX expression and low annualized bleeding rates among patients with #hemophilia B. Published in @nejm.org.

https://bit.ly/4ucPZ4W

#hemophiliaB

Patients With Hemophilia B Report Improvement in QoL After Gene Therapy A majority of patients with hemophilia B reported that their impression of life with hemophilia was “greatly improved” after gene therapy with fidanacogene elaparvovec.

After a single infusion of fidanacogene elaparvovec, a majority of patients with #hemophiliaB reported that their impression of life with #hemophilia was “greatly improved” and demonstrated gains in health-related QoL measures. From @jthjournal.bsky.social.

https://bit.ly/49kPq0D

Gene Therapy Shows Durable 5-Year Protection in Adults With Hemophilia B One infusion of etranacogene dezaparvovec-drlb for hemophilia B provides lasting bleed protection, stable FIX for 5 years, and a favorable safety profile.

Nearly all (94%) responders were able to discontinue continuous prophylactic #FIXTherapy, and exogenous FIX use dropped by 96%.

Read here: https://bit.ly/4a6LqS2

#RareDisease #Hemophilia #Hematology #Hemgenix® #HemophiliaB

Turning genes into medicines - Nature Medicine The first clinical trial of a gene therapy infusion for hemophilia B revealed unexpected findings.

A landmark clinical trial of gene therapy infusion for hemophilia B revealed surprising findings—reshaping the future of genetic medicine.
Read the full Turning Point essay in Nature Medicine: https://ow.ly/RVST50XvVs0
#GeneTherapy #HemophiliaB #NatureMedicine

Fidanacogene Eleparvovec Improves Clinical Parameters in Hemophilia Gene therapy remains a promising therapeutic innovation in the management of patients diagnosed with hemophilia.

#GeneTherapy with fidanacogene eleparvovec is useful for improving the overall health and functional status of patients with #HemophiliaB, in addition to elevating health-related quality of life parameters. Study in @jthjournal.bsky.social

Read more: https://bit.ly/47Orssr

#RareDisease #Hemophilia

Novo Nordisk’s stock rises with broad US FDA approval for Alhemo Alhemo wins FDA nod for hemophilia A and B without inhibitors, showing up to 86% fewer bleeds in a Phase III trial.

The US FDA has expanded approval for Novo Nordisk’s Alhemo, offering a once‑daily, subcutaneous option for hemophilia A & B patients without inhibitors. In trials, it cut bleeding episodes by up to 86%.
#news #HemophiliaCare #FDAApproval #Hemophilia #HemophiliaA #HemophiliaB #BleedingDisorders

Novo Nordisk’s stock rises with broad US FDA approval for Alhemo Alhemo wins FDA nod for hemophilia A and B without inhibitors, showing up to 86% fewer bleeds in a Phase III trial.

The US FDA has expanded approval for Novo Nordisk’s Alhemo, offering a once‑daily, subcutaneous option for hemophilia A & B patients without inhibitors. In trials, it cut bleeding episodes by up to 86%.
#news #HemophiliaCare #FDAApproval #Hemophilia #HemophiliaA #HemophiliaB #BleedingDisorders

🎉 Congrats to member hospital Loma Linda University Children's Hospital for becoming the first in California to offer FDA-approved immunotherapy for Hemophilia B—giving patients a more convenient, less invasive treatment option.

🔗 bit.ly/3XRmDut
#HemophiliaB #CCHA #MedicalInnovation #PatientCare

My most important take-home from long-term follow-up of Spark/Pfizer #hemophiliaB #AAV gene therapy study is that alcohol consumption leads to loss in activity (likely due to liver regeneration).

www.nejm.org/doi/pdf/10.1...

An integrated multitool analysis contributes elements to interpreting unclassified factor IX missense variants associated with hemophilia B Dissection of genotype–phenotype relationships in hemophilia B (HB) is particularly relevant for challenging (mild HB) or for HB-associated but unclassified factor (F)IX missense variants.

Researchers in Italy and China used a multitool #bioinformatic approach to identify new F9 gene mutations that cause #HemophiliaB and explored their effects on disease severity.
www.jthjournal.org/article/S153...

See also: www.jthjournal.org/article/S153...

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,...

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/...

Steven Pipe, MD, Professor of Pediatrics and Pathology at the University of Michigan, discusses positive follow-up results in patients with hemophilia B treated with Hemgenix (etranacogene dezaparvovec-drlb).
checkrare.com/positive-fol...

#CheckRare #HemophiliaB #RareHematology #RareDisease

My main concern is what is the significance of elevated liver enzymes..which in this trial often went untreated and persisted?

Does this indicate there's nothing to worry about after all? #hemophiliaB #genetherapy

Maybe there is HOPE for #HemophiliaB gene therapy Hemgenix by #CSL.AX (licensed from #$QURE) maintains consistent Factor IX levels (at least the 'mean') over 4 years; after

year 1: 41.5 IU/dL
y2: 36.7
y3: 38.6
y4: 37.4

CSL also sees increased uptake.

finance.yahoo.com/news/csl-beh...

Man first in Indiana to receive gene therapy for hemophilia B Man becomes first in Indiana to undergo gene therapy for hemophilia B. The procedure aims to address the root cause of the disorder.

This week at Innovative Hematology a 47-year-old man made history as the first person in Indiana to undergo gene therapy for hemophilia B.

wishtv.com/news/health-...

Special thanks to WISH-TV.

#genetherapy #hemophilia #hemophiliab #hematology
#CSL

Multiplex, multimodal mapping of variant effects in secreted proteins bioRxiv - the preprint server for biology, operated by Cold Spring Harbor Laboratory, a research and educational institution

I am beyond thrilled to share this preprint with Nicholas Popp from Doug Fowler’s lab. I can’t wait to see where MultiSTEP goes next! 🧬🔬

Take a look here! ⤵️
www.biorxiv.org/content/10.1...

#deepmutationalscan #dms #vampseq #factorix #factor9 #hemophiliab