🧬The second question of the day focuses on how patient-reported outcomes reflect clinical benefit after Casgevy therapy.

#Casgevy #GeneTherapy #CRISPRGeneEditing #SickleCellGeneTherapy #SickleCellDisease #Hematology #CellTherapy #TranslationalMedicine #ClinicalTrials #RareDiseases #BloodDisorders

🧬 Today’s first question focuses on interpreting hemoglobin expression patterns following Casgevy treatment.

See the answer and explanation on the next slide!

#Casgevy #GeneTherapy #CRISPRGeneEditing #SickleCellDisease #Hematology #CellTherapy #TranslationalMedicine #ClinicalTrials #RareDiseases

Additional results from the Exa-cel study.

Focus: CLIMB SCD-121 Trial

#Casgevy #GeneTherapy #CRISPRGeneEditing #SickleCellGeneTherapy #SickleCellDisease #Hematology #CellTherapy @ash.hematology.org @asgct.bsky.social @genesndiseases.bsky.social @bsochaem.bsky.social @ash.edu.hematology.org

Our session with Dr. Thomas Coates is in 2 weeks!

Don’t miss this opportunity to hear from a top expert in the field!

🔗Register now:
hemehub.org/live-events

#Casgevy #GeneTherapy #CRISPRGeneEditing #SickleCellGeneTherapy #SickleCellDisease #Hematology #CellTherapy #TranslationalMedicine

Further results from the Exa-cel study.

Focus: CLIMB SCD-121 Trial

#Casgevy #GeneTherapy #CRISPRGeneEditing #SickleCellGeneTherapy #SickleCellDisease #Hematology #CellTherapy #TranslationalMedicine #ClinicalTrials #RareDiseases #BloodDisorders #StemCellTherapy #HematopoieticStemCells

🩸 Today’s question explores laboratory indicators of treatment success with Lyfgenia, focusing on the clinical significance of normalized bilirubin and reticulocyte counts.

See the answer and explanation on the next slide!

#Casgevy #GeneTherapy #CRISPRGeneEditing #SickleCellDisease #Hematology

🩸 Today’s question addresses long-term safety considerations for patients treated with Lyfgenia, focusing on which risks require ongoing surveillance.

#Casgevy #GeneTherapy #CRISPRGeneEditing #SickleCellGeneTherapy #SickleCellDisease #Hematology #CellTherapy #TranslationalMedicine #ClinicalTrials

Additional facts about the Exa-cel (Exagamglogene Autotemcel, Casgevy) Study

Focus: CLIMB SCD-121 Trial

#Casgevy #GeneTherapy #CRISPRGeneEditing #SickleCellGeneTherapy #SickleCellDisease #Hematology #CellTherapy #TranslationalMedicine #ClinicalTrials #RareDiseases #BloodDisorders

Facts about the Exa-cel (Exagamglogene Autotemcel, Casgevy) Study

Focus: CLIMB SCD-121 Trial

#Casgevy #GeneTherapy #CRISPRGeneEditing #SickleCellGeneTherapy #SickleCellDisease #Hematology #CellTherapy #TranslationalMedicine #ClinicalTrials #RareDiseases #BloodDisorders #StemCellTherapy

🩸 Today’s question explores the expected clinical trajectory and hemoglobin response in patients following treatment with Lyfgenia.

See the answer and explanation on the next slide!

#Casgevy #GeneTherapy #CRISPRGeneEditing #SickleCellDisease #CellTherapy #Hematology #ClinicalTrials

SCD and Gene Therapy:

Additional facts about the LentiGlobin Study

#Lyfgenia #GeneTherapy #CRISPRGeneEditing #SickleCellDisease #Hematology #CellTherapy #RareDiseases #BloodDisorders #MedicalEducation #InternalMedicine

From Canada with AI robotic self-driving laboratory👹

LUMI-Lab
Autonomous development of #LipidNanoParticle for #CRISPRGeneEditing delivery

LUMI-6 (brominated lipid, intratracheal)➡️
🐭Lung editing efficiency (epithelial 20.3%)

@bowang87.bsky.social cial bioRxiv 2025
www.biorxiv.org/content/10.1...