La prima terapia CRISPR funziona ma va a rilento, ecco perché A due anni dall’approvazione commerciale di Casgevy solo una sessantina di persone affette da anemia falciforme o talassemia hanno potuto beneficiarne, per via di un problema tecnico che la prossim…

Il primo trattamento CRISPR approvato nel mondo è stato salutato (giustamente) come un evento storico, ma a due anni di distanza dall'autorizzazione sono troppo poche le persone che hanno potuto riceverlo. Circa 60. Cosa non ha funzionato? E c'è rimedio? #casgevy crispr.blog/2026/02/09/l...

We welcome the SMC decision to recommend exa-cel (Casgevy) for severe sickle cell disorder in Scotland.

For more information and to submit your expression of interest for our gene therapy webinars, visit:
www.sicklecellsociety.org/gene-therapy...

#SickleCell #Casgevy #GeneTherapy #Scotland

Medicina del mañana: innovaciones médicas y biotecnología clave Cómo la biotecnología, la genómica, CRISPR y el ARNm están transformando la medicina: terapias avanzadas, diagnóstico preciso y salud del futuro.

De laboratorio a paciente en 11 años. Así pasó CRISPR de ser un hallazgo científico a una terapia aprobada que cura la anemia falciforme. La velocidad de la innovación médica se ha disparado
www.cincofrentes.com/2026/01/tecn...

#CRISPR #TerapiaGénica #MedicinaDePrecisión #Casgevy #Ciencia #Genética

ToolGen Secures U.S. Patent and Initiates Lawsuit on CASGEVY® for Fair Recognition ToolGen, a leader in genome editing, secures a U.S. patent for CRISPR technology and launches a lawsuit to protect its innovations, particularly CASGEVY®.

ToolGen Secures U.S. Patent and Initiates Lawsuit on CASGEVY® for Fair Recognition #South_Korea #Seoul #ToolGen #CRISPR #CASGEVY

🧬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

General Facts about Gene Therapy:

#LentiGlobin and #Exa-cel studies

#Casgevy #Lyfgenia #GeneTherapy #CRISPR #SickleCellDisease #Hematology #CellTherapy #ClinicalTrials
@ash.edu.hematology.org @asgct.bsky.social @cfgtedinburgh.bsky.social @genesndiseases.bsky.social
@bsochaem.bsky.social

🧬 Today's second question highlights the significance of neutrophil engraftment timing in #Casgevy studies.

See the answer and explanation on the next slide!

#GeneTherapy #SickleCellDisease #Hematology #CellTherapy #TranslationalMedicine #ClinicalTrials #RareDiseases #BloodDisorders #StemCells

🧬 Today's first question focuses on the safety profile of #Casgevy, examining which adverse events have not been reported in clinical trials to date.

See the answer and explanation on the next slide!

#GeneTherapy #SickleCellDisease #Hematology #CellTherapy #RareDiseases #ClinicalTrials

🧬The second question of the day explores key predictors of lasting clinical benefit after Casgevy therapy.

See the answer and explanation on the next slide!

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

Further results from the Exa-cel study.

Focus: CLIMB SCD-121 Trial

#Casgevy #GeneTherapy #SickleCellDisease #Hematology #CellTherapy #BloodDisorders @ash.edu.hematology.org @asgct.bsky.social @dgsomucla.bsky.social @dukemedschool.bsky.social @harvardmed.bsky.social @yaleschoolofmed.bsky.social

🧬 Second question of the day focuses on understanding breakthrough vaso-occlusive events in patients treated with Casgevy and what they reveal about post-therapy physiology.

See the answer and explanation on the next slide!

#Casgevy #GeneTherapy #SickleCellDisease #Hematology #CellTherapy

🧬 Today’s first question highlights the importance of pancellular HbF expression after Casgevy.

See the answer and explanation on the next slide!

#Casgevy #GeneTherapy #SickleCellDisease #Hematology #CellTherapy #TranslationalMedicine @bsochaem.bsky.social @asgct.bsky.social @ash.hematology.org

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

ToolGen Files Patent Infringement Lawsuit Against Lonza in the Netherlands ToolGen, a leader in genomic editing technology, has initiated a patent infringement lawsuit against Lonza Netherlands, securing its intellectual property rights.

ToolGen Files Patent Infringement Lawsuit Against Lonza in the Netherlands #Netherlands #ToolGen #CRISPR #The_Hague #CASGEVY

ToolGen Initiates Legal Action Against Lonza for Patent Violations in the Netherlands ToolGen has filed a lawsuit against Lonza in the Netherlands for patent infringement related to crucial genome editing technology. This legal move enhances ToolGen's IP protection.

ToolGen Initiates Legal Action Against Lonza for Patent Violations in the Netherlands #Netherlands #ToolGen #The_Hague #CASGEVY #CRISPR-Cas9

This paper draws on ethnographic observation and discourse analysis of public conferences on genome editing and meetings of the FDA on the evaluation of #Casgevy, the first CRISPR-based treatment for #sicklecell to be approved.

Vertex, CRISPR Therapeutics Land On TIME's Most Influential Companies List - IndiaWest Journal News Vertex, CRISPR Therapeutics Land On TIME's Most Influential Companies List.

Vertex, CRISPR Therapeutics Land On TIME's Most Influential Companies List

Full Story: indiawest.com/vertex-crisp...

#VertexPharmaceuticals #CRISPRTherapeutics #Casgevy #SickleCellTreatment #BetaThalassemiaCure #GeneEditingInnovation #FDAApprovedTherapy #ExaCel

This is more than innovation — it’s equity in action.
Let’s make sure no one is left behind. ✊🏾

#SickleCellAwareness #GeneTherapy #Casgevy #Lyfgenia #HealthEquity #CRISPR #MedicaidMatters #BlackHealthMatters #FutureOfMedicine #CMS #ValueBasedCare #MedicalInnovation #ChronicIllnessWarrior

CASGEVY: Rethinking Drug Costs Through the Lens of Public Access Maria Klimenko considers the extent to which the price of innovative treatments like CASGEVY, a type of gene therapy, should reflect not just the cost of development, but also their broader societa…

#CASGEVY: Rethinking #Drug Costs Through the Lens of Public Access - by Maria Klimenko.
wp.me/p38o3S-1QO

ToolGen Takes Legal Action Against Vertex for Patent Infringement in the UK ToolGen has filed a lawsuit in the UK against Vertex Pharmaceuticals for patent infringement related to their groundbreaking CRISPR technology used in the CASGEVY therapy.

ToolGen Takes Legal Action Against Vertex for Patent Infringement in the UK #None #United_Kingdom #ToolGen #Vertex_Pharmaceuticals #CASGEVY

ToolGen Initiates Patent Infringement Lawsuit Against Vertex in the UK Over Genome Editing Technology ToolGen, Inc. has filed a lawsuit against Vertex Pharmaceuticals for patent infringement related to its CRISPR RNP technology used in CASGEVY, a pioneering genome editing therapy.

ToolGen Initiates Patent Infringement Lawsuit Against Vertex in the UK Over Genome Editing Technology #London #United_Kingdom #ToolGen #Vertex #CASGEVY