#BetaThalassemia hashtag - Bluesky

New studies in ASH’s Blood Advances show significant quality-of-life gains for patients with #SickleCell & #BetaThalassemia.

From hospital beds to classrooms & workplaces, read how CRISPR is giving new opportunities to pts.

#GeneTherapy #ExaCel #CRISPR #Hematology

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Agentic Tribune

@agentictribune.bsky.social

9 months ago

Breakthrough CRISPR Gene Therapy Casgevy Gains Global Approval for Blood Disorders CRISPR therapy Casgevy approved in UK, US, EU to treat sickle cell and beta-thalassemia, marking a gene-editing milestone.

Breakthrough CRISPR Gene Therapy Casgevy Gains Global Approval for Blood Disorders

#genetherapy #crispr #sicklecell #betathalassemia

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Donna E

@edwardsdna.bsky.social

11 months ago

First Patient to Receive Gene Therapy ‘Cure’ for Beta-Thalassemia Living Pain-Free In 2020, GNN reported that CRISPR gene editing technology was being used to develop a medicine for beta-thalassemia, a genetically-inherited blood disorder. Now though, another medicine—a ‘cure’—has already been used to treat a young girl, and more individuals are lined up for treatment. Developed at the Children’s Hospital of Philadelphia (CHOP) and called Zynteglo, the […] The post First Patient to Receive Gene Therapy ‘Cure’ for Beta-Thalassemia Living Pain-Free appeared first on Good News Network.

First Patient to Receive Gene Therapy ‘Cure’ for Beta-Thalassemia Living Pain-Free: In 2020, GNN reported that CRISPR gene editing technology was being used to develop a medicine for… @goodnewsnetwork.org #GeneTherapy #BetaThalassemia #CRISPR #HealthcareInnovation #MedicalBreakthrough

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Cozzy Energy Solutions

@energysolutions.bsky.social

1 year ago

CRISPR Therapeutics Announces Promising Trial Results for Gene Therapy Treatment for Sickle Cell Disease and Beta-Thalassemia - Cozzy Energy Solutions CRISPR Therapeutics Announces Promising Trial Results for Gene Therapy Treatment A leading gene editing company has made significant strides in developing a treatment for two inherited blood disorders. CRISPR Therapeutics AG announced positive results from its clinical trial of CTX001, a dual CRISPR-Cas9 gene therapy designed to correct genetic mutations that cause sickle cell disease and beta-thalassemia. The trial involved 21 patients treated with CTX001 using an electroporation-based delivery method. The outcomes were encouraging, with no adverse events reported among the recipients. Furthermore, significant improvements in hematological parameters were observed, including a notable reduction in transfusion requirements. These results suggest that CTX001 may offer a transformative treatment option for individuals affected by these conditions. According to Dr. David Liu, Chief Scientific Officer at CRISPR Therapeutics, "Today's results demonstrate the promise of CTX001 as a potentially game-changing therapy." The company plans to submit a Biologics License Application (BLA) in the United States and Europe pending regulatory review. The development of CTX001 marks an exciting milestone for gene editing technology. Its potential to transform the lives of patients with sickle cell disease and beta-thalassemia is substantial, offering new hope for those affected by these conditions.

CRISPR Therapeutics Announces Promising Trial Results for Gene Therapy Treatment for Sickle Cell Disease and Beta-Thalassemia #PJM #CRISPR #SickleCellDisease #GeneTherapy #BetaThalassemia #GeneticDisorders

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American Society of Gene + Cell Therapy

@asgct.bsky.social

1 year ago

🩸 January is #NationalBloodDonorMonth! Many rare blood disorders are managed by receiving regular blood transfusions. We honor those who are able to donate their time and give hope to people with disorders like #SickleCell and #BetaThalassemia. patienteducation.asgct.org/gene-therapy...

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Manal Mehta

@manalmehta.bsky.social

5 years ago

🚨🚨 $SGMO CATALYST ALERT: @Sanofi has dosed first #Sicklecell patient. Data: "start of next year." #BetaThalassemia w/ Sickle data to move products forward together. "work going between us and $SNY...we are confident that there's a way forward here"

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