#casgevy — Public Fediverse posts

NEW: #Vertex Pharmaceuticals is suing the gov over blocking access to #fertility preservation services for people getting #Casgevy, the first #CRISPR treatment, which treats #sicklecell & #betathalassemia

Currently, Vertex offering to help patients access these services illegal under anti-kickback laws

via @STAT
https://www.statnews.com/2024/07/15/vertex-casgevy-sickle-cell-infertility-hhs-lawsuit-fertility-preservation/

#health #healthcare #genetherapy #medicine

¿Quieres conocer cómo hemos llegado a aprobar #Casgevy, la primera terapia #CRISPR para tratar enfermedades, desde una investigación básica inicial en arqueas de las salinas de Santa Pola?
Te lo cuento a traves de 10 hitos científicos ocurridos durante 30 años en mi blog #gen_ética en @Naukas_com
https://montoliu.naukas.com/2023/12/17/desde-las-salinas-de-santa-pola-a-casgevy/

Estupendo artículo de @manuelansede hoy en @materia_ciencia @el_pais resumiendo la revolución #CRISPR desde los inicios con Francis Mojica en las salinas de Santa Pola hasta la aprobación de la primera terapia #Casgevy aprobada ya en RU, EEUU y pronto UE.
https://elpais.com/ciencia/2023-12-17/nace-la-medicina-crispr-con-la-promesa-de-salvar-millones-de-vidas.html

Hoy la Agencia Europea del Medicamento (EMA) ha recomendado la autorización de #Casgevy la primera terapia #CRISPR para tratar anemia falciforme y beta talasemia. He publicado una opinión en Science Media Centre ES
https://sciencemediacentre.es/reaccion-la-ema-da-luz-verde-al-primer-medicamento-en-la-ue-que-utiliza-la-edicion-genetica-de

I thought that the fantastical price tag might be keeping some patients from getting #Zynteglo

Turns out, that doesn't seem to be the case (yet), as I detail in a sister piece about Bluebird and the #insurance logistics behind these therapies:

https://www.statnews.com/2023/12/08/lyfgenia-sickle-cell-gene-therapy-bluebird-vertex-crispr-casgevy/

#health #healthcare #scd #crispr #betathalassemia #transplant #lyfgenia #casgevy #healthinsurance #pharma #bigpharma #biotech

Confirmado. El precio de adquisición de #CASGEVY la nueva terapia #CRISPR producida por @vertexpharma (junto a @crisprtx) para tratar la anemia falciforme y la beta talasemia en EE.UU. en pacientes de 12 o más años será de 2,2 millones de dólares (2,2M$)
https://www.sec.gov/ix?doc=/Archives/edgar/data/0000875320/000087532023000054/vrtx-20231208.htm

From Alex Dainis Ph.D. (Wish she would post on Mastodon, she's so good ☹️) about the "very first approved CRISPR gene editing therapy in the US. Breaking genetics news, so I had to talk about it. Tell me what you want to know!"

https://x.com/AlexDainis/status/1733321710991257796?s=20

#genetherapy #crispr #geneediting #genetics #sicklecell #casgevy

#US #approves first #geneediting #treatment, #Casgevy, for #SickleCellDisease.

#SickleCell, an inherited #blood #disorder, causes #redbloodcells to become misshapen half moons that get stuck inside #blood #vessels, #restricting #bloodflow and causing what are known as #paincrises.

#Women #Transgender #LGBTQ #LGBTQIA #Health #Healthcare #Progress #BlackMastodon

https://www.cnbc.com/2023/12/08/casgevy-first-crispr-gene-editing-treatment-approved-in-us.html

Answers to questions about the first CRISPR-based medicine:
https://www.statnews.com/2023/11/16/crispr-vertex-sickle-cell-beta-thalassemia-casgevy-approval/

#health #healthcare #medicine #biotech #pharma #crispr #genetherapy #geneediting #scd #SickleCell #casgevy #lyfgenia

Most of the sickle cell community is African-American, and with that comes a lot of medical mistrust, especially for a brand-new technology.

From a sickle cell patient herself:
https://www.statnews.com/2023/12/08/casgevy-lyfgenia-vertex-bluebird-sickle-cell-gene-therapy-approval-patients/

#health #healthcare #medicine #biotech #pharma #crispr #genetherapy #geneediting #scd #SickleCell #casgevy #lyfgenia #healthequity

Adam Feuerstein spoke with #CRISPR Therapeutics CEO Sam Kulkarni on the approval and future of CRISPR-based medicines:
https://www.statnews.com/2023/12/08/crispr-therapeutics-ceo-samarth-kulkarni-interview/
#health #healthcare #medicine #biotech #pharma #crispr #genetherapy #geneediting #scd #SickleCell #casgevy

Don't miss the rest of STAT's coverage of this historic moment:
https://www.statnews.com/2023/12/08/fda-approves-casgevy-crispr-based-medicine-for-treatment-of-sickle-cell-disease/

#health #healthcare #medicine #biotech #pharma #crispr #genetherapy #geneediting #scd #SickleCell #casgevy #lyfgenia #zynteglo

Today the FDA approved 2 gene therapies to treat sickle cell disease, including the first CRISPR-based therapy.

Bluebird Bio, who has the ~$1M more expensive and not-CRISPR therapy, has a single card left to play in this competition: It's been conducting a year-long dress rehearsal for this moment

https://www.statnews.com/2023/12/08/lyfgenia-sickle-cell-gene-therapy-bluebird-vertex-crispr-casgevy/

#health #healthcare #medicine #biotech #pharma #crispr #genetherapy #geneediting #scd #SickleCell #casgevy #lyfgenia #zynteglo

The Food and Drug Administration on Friday approved a powerful 👉treatment for 🔸sickle cell disease🔸, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black.
The therapy, called "Casgevy", from Vertex Pharmaceuticals and CRISPR Therapeutics, is the ⚠️first medicine to be approved in the United States that uses the gene-editing tool 🔹CRISPR🔹, which won its inventors the Nobel Prize in chemistry in 2020.

The approval marks the first of two potential breakthroughs for the inherited blood disorder.

The FDA on Friday also approved a 👉second treatment for sickle cell disease, called "Lyfgenia", a gene therapy from drugmaker Bluebird Bio.
Both treatments work by genetically modifying a patient’s own stem cells.

Until now, the only known cure for sickle cell disease was a bone marrow transplant from a donor, which carries the risk of rejection by the immune system, in addition to the difficult process of finding a matching donor.
#casgevy #lyfgenia #crisper #Sicklecelldisease #sicklecell
https://www.nbcnews.com/health/health-news/fda-approves-cure-sickle-cell-disease-first-treatment-use-gene-editing-rcna127979

The '@MHRAgovuk has authorised world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia.'
'#Casgevy is the first medicine to be licensed that uses the innovative gene-editing tool #CRISPR.'
https://www.gov.uk/government/news/mhra-authorises-world-first-gene-therapy-that-aims-to-cure-sickle-cell-disease-and-transfusion-dependent-thalassemia

And here's our coverage of the FDA's concerns about potential off-target edits as it considers Casgevy for US approval by Dec 8:

https://www.statnews.com/2023/10/27/fda-has-some-safety-concerns-about-crispr-based-sickle-cell-treatment-documents-show/

#crispr #health #healthcare #medicine #biotech #scd #genetics #casgevy

Here's the announcement from this morning:
https://www.statnews.com/2023/11/16/u-k-approves-worlds-first-crispr-based-medicine/
#crispr #health #healthcare #medicine #biotech #scd #genetics #casgevy

Today the UK approved the first CRISPR-based treatment: A gene-editing treatment for beta thalassemia and sickle cell disease

We've been reporting on CRISPR for years @STAT. Here are the answers to your questions about Casgevy:

https://www.statnews.com/2023/11/16/crispr-vertex-sickle-cell-beta-thalassemia-casgevy-approval/

#crispr #health #healthcare #medicine #biotech #scd #genetics #casgevy