Brittany TrangJul 15, 2024

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

Vertex sues U.S. government over access to fertility support services for Casgevy patients

CRISPR treatment for sickle cell disease carries a high risk of infertility. Vertex wants to preserve patient fertility, but a federal rule poses problems.

STAT
Lluis MontoliuDec 17, 2023
¿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/
Desde las salinas de Santa Pola a Casgevy - Gen-Ética

En 30 años, desde 1993 a 2023, hemos transitado desde un descubrimiento de investigación básica, la existencia de curiosas repeticiones de secuencias de ADN en el genoma de microorganismo procariotas, […]

Gen-Ética
Lluis MontoliuDec 17, 2023
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
Nace la medicina CRISPR, con la promesa de salvar millones de vidas

La aprobación de la primera terapia de edición del ADN abre la puerta a una revolución en el tratamiento de multitud de enfermedades, como el cáncer

El País
Lluis MontoliuDec 15, 2023
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
Reacción: la EMA da luz verde al primer medicamento en la UE que utiliza la edición genética de CRISPR

La Agencia Europa del Medicamento ha recomendado la aprobación del primer medicamento en la UE que utiliza la técnica de edición genética CRISPR/Cas9.

SMC España
Show threadBrittany TrangDec 13, 2023

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

Tough road ahead for Bluebird Bio despite FDA approval for sickle cell therapy

The FDA's approval of two gene therapies sets the stage for intense competition between Bluebird Bio and the partnership of Vertex and CRISPR Therapeutics.

STAT
Lluis MontoliuDec 10, 2023
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
Inline XBRL Viewer

kurtshDec 9, 2023

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

Alex Dainis, PhD 🧬 (@AlexDainis) on X

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

X (formerly Twitter)
Susan Larson ♀️🏳️‍🌈🏳️‍⚧️🌈Dec 8, 2023

#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

Show threadBrittany TrangDec 8, 2023

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

Key questions (and answers) about the historic approval of a CRISPR-based medicine

Here are some key questions (and answers) about the historic approval of a CRISPR-based medicine.

STAT
Show threadBrittany TrangDec 8, 2023

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

After living with sickle cell disease for 39 years, I’m both excited and skeptical about the newly approved gene therapies

What a sickle cell disease patient thinks is the biggest challenge facing a newly approved gene therapy.

STAT