“U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older.”
February 7, 2026 at 2:53 AM
“U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older.”
Vertex is running out of time to establish Casgevy as a transformative treatment for sickle cell. www.statnews.com/2026/02/05/v...
Vertex’s CRISPR treatment for sickle cell disease hits unexpected roadblock
Vertex is running out of time to establish Casgevy as a transformative treatment for sickle cell.
www.statnews.com
February 6, 2026 at 6:05 PM
Vertex is running out of time to establish Casgevy as a transformative treatment for sickle cell. www.statnews.com/2026/02/05/v...
Interesting piece:
"Vertex’s CRISPR treatment for #sicklecell disease hits unexpected roadblock - Although Casgevy can cure, a basic hospital procedure is slowing uptake and threatening its edge" www.statnews.com/2026/02/05/v...
"Vertex’s CRISPR treatment for #sicklecell disease hits unexpected roadblock - Although Casgevy can cure, a basic hospital procedure is slowing uptake and threatening its edge" www.statnews.com/2026/02/05/v...
Vertex’s CRISPR treatment for sickle cell disease hits unexpected roadblock
Vertex is running out of time to establish Casgevy as a transformative treatment for sickle cell.
www.statnews.com
February 5, 2026 at 5:54 PM
Interesting piece:
"Vertex’s CRISPR treatment for #sicklecell disease hits unexpected roadblock - Although Casgevy can cure, a basic hospital procedure is slowing uptake and threatening its edge" www.statnews.com/2026/02/05/v...
"Vertex’s CRISPR treatment for #sicklecell disease hits unexpected roadblock - Although Casgevy can cure, a basic hospital procedure is slowing uptake and threatening its edge" www.statnews.com/2026/02/05/v...
Ex-bench: I watch $VRTX; Vertex nails ex-vivo CRISPR HSC editing (Casgevy proof), HDR/indel control is focus, market underprices modal upside - I predict 40% rerate. analysi
February 2, 2026 at 1:04 PM
Ex-bench: I watch $VRTX; Vertex nails ex-vivo CRISPR HSC editing (Casgevy proof), HDR/indel control is focus, market underprices modal upside - I predict 40% rerate. analysi
Watching $VRTX; Vertex HDR CRISPR in HSCs undervalued - focus/exposure risk. analysi. https://www.biopharmadive.com/news/ASH-Vertex-Casgevy-childen-sickle-cell-thalassemia/807182/
January 22, 2026 at 2:06 PM
Watching $VRTX; Vertex HDR CRISPR in HSCs undervalued - focus/exposure risk. analysi. https://www.biopharmadive.com/news/ASH-Vertex-Casgevy-childen-sickle-cell-thalassemia/807182/
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
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
January 19, 2026 at 10:30 AM
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
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
The company in 2026 is seeking approval from global regulators for Casgevy and two cystic fibrosis drugs in younger patients.
Vertex Says Casgevy Topped $100M Sales in 2025, Sees 'Blockbuster' Potential
www.precisionmedicineonline.com
January 13, 2026 at 4:11 AM
The company in 2026 is seeking approval from global regulators for Casgevy and two cystic fibrosis drugs in younger patients.
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
www.cincofrentes.com/2026/01/tecn...
#CRISPR #TerapiaGénica #MedicinaDePrecisión #Casgevy #Ciencia #Genética
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.
www.cincofrentes.com
January 7, 2026 at 5:42 PM
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
www.cincofrentes.com/2026/01/tecn...
#CRISPR #TerapiaGénica #MedicinaDePrecisión #Casgevy #Ciencia #Genética
Casgevy can be used to tackle treat sickle cell disease and beta thalassaemia
Abu Dhabi clinic uses life-changing gene therapy to treat blood disorders in UAE first | The National
www.thenationalnews.com
January 6, 2026 at 8:15 AM
Casgevy can be used to tackle treat sickle cell disease and beta thalassaemia
Day 4: Kavita Mehta
Has spent her life needing transfusions and medication to keep her alive. But now she has become one of the first patients to receive a gene-editing treatment, Casgevy, on the NHS.
Cut from the i 1.1.26.
Article by Jane Kirby.
Photo uncredited.
Has spent her life needing transfusions and medication to keep her alive. But now she has become one of the first patients to receive a gene-editing treatment, Casgevy, on the NHS.
Cut from the i 1.1.26.
Article by Jane Kirby.
Photo uncredited.
January 4, 2026 at 10:27 PM
Day 4: Kavita Mehta
Has spent her life needing transfusions and medication to keep her alive. But now she has become one of the first patients to receive a gene-editing treatment, Casgevy, on the NHS.
Cut from the i 1.1.26.
Article by Jane Kirby.
Photo uncredited.
Has spent her life needing transfusions and medication to keep her alive. But now she has become one of the first patients to receive a gene-editing treatment, Casgevy, on the NHS.
Cut from the i 1.1.26.
Article by Jane Kirby.
Photo uncredited.
Casgevy uses the patient's own stem cells which are removed, edited over a six-month period so they produce haemoglobin, then reintroduced into the body via infusion. In international clinical trials the technique removed the need for blood transfusions for at least a year in 98% of patients.
January 3, 2026 at 11:26 AM
Casgevy uses the patient's own stem cells which are removed, edited over a six-month period so they produce haemoglobin, then reintroduced into the body via infusion. In international clinical trials the technique removed the need for blood transfusions for at least a year in 98% of patients.
Last year the NHS became one of the first health systems in the world to use the therapy called Casgevy, for beta thalassaemia, which mainly affects people of Asian, Mediterranean, and Middle Eastern descent.
January 3, 2026 at 11:26 AM
Last year the NHS became one of the first health systems in the world to use the therapy called Casgevy, for beta thalassaemia, which mainly affects people of Asian, Mediterranean, and Middle Eastern descent.
Ex-researcer: I watch Vertex; $VRTX CRISPR HDR in HSCs, focus. https://www.biopharmadive.com/news/ASH-Vertex-Casgevy-childen-sickle-cell-thalassemia/807182/
December 31, 2025 at 1:05 PM
Ex-researcer: I watch Vertex; $VRTX CRISPR HDR in HSCs, focus. https://www.biopharmadive.com/news/ASH-Vertex-Casgevy-childen-sickle-cell-thalassemia/807182/
Sickle cell patients can now receive CRISPR gene therapy, as Casgevy gains regulatory approval in the US and UK. This milestone marks a major step forward in the Biology Century, where programming life could account for 40% of manufacturing by 2040.
December 29, 2025 at 4:40 PM
Sickle cell patients can now receive CRISPR gene therapy, as Casgevy gains regulatory approval in the US and UK. This milestone marks a major step forward in the Biology Century, where programming life could account for 40% of manufacturing by 2040.
There’s fair concern around a few African ancestry associated-SNPs w Casgevy. Suspect if in enchancers/promoters, deleterious effects would take much time to tell tho.
December 24, 2025 at 3:45 PM
There’s fair concern around a few African ancestry associated-SNPs w Casgevy. Suspect if in enchancers/promoters, deleterious effects would take much time to tell tho.
Vertex Presents New CASGEVY® Data in Patients 5-11 Years and Announces Plan for Global Regulatory Submissions
Vertex Presents New CASGEVY® Data in Patients 5-11 Years and Announces Plan for Global Regulatory Submissions
On December 6, 2025, Vertex Pharmaceuticals Inc. (“Vertex”) announced data from several studies demonstrating the clinical benefits of CASGEVY®...
www.jdsupra.com
December 22, 2025 at 10:17 PM
Vertex Presents New CASGEVY® Data in Patients 5-11 Years and Announces Plan for Global Regulatory Submissions
Vertex Presents New CASGEVY® Data in Patients 5-11 Years and Announces Plan for Global Regulatory Submissions
Vertex Presents New CASGEVY® Data in Patients 5-11 Years and Announces Plan for Global Regulatory Submissions
On December 6, 2025, Vertex Pharmaceuticals Inc. (“Vertex”) announced data from several studies demonstrating the clinical benefits of CASGEVY®...
www.jdsupra.com
December 22, 2025 at 10:17 PM
Vertex Presents New CASGEVY® Data in Patients 5-11 Years and Announces Plan for Global Regulatory Submissions
Vertex Presents New CASGEVY® Data in Patients 5-11 Years and Announces Plan for Global Regulatory Submissions
Vertex Presents New CASGEVY® Data in Patients 5-11 Years and Announces Plan for Global Regulatory Submissions
On December 6, 2025, Vertex Pharmaceuticals Inc. (“Vertex”) announced data from several studies demonstrating the clinical benefits of CASGEVY®...
www.jdsupra.com
December 22, 2025 at 10:17 PM
Vertex Presents New CASGEVY® Data in Patients 5-11 Years and Announces Plan for Global Regulatory Submissions
ToolGen Files Complaint Against Vertex Concerning Gene Therapy Casgevy®
ToolGen Files Complaint Against Vertex Concerning Gene Therapy Casgevy®
On November 18, 2025, ToolGen, Inc. (“ToolGen”) filed a complaint (“Complaint”) in the District of Massachusetts alleging infringement of its...
www.jdsupra.com
December 9, 2025 at 7:31 PM
ToolGen Files Complaint Against Vertex Concerning Gene Therapy Casgevy®
ToolGen Files Complaint Against Vertex Concerning Gene Therapy Casgevy®
ToolGen Files Complaint Against Vertex Concerning Gene Therapy Casgevy®
On November 18, 2025, ToolGen, Inc. (“ToolGen”) filed a complaint (“Complaint”) in the District of Massachusetts alleging infringement of its...
www.jdsupra.com
December 9, 2025 at 7:31 PM
ToolGen Files Complaint Against Vertex Concerning Gene Therapy Casgevy®
ToolGen Files Complaint Against Vertex Concerning Gene Therapy Casgevy®
ToolGen Files Complaint Against Vertex Concerning Gene Therapy Casgevy®
On November 18, 2025, ToolGen, Inc. (“ToolGen”) filed a complaint (“Complaint”) in the District of Massachusetts alleging infringement of its...
www.jdsupra.com
December 9, 2025 at 7:31 PM
ToolGen Files Complaint Against Vertex Concerning Gene Therapy Casgevy®
The gene therapy exagamglogene autotemcel (exa-cel; Casgevy) -- already approved for patients 12 years and older with TDT and sickle cell disease -- led to durable clinical benefits in kids ages 5 to 11 years, two phase III trials showed. #ASH25
www.medpagetoday.com/meetingcover...
www.medpagetoday.com/meetingcover...
Gene Therapy Also Effective in Younger Children With Inherited Blood Disorders
Exa-cel could provide benefit before kids develop chronic organ damage
www.medpagetoday.com
December 8, 2025 at 5:14 PM
The gene therapy exagamglogene autotemcel (exa-cel; Casgevy) -- already approved for patients 12 years and older with TDT and sickle cell disease -- led to durable clinical benefits in kids ages 5 to 11 years, two phase III trials showed. #ASH25
www.medpagetoday.com/meetingcover...
www.medpagetoday.com/meetingcover...
Vertex CRISPR therapy hits early goal in children with blood disorders
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake. Elevate your perspective with NextTech News, where…
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake. Elevate your perspective with NextTech News, where…
Vertex CRISPR therapy hits early goal in children with blood disorders
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake. Elevate your perspective with NextTech News, where innovation meets insight. Discover the latest breakthroughs, get exclusive updates, and connect with a global network of future-focused thinkers. Unlock tomorrow’s trends today: read more, subscribe to our newsletter, and become part of the NextTech community at NextTech-news.com
nexttech-news.com
December 7, 2025 at 10:49 AM
Vertex CRISPR therapy hits early goal in children with blood disorders
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake. Elevate your perspective with NextTech News, where…
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake. Elevate your perspective with NextTech News, where…
Vertex CRISPR therapy hits early goal in children with blood disorders
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake. Elevate your perspective with NextTech News, where…
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake. Elevate your perspective with NextTech News, where…
Vertex CRISPR therapy hits early goal in children with blood disorders
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake. Elevate your perspective with NextTech News, where innovation meets insight. Discover the latest breakthroughs, get exclusive updates, and connect with a global network of future-focused thinkers. Unlock tomorrow’s trends today: read more, subscribe to our newsletter, and become part of the NextTech community at NextTech-news.com
nexttech-news.com
December 7, 2025 at 10:49 AM
Vertex CRISPR therapy hits early goal in children with blood disorders
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake. Elevate your perspective with NextTech News, where…
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake. Elevate your perspective with NextTech News, where…
Citizens Maintains Market Outperform on CRISPR Therapeutics (CRSP) Despite Casgevy Slowdown
Citizens Maintains Market Outperform on CRISPR Therapeutics (CRSP) Despite Casgevy Slowdown | BeyondLINK
beyondlink.live
December 1, 2025 at 1:19 AM
Citizens Maintains Market Outperform on CRISPR Therapeutics (CRSP) Despite Casgevy Slowdown