Vertex Pharmaceuticals
Boston
Massachusetts
United States
863 articles about Vertex Pharmaceuticals
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Vertex Announces Approval of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™, for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) in Kingdom of Saudi Arabia
1/9/2024
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the Saudi Food and Drug Authority (SFDA) granted Marketing Authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)
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The patient deaths were deemed unrelated to Vertex’s investigational islet cell therapy, but the study will be halted while regulatory authorities and an independent data monitoring committee review the findings.
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5 FDA Decisions to Watch in Q1
1/2/2024
Iovance’s lifileucel for advanced melanoma and Madrigal’s resmetirom for nonalcoholic steatohepatitis are just a couple of the key decisions on the FDA's docket this quarter. -
As Vertex and CRISPR Therapeutics’ exa-cel and Verve Therapeutics’ VERVE-101 move forward, questions remain about possible drawbacks of such therapies.
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Vertex to Present at the 42nd Annual J.P. Morgan Healthcare Conference on January 8
12/18/2023
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Dr. Reshma Kewalramani, Chief Executive Officer and President, will present at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 11:15 a.m. ET/8:15 a.m. PT.
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Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
12/15/2023
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY™ .
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A week after securing FDA approval, a European Medicines Agency committee has endorsed Vertex and CRISPR Therapeutics’ Casgevy for sickle cell disease and transfusion‑dependent beta thalassemia.
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Vertex Announces Positive Results From Phase 2 Study of VX-548 for the Treatment of Painful Diabetic Peripheral Neuropathy
12/13/2023
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from its Phase 2 dose-ranging study of the selective NaV1.8 inhibitor VX-548 in people with painful diabetic peripheral neuropathy (DPN).
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The company’s non-opioid painkiller candidate VX-548 decreased Numerical Pain Rating Scale scores by at least two points after 12 weeks in a mid-stage diabetic peripheral neuropathy study.
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Editas Medicine and Vertex Pharmaceuticals Enter into Non-exclusive License Agreement for Cas9
12/13/2023
Editas Medicine, Inc. and Vertex Pharmaceuticals entered into a license agreement.
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The deal follows the FDA approval of Vertex’s gene-editing sickle cell treatment and Editas’ earlier legal battle over rights to the technology.
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Vertex and CRISPR Therapeutics Announce US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease
12/8/2023
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).
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Friday’s FDA approval of Vertex-CRISPR’s Casgevy and bluebird bio’s Lyfgenia has immediately revealed startling differences between these two gene therapies: price and a black-box warning.
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This week, the FDA could approve the first CRISPR-edited therapy in the U.S., while two other companies await decisions on topical drugs.
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Health Canada Grants Market Authorization for KALYDECO (ivacaftor) for Eligible Children With Cystic Fibrosis Ages 2 Months and Older
11/29/2023
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has granted Market Authorization for the expanded use of PrKALYDECO® (ivacaftor) for the treatment of cystic fibrosis (CF) in children ages 2 months and older weighing at least 3 kg who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R or R117H.
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RoslinCT to manufacture exagamglogene autotemcel (exa-cel) for Vertex Pharmaceuticals Inc following an extensive development collaboration
11/28/2023
RoslinCT to manufacture exagamglogene autotemcel (exa-cel) for Vertex Pharmaceuticals Inc following an extensive development collaboration RoslinCT will manufacture world’s-first CRISPR-based therapy.
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European Commission Approves KAFTRIO® in Combination With Ivacaftor for the Treatment of Children With Cystic Fibrosis Ages 2 Through 5
11/23/2023
Vertex Pharmaceuticals Incorporated announced that the European Commission has granted approval for the label expansion of KAFTRIO® in a combination regimen with ivacaftor for the treatment of children with cystic fibrosis ages 2 through 5 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.
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The U.K.’s conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel raises some potential safety concerns with the risk of off-target effects.
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Vertex & CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
11/16/2023
Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that the United Kingdom Medicines and Healthcare products Regulatory Agency has granted conditional marketing authorization for CASGEVY™, a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease and transfusion-dependent beta thalassemia.
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The U.K.’s Medicines and Healthcare products Regulatory Agency on Thursday provided conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel.