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678 articles with Vertex Pharmaceuticals
Vertex and CRISPR Therapeutics Present New Data in 22 Patients With Greater Than 3 Months Follow-Up Post-Treatment With Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at European Hematology Association Annual Meeting
Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced new data on 22 patients, with follow-up of at least 3 months, and ranging from 4 months to 26 months, treated with the investigational CRISPR/Cas9-based gene-editing therapy, CTX001, that show a consistent and sustained response to treatment.
Shares of Vertex fell more than 13% in premarket trading after it announced that it will not advance a novel small molecule corrector of the Z-AAT protein into a Phase III study.
CORRECTING and REPLACING Vertex Announces U.S. FDA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations
Vertex Pharmaceuticals Incorporated announced the U.S. Food and Drug Administration approved expanded use of TRIKAFTA® to include children with cystic fibrosis ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene or a mutation in the CFTR gene that is responsive to TRIKAFTA based on in vitro data.
June is turning out to be a busy month for PDUFA dates for the U.S. Food and Drug Administration. Here’s a look at this week’s dates.
Vertex and CRISPR Therapeutics to Present New Clinical Data on Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001™ For Severe Hemoglobinopathies at the Annual European Hematology Association Virtual Congress
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced two abstracts detailing updated data from the ongoing CTX001 clinical trials have been accepted for presentation during the European Hematology Association (EHA) 2021 Virtual Congress. Abstract #EP736 entitled “ CTX001 for Sickle Cell Disease: Safety and Efficacy Results from the Ongoing CLIMB SCD-121
5/11/2021“The recent progress of cystic fibrosis drugs has been amazing and, in my case, miraculous,” said Kelly Peters, who lives with cystic fibrosis. “The new drugs are not a cure, but they feel pretty close.”
Vertex to Present at the Bank of America Securities 2021 Virtual Health Care Conference on May 13
BioSpace takes an in-depth look at the Top 25 of Q1.
Vertex and CRISPR Therapeutics Announce Priority Medicines (PRIME) Designation Granted by the European Medicines Agency to CTX001™ for Transfusion-Dependent Beta Thalassemia
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of transfusion-dependent beta thalassemia (TDT).
Executives at pharmaceutical companies are known for receiving large salaries. When bonuses and stock options are factored into the picture, their annual compensation can jump into the range of tens of millions of dollars, like Pfizer’s Albert Bourla, Alex Gorsky from Johnson & Johnson, and Astra...
Under the terms of the expanded deal, Vertex will be responsible for 60% of the costs of developing, manufacturing and commercializing CTX001 with support from CRISPR Therapeutics, and will receive 60% of profits from global sales.
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the full year and fourth quarter ended December 31, 2020 and provided full-year 2021 financial guidance.
The U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug application that will allow Boston-based Vertex Pharmaceuticals to proceed with a clinical trial of its investigational stem cell-derived, fully differentiated pancreatic islet cell treatment for type 1 diabetes (...
Vertex Announces FDA Clearance of Investigational New Drug (IND) Application for VX-880, a Novel Cell Therapy for the Treatment of Type 1 Diabetes (T1D)
- Vertex will initiate a Phase 1/2 clinical trial in first half of 2021 - - VX-880 is the first stem cell-derived therapy evaluating fully differentiated pancreatic islet cells for the treatment of T1D -
Vertex Announces U.S. FDA Acceptance of Supplemental New Drug Application for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations
Vertex Announces U.S. FDA Acceptance of Supplemental New Drug Application for TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations
On Monday, at the JP Morgan Healthcare Conference held virtually, Vertex Pharmaceuticals’ chief executive officer Reshma Kewalramani said the company is looking to buy “mid- and late-stage assets.”
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at the J.P. Morgan Healthcare Conference on Monday, January 11, 2021 at 9:10 a.m. ET (6:10 a.m. PT). The audio portion of management’s remarks can be accessed live through Vertex’s website, www.vrtx.com , in the “Investors” section under the “News and Events” page. A replay of the conference webcast will be archived on the compa
Vertex Announces New Drug Submission for Investigational Triple Combination Medicine for the Treatment of Cystic Fibrosis Has Been Accepted for Priority Review by Health Canada
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced its New Drug Submission for TRIKAFTA ® , Vertex’s investigational triple combination medicine, has been accepted for Priority Review by Health Canada for the treatment of cystic fibrosis (CF) in people ages 12 years and older. “We are pleased this submission has been accepted for Priority Review by Health Canada, and we anticipate this accelerated review pr
Skyhawk Therapeutics and Vertex Pharmaceuticals inked a strategic research collaboration and licensing deal focused on developing novel small molecules that modulate RNA splicing.
Vertex Announces FDA Approvals of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) for Use in People With CF With Certain Rare Mutations
Vertex Announces FDA Approvals of TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO ® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO ® (ivacaftor) for Use in People With CF With Certain Rare Mutations - More than 600 people with certain rare CF mutations are now eligible for TRIKAFTA, SYMDEKO or KALYDECO - BOSTON--( BUSINESS WIRE )-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Ad