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Toggle Summary Vertex Presents Positive, Updated VX-880 Results From Ongoing Phase 1/2 Study in Type 1 Diabetes at the European Association for the Study of Diabetes 59th Annual Meeting
- All patients treated with VX-880 in Parts A and B have follow-up data beyond Day 90 and have demonstrated islet cell engraftment and glucose-responsive insulin production - - All patients showed improvement across all measures of glucose control, including decreases in HbA1c, increases in blood
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Toggle Summary Vertex Receives CHMP Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor for Children With Cystic Fibrosis Ages 2 Through 5
-If approved, more than 1,200   children would be newly eligible for a medicine that could treat the underlying cause of their disease- BOSTON --(BUSINESS WIRE)--Sep. 15, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee
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Toggle Summary Vertex Researchers Awarded 2024 Breakthrough Prize in Life Sciences
- Paul Negulescu , Fredrick Van Goor and Sabine Hadida receive Breakthrough Prize for developing transformative medicines for people with cystic fibrosis – BOSTON --(BUSINESS WIRE)--Sep. 14, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that three of its researchers have
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Toggle Summary Vertex to Participate in the Morgan Stanley 21st Annual Global Healthcare Conference on September 13
BOSTON --(BUSINESS WIRE)--Aug. 30, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in the Morgan Stanley 21 st Annual Global Healthcare Conference. Dr. Reshma Kewalramani , Chief Executive Officer and President, and Charles Wagner , Executive Vice
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Toggle Summary Vertex Appoints E. Morrey Atkinson, Ph.D., to EVP and Chief Technical Operations Officer, Head of Biopharmaceutical Sciences and Manufacturing Operations
BOSTON --(BUSINESS WIRE)--Aug. 29, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that E. Morrey Atkinson , Ph.D., has been appointed Executive Vice President, Chief Technical Operations Officer, Head of Biopharmaceutical Sciences and Manufacturing Operations, effective
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Toggle Summary Vertex Announces Publication of VX-548 Positive Phase 2, Proof-of-Concept Results in New England Journal of Medicine
- First published manuscript of VX-548 preclinical and clinical trial results in a peer-reviewed journal - BOSTON --(BUSINESS WIRE)--Aug. 3, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced publication in the New England Journal of Medicine (NEJM) of preclinical data and
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Toggle Summary Vertex Reports Second Quarter 2023 Financial Results
— Product revenue of $2.49 billion , a 14% increase compared to Q2 2022 — — Company raises full year 2023 product revenue guidance to $9.7 to $9.8 billion — — FDA has accepted the exa-cel BLAs in both severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT); Priority Review
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Toggle Summary European Commission Approves ORKAMBI® (lumacaftor/ivacaftor) for the Treatment of Children With Cystic Fibrosis Ages 1 to <2 Years Old
-Nearly 300 children with cystic fibrosis and two copies of the F508del mutation are now eligible for the first time for a medicine that can treat the underlying cause of their disease- LONDON --(BUSINESS WIRE)--Jul. 5, 2023-- Vertex Pharmaceuticals today announced that the European Commission has
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Toggle Summary Vertex to Announce Second Quarter 2023 Financial Results on August 1
BOSTON --(BUSINESS WIRE)--Jun. 30, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its second quarter 2023 financial results on Tuesday, August 1, 2023 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
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Toggle Summary Vertex and Lonza to Build Dedicated Manufacturing Facility for Type 1 Diabetes (T1D) Cell Therapies
- Following positive Phase 1/2 results in Vertex’s VX-880 program, Vertex and Lonza announce strategic manufacturing collaboration - - Dedicated large-scale facility will support commercial production of Vertex’s T1D cell therapy portfolio - - Approximately 300 new jobs anticipated at Lonza
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Toggle Summary Vertex Presents Positive VX-880 Results From Ongoing Phase 1/2 Study in Type 1 Diabetes at the American Diabetes Association 83rd Scientific Sessions
–   All six patients treated with VX-880 engrafted islet cells, produced endogenous insulin (C-peptide) and had improved glycemic control while reducing or eliminating insulin use – –   The two patients with at least one year of follow-up met the criteria for the primary endpoint of elimination of
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Toggle Summary Vertex Presents Data Demonstrating Significant Benefits of Long-Term Treatment With CFTR Modulators at the European Cystic Fibrosis Conference
-Interim results of largest real-world study of TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) showed sustained improvement in lung function, reduction in pulmonary exacerbations frequency and lower rates of lung transplant and death for people with cystic fibrosis- - Twelve
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Toggle Summary Positive Results From Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology Association (EHA) Congress
- Both trials met the primary and key secondary endpoints at the pre-specified interim analysis - - Data continue to demonstrate transformative and durable benefit - - Safety profile consistent with busulfan conditioning and   autologous hematopoietic stem cell transplant - BOSTON &amp; ZUG,
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Toggle Summary FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
- First CRISPR gene-editing filings to be accepted for review by FDA - - FDA grants Priority Review for severe sickle cell disease (SCD) and Standard Review for transfusion-dependent beta thalassemia (TDT) – - PDUFA target action date of December 8, 2023 , for SCD and March 30, 2024 , for TDT –
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Toggle Summary Vertex to Participate in the Goldman Sachs 44th Annual Global Healthcare Conference on June 13
BOSTON --(BUSINESS WIRE)--May 30, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in the Goldman Sachs 44 th Annual Global Healthcare Conference. Dr. Reshma Kewalramani , Chief Executive Officer and President, and Stuart A.
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Toggle Summary Vertex Announces U.S. FDA Approval for KALYDECO® (ivacaftor) to Treat Eligible Infants With CF Ages 1 Month and Older
-First and only CFTR modulator approved for this age group- BOSTON --(BUSINESS WIRE)--May 3, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved KALYDECO ® (ivacaftor) for use in children with cystic fibrosis (CF) ages 1
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Toggle Summary Vertex Reports First Quarter 2023 Financial Results
— Product revenue of $2.37 billion , a 13% increase compared to Q1 2022 — — Company reiterates full year 2023 financial guidance, including product revenue guidance of $9.55 to $9.7 billion — — TRIKAFTA approved in U.S. for children with cystic fibrosis 2 to 5 years of age — — Pipeline advancement
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Toggle Summary Vertex Receives CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 1 to <2 Years Old
- If approved by the European Commission, nearly 300 children with cystic fibrosis and two copies of the F508del mutation would be eligible for the first time for a medicine&nbsp;that can treat the underlying cause of their disease - LONDON – 26/27/28 April 2023 – Vertex Pharmaceuticals (Nasdaq: VRTX)
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Toggle Summary Vertex Announces U.S. FDA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 2 Through 5 With Certain Mutations
-About 900 children with cystic fibrosis will now have a medicine to treat the underlying cause of their disease for the first time- BOSTON --(BUSINESS WIRE)--Apr. 26, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved the
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Toggle Summary Vertex to Present at the Bank of America Securities 2023 Health Care Conference on May 9
BOSTON --(BUSINESS WIRE)--Apr. 25, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at the Bank of America Securities 2023 Health Care Conference on Tuesday, May 9, 2023 at 9:20 a.m. PT / 12:20 p.m. ET .
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Toggle Summary Vertex to Announce First Quarter 2023 Financial Results on May 1
BOSTON --(BUSINESS WIRE)--Apr. 10, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its first quarter 2023 financial results on Monday, May 1, 2023 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
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Toggle Summary Vertex and CRISPR Therapeutics Complete Submission of Rolling Biologics License Applications (BLAs) to the US FDA for exa-cel for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
-EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review procedure has begun as of January 2023 - BOSTON &amp; ZUG, Switzerland --(BUSINESS WIRE)--Apr. 3, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced
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Toggle Summary Vertex and CRISPR Therapeutics Announce Licensing Agreement to Accelerate Development of Vertex’s Hypoimmune Cell Therapies for the Treatment of Type 1 Diabetes
- Vertex to receive non-exclusive rights to CRISPR Therapeutics’ CRISPR/Cas9 to accelerate development of potentially curative cell therapies for T1D- - CRISPR Therapeutics to receive $100M upfront payment plus milestone and royalty payments on potential future gene-edited hypoimmune T1D products-
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Toggle Summary Vertex Announces Publication in New England Journal of Medicine of Results from Phase 2 Study of Inaxaplin (VX-147)
BOSTON --(BUSINESS WIRE)--Mar. 16, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced publication in the New England Journal of Medicine   (NEJM)   of results from preclinical studies and a Phase 2 study evaluating the efficacy and safety of inaxaplin (VX-147) on top of
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Toggle Summary Vertex Announces FDA Clearance of Investigational New Drug Application for VX-264, a Novel Encapsulated Cell Therapy for the Treatment of Type 1 Diabetes
  – Vertex to initiate a Phase 1/2 clinical trial in the coming months – – VX-264 is the second investigational program in Vertex’s pipeline containing stem cell-derived, fully differentiated pancreatic islet cells for the treatment of type 1 diabetes – – VX-264 program does not require
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Toggle Summary Vertex to Present at Cowen’s 43rd Annual Health Care Conference on March 7
BOSTON --(BUSINESS WIRE)--Feb. 21, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at Cowen’s 43 rd Annual Health Care Conference on Tuesday, March 7, 2023 at 2:50 p.m. ET . A live webcast of management's remarks will be available through the
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Toggle Summary Vertex Reports Fourth Quarter and Full Year Financial 2022 Results
— Full year product revenue of $8.93 billion , an 18% increase compared to full year 2021 — — Full year GAAP and non-GAAP net income increased 42% and 53%, respectively, versus 2021— — Company provides full year 2023 product revenue guidance of $9.55 to $9.7 billion — — Exa-cel regulatory
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Toggle Summary Vertex to Announce Fourth Quarter and Full Year 2022 Financial Results on February 7
BOSTON --(BUSINESS WIRE)--Jan. 17, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its fourth quarter and full year 2022 financial results on Tuesday, February 7, 2023 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
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Toggle Summary Vertex to Present at the 41st Annual J.P. Morgan Healthcare Conference on January 9
BOSTON --(BUSINESS WIRE)--Jan. 4, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at the 41 st Annual J.P. Morgan Healthcare Conference on Monday, January 9, 2023 at 11:15 a.m. ET / 8:15 a.m. PT .
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Toggle Summary Vertex Announces Investigational New Drug (IND) Application for VX-522, mRNA Therapy for People With Cystic Fibrosis, Cleared by FDA
- Novel, inhaled mRNA therapy intended for the ~5,000 people with CF who cannot benefit from CFTR modulators - - VX-522 clinical trial in people with CF to initiate in coming weeks - BOSTON --(BUSINESS WIRE)--Dec. 12, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that
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Toggle Summary Vertex and CRISPR Therapeutics to Present at the American Society of Hematology (ASH) Annual Meeting and Exposition
- Oral presentation of encore clinical data from Phase 3 clinical trials of exa-cel - - Oral and poster presentations on quality-of-life impacts of sickle cell disease or transfusion-dependent beta-thalassemia and burden of hematopoietic stem cell transplant - BOSTON and ZUG, Switzerland
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Toggle Summary Vertex and Entrada Therapeutics Establish Collaboration to Discover and Develop Endosomal Escape Vehicle (EEV) Therapeutics for Myotonic Dystrophy Type 1 (DM1)
- Entrada to receive $224 million upfront payment and $26 million equity investment, as well as potential milestone payments and royalties - - Global collaboration includes ENTR-701, Entrada’s EEV-investigational candidate for the treatment of DM1 - BOSTON --(BUSINESS WIRE)--Dec.
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Toggle Summary Vertex to Participate in Upcoming Investor Conferences
BOSTON --(BUSINESS WIRE)--Nov. 9, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced its participation in the following upcoming investor conferences: Jefferies London Healthcare Conference on Wednesday, November 16, 2022 , at 9:10 a.m. GMT ( 4:10 a.m.
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Toggle Summary Vertex Announces Partnership with Basketball Hall-of-Famer Alonzo Mourning to Raise Awareness of APOL1-Mediated Kidney Disease (AMKD)
BOSTON --(BUSINESS WIRE)--Nov. 4, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the company has partnered with basketball Hall-of-Famer Alonzo Mourning to launch “Power Forward” ( www.PowerForwardTogether.com ), an educational initiative aimed at raising awareness
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Toggle Summary Vertex to Present New Data on its Portfolio of Cystic Fibrosis Medicines at the 2022 North American Cystic Fibrosis Conference
- New clinical data show TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) continues to deliver significant benefit across a variety of outcomes and over the long term in people with CF - BOSTON --(BUSINESS WIRE)--Nov. 3, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
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Toggle Summary Vertex Reports Third Quarter 2022 Financial Results
— Product revenue of $2.33 billion , an 18% increase compared to Q3 2021 — — Company increases full year 2022 product revenue guidance to $8.8 billion to $8.9 billion — — Significant progress in mid- and late-stage pipeline, with near-term commercialization opportunities: Initiating global exa-cel
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Toggle Summary Vertex to Announce Third Quarter 2022 Financial Results on October 27
BOSTON --(BUSINESS WIRE)--Oct. 13, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its third quarter 2022 financial results on Thursday, October 27, 2022 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
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Toggle Summary Vertex Advances Program Targeting Alpha-1 Antitrypsin Deficiency
 - Initiates first-in-human trial of next-wave AAT corrector, VX-634 - - Phase 2 study evaluating the impact of longer-term treatment with VX-864 to be initiated in the coming weeks - - Additional AAT correctors to enter the clinic starting in 2023 - BOSTON --(BUSINESS WIRE)--Oct.
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Toggle Summary Vertex and CRISPR Therapeutics Announce Global exa-cel Regulatory Submissions for Sickle Cell Disease and Beta Thalassemia in 2022
- Exa-cel will be submitted to the U.S. FDA for rolling review beginning in November, with completion of the U.S. submission package in Q1 2023 - - EMA and MHRA submissions are on track for Q4 2022 - - Exa-cel granted Fast Track, Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug
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Toggle Summary Vertex Appoints Jonathan Biller as Chief Legal Officer
BOSTON --(BUSINESS WIRE)--Sep. 8, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Jonathan Biller has been appointed Chief Legal Officer, effective September 19, 2022 . Mr. Biller will report directly to Vertex’s Chief Executive Officer and President, Reshma
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Toggle Summary Vertex Announces U.S. FDA Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 12 to <24 months
-With this approval, approximately 300 children with two copies of the F508del mutation will have a medicine to treat the underlying cause of their disease for the first time- BOSTON --(BUSINESS WIRE)--Sep. 2, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S.
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Toggle Summary Vertex to Participate in the Wells Fargo Healthcare Conference on Friday, September 9
BOSTON --(BUSINESS WIRE)--Aug. 31, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the company will participate in a fireside chat at the Wells Fargo Healthcare Conference on Friday, September 9, 2022 at 11:00 a.m. ET . A live webcast of management’s remarks will be
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Toggle Summary Vertex Reports Second Quarter 2022 Financial Results
— Product revenues of $2.20 billion , a 22% increase compared to Q2 2021 — — Company raises full year 2022 product revenue guidance to $8.6 to $8.8 billion — — Recent exa-cel and VX-880 clinical data presentations demonstrate transformative potential for patients with sickle cell disease, beta
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Toggle Summary Vertex Advances VX-548 in Acute and Neuropathic Pain
- VX-548 advances into pivotal development for people with acute pain ; Phase 3 program to initiate in Q4 2022 - - Phase 2 dose-ranging trial in neuropathic pain expected to initiate by year end - -Breakthrough Therapy Designation granted by FDA - BOSTON --(BUSINESS WIRE)--Jul.
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Toggle Summary Vertex and Verve Therapeutics Establish Collaboration to Discover and Develop an In Vivo Gene Editing Program for Liver Disease
Verve to Receive $25 Million Upfront Payment and $35 Million Equity Investment, as Well as Potential Milestones and Royalties BOSTON and CAMBRIDGE, Mass., July 20, 2022 (GLOBE NEWSWIRE) -- Vertex Pharmaceuticals Incorporated&nbsp;(Nasdaq: VRTX) and Verve Therapeutics, Inc.
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Toggle Summary Vertex to Announce Second Quarter 2022 Financial Results on August 4
BOSTON --(BUSINESS WIRE)--Jul. 18, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its second quarter 2022 financial results on Thursday, August 4, 2022 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
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Toggle Summary Vertex to Acquire ViaCyte, With the Goal of Accelerating its Potentially Curative VX-880 Programs in Type 1 Diabetes
- ViaCyte brings tools, technologies and assets with potential to accelerate development of VX-880, Vertex’s fully differentiated, insulin-producing, stem cell derived islets - - ViaCyte to be acquired for $320 million in cash- BOSTON --(BUSINESS WIRE)--Jul.
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Toggle Summary Vertex Announces FDA Has Lifted the Clinical Hold on VX-880 Phase 1/2 Clinical Trial for the Treatment of Type 1 Diabetes
BOSTON --(BUSINESS WIRE)--Jul. 5, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated
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Toggle Summary Vertex and CRISPR Therapeutics Present New Data on More Patients With Longer Follow-Up Treated With exagamglogene autotemcel (exa-cel) at the 2022 European Hematology Association (EHA) Congress
–   Data from 75 patients with transfusion-dependent beta thalassemia or severe sickle cell disease with follow-up of up to 37.2 months continue to demonstrate that exa-cel has the potential to be a one-time functional cure – – Safety profile generally consistent with myeloablative conditioning and
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Toggle Summary Vertex to Present Data Demonstrating Significant Benefits of Long-Term and Early Treatment With CFTR Modulators at the European Cystic Fibrosis Conference
- TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) real-world safety and effectiveness interim results show   improved lung function and significant reductions in risk of pulmonary exacerbations, lung transplant and death for people with cystic fibrosis (CF) - - Study in people with CF
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